Interview with David Panzirer and Dana Ball – April 21, 2014

David Panzirer was named a trustee of the Helmsley Charitable Trust (HCT) in 2007, just five months after learning that his six-year-old daughter Morgan had been diagnosed with type 1 diabetes. These events launched more than a year and a half of intensive, full-time due diligence about the disease, which culminated in the Trust’s launching of the T1D Program. Today, it represents the largest private funding program in the world dedicated to advancing type 1 diabetes research, treatment, and services – in 2013, approximately $50 million per year. From the beginning, patient advocate Dana Ball worked tirelessly with David to build and ultimately direct the Trust’s T1D program, as well as to co-found the influential T1D Exchange. Dana is now the CEO of Unitio, a new standalone non-profit that oversees the T1D Exchange; it receives funding from the Helmsley Charitable Trust and other non-profit and corporate partners.

In just a bit over seven years since its inception, HCT’s T1D program has truly changed the face of type 1 diabetes by funding ~$200 million in four project areas: Research, Systems, Technology, and Outreach. From seeding the initial artificial pancreas team at JDRF in 2008 to creating the 26,000+ patient T1D Exchange registry in 2009 to launching support for diabetes camps starting in 2011, David, Dana, and the HCT/T1D Exchange teams have plowed full steam ahead with one single mission in mind: improving the lives of people affected by type 1 diabetes. And instead of fragmenting the ecosystem, David and Dana have emphasized collaboration and partnership through their joint work with the NIH, ADA, JDRF, large and small industry players, academia, and small and medium-sized non-profits.

Our series of conversations with the pair spanning nearly ten hours in all was fascinating on a number of levels, and we have divided the content into several sections below. Following background on HCT and the T1D Program, you can read the duo’s views on five key areas including (i) funding and philanthropy in type 1 diabetes; (ii) the direction and future of type 1 diabetes; (iii) the founding and vision of the T1D Exchange; (iv) diabetes technology and automating insulin delivery; and (v) some of the field’s biggest debates. The Appendix offers an additional glimpse into David and Dana’s stories, background, and views on advocacy and a cure for type 1 diabetes.

Main Interview

Background on the Helmsley Charitable Trust (HCT) and the T1D Program

KELLY CLOSE: Thank you so much for sitting down with us today. I remember really vividly when I met both of you in New York in 2008 at the suggestion of Dr. Bruce Buckingham. And we sat in that diner on Lexington Avenue and didn’t stop talking about type 1 for two hours straight! It’s amazing to think about how you have influenced the landscape of type 1 diabetes – we’re in such a more promising position in type 1 today, and the HCT in particular has helped make so many strides. To start off, can you share some background on the formation of the Helmsley Charitable Trust?

DAVID: Absolutely. My grandmother, Leona Helmsley, amassed a huge fortune through real estate. Upon her death in 2007, her will dictated that virtually all of her assets be liquidated and transferred into a charitable trust. Today, it is worth between $5 and $6 billion dollars. We’re the 12^th biggest foundation in the world. I didn't grow up with money, so it’s surreal what her legacy has enabled.

My grandmother appointed five trustees before her death, including her brother (who has since passed away), Walter (my half-brother), John Codey, Sandor Frankel, and myself. John was a close friend and advisor of my grandmother’s, and Sandor is a lawyer with whom she worked closely.

KELLY CLOSE: It’s an incredible story. As we understand it, the Trust was being put together right around the time your daughter, Morgan, was diagnosed with type 1 diabetes.

DAVID: My daughter was diagnosed five months before Leona passed – March 23, 2007 at 4:33 pm. [Chuckles ruefully] Not that I remember the exact time … At that point, Leona had given $5.0 million to two different diabetes causes, to the JDRF as well as to another organization. This was based on Morgan’s diagnosis, of course. Those were the very initial grants, before I was even really involved in helping operate the Trust. We started as Trustees in April 2008.

ADAM BROWN: The Trust has seven areas of focus – how did the trustees initially narrow it down?

DAVID: It was labor intensive. Each of the trustees came to the table with different things that were relevant to them. We did a heavy amount of due diligence on a number of these ideas and as a group have approved the programs that emerged. I obviously was very interested in exploring what the Trust could do in the type 1 diabetes space.

KELLY: Thank you! We owe so much to you for that as a community. Little did I know that five years after founding Close Concerns and a year after founding diaTribe, that the scope of type 1 diabetes would change so much globally due to one person’s perspective, feeling, and intuition for our world. What drove your decision to become so personally involved in the development of HCT’s program?

DAVID: I made this call fairly soon after my grandmother died. I’m not a religious person, but the fact that I was appointed as a Trustee just five months after my daughter was diagnosed with type 1 diabetes was a real message to me as to what I should be doing with my life. At that point, I was still working in real estate, at RFR Realty, but I immediately started doing diligence in diabetes – that is what led us to our conversation with you, very early.

KELLY: Yes, in 2008. That was also a formative meeting for me, when I began to quickly understand what you had the power to do for type 1 at the Helmsley Charitable Trust. Although – you redefine this each year with what you make possible! Thinking about Morgan - when did she find out that type 1 diabetes was going to be a primary thrust of the Charitable Trust?

DAVID: She was six or seven years old. Listen, she just wants to do her thing. She knows I’m working with diabetes. She knows I’m trying to make life easier. She never asks details – never. Morgan obviously knows I travel a lot, but she hasn’t shown an interest in the actual details of what I do. I kind of feel like diabetes is enough of her life, and I don’t need to push that with her. If she’s interested, she’s interested. She’s read some of the articles outlining what’s going on at the Trust, so she’s got a sense. But she’s never said anything.

KELLY: What Morgan is responsible for is incredible – I can’t think of another child anywhere in diabetes that is actually changing the scope of our field the way that she is and has, due to her diagnosis and your drive. Her place in history is pretty monumental.

DAVID: She’s certainly the driver. When I first started out my singular drive was to find a cure for my daughter. But I soon came to understand that my responsibility is a lot bigger than just for my child.

HANNAH DEMING: How large is the Trust’s T1D program?

DAVID: To maintain our nonprofit status, overall, we must give away 5% a year of the ~$5.0 billion HCT endowment ($250 million). The T1D program currently funds around $50 million a year. Since our first grants, we’ve funded about $200 million to date.

KELLY: It’s remarkable what you have brought us. To give our readers some context, can you discuss how that funding compares to NIH, JDRF, ADA, and others? When did you learn what all these organizations were funding?

DAVID: HCT’s T1D Program represents approximately 10% of the available T1D budget when you include NIH/NIDDK, JDRF, ADA and DRI. We think it’s about $500-600 million per year from the sources in the US. That’s a big number and a big responsibility for the Trust staff. Overall, I think the right strategy is happening – HCT, JDRF, and others engage the NIH/NIDDK and try to work collaboratively to really understand the global strategic plan.

KELLY: Some worry that type 1 gets less funding because private foundations have stepped in. How do you think about that?

DAVID: I am not sure that is the case. We can only control what we do, and we need all the help we can get. Making this disease easier to deal with and ultimately curing it is not an easy task and I don’t think anyone who understands the landscape thinks we have this under control, and if they do, they are wrong.

HANNAH: That’s well understood by us. It is amazing all that your team at HCT is responsible for! Can you tell us a bit more about this?

DAVID: HCT is about an 80-person organization in total, and the diabetes team is ten people. The Trust’s T1D program is the largest in terms of staff because it was the first one out of the gate. The second HCT program is also healthcare related – the brilliant rural healthcare program in South Dakota.

Funding and Philanthropy

KELLY: There’s so much to ask! Let’s start with what everyone wants to know. How do you choose who and what to fund through the Helmsley Charitable Trust’s T1D program? What's most important?

DAVID: We start with our four main program areas: Research, Technology, Systems, and Outreach. Each area receives a budget and a strategy. Without getting into too much detail, we do view technology as the way to impact people with T1D in the near term (5-7 years). We also create goals for each area, so we choose what to fund based on what we believe will give us the best chance of achieving our goals for that particular area.

We have a program and process to ensure funding decisions are driven by a strategic plan based on how HCT feels they can make the biggest impact for people with T1D and effectively influence the type 1 ecosystem. Most of HCT grants have been directed, providing funding to support a specific project with clearly defined goals, budget, and milestones – it makes sense to identify the gap or question and find the best person/people to work together to solve the problem. Talent is key and most projects involve senior and junior team members – this provides wisdom and expertise along with new ideas and innovation. What is most important is a well-defined plan to address the problem and a timeline and deliverables so funders feel confident a conclusion will be reached. Ultimately, could this move a better solution for patients with diabetes forward? HCT limits funding to three-year grants, because it’s long enough but it’s short enough. Three years is long enough to get going and then monitor it closely to make go, no-go decisions.

HANNAH: How did you come up with Research, Technology, Systems, and Outreach? Can you say roughly how these areas are divided in terms of requests for funds and how you ultimately fund them? And, how do all your team members work together? It sounds like they are doing an enormous amount, for ten people.

DAVID: It really was an evolution of our learning. I started out thinking we were going to cure type 1. After months of diligence I realized a cure wasn’t around the corner and we needed to ease the burden of managing this disease. At the Trust, we feel that the best way to do this is through technology. The Systems portfolio (which is predominately the T1D Exchange) came about with Dana repeatedly asking the question, “Where is the human data?” And Outreach is our near-term win. Thankfully, Dana was smart enough to realize that I am a hard charging type A personality and all the things we are doing take time to see results, so Outreach is our instant gratification. We send under privileged, underserved children to diabetes camps. We also help a couple international organizations get supplies into people’s hands in parts of the world where type 1 is a death sentence.

ADAM: We’d love to learn a little more about the trends in your philanthropy. How many request for funds does HCT receive each year and how does the “mix” change? Where do you see the growth in need?

DAVID: Aside from the few open requests for proposal (RFPs) we do per year, the Trust funds by invite only.

HANNAH: What do you think are the unique challenges in diabetes vs. other disease categories that HCT funds?

DAVID: One of the major challenges of type 1 diabetes is there are not enough people with type 1 diabetes to attract the big dollars from the large pharmaceutical companies. We are not quite an orphan disease, but we don’t have tons of people like in type 2 diabetes, where there are nearly 30 million people who are all living longer every year and represent an increasingly attractive market. If you are the CEO of a major pharma company, your job is to return dollars to shareholders. If you have $1 billion in R&D to invest, you will put that money to work in type 2 vs. type 1 every time because of the many more end users of your potential products. The phenomenon is exacerbated even more in the type 1 technology space – pumps and continuous glucose monitoring – because only about 30% of people with type 1 diabetes use pumps and less than 10% use CGM.

DANA: Type 1 diabetes is a complicated small disease that is fragmented by what is required to treat the disease in the short term. We need devices, drugs, and strong clinical support because of the demands of patients with diabetes assuming the role of their pancreas. We still don’t know the cause and there no clear targets for addressing the cause/effect of type 1 diabetes today. This has created a complex matrix that dilutes research efforts and financial resources. With so many questions, needs and opportunities, finances are strained. This is further complicated by a lifetime of diabetes and changing treatment needs and health concerns over time.

KELLY: No doubt there’s substantial complexity. We like to think that although type 1 represents a smaller population, there are lots of funds that could be saved and redirected if type 1 patients were much better managed or if type 1 were eradicated. Do you have a sense of how much is being spent on type 1 annually in the US and overseas and how much could be saved? We are asked that a lot and we haven’t been able to calculate it, though we’re hoping that the recent NEJM piece by Dr. Edward Gregg will lead us closer to understanding prevalence and costs of diabetes complications in type 1 specifically.

DANA: I have heard lots of numbers kicked around, but I don’t think we have a way to calculate a real number for costs. If we could eliminate complications and severe hypoglycemic events, that would have a dramatic impact on the dollars spent. This is one of the tasks we are looking at – how to quantify this so we can use that information when talking to the regulators and payers.

HANNAH: We’d love to talk to you more about your partnerships, which is one way HCT clearly addresses the complexity. When working with manufacturers, what are the biggest challenges? What are the biggest benefits? What advice do you have about what makes a company a good partner?

DAVID: The biggest challenge with the larger companies is how slowly they move. The benefit is they are the only entities with the wherewithal to manufacture and distribute products and deliver them to people. The ideal structure is a very open and direct relationship where the companies will show us their pipeline and priorities, and we have the opportunity to accelerate products to people with type 1 diabetes. In a perfect world, the company will be considering doing something in the type 1 diabetes space, but with a lack of impetus to move forward, our funding can sometimes provide the push to move things along. We have also been told by some of the company executives that when they partner with nonprofits like us, those funded programs become much harder for the company to cut later. That’s the case for many reasons, including that there is usual a financial penalty back to us for doing so.

KELLY: Hmm, that’s a big responsibility. And a big opportunity of course – funding from HCT really comes with a “halo” effect. How else can companies be good partners?

DANA: As David was saying, the biggest challenges are the speed for companies to engage and respond to nonprofit opportunities, and the ability of companies to trust us enough to be transparent about their pipeline. Depending on the size of the company, it’s often challenging to identify the internal champion that can help bridge organizational divisions. The greatest benefit is that industry has the capital and expertise to commercialize and support new solutions. The best partnerships are built on trust and aligned missions where nonprofit resources can be leveraged to accelerate the delivery of better care, products, and therapies. The best deal is mutually beneficial deal that ensures the patient voice is present, has a clearly defined program with deliverables, could decrease the time for development, and includes a return on the investment to seed additional research or establish financial support programs for un/underinsured people with diabetes.

KELLY: We’d love to know more about competition for funds each year and the size of requests, though we understand that these requests are by-invite only. So to ask it another way, on the corporate front, how do you feel about the balance between innovation coming from large companies and smaller companies? What do you see as strengths and weaknesses of working with each? How do you think about ways in which HCT and T1D Exchange can make the most difference?

DAVID: The larger companies have the ability to commercialize products, but move more slowly than the smaller companies. The smaller companies are often fighting for their own survival and depending upon who has invested in them, might have different timelines for products that may or may not be aligned with people’s needs. I think philanthropic dollars should be high-risk investments. Venture capital (VC) dollars have, for the most part, gone away and philanthropic dollars must help fill that void. HCT can help by funding the smaller companies’ R&D and give them a better investment partner than VC partners, who typically look for quick exits. With the larger companies, HCT can invest to help them prioritize their pipelines.

DANA: All companies want to innovate. At T1D Exchange, we find smaller companies are traditionally rich with entrepreneurial spirit and innovation, but short on financial resources and often struggle with business and commercialization expertise. Larger companies want to innovate as well, but are often stymied by the confines of their corporate structure, business/shareholder/market/risk issues, competition for budget, lack of vision, and evidence. Throughout my career I have worked strategically with both small and large companies and feel it’s a continuum – sometimes the biggest bang for the buck is supporting a development project in a start-up with the goal to collaborate later with an industry partner for commercialization.

Alternatively, there may be an opportunity to spur innovation in a large company and provide the passion and capital/resources to move something that isn’t a priority for the company. HCT and the T1D Exchange have the ability to work with other type 1 diabetes funders and stakeholders to do what’s best for patients with diabetes – and since HCT doesn’t fundraise, it can take more risk. It’s also important to note sometimes grantees need more than what they think they need – at HCT and the Iacocca Foundation, we funded equipment and provided funding to hire expert consultants and teams of talented people to support our projects and programs. I think we’ve gone from a decade of not talking to industry to real collaborations that are now happening.

HANNAH: It’s been incredible from the perspective of patients and healthcare providers to see the investments. How much research do you do before deciding which companies’ projects to invest in?

DANA: I’ve always managed trusted relationships with companies and they have been transparent with what they have in development. If the company has the data, has invested enough money, and has internal support to move something forward, funders can help de-risk it. That’s when the advocacy groups or nonprofit groups can step in and say, “We’ll help you move that along even faster and create awareness.” What that does is two things. One, it’s a little bit of money, which is important. But more importantly, it sends a signal to the company leadership that there’s an unmet need and that patients want this product or device.

ADAM: Switching gears a bit, what are the most misunderstood funding needs? 

DAVID: I think people need to understand the business side of type 1 diabetes. I have heard many people criticize us and JDRF among others for funding large companies. What needs to be understood is that the business model for these companies in the type 1 diabetes device space is not very good. There are roughly 500,000 using pumps and less than 10% of type 1s on CGM. If it costs about $500 million to get a product to market, the numbers just don’t work for company executives whose job it is to return shareholder profits. This is especially true on the insulin pump front, where Medtronic has over 65% market share, leaving the others to fight over the remaining 35%. Further complicating the pump market is that the existing user base gets a new pump every three or four years. These factors make it very tough. We want to push/incentivize/de-risk promising new devices and therapies so the companies can see value in a market that they otherwise wouldn’t and, ultimately, develop technologies that will improve the lives of people with T1D.

DANA: First, type 1 diabetes is complicated, meaning that discovery and development is hard and takes a lot of money. It’s also a small disease, as we said, meaning we need strong evidence and an army of engaged clinicians and patients to ensure the consumer market is viable. People often forget the need to start with the end in mind – we want to develop a drug or device to treat type 1 diabetes on the path to a cure. This means it’s a business and sometimes when a question is answered, we need to go in a different direction – this can’t be about supporting academic careers forever, because science and medicine will be disrupted and new talent, tools, and skills are needed to move forward. We can’t apologize if we want to make a difference.

KELLY: Of course. Can you name some careers that you’ve been excited to help push ahead?

DAVID: Dr. Matthias Hebrok (Director, Diabetes Center, UCSF), Dr. Rene Maehr (Assistant Professor, University of Massachusetts Medical School), Peter Nerothin (Founder/President, Insulindependence), and Christina Roth (Founder/CEO, College Diabetes Network) – those are just a few of the many we could mention.

HANNAH: As a budding researcher, that is so exciting to hear. Can you also tell us more about how you decide which non-profits to fund?

DAVID: We only have one criterion on what/who we fund, and that is what is best for people with type 1 diabetes. We fund those entities, nonprofit or for-profit, that give us the best chance of achieving our goals within each program area. But in this realm, you must stay disciplined and focused, because if you don’t, your approach becomes like a shotgun – in my opinion that’s less effective. When you maintain focus, it’s a lot more like using a cannon.

DANA: I like to partner with entities that have the best combination of talent, experience, success, clear vision, ability to execute, and willingness to truly collaborate. My projects as a funder and on the nonprofit side at the T1D Exchange are always active projects – we don’t sit back and wait for the results; we take an active role.

I’m in type 1 diabetes today because I really believe in the approach – the Helmsley Charitable Trust funds could have fragmented the community and made it worse. Instead, David has worked with everyone in the community and brought them together. I can’t think of another person that I know in type 1 diabetes, or even in philanthropy, other than David that could have done what he’s accomplished.

KELLY: It has been remarkable – he has changed our landscape forever. And, even in type 1 – especially in type 1! – it’s a complicated landscape. Can you talk about when funding projects, how you balance cure vs. complications vs. better type 1 treatment?

DAVID: I am not a big believer in “cure,” and if there is going to be a cure, it is certainly not on the horizon. With that in mind, care is key. In my opinion, the way to impact care is to make this disease easier to manage, and I am convinced technology is the key to doing this. We believe CGM is the gating factor towards being able to begin to automate insulin delivery. We have invested in three companies around CGM technology: Medtronic, BD, and Dexcom. We are continuing to look at others – academic projects and for-profit. We have also put some money to work in earlier technologies that we think will be needed down the road, such as a stabilized glucagon.

DANA: I think it’s important to balance the goal of cure with the need and responsibility to improve quality of life for patients with diabetes and reduce the burden of managing type 1 diabetes. Short term, there is a lot of potential in automating insulin delivery and testing drugs that may improve glycemic variability and the amount of insulin needed to manage type 1 diabetes. Research, development, and care demands stretch the funding community’s resources – as we move our short term goals forward, we are also developing systems, like the T1D Exchange, that can gather enormous amounts of data on human disease so we can be more strategic about prevention and complications.

KELLY: This is an incredible responsibility. David, can you talk more about the key people on your team and how you all evaluate the outcomes of your funding?

DAVID: With all projects, we always start with a goal. As the HCT program has matured, we have developed our team based on our focus areas. The program has defined which expertise we need in house vs. which expertise we can hire on a consulting basis. We now have two PhD scientists on staff to help us parse thru all of the findings on the research landscape: Gina Agiostratidou and Ben Williams. We hired a mechanical engineer named Marc Anderson – he has specific type 1 experience to help drive the technology portfolio.

In terms of how we measure impact, it depends on which of the four areas we’re talking about:

• With Technology, we measure impact by how quickly we are helping the companies to accelerate their timelines in getting better products to people living with type 1 diabetes.
• Systems refer to tools and resources that we have funded that help all stakeholders working in type 1 diabetes, like the T1D Exchange. The fact that the T1D Exchange is the database now cited for all type 1 diabetes in the US is very impressive. We believe the type 1 diabetes community as a whole is better off with the T1D Exchange than without it. (Editor’s note – to say the least!)
• Outreach is our near-term win: funding the smaller organizations and the diabetes camps allows us to make an immediate impact, while the other areas tend to take more time.
• In Research we measure impact by moving things forward or moving on in a timely and efficient manner, all the while having a focus on the goal – getting a drug or therapy/intervention into people living with type 1 diabetes. We’re also focused on getting a traditionally siloed research community to work together and share data across institutions and disciplines.

KELLY: How do you think about the growth in type 2 diabetes competing with funding for type 1?

DAVID: It is a major challenge. The companies focus on type 2 because of the sheer numbers of end users – type 1 is a small market. However, as type 2 grows and a small fraction of that population start to use pumps and CGMs, that will ultimately help people with type 1 diabetes by increasing the number of people using these devices.

DANA: Years ago, we split type 1 and type 2; I think the future will prove there are many forms of diabetes ranging from the most severe form of autoimmune diabetes to insulin resistance in the aging population – eventually, most people will get diabetes if you live long enough. There is potential value to use type 2 drugs to improve control, insulin use, weight, resistance, and mood in type 1s. I think it’s a diabetes universe with many subtypes, and we need to do a better job of telling this story to patients, clinicians, and the public at large.

KELLY: In terms of supporting early scientific careers in diabetes, how do you look at that today and in the future?

DANA: I still think it’s a huge problem. I don’t think that there are enough resources and incentives to attract extraordinarily talented people to type 1 diabetes. Talented molecular biologists are needed for diabetes, as they are for other diseases. I worry that other diseases like cancer have more money to recruit the young talent. There are only so many brilliant, new, young people. And the brightest and best discoveries happen when you’re younger. There’s a pool of talented people coming into the world of research, but if all the money is in cancer, you’re going to have to make a decision: “Do I want to go into a field where there isn’t a lot of funding or do I want to go to a field where there is a lot of funding?”

HANNAH: That’s illuminating. And a bit depressing. How can HCT help support academic research? How much time does HCT think researchers should spend fundraising?

DAVID: We do support academic research, but do so strategically. In most of our academic grants the teams consist of senior scientists and clinicians that work with and mentor fellows, post docs and junior scientists – we have already seen junior careers launched through our funding. We also push our investigators to always have their eye towards the clinic. We are not interested in funding science just for the sake of science; we want things to progress towards people in the clinic.

DANA: It’s hard to say how much time the researcher should spend on fundraising, but we understand there is a lot of work in securing funds, which limits the ability to be productive. It seems this is an institution question, and perhaps academic institutions should be expanding their model to invest in providing new support to researchers who are developing IP and moving bench projects towards the clinic. Clearly there are for-profit/nonprofit partnerships and we should study which programs work best and why.

KELLY: With declining dollars for research funding, do you think the NIH will have the same number of programs going forward – just thinner – or is it your impression that NIH is narrowing their focus?

DANA: Over time, what I’ve watched is really a change in how they have operated. I think that the NIH/NIDDK is incredibly collaborative and completely transparent with us, with JDRF, and with other patient advocacy groups. They want everyone to understand what they’ll be funding and their funding cycles. I think that that’s a fantastic development – they really are partners now.

As far as the budget, you know the size of our government and the healthcare demands of this country are a tsunami. I think it would be a bad time to stop funding from the government. There are certain programs in certain parts of the ecosystem that would implode if they didn’t have the government support. I’m hoping that the changes that may have to be made don’t disrupt our space. Sometimes change is okay because it makes us work smarter. And maybe it’s an opportunity to think about restructuring. If so, I would hope it would involve patient advocacy groups and other stakeholders in the space – they can really think about what’s critical to fund and what can other stakeholders potentially pick up.

DAVID: The decline in dollars is clearly a major loss for people with type 1 diabetes. I agree it can force us to work smarter, but in the end there are less dollars flowing into the area. My sense is that many people outside NIDDK in the government believe type 1 diabetes is a safe and easily managed disease – they have no idea how complex type 1 is and how many programs are required to move both devices and therapies forward.

KELLY: Right. That is really worrying. Do you see people starting to mobilize and think about that change being made?

DANA: Yes. In diabetes, there has been a shift over the past several years – funders collaborate prospectively and are working on problems together. Jeffrey Brewer has done a great job leading change at JDRF. Outside type 1 diabetes, groups like FasterCures are working in DC all the time with all stakeholders, all disease groups, all patient advocacy groups, companies, and the government. David and I recommended that HCT become the title sponsor for the initial FasterCures Partnering for Cures Conference (P4C), which has become one of the best conferences in the biotech space. It’s the only one where philanthropists, governments, disease advocacy groups, and investment companies all come together. This year there were about 1,000 people at the conference. It’s the only meeting I can go to outside of type 1 diabetes meetings, and it’s because I want to, not because I have to. P4C has developed a great platform for collaboration and how stakeholders can partner for success.

KELLY: What's a recent model of highly effective funding in the field of philanthropy? Within diabetes?

DAVID: What the Cystic Fibrosis Foundation has done has really paved the way for what all of us are trying to achieve. They are the gold standard we all strive to duplicate. I was fortunate enough to spend some time during our diligence with their President and CEO, Dr. Robert Beall. Bob created a registry for Cystic Fibrosis in the 1960s; his is one of the oldest and most compelling. We learned a great deal from Bob and his team.

DANA: Within diabetes, the HCT/JDRF partnership to automate insulin delivery is an example of a partnership that is working very well. HCT’s first grant to JDRF was to build the artificial pancreas team under the great Dr. Aaron Kowalski’s leadership, and they have been able to work closely to move the field forward. There are many HCT/JDRF funded projects; I also like the work between JDRF, ADA, and HCT on Standards of Care. In addition, their work on the Special Diabetes Program renewal is evidence of the community working together.

KELLY: These are very exciting and important initiatives to patients. Thank you for all the work on these fronts. Can you tell us who are the other leaders in diabetes and non-profit that you most admire and learn from?

DAVID: I am obviously very fond of my mentor and one of my closest friends, Dana Ball, who has taught me so much – not only about type 1 diabetes but also about life. I admire people who have taken it upon themselves to have an impact. People like Dr. Ed Damiano, Jeffrey Brewer, Dr. Mark Atkinson, and many, many others who are devoted to this cause. I think the people who have had a significant impact on me and have helped me in my diligence from the corporate world would be Linda Tharby from BD and Terry Gregg from Dexcom just to name two, as there are too many to list here. I have learned tons from each of them. I also admire the excellent team we’ve been fortunate to pull together here at the Trust. They are ridiculously knowledgeable and driven when it comes to making sure that the Trust’s resources have the greatest impact in moving the ball forward for people with T1D.

DANA: I have enjoyed and respect everyone in type 1 diabetes, particularly the clinicians in our T1D Exchange Clinic Network, T1D investigators, the teams at JDRF, ADA, and our corporate members. Terry Gregg at Dexcom is a leader who persisted, Linda Tharby from BD and Katie Szyman from Medtronic are dedicated and share our mission. We were very happy to see Dr. David Kendall land at Lilly, Dr. Henry Anhalt with Sanofi. Just a few examples and of course you, Kelly, and your team who keep the world up to date on what is happening, what’s working and what’s not, and your patient work with diaTribe – thank you. There are many other strong leaders in the diabetes advocate patient community and there are everyday leaders who care for their families and show up every day with a smile even after a day of horrible lows and chasing highs. In nonprofit more generally, Dr. Bob Beall (CEO, Cystic Fibrosis Foundation), Kathy Guisti (CEO, Multiple Myeloma Research Foundation), Debi Brooks (Co-Founder, Michael J. Fox Foundation), Michael Milken (Chairman, Milken Institute), and Faster Cures.

KELLY: Thank you. We really appreciate you discussing some of the models in for-profit and not-for-profit.

The Future of Type 1 Diabetes

KELLY: What do you think will be the most important advance in type 1 diabetes in the next five years? The next 10 years?

DAVID: I think the most important advance in the next five years will be a CGM that will be accurate enough to begin to automate some insulin delivery. I will never forget a slide I saw in presentation by Dr. Aaron Kowalski many years ago, that said “someone with an A1c of 7.0% has a normal blood glucose (70-120 mg/dl) 30% of the time. I have always said if we can automate basal rates overnight and keep people euglycemic overnight, that this would have a dramatic impact on those living with type 1 diabetes. I would hope that in the next ten years, we would move closer towards automation, but I don’t believe you will ever have a device that you put on people and say, ‘Go live your life like you don’t have diabetes.” I don’t believe a fully automated system is achievable. I don’t think you can remove the patient entirely from the equation. I do think we can have dramatic improvement from where we are today, but I think type 1 diabetes is way too complex to fully automate. You’ll never ever see the term “artificial pancreas” or anything like that coming out of the Trust. I’ve told everybody I'd kill it.

KELLY: And I don’t think anyone minds if insulin isn’t fully automated; if we have to push a button to say we’re eating a meal or exercising – I’d guess that’s fine by patients. That’s a high class problem compared to using insulin now, where it has such a narrow therapeutic window and where it’s so dangerous. Dana?

DANA: In the next five years, incredibly accurate, easy to wear, connected sensors. I believe the future of personalized care and precision medicine for patients with diabetes is founded on sensor use and adoption. In addition, I hope to see progress towards automating insulin delivery and reducing the burden on patients through technology – low glucose suspend and treat to range systems; testing and greater awareness of type 2 drugs for type 1 diabetes; and standardization of device data. On the more basic science side, major advances in the next five years would include clinical progress with cellular therapies and staging type 1 diabetes through deep characterization of patients (i.e., to inform new discovery work for study/trial design). I hope we also see patients come to understand that they are the missing piece and they need to take action, whether that’s getting involved in a study, donating blood, or becoming part of our type 1 diabetes army.

In the next 10 years I hope we’ll see an automated system to deliver insulin with CGM that integrates into handhelds; testing glucagon as a therapy; drug combination work progressing towards a type 1 diabetes cocktail; a biological pancreas (cells in a device); diabetes data and electronic medical record (EMR) integration; decision support for clinicians; a viable glucose responsive insulin; new validated targets and mechanisms for drug discovery; and continued support by patient and family members or research.

ADAM: How would each of you define a cure for type 1 diabetes? Would putting a pump on every three days count as a cure, if insulin delivery could be largely automated?

DAVID: I define a cure as though you live your life like you never had the disease.

DANA: I think an automated insulin delivery system is in the cure family. I think it cures the burden. HIV patients that were once dying can now take three pills at bedtime. They understand intellectually that the disease is not gone, but they are not the same as they were before. I don’t define a cure as permanently reversing type 1 diabetes with no management required, whether it’s a medication or whether it’s a device. I think a “cure” is anything that relieves an individual from the burden of managing type 1 diabetes and controlling blood glucose. Diabetes is time, it’s stress, it’s money, it’s worry, and it’s the damage of not having your blood glucose under control.

HANNAH: What is the biggest thing the type 1 diabetes field is missing? 

DAVID: I think that the groups working in type 1 diabetes are very siloed; they don’t always work together and I think a huge reason for this is the competition for fundraising dollars. We have started and continue to help to build bridges and work together with all entities that have an interest in improving the lives of patients with diabetes.

DANA: I agree – too many competing interests and most people want to operate independently.

KELLY: Advocates should all think about that a little more. Switching gears a bit, can we talk about what you see the top areas of unmet need in type 1 diabetes globally?

DAVID: The fact that in some places in the world type 1 diabetes is a death sentence is horrible. We need to get people access to the tools required to stay alive. A stabilized insulin that isn’t affected by heat or cold would be awesome.

DANA: Agreed – we definitely need far better global care and access to insulin. In my view, the other major areas of unmet need include funding and expertise for translation studies; funding for phase 2 studies; data and care standards; new endpoints for studies (e.g., time-in-range); quality of life; and improving reimbursement and the financial burden of managing type 1 diabetes both in the US and around the world.

ADAM: Big picture, what do you each see as the biggest challenge or threat the field of type 1 diabetes faces in the next five or ten years? 

DAVID: As far as within industry, we need to try and increase the number of people using technologies to manage type 1 diabetes – the companies need to see that the numbers are increasing and there is money to be made in type 1 diabetes technology products. If industry cannot see a way to make a profit in the space, the innovations will continue to be very slow.

DANA: Tailored therapies will create subsets of patients to market therapies. How will we incentivize financial investment and industry engagement? How do we prepare for success and accumulate the evidence to address a stressful reimbursement environment? Will patients show up and embrace changing care and treatment options? Who will care for the population with type 1 diabetes?

KELLY: That sets up our next question really well. Please talk to us a little more about how you are thinking about type 2 diabetes drugs for type 1 diabetes?

DANA: I think there’s a lot of promise. It’s not going to be an insulin-only world. In terms of moving forward, I think the trick is looking at what’s already been learned from some of these studies, and then creating partnerships with the companies that have the drugs. We can run some very smart clinical trials to figure out who’s going to respond to these drugs and why. We also need to educate everyone that it’s not an insulin-only world.

Throughout my career for 13 years, I never said type 1 or type 2. I always thought, “It can’t be right.” So many in the type 1 field were saying, “Well, this drug isn’t important. This is for the type 2 overweight population. It’s never going to help type 1. Why would we invest in this?” And I kept saying, “We don’t know that.” Glycemic variability! It’s glucose, and there’s a connection here – I think in many ways we’ve done a disservice. Now the world thinks in black and white, type 1 and type 2, and here we are. Now 13 years later, I’m glad I never said a word, because now I’m knocking on all the companies’ doors and saying, “Come in, we want to design a study because your drug may be able to help people with type 1.”

But we have an uphill battle to reeducate people. Many think that type 1 diabetes is insulin-only, and these drugs are for type 2s only. I think we have to figure out how to get ADA, JDRF, ad agencies, and HCT funding some of this. It’s now coming together – people with type 1 may benefit from type 2 drugs, and people with type 2 should start insulin earlier.

KELLY: When you think about type 2 drugs for type 1 diabetes, what drug classes intrigue you the most?

DANA: Metformin is the low-hanging fruit, because it’s so safe and it’s safe for kids. If we talk about changing the perception that type 1 diabetes is an insulin-only system, metformin is so important. The world has also changed and type 1 isn’t a thin person disease anymore; there are plenty of people with an increased BMI that are going to benefit from metformin. [Editor’s Note: Courtesy of a $2.8 million JDRF grant, the T1D Exchange is studying the use of metformin in overweight adolescents with type 1 diabetes].

KELLY: That is so good to hear; it will be so interesting to see that how these trial results emerge since having a drug for type 1 that also happened to be anti-cancer would certainly be a benefit. And besides metformin?

DANA: Right now, we’re focused on DPP-4 inhibitors, GLP-1 agonists, SGLT-1 and SGLT-2 inhibitors. This is going to take time and we need to figure out the right patients for these different classes of drugs. We need to escalate the conversation.

KELLY: In terms of glucose responsive insulin, did you think about investing in SmartCells or other smart insulins? Merck wound up investing $500 million, which seems like the kind of investment you are glad to see.

DANA: I was at HCT when we learned about SmartCells. JDRF was already involved and it was early, not a lot of data, and they had enough interest and support. Now, it’s inside of Merck and it’s really quiet. When I was a funder, I believed if we could influence the speed or influence the company through a partnership with funding, we pursued it. If an opportunity had enough resources or another funder was committed, we traditionally passed and monitored the progress. I think glucose responsive insulin is a potential game changer for people with type 1 diabetes.

KELLY: When you look at the future of type 1 diabetes, do you think a lot of care delivery innovation needs to happen? Or is it an exception to this growing theme?

DANA: I don’t worry about it as much for type 1 diabetes, because the average type 1 adult sees their physician twice a year for 20 minutes. So that’s 40 minutes out of the year that a physician is involved with a patient’s care. Type 1 is a primarily patient- or family-driven disease on a day-to-day, hour-to-hour basis. I think we need to improve the technology to relieve the burden. Ideally, the device data would go to straight to the medical record, and when you go in to see your provider, you wouldn’t have to do anything. The nurse would print a report out and the doctor would look at it in advance. Then, the doctor could sit down and say, “So how are things?”

DAVID: Type 1 diabetes is the only disease I know of that patients and their caregivers are making dosing decisions of a drug that if you get it really wrong, could kill you – and they are making these decisions 24/7/365. Type 1 diabetes is a relentless grind that never pauses. Ultimately patients and their caregivers become the lead in managing their disease if they are going to maintain good glycemic control. We strive to get technology to market that can ease the burden of managing this disease.

KELLY: What about the artificial/bionic pancreas – to what degree can that ease the burden, in your mind?

DANA: If it’s smart enough, yes, because it will provide real value to patients – we have seen the power of automating insulin delivery through the studies this year. Part of the problem with current systems is they require more time and work for physicians – time for printing, reading, analyzing, interpreting, and giving you advice on what to do. Not all physicians are going to support the additional administrative work.

KELLY: Then it’s also about influencing the decision makers on reimbursement. More investment in HCP time now could reap great rewards later; this should very obviously be based on the landmark results of the DCCT.

DAVID: I think automated insulin delivery (I hate the name “artificial pancreas”)/bionic pancreas has the potential to revolutionize care of people living with type 1 diabetes. We know from data in the Exchange that the large majority of people are failing to meet their A1c goals. In most cases it isn’t for lack of trying. People with type 1 must become a part-time pancreas, a job that is exquisitely done by the body in people that don’t have diabetes.

T1D Exchange

KELLY: Looking back at what you did in deciding to make the T1D Exchange registry, you were way ahead of your time. Can you take us back?

DANA: Back in 2002, when I started in type 1 diabetes at the Iacocca Foundation, the most important thing was to understand human disease – this is why we supported Dr. Denise Faustman. Dr. Faustman created a large cohort of type 1 patients. She had hundreds of people traveling to her lab from all over the US and she had patients, data and samples. As a grant maker, as the guy who invested a philanthropist’s money, it was important for me to fund projects based on evidence. Without really understanding what was happening in people with type 1 diabetes, I didn’t sleep at night. Each grant seemed like a long shot and we were funding just another investigator with mouse data saying, “I think this is going to be the cure.” And I sat there with my arms crossed asking, “How do you do this?”

I wanted a compass. I wanted a large database and an engine to inform what we know about type 1 diabetes and where to go, “Now we have the evidence on these patients. We have a collection of biosamples that we know everything about. Now, who’s the right researcher to do the analysis? Who’s the right company with the right drug portfolio to partner with and what is the right study?”

ADAM: In other words, T1D Exchange is sort of a GPS in your toolbox?

DANA: That’s right. When we started this in 2008, David said, “Why do I need this thing?” I said, “The Exchange is a compass to drive our research and accelerate studies.” Say investigator A comes to you and says, “Our drug hits a target in the mouse and we think it will work in type 1 patients” Well, in the near future, we can look in T1D Exchange and say, “How many patients have this marker and if we don’t have the answer, we can get it.” The T1D Exchange network of over 70 clinics also provides a network for clinical studies – we can now go from concept to trial very quickly.

And even for companies, if we grow the registry and data large enough, they can look at a drug target or a mechanism in the patient population. Think of all the wasted money that goes into drug development. Wouldn’t it be better to know earlier if something is only going to work in 2% of the patients? I believe in the richness of the data over time will allow us to understand more about clinical relevance and through small, well-designed studies, understand what’s worth moving forward. Development time and access to data and patients are an expensive obstacle we hope to solve with T1D Exchange.

KELLY: How has the Exchange evolved since its creation? How large is the current team?

DANA: The Exchange was incubated with HCT funding from February of 2010 until February of 2012. It was incubated knowing that it ultimately had to be under a separate nonprofit organization. It took two years to achieve that nonprofit status, but we were finally granted it in June 2013. [Editor’s Note: The T1D Exchange is the first program of the newly formed non-profit, Unitio.] Under that initial three-year grant from the Trust, the Exchange was in design, development, and testing mode. David, myself, Marie Schiller, and Dr. Roy Beck were the executive committee overseeing design and development of the Exchange. Original projections for the build and development of the Exchange over the first three years were projected up to $50 million, depending on the budget for seeding clinical and Biorepository studies and organizational development for the nonprofit to manage and maintain the Exchange when complete. We were very pleased that less money was needed.

In June of 2012, I transitioned from HCT to lead T1D Exchange, based out of Boston. The T1D Exchange currently has 44 full time employees in three locations – 15 of those are in the business and Glu offices based in Boston, 22 are at the Jaeb Center for Health Research based in Tampa, Florida (responsible for the activities of the Clinic Registry and Clinic Network), and seven staff members are at the Biobank Operations Center in Seattle. Dr. Roy Beck is the director of the Jaeb clinical coordinating center, and Dr. Carla Greenbaum is the director of the Biobank.

KELLY: That’s amazing that you recruited Dr. Greenbaum – she’s incredible.

DANA: Yes – beyond her broad clinical expertise and experience in type 1 research, her sweet spot is “What is the question we’re trying to answer?” She really understands protocol and cohort development. Dr. Greenbaum’s team supports studies involving biosample collection, analysis, and distribution of samples for research purposes.

KELLY: What are you most proud of now that T1D Exchange is established? 

DAVID: The T1D Exchange Registry data have been presented all over the world and the data is being used to help direct all who play in the type 1 diabetes space.

DANA: I’m proud that it was created by the community, for the community, and now the community is seeing the benefits and working with the Exchange as a facilitator of research. I’m also very proud that the clinical community has given so much to make this happen, and I’m blown away by their commitment and collaboration.

ADAM: How has the T1D Exchange data affected type 1 diabetes? In your view, what has been or would ultimately be a home run?

DANA: We now know what we used to guess about in the US – we have evidence. I think the data has educated us and our clinicians about the reality of the state of type 1 diabetes care and outcomes in 2014. I also think the registry has created a platform for clinicians and patients to learn more about the person with diabetes.

A home run is data that is leveraged to create change – better care, leveraged when necessary for regulatory and reimbursement issues, and educating the public about the challenges of living with type 1 diabetes. Another home run is data that informs studies – we develop protocols with our clinic KOLs, we use the network to execute studies, Glu members help industry understand what they want and need in products and therapies, and we are working on supporting type 1 breakthroughs from beginning to end.

DAVID: When you start with a goal of delivering a drug, device or therapy to market, you have two gatekeepers: the FDA and the payers. Both need data for different reasons – the FDA to prove that it is safe and the payers that need proof that it is effective. Dana had the vision when creating the Exchange so that it could fill this huge void.

KELLY: What has been the hardest thing about developing T1D Exchange? To what degree did you understand how complicated it was before you did it?

DANA: Honestly, it’s good that I hadn’t quite realized the enormity of the complexity. If I knew how complicated it would be, I may not have done it. I don’t want to think about that! It’s complicated because it’s a model where the sum of the parts is greater than the individual parts. So we had to figure out how to co-create it with the type 1 stakeholder community.

DAVID: The T1D Exchange model only existed in pieces. Dana was the one with the vision to put them all together. We have learned tons along the way. As Dana always says “it isn’t easy building it while we are flying it”.

KELLY: What has been the biggest surprise?

DANA: Two years ago when we started the conversation, I thought industry would jump up and down and want to work with us immediately. I thought they would want to work with us from discovery to delivery of new therapies and devices. We had patients, we had the network, we had everything. I was surprised how long it took to get these first industry partnerships moving along. I underestimated the challenges of working within the silos of the companies to try to take a cohesive approach to developing better drugs and devices for patients.

HANNAH: Does industry have more of an incentive to collaborate between internal silos, as we’ve seen at J&J with Animas and Janssen working together on the sales side?

DANA: I’m not an industry guy, so I just thought if you’re trying to save money and work smart, T1D Exchange would be a slam dunk. And instead, it was a little bit of “No.” We heard, “This is how we work” and “Here’s our path to working with nonprofits.” The speed of change and adoption for companies was a little bit slower than I would have expected. I’ve come to appreciate it takes time to get the teams on the same page and demonstrate the value of thinking with the patient in mind from the beginning.

KELLY: At the Cleveland Clinic Medical Innovation Summit, we heard how big pharma companies are looking to make the risky bets later and later in the process. This seems to be increasingly important.

DANA: Yes, and for a small disease like type 1 diabetes, the more we can de-risk it and add some positive data based on patients, the closer they will come. There are interesting models we’re exploring with industry. If all the companies are looking for new drug targets, we should de-risk that for them. We have samples, we have patients, and we have partners with money. If we could do more discovery work and flood the field with new targets, then they could take it inside their walls and see what they have for molecules. I think nonprofit research groups have proven they can be change agents and successfully lower the risk for industry.

HANNAH: How do you prioritize research questions to look at in T1D Exchange?

DANA: It has to be a dynamic approach. The first phase of creating a registry was to get a baseline understanding of the state of how people were living with type 1 diabetes. Now, Dr. Roy Beck and I get on the phone and say, “What are the best uses of the Exchange and what are the most important questions we can address?” My top two areas are automating insulin delivery and exploring available type 2 drugs for type 1 diabetes. We’re always thinking about what are our top priorities and what’s the Exchange data needed to support those priorities.

We have to think about how to keep the data fresh and relevant. First, we have to understand device data on an annual basis because the world’s going to change quickly every year that we go forward. Second, we’re going to have to understand everything about patients and we’re going to have to understand a world beyond just insulin. We also have to think ahead from a health economics perspective. If we’re going to see strong efficacy and improvement in patient outcomes because of using a non-insulin-only approach to type 1 diabetes, we want to make sure that we have the health economics data ready for anyone that needs it.

I always think in ten years – what’s going to happen and what else do we need? I also worry about NIH/NIDDK funding, especially the Special Diabetes Program. What if we had to fill the gaps? What if we had to do more? We’re trying to think about ways to prepare.

KELLY: Can you detail how the T1D Exchange data collection process works? I always thought that there was some magic way you were getting all of the data from the Exchange. My imagination pictured data from all the centers landing in Dr. Beck’s office in real time at his fingertips. In other words, he could tell us how many severe hypos there were this morning.

DANA: No. It’s not real time. The problem is that every Electronic Health Record (EHR) isn’t the same, they aren’t interoperable, and to thoroughly understand type 1, you need more than just the EHR data. We find that even within EHR companies like EPIC, each version of EPIC isn’t necessarily the same – they are not interoperable and not all data we need is standardized. There are good standards for medications and labs, but many data don’t have standards. In some ways, we are creating standards for type 1 diabetes. Additionally, not every institution uses EPIC and some clinics use their own homegrown EHRs. In the US, we have not solved this problem. It’s absolutely ridiculous. So we decided to spend the money and the time to do it manually.

How we operationalized the T1D Exchange registry was to pay clinic coordinators at our clinics to enter everything that’s in the EMR into the registry database using a tablet. We also collect comprehensive patient entered data about the registry patients. A1cs were entered retrospectively for ten years, if they existed – for about 100% of the over 26,000 registry members. The patient data is updated annually and we use custom modules to support additional data as needed.

ADAM: Did you think about designing the system from the start so it could pull data straight from the EHR?

DANA: We looked at EHR integration when we started developing the registry four years ago. EHR companies were busy growing their businesses and focusing on market share created by meaningful use requirements. We realized it wasn’t the right time and it was tough to get a meeting or even have a conversation with them. Now that that’s settling down, we are working on a pilot project to test an integration plan to automate data collection.

KELLY: It’s impressive what you’ve been able to do with the current system given the constraints and the lack of interoperability.

DANA: We knew there was such an unmet need that it warranted the investment to manually create the registry and maintain it through annual data collection. We have collaborated with The Cystic Fibrosis Foundation – who also uses manual collection for their registry – and other groups who are trying to solve the interoperability issues. We have found the International Diabetes Center and Cincinnati Children’s Hospital to be great collaborators on the pilot project in development.

ADAM: What is the biggest obstacle to getting EHR integration? Have you seen improvements in EMRs over time?

DANA: Time and money. They’re all different. Unfortunately, I haven’t seen that many improvements in EHRs. I think one of the biggest problems for EHR adoption is actually cultural – physicians, depending on their age, didn’t go to school to become a data entry clerk and I think they resent it. Physicians went to school to get into medicine, because they wanted to treat patients. The current generation of clinicians grew up using technology and devices, so it doesn’t seem to be such an issue. I also think that current EHR technology needs to evolve and become smarter.

While at HCT, we funded Dr. Richard Bergenstal’s ambulatory glucose profile (AGP) project, which is now moving forward and gaining device industry support. The AGP project is one example of preparing for automating data capture seamlessly and providing a standard to improve clinic visit workflow and time with patients. If we can integrate AGP with EHR technology and work on decision support tools, we should be able to increase the value of EHR systems and improve patient/physician interaction. A new group, Tidepool, led by Howard Look out in San Francisco, has made a loud entry in this space and brings a lot of energy and new expertise. They are moving quickly, and it will be interesting to see what they do with their platform.

KELLY: The T1D Exchange hypoglycemia data has become a classic slide. Did you expect to see hypoglycemia this frequent in people with type 1? How can you strengthen the data?

DAVID: I think the hypo data has been very eye opening to everyone, including us. When we presented the data to the FDA it was truly impactful. We now have proof that type 1 diabetes is not a safe and managed disease with an average A1c across our almost 27,000 people at 8.3%, with about 7% of them having a severe hypo within the last 12 months at the time of data collection. Clearly not safe and not achieving outcomes.

ADAM: What do you feel is the most key data point to set us up for success with the artificial pancreas, especially to help a lot of patients get access and reimbursement?

DANA: It has to be outcomes data. I think that we have to really start to push on new endpoints – glycemic variability and a better quality of life scale. I think the FDA appreciates the data and it helps support their decision process. But when it comes to payers, it’s all about data of keeping patients out of the emergency room. You need to prove that a little bit more money spent upfront will help overall healthcare costs. We just have to raise our efforts to meet the higher bar. And we can’t think of it as an afterthought – we can’t be naïve enough to create a system or create a product or create a device and not think of the end game: getting it reimbursed. I always think, “What is the irrefutable data that we need to capture over the next three to five years to prepare for these new therapies and devices?”

DAVID: What is key is to show that a device can eliminate a big chunk of severe hypos or improve people’s outcomes, which is measured by A1c right now and absence of complications – if you do that, you dramatically increase the chances that the payers will pay for it. Ultimately, it will be cheaper than paying for all the hospitalizations from severe hypos or complications stemming from poor glycemic control.

KELLY: Absolutely. When you talk about improving outcomes, do you mean showing fewer long-term complications?

DANA: I think outcomes is all inclusive. When I think about outcomes, I think about how people can live their lives as normally as possible while managing type 1 in a prudent way. Ultimately, that will allow them to avoid long-term complications and short-term severe hypos. A huge component that is often overlooked is quality of life. People who live with this disease don’t want to be defined by it.

KELLY: When it comes to quality of life or productivity measures, what can you do in the Exchange to make those much more real?

DANA: Our goal is to develop new data that is not only substantial, but irrefutable. If the data is ignored and we can’t find a path forward, we will have the evidence, and the patients and community can be a change agent: “Now we have something to get really pissed off about.” We would have something to show up in DC and stand outside and say, “We did this right every step of the way. We engaged all the stakeholders and we provided the data.” We did this in HIV decades ago and the time may come to take an aggressive stand in type 1 diabetes.

KELLY: What data are you going to be publishing soon coming out of the T1D Exchange?

DANA: I think the data from our study on C-peptide is interesting. Not only that some people still produce it, but then following them over time and tying it to phenotypic data. We’re hoping to see trends for what’s going on and see if it means anything. We’re also doing a really interesting study on hypoglycemia unawareness in older type 1 patients. It’s something we didn’t expect to be as bad, but the registry data showed that 15% of people 65 and over reported a severe hypoglycemia episode (seizure or loss or consciousness) in a one-year period. Many of these people are living alone and they have hypoglycemia unawareness; this is a huge unmet need.

KELLY: That could enable so much, especially with CMS and reimbursement for CGM in the elderly.

DANA: Right. Before, we just knew by our guts that these things were problems. But you can’t go to the FDA or CMS with a gut instinct. The power of the data is huge, and I think everyone is receptive to data and driven by data. But I worry about the future. Is the data going to be enough given the current economy, the healthcare crisis, and the cost of delivering healthcare? In public settings, payers, companies, and CMS say that we want better outcomes for less money. Those are homeruns.

ADAM: What strategies do you have to get people without a personal connection to T1D invested in the cause?

DANA: People like to see a return on their donations and see something tangible that is solving a problem or improving the lives of people, especially children with chronic disease. The Exchange and Glu are tangible resources that bring the community together around data and evidence. Patients have traditionally written checks and thought of research as something in a lab. We aim to change that and create awareness that they are central to solving type 1 diabetes. We need them to give their time for research by answering questions and participating in studies – especially observational studies over the next five years.

KELLY: How are you learning from the European national registries?

DANA: Dr. Roy Beck has collaborated with the German and Austrian Prospective Diabetes Follow-up Registry to compare pump use and glycemic control outcomes in young children <6 years of age. We have also established that there is interest from several countries in an international collaboration to compare glycemic control among people with type 1 diabetes. We hope that this initial collaboration will lead to further joint work with the aim of improving quality of care.

HANNAH: Biomarkers are getting more attention, but it seems to have been extremely challenging in type 1 diabetes. How wide do you cast the net, especially when you’re trying to think prospectively and aren’t even sure what your questions will be in the future? How do you balance the economics of looking at a lot of different potential human biomarkers with cost-effectiveness?

DANA: The great thing about the Exchange is that if you have samples, there are a lot of investigators with ideas for good studies, and some have money to do the studies. It’s not our job to execute all the biomarker studies, but rather to procure the samples and work with the best teams with the best ideas. We are looking forward to announcing the formal opening of the Biobank in spring 2014. We plan on working with the scientific community to define new biomarker studies and hope to work with the funding community to collaborate on targeted requests for application. The hope is to work with NIDDK, JDRF, ADA, Helmsley, and industry to leverage this important resource.

HANNAH: What do you hope T1D Exchange looks like in five or ten years? 

DANA: We want to integrate our registry data collection module with EMRs in as many clinic sites as possible so we can cost effectively include all patients in the registry – I would be very happy if we can grow the registry to 50,000 patients. We are also exploring how to work with the clinics to define metrics for measuring outcomes and develop a quality improvement program to measure and improve outcomes for our patients. We are very interested in exploring global opportunities and my hope is we will have sites outside the US that will collect similar data and provide locations for improving care and supporting clinical studies. Developing T1D Exchange as the network for accelerating new products and therapies would be a win-win for patients, clinicians, sponsors, and healthcare.

DAVID: If we can automate the data collection through EHRs I would hope we can double Dana’s 50,000-patient goal. T1D Exchange also has the patient community, Glu, which can answer questions in very short order and be an incredible asset to those living with type 1 and their caregivers.

ADAM: Now that T1D Exchange is its own non-profit organization, how do you think about long-term sustainability?

DANA: The sustainability plan for the Exchange comes from three areas. One is from industry, one is from philanthropy (e.g., JDRF, the Helmsley Charitable Trust, grants), and the last is general fundraising. It’s a crowded space, and our goal isn’t to compete against other organizations. We believe the unique nature of the Exchange will attract new resources for type 1 diabetes

KELLY: How far in the future have you modeled out for how much T1D Exchange will need?

DANA: We have planned for phase 2, which is through 2016. Right now, it costs about $12 million per year to run all components of the Exchange. Most of the cost is full time staff, clinical expertise, study design and management, supporting the clinic network and data collection. I’m really hoping we can get it down to $10 million per year through automating data collection over time.

Diabetes Technology

KELLY: The Helmsley Charitable Trust’s T1D Program has a major focus on technology. How did that start?

DAVID: It became clear to us early on that the cure wasn’t around the corner and we needed to provide better care for people today. It was also obvious to us that technology was the way to do so.

DANA: David drove the technology interest and he helped me understand the importance of devices. Whenever I came to New York for work, I’d stay at his house, which provided a real understanding of what it means to have a child with type 1 diabetes. The most surprising thing to me was the family impact of having a child with type 1 diabetes; not the impact on his daughter. But seeing all the devices, I still thought, “This is crazy.” This could be much better – the devices should be smarter, and if they were, they could actually relieve some of burden for families and people with type 1 diabetes. This is the future.

KELLY: You mentioned the biggest barrier to bringing better technology to market is industry investment. How can HCT help address commercial viability?

DAVID: The biggest barrier is that it doesn’t get the big industry dollars that a product needs to come to market. Industry doesn’t invest the big dollars because they don’t see the commercial viability of the products (i.e., where they can make a profit). It really goes back to the business model mentioned earlier. In my opinion the single biggest barrier to having better technologies on the market sooner is the number of end users on devices is not enough. The business model to commercialize a pump or a CGM is a very tough model and a dramatic increase in end users would attract more attention from big pharma.

DANA: I see three main barriers – first is development of the system assuming we have rock solid CGM options. Second, we need clinician and patient support and interest to create the market. Last talent and finances will be required to move this product through the process. I think HCT has done a great job of working with NIDDK, JDRF and the artificial pancreas community to make major investments from different positions. While I was at HCT, we funded early stage innovation to feed the pipeline, partnered with JDRF to leverage resources and expertise to move the field forward, and formed industry partnerships. Funding to date may be adequate, but this area will need major funding over the next five years. This will take a big push when the time is right.

KELLY: Lots of things need to go right. What is the biggest barrier to a future with automated insulin AND glucagon delivery?

DAVID: The absence of a commercially viable, dual-hormone pump. Despite what Tandem says, that’s a research project and not a commercialization project. The dual-hormone pump is a bigger problem than stabilizing glucagon. I think the stabilized glucagon is going to get going between Xeris and Latitude, and the other two big companies at play in this space.

ADAM: How can HCT help get more patients on diabetes technology? What are the biggest barriers?

DAVID: We need the help of the entire community and organizations like yours. I think a lot of people have checked out on this disease because they are tired of the noise of a cure coming in five years. We need a huge campaign that shows these new devices can actually ease the burden and make type 1 diabetes more manageable. The more people that use the devices, the more of an incentive there is for companies to innovate – the business model gets better with more end users.

DANA: I think change makes patients with diabetes anxious. Glycemic control is challenging and this is a 24/7 problem. Once patients have a routine that keeps them safe and provides some predictability, one day of anxiety about learning new methods is challenging, let alone a week or a month. I think we also need to work on awareness for clinicians, especially PCPs who see adult type 1s. If physicians aren’t educated and supporting we won’t make progress.

ADAM: You're working really hard on CGM/BGM data standardization with the International Diabetes Center – how is that going?

DAVID: It is going really well. The team at the International Diabetes Center is great. I think the challenge has been pushing the companies to open up the data so that we can get a standard report created regardless of which devices you are using. The analogy I have heard is that of an EKG, which gives a standard readout no matter where you are in the world. We need that type of report for glucose and insulin data.

KELLY: Moving back to global needs, including access to insulin, we know the HCT has been a long-time supporter of Life for a Child. How do you balance underserved populations vs. those who have access? And how do you weigh giving to organizations directly vs. influencing others?

DAVID: It is very tough. We do fund amazing organizations like Life for a Child and Ayuda, but we too have a limited budget that we review and try and stick to based on our programmatic areas and goals. It’s depressing to hear about areas of the world where people are dying due to lack of access to insulin.

Debates in Diabetes

ADAM: Glucagon vs. insulin-only closed-loop delivery?

DAVID: I believe in the dual hormone approach because I have seen a lot of Dr. Ed Damiano’s work and results, and it more closely mimics the body’s natural functions. As I have heard some say, insulin alone is like driving a car without the brake pedal.

DANA: I love the combination, but it’s early and we don’t have an approved stable glucagon. This will continue to be an interesting debate over the next five years.

KELLY: Will an artificial pancreas be approved in the US?

DAVID: As I said before, I do not believe a fully automated artificial pancreas is attainable. That being said, I do believe we can improve dramatically what is being done today. I don’t think you can ever completely remove the person with type 1 diabetes out of the equation. I believe the issues are more the companies than the FDA. We need the companies to aggressively step forward and commit all of their resources towards commercializing insulin automation. I think the companies could be doing more, but the business model isn’t such that the companies want to put big dollars to work in this area.

DANA: When it’s ready to go, I think it will be approved. My new worry is what is the cost, will payers cover the device, and how high will the financial burden be on patients?

HANNAH: How do you think about FDA in terms of roadblocks to new therapies for type 1 diabetes? 

DAVID: Industry is the biggest roadblock. For all the reasons I’ve mentioned, nobody in industry is pushing full speed ahead to automate insulin delivery.

DANA: Everyone needs to do his or her job. FDA’s job is to protect individuals by ensuring therapies are tested and safe. Our job is to make sure we have evidence to support the process – “irrefutable data.” I think our healthcare system is under financial pressure and payers have shared they are looking for superior solutions at no additional cost – they say, “no new money.”  This is going to be tough as we prepare to bring new solutions forward and the community needs to work together to prepare accordingly. Patients have to become aware that they will have a big role in the near future when challenges arise. I believe it’s time to ask payers for guidance documents so we have something to drive towards.

KELLY: Will glucose responsive insulin ever happen?

DAVID: I think if you told people with type 1 diabetes you could have a once-a-day injection that would normalize blood glucose and eliminate hypos, they would be all over it. That is the promise of glucose responsive insulin. It is in very early stages and has very long time before it is a therapy in people, if at all. I am not sure if it will ever happen; give me a crystal ball and I will let you know. If we knew the steps to take to make this a reality, it would have happened a while ago; it is not that easy.

DANA: God I hope so! I agree with David, this would be an amazing breakthrough for patients with diabetes.

ADAM: Type 1 diabetes prevention vs. cure?

DAVID: I am not a “cure” guy. As a society, we have never reversed a chronic disease. If you look at history, we have eradicated some diseases via vaccination or prevention (e.g., polio). Although we do and will continue to do cure-based research, we have started to put a focus on prevention. As a parent of a child with type 1 diabetes, I hope that I am wrong. But I would bet a lot of dollars that a cure will not happen, and if it does, it isn’t even on the horizon now. Our major focus is giving people with type 1 diabetes better tools to manage their disease, so when there are therapies that come, they will be healthy enough to receive them.

DANA: With the right work over the next decade, we could see potential cures in decades to come, depending on your cure definition. It will be more than likely easier to ultimately prevent type 1 diabetes, but prevention is a tough conversation because our donor community lives with the disease today. Type 1 diabetes is time consuming and a burden – patients need solutions now! I think we have new tools and resources to better characterize patients at risk and learn how the disease develops, why some people develop disease, and what protects those that don’t get type 1 diabetes. My guess is there is a lot of work in the at-risk population that has to happen first before we will be able to move forward more effectively.

Closing Thoughts

KELLY: Dana, will you stay in type 1 diabetes for another decade?

DANA: I think it’s going to be a very good decade for us. I wake up every day excited about the willingness of our community to work together – from the government, industry and the funding community, to families and individuals. Automating insulin delivery requires technology. How fortunate are we that we’re in the best era of technology development? There’s no doubt in my mind – we will be successful in type 1 diabetes. Now we have the Exchange and the data. All the pieces are coming together. I think it’s going to be a decade where we see less burden and less worry for people with type 1 diabetes. I think we’re sitting on the cusp – we’re armed with all the tools and it’s like we’ve broken ground. We have the foundation of the house, and now we have to figure out what type of house we want and what’s on the first floor. I’m hooked. All of the seeds I’ve planted for over a decade are finally moving. I think it’s going to be an awesome decade.

KELLY: David, is there a finish line in your mind? What does it look like for you and the T1D Program?

DAVID: The finish line for me would be to try and reverse or prevent type 1 diabetes and make it so that future generations don’t have to worry about the disease at all. Recognizing that is a long way off, I would settle for getting tools to people with T1D that significantly ease the burden of managing this disease. My immediate aspiration is to try to automate basal rates overnight, so that people can sleep through the night without fear and without running high to avoid severe hypos. I would like to take nighttime from a period of extreme worry to a period of pristine blood glucose control. I wholeheartedly believe that this is an attainable goal that would have an absolutely dramatic effect people’s outcomes.

KELLY: What do you want the legacy of HCT to be?

DAVID: I want the legacy to be similar to that of the Howard Hughes Foundation. A grant from them is extremely prestigious – it’s one of the most well respected non-profits out there – and immediately signals that the work being supported is important and worthwhile. I want it to mean the same thing when you get a grant from HCT. And most of all, I would like the Trust defined by the positive impact of what we have helped create. The legacy of my grandmother is going to be what it’s going to be. But the legacy of the Helmsley Charitable Trust should be influenced based on what we do, not based on stories. What our grantees have achieved should be the story.

As far as the T1D program, I want the legacy to be that we have put our hearts and souls into easing the burden of managing this disease. I want to be part of the solution that makes this disease easily treatable and keeps people healthy while we wait for that elusive cure.

DANA: I hope the HCT legacy becomes a place where passion meets strategic execution. In the very near future, I hope patients with diabetes will have a product in their hands that has been touched by HCT and co-created with the community.

Appendix

The David and Dana Story

KELLY: How did the two of you meet?

DANA: In the spring of 2008, I was winding down my work at the Iacocca Foundation. At 84 years old, Lee decided he was finally going to retire. It was a difficult time for me – Lee was my mentor and a true motivator and disruptive force in type 1 diabetes. I was in the process of downsizing the Foundation operations and working with the Iacocca family to figure out what the next generation of the Iacocca Foundation would look like.

DAVID: At that time, I had watched a YouTube video of Charlie Rose interviewing Lee Iacocca. At the end of the interview, Lee Iacocca was talking about a foundation for type 1 diabetes. That night, I wrote Lee a letter and asked him for help. That letter landed on Dana’s desk. I wanted to meet with Lee and ask him for advice on how to best invest in type 1 diabetes research – how was he going to cure diabetes? I followed up and called Dana.

KELLY: What was the phone call like?

DANA: It was an interesting first call because this was all new to David and it was clear he was on a mission. His daughter had only been diagnosed for a short amount of time – about a year. And you know David today: he’s tenacious, he’s driven, he’s in a hurry, and he wants to get things done. He really wanted to meet with Lee and he had started the process of asking everyone working in type 1 what they thought was the right solution for the Helmsley Charitable Trust program.

It ended up being a two-hour conversation. David told me all about his daughter, Morgan – when she was diagnosed and how she was doing. At the end of the phone call, I said, “David, this isn’t easy. It’s a very complicated disease and welcome to the drug discovery business.”

KELLY: After the call, did you and David meet with Lee?

DAVID: Yes. We met with Lee Iacocca in April or May of 2008. I remember it being really hot because I was wearing a suit, and I remember sweating as I was walking over there. I remember thinking that I was on my way to meet a legend; Lee is an icon. Dana met me in the lobby and we went up to Lee’s apartment. He opened the door and put his arm around me and said, “Come on in and let’s talk about your daughter.”

DANA: David told him all about Morgan and explained that he was a trustee of his grandmother’s Charitable Trust and he wanted to do something in diabetes. And Lee said, “Well, I think you should have Dana take you on a tour and give you an education about diabetes.” And so we planned a trip to the West Coast where we went from San Diego to San Francisco. I think we had 13 site visits over five days. We went from a small lab working in biomaterials to a basic research lab, stopped at Peter Butler’s Lab in LA, and met with Dr. Anne Peters. One of the stops in LA was also with the Milken Institute, looking at best practices and models in philanthropy. Then we worked our way up to San Francisco and went to UCSF and Bayhill. I wanted to give David a tour that ranged from basic research on the drug side, to devices, to biomarkers and biomaterials in the academic setting, to a small biotech company.

DAVID: An encounter with Anne Peters was one I’ll never forget. We bumped into her at the airport just before our flight from LA to San Francisco. Anne changed her seat so that she was next to me, and handed me a sheet of paper with about 15 names (I still have that sheet of paper in my desk). She said, “These are the people you need to contact to do your diligence.”

KELLY: And this was just a five-day trip in 2008?

DANA: Yes. We started the week as strangers and ended it like brothers.

KELLY: Incredible. What was your impression of each other after that trip?

DANA: David’s an amazing human being who just wants to help people with diabetes. He doesn’t care who has the answer. He doesn’t care how. He just wants to get it done. He has no ego. He’s compassionate, he’s caring, he’s dedicated, and he works around the clock. He never stops, even from the first time I met him and he was like a sponge. As I was teaching him, I couldn’t get the words out of my mouth fast enough that he was asking the next question. David was a bull in a china shop on that trip. He was very funny, but in the kindest way. He was just tenacious. He wanted to know everything.

DAVID: I pride myself on what I call my gut instinct. It was very clear to me after spending a week with Dana that he was an incredibly smart, genuine person who is also a visionary. I knew this was someone that I wanted to work with long term. Most importantly Dana had tons of experience in the very field I was trying to impact. Dana doesn’t like to admit this, but he is just an older, mellower version of me. I am completely convinced that Dana and I meeting was the work of a higher authority. We were meant to work together.

ADAM: Besides becoming closer, what was the biggest learning from that trip?

DANA: Showing David the diversity of just how complicated the problem was. I think that journey also showed him how you could spend a lot of money really quickly – there’s so much to try to develop for type 1 diabetes, which is a big part of our problem. We’re not like cystic fibrosis, where we have one gene and we’re developing drugs. It’s not even like multiple myeloma, which is screening drugs and bringing them to the clinic and seeing whether personalized medicine approaches are going to work or not. We’re trying to develop an insulin delivery, glucose regulating automated system to strap on to a human being. We’re trying to develop autoimmune therapies. We’re trying to develop cells. It’s not like any other disease. I think that first trip and all those little site visits were really important – I wanted to overwhelm him a little bit.

DAVID: For me it was really the beginning of my education. I had not yet started my diligence on how to have an impact when I met Dana. I was completely naïve. I knew I had to care for a child with type 1 diabetes, but had no clue how to get a drug or a device to market. At the beginning I just thought money could solve the problem. It just isn’t that simple. If we knew what to do, we would be done. I really had my eyes opened on this first trip.

Q: Dana, once that trip was over, how did you end up working with David and the Trust?

DANA: At the end of that trip, David and I got on the plane and David said, “I’d love to have you come work with me.” And I said, “I hope I’ve been able to help you, but I’m not interested.” I actually wanted to do something else. I didn’t know exactly what I was going to do next but I actually didn’t think that that the type 1 diabetes community would ever come together. And it was frustrating. It was challenging to work because it was very much siloed. Part of my goal of helping David in the beginning was to teach him everything I learned over my seven years working for the Iacocca Foundation. At the end of the trip, we said goodbye. He said, “I’d love to talk more.” I said, “Sorry, I have a job. Thank you very much. You know that I’m happy to answer questions.” And that was it.

KELLY CLOSE: David, what did you do at that point?

DAVID: Dana had expressed a desire to do something other than type 1 diabetes, and I respected that view. But I was also very open to trying a new way of doing things. Dana said that I could reach back out to him with follow up questions, which there were many. The relationship continued to grow through these conversations. Once I knew Dana had left Iacocca, I reached out to him with a plea to help me devise and implement a plan. I asked him to consult for the Trust and convinced him to give me a year. Dana agreed!

KELLY: Dana, was it hard to resist working with him?

DANA: He never hounded me about it. He respected that one time when I said, “No, I can’t do that.” And then six months later, September of 2008, is when I left the Iacocca Foundation. David convinced me to consult and I started coming to New York and consulting with him on doing the landscape and strategic plan for the T1D program that fall. We continued the site visits and we met with the government, the FDA, and we started meeting regularly with JDRF. For close to a whole year, we continued the due diligence period to really understand what was the highest and best use of Helmsley funding in this ecosystem of type 1 diabetes.

And then in the spring of 2009, we sat down and said, “Okay, what is the type 1 diabetes program going to be?” There were a lot of things that were happening at that same time, and the roadmap became a bit of a moving target. We were waiting for anti-CD3 results. I was trying to get my head around the device space. We knew healthcare changes were coming. There were a lot of moving parts when we put together the program in 2009. We decided that it needed to have a strong technology program, a very targeted program on beta cell development, the outreach program to help advocates (e.g., camps, community support), and then the systems part of the program, which is how we funded the T1D Exchange.

KELLY: When you look back on the evolution of your relationship – from that first meeting to where things are today – how would you summarize it?

DANA: I think the most important part of the David and Dana story is that it’s about two people coming together – each bringing important history and important resources – to create this program at the Helmsley Charitable Trust.

For me, it was my experience as part of HIV/AIDS community and the story of how HIV showed up, could be controlled in only 13 years, and how patients and the community could demand better solutions. I brought this experience to the Iacocca Foundation, but had almost given up on type 1. For David, it was about the passion of a dedicated parent who stopped everything in his life when his child was diagnosed with type 1 diabetes. He put everything down that he cared about, and because of this important opportunity related to the Helmsley Charitable Trust, rolled up his sleeves. He traveled nonstop and was open to listening and learning. Never once did I make a decision that he said, “No, I want to do this.”

We were true, trusted partners from the beginning, and it was based on evidence. It’s based on “What’s the government funding?” “What does JDRF need?” “What does the community need?” and “What do the companies need?” It had to be an ecosystem approach where we only had one goal – to find the best and brightest people to answer the questions and develop more evidence to move on to the next question.

ADAM: David, what does your average day look like? Dana, what does your average day look like?

DAVID: Wake up at 5 am, get on the train at 5:40 am. I go through emails and read any documents I haven’t gotten through from the night before. I go to the gym from 6:45 to about 8:15 am. After I get to office, I have various meetings and calls during the day. I do spend part of my time on estate matters, as I am an executor of the Helmsley estate. I have never had less free time in my life. I try and leave the office a little after five so I can catch the train home. I work for the hour commute on my laptop doing emails and reading. I get home around 6:30 pm so I can eat dinner with my family. I travel a decent amount, so when I am in NY, I make it my business to get home to spend time with my wife and children. After putting the kids to bed and spending some time with my wife, I generally check emails again, do any reading I have not gotten through, and go to bed. I wake up two times per night to test my daughter to make sure she is ok – nighttime is the easiest time to get good control, as there are no meals to deal with and no residual insulin from those meals.

DANA: Every day starts early, which is when it’s quiet enough to read emails and try to write what I’m supposed to be writing. Now that I’m back in Boston, I get to walk to work, which is always a great way to kick off the day. Each week is filled with a mix of internal staff meetings for organizational needs, program development and progress, Glu strategy and events, and calls/meetings with T1D Exchange non-profit and industry members around developing projects. I spend a lot of time with Drs. Roy Beck and Carla Greenbaum working on clinical and research strategies and opportunities. We discuss what projects the T1D Exchange is uniquely positioned to support and what resources and partners we will need in the future to accelerate research and development projects. I get a couple hours of quiet after my staff leaves, which is when there is usually time to catch up on work and try to get out by 7 pm so I can attempt to balance home and work – I’m told I’m failing the balance part.

Partnering with JDRF and ADA

KELLY: JDRF is responsible for raising a lot of money every year. And it is symbiotic; obviously they’re raising more money because of what you’re doing. Can you tell us what it’s like to work together?

DANA: When I worked at HCT, we had quarterly strategic meetings with JDRF from the beginning. We wanted to be efficient with every dollar we had and we wanted to make an impact just as soon as possible. When David and I met with JDRF to identify our first grant, we met Jeffrey Brewer and Dr. Aaron Kowalski. We had a specific meeting on how to improve technology and devices. Jeffrey Brewer was a board member and a volunteer at the time, working closely with Aaron on their artificial pancreas program. When we walked out of the meeting, I said to David, “We need to hire Aaron a whole team of people to build the program that he wants to do.” And so that was our first grant to JDRF back in 2009 – that allowed them to hire five people to build their artificial pancreas team, to be able to do the diligence, build the landscape, the roadmap, and create the artificial pancreas program. It’s a good example of using philanthropic dollars not to fund a research project, but to look at a problem and say, “This is how we could help JDRF.” That grant was about $2.5 million – all personnel and travel. And for us, because we were starting this organization, we created a collaboration with JDRF leveraging their expertise instead of hiring a team of consultants – it was a win-win for both organizations. That first grant and that investment for the artificial pancreas team provided so much return.

KELLY: How do JDRF and the Helmsley Charitable Trust leverage each other’s resources?

DAVID: We have a very close relationship with JDRF. After the NIH, JDRF is the largest funder in the space and we share everything with them. We will partner with any organization if we think they give us the best chance at achieving our goals. We are completely agnostic as to who our partner is as long as our goals are aligned.

DANA: While I was at the HCT we developed a collaborative approach to funding and almost every grant that we award is a directed grant. As JDRF and HCT identify funding opportunities, we work closely to leverage each other’s interests when aligned. Since forming our collaboration in 2009, we have jointly funded 11 projects, totaling more than $60 million.

ADAM: How do you see the collaboration between HCT, the T1D Exchange, and JDRF evolving in the new partnership with PureTech?

DANA: T1D Exchange will bring our scientific, clinical, and patient engagement capabilities to the T1D Innovations collaboration.

DAVID: HCT isn’t involved with the PureTech/JDRF T1D Innovations Fund. It’s still very early in the collaboration and I expect we’ll all use our resources depending on the stage of the technology and figure out who has the right resources at the right time. If someone develops intellectual property that is really interesting, the T1D Innovations Fund is an innovative resource to think about the development and commercial pathway. We think it’s really important to start with the beginning in mind. We also have to keep in mind, “How many patients are going to want this?” Not only does it have to be effective, but is the market there?

KELLY: Have you had people saying, “Can I just add my money to the Helmsley Charitable Trust and know that my money is going to go toward type 1 research?”

DAVID: It’s interesting you say that. The short answer is that yes, people have asked. We don’t have a mechanism – we can’t take money in. We can give money out. We’ve had folks talk about partnering with us and putting their money alongside of us, but letting the Trust do the diligence towards the work. We are exploring different avenues of being able to partner with people.

KELLY: And can you talk a bit about your relationship with ADA?

DANA: When David and I first started to work in the community, we collaborated with a group of type 1 funders and created an informal group called the T1D Alliance. The group includes JDRF, Helmsley, ADA, DRI and several private funders. The group continues to meet annually to understand who’s doing what, who’s funding what, and where the gaps are. I started my relationship with the ADA through these meetings and now work with them through T1D Exchange. They continue to be a fantastic collaborator.

DAVID: We have done several projects with the ADA. Helmsley funded the prelude to revising the Standards of Care, and the sourcebook just came out late last year. We identified a huge gap in the Standards of Care for people with type 1 diabetes, and we approached ADA to understand what was involved with changing the Standards of Care. Drs. Anne Peters and Lori Laffel led this project, and now we’re already working on the next update. The Standards of Care were far from where they should have been. We pushed really hard and then we met in the middle and said, “Well let’s get this far in phase one and move them forward in phase two.” So as much as we work with JDRF on the research side, both with the Exchange and Helmsley, I think we are developing additional relationships with ADA on the clinical side.

KELLY: What did you want to push more that wasn’t included in the guidelines?

DAVID: I think it’s important to say that we identified an issue with the Standards of Care, and correcting the problem was a joint effort where Helmsley funded it, but JDRF and ADA collaborated along with many leading clinicians. It’s a good example of a collaborative project where several stakeholders were working together.

KELLY: How will the Standards of Care be rolled out? There is a greater focus on individualization of medicine, but providers often don’t know how to do it in practice.

DANA: I think standards in writing are very different from adoption, and it’s going to be challenging – there are disparities of care and physicians that see type 1 patients, especially depending on demographic and geographic factors. I think it’s easier to set the standards; it’s harder to deliver a plan for adoption.

KELLY: Whose job is that?

DANA: I think it’s the professional organizations collectively who should all be supporting and driving the standards. They should also streamline the process of the translation of abstracts and presentations and publications to clinical care. I worry a little bit about that; we know the data, but whose job is it to really translate that and then deliver it from a healthcare delivery best practice standpoint?

KELLY: With the HIV movement, it really helped that there was largely one actor that was able to coordinate all the actions. In diabetes, it’s more fragmented. Would the type 1 diabetes movement do better if the ADA was solely focused on type 2 diabetes so that JDRF could act as a sole player?

DANA: I think dividing and conquering is a very good thing in really defining who’s best suited to focus and commit to certain parts of the process. I believe the two organizations have figured out how to collaborate around their respective areas of expertise in type 1 and both play unique and special roles. If you ask JDRF if they want to run a conference and take over publications, they’re not interested in that because ADA does an amazing job. I don’t think this is something we have to worry about because there is communication and there are regular meetings with NIDDK, ADA, JDRF, and the Helmsley Charitable Trust. I think over time, if we continue down this path, there will be more collaborations.

DAVID: As Dana notes, we do collaborate with both JDRF and ADA and will continue to do so any time we think they are the best option to get us to our goal. At Helmsley we are very fortunate to be in a position where we do not have to fundraise.

KELLY: Which of the professional organizations is the most impressive to you?

DANA: Honestly, I haven’t worked with them enough to really make a judgment. My general feeling is that there’s a lack of innovation in the professional organizations. I don’t know if it’s because they don’t have the funding or they don’t have the vision. I don’t know if they’re older organizations that are still operating based on a dated healthcare system and career trajectory.

DAVID: Different times call for a different approach. The status quo isn’t acceptable and we need to be prepared to step out of the norms and try something different.

KELLY: How do you feel about the leadership at ADA? Being the Chief Scientific Officer there is a hard job. Do you think that Dr. Bob Ratner is doing a lot of good things for type 1? He was certainly a very good clinical trialist.

DANA: I like Bob a lot. The current leadership is engaged with the T1D Exchange and having conversations with us around how to leverage the evidence and the data. They like the quality of what we’ve created – “Before T1D Exchange we couldn’t have this conversation. Now we can.”

I think it’s all about what data and evidence can do for our community and these organizations. It gives them the evidence to really change the way they think. Once you can see the data and can start cutting and slicing the data, the picture becomes clearer. Then suddenly people really say, “There’s an opportunity here to do this, where before we couldn’t see that.”

A Biological Cure and Prevention of Type 1 Diabetes

KELLY: How many different cure approaches will you be satisfied with? Not every patient would probably be able to use an automated insulin delivery system.

DANA: I think about tailored cures – because of age, duration of diabetes, and stages of life, you need different cures for the burden of type 1 diabetes. At different times where the risk-benefit may be there, you can do something big and bold. Other times you are treating children and adolescents and pregnant women. It has to be multiple cures and it has to be multidimensional treatment and cures.

HANNAH: What is acceptable risk to cure type 1 diabetes? 

DANA: Investing in type 1 diabetes has been very risky and there have been many expensive programs that have failed in the clinic. I like the concept of informed risk – we have many new resources like the T1D Exchange that are developing strong evidence to support the development and translation process. I think we need to work smart, use well characterized patients for studies, and collect as much data over time on a large primary cohort. Over time the data will have more utility. I believe this approach will allow us to reduce risk and support an incremental approach to testing and validating new therapies. Improving efficiency and efficacy will increase odds of success.

DAVID: I believe it is philanthropy’s role to fund some of the riskier projects that others won’t fund. We can fund very long term projects that are not all that appealing to a lot of funders. Like everyone else, I want a solution now, but taking that short-term view hasn’t yielded a whole lot. We are trying to look at things a little differently.

KELLY: Do you think all the progress on the automated insulin delivery front makes the biological cure researchers nervous?

DANA: I think there will be disruptive solutions that have real utility to patients and they will decide what they want. When it happens, we’ll celebrate each one.

DAVID: I don’t think there are many people today that would say you are cured if you can automate insulin delivery via technology. In my mind, beginning to automate insulin delivery is the way to keep people healthy while the biological researcher chases the elusive cure.

KELLY: You mentioned you funded Dr. Faustman’s work at the Iacocca Foundation; how do you feel about her work?

DANA: We didn’t support Dr. Faustman’s work while I was at the Trust. The primary reason is because she’s very well-funded.

KELLY: How much funding does she have?

DANA: My recollection is around $20 million for basic research and phase 1 of the BCG study. She’s well-funded and has the ability to raise a lot of money on her own. She has a huge following.

KELLY: Can you say to what degree you are philosophically supportive and optimistic about what Dr. Faustman and other prominent researchers are doing?

DANA: Philosophically yes, for Dr. Faustman. I’m in support of the process she created to understand the cause of diabetes and the patients – she used her data and biosample collection. I think she is absolutely thinking the right way. I don’t think we have the evidence, and I question if we will have the evidence, that BCG has any clinical effectiveness in type 1 patients. I think that it’s taking a very long time to get this amount of evidence, and the world has changed a great deal in that very long runway. It’s complemented by what we learned from anti-CD3 and the economics of bringing therapies to the market for type 1 diabetes.

KELLY: What was your reaction to anti-CD3 and what do you think about it today in terms of prevention? It’s still very controversial.

DANA: The dosing was complicated and it seems like too big of a hammer. And it was too broad and not specific enough. We didn’t fund any of the anti-CD3 work at the Iacocca Foundation. It seemed like too much risk. There was also not enough funding to do it, so we didn’t participate.

By 2006 or 2007, I was then saying, “Autoimmunity – is that all?” Even if you have drugs to suppress autoimmunity in patients with type 1 diabetes, these cells are not magically going to regenerate. By then, I started to shift towards thinking that this is not a one-dimensional disease. I think it’s multiple pathways, and I think it’s multiple processes. It may be that these single approaches are not going to work. It was kind of going back to HIV – it wasn’t until we started to think about multiple drug combinations that we made progress.

ADAM: We’ve been hearing about the need for multiple drugs for quite some time. Has that moved fast enough?

DANA: I don’t think it’s moved much. That’s part of why I wanted the Exchange. We need to study a large cohort of people and take them apart biologically over time to really understand what’s causing this disease. That will allow us to work smart. At this point, I don’t think we know enough. We keep trotting down the same path doing what we’ve been doing as long as I’ve been in type 1 diabetes, and that’s why the Exchange is so important.

KELLY: So some of this is on ice until you figure out more?

DANA: I think we want to work smart, which means let’s take a step back and really look at what’s available today that may have clinical effectiveness. We will continue to have more evidence to guide research strategies in type 1 diabetes and our goal at the Exchange is create an open resource that quickly tells a story: “This is what’s happening in patients.” Here are samples that we have from well-characterized patients. Let’s glean information about what’s happening in patients and use that to guide a logical, incremental program of testing drugs.

HANNAH: There was some desire to fund another anti-CD3 trial – did you consider it?

DAVID: I can only tell you that we took a look at this and decided not to fund it. I believe they need an industry partner who is committed to bring this to the market, and to my knowledge, that partner doesn’t exist today.

KELLY: Aside from curing type 1, what about prevention? That is very relevant to your other daughter, Caroline, right?

DAVID: Yes. She knows she’s at high risk. She was always asking me, “Dad, do you think I’m going to get diabetes?” I would say, “I hope not.” When George Eisenbarth was still around, he tested her blood outside the TrialNet lab. She has a protective allele – one of the two. It’s the lesser of the two, according to George. She now has her own meter, and we test her fasting glucose about once every two weeks.

KELLY: How is HCT thinking about prevention of type 1 diabetes?

DAVID: We recently hired a consultant to do a landscape for us on prevention. Prevention is big right now. We’ve never successfully reversed a chronic disease. I wouldn't bet that we’re going to do type 1 anytime soon. We’ve decided that we are not going keep on throwing darts at the wall when we don’t even know what causes the disease. We might want to start thinking about prevention vs. reversal. If we can reverse it, we can help quite a few people, but if we can prevent it, the potential for help is essentially infinite. This wouldn’t necessarily help my daughter, but what about her children and future generations?

We want to look at steps that other funders wouldn’t ordinarily consider. Take the T1D Exchange, for instance. A GRAS study – generally regarded as safe (e.g., Vitamin D) – is going to be huge and cost a boatload of money. It might be best to give everybody everything and see if there’s efficacy. Then you back stuff off. We have a great chance through the Exchange.

Dana's Early Experience in HIV/AIDS

KELLY: Dana, you have had an amazing career in advocacy that started with the AIDS movement. Can you tell us more about that?

DANA: I’m an enigma even to myself. Most important, I grew up in poverty in a highly dysfunctional family in Southern Maine where no one had a college education and most people did not do well. But somehow, I knew I was different and I knew by the time I was six or seven that I had to get out of that family because I saw the world in a very different way.

And so from a very early age, I saw windows and not walls. I think this is what makes me different – it served me well in the HIV/AIDS era and in type 1 diabetes with the Exchange, it’s because of this, I see opportunities and I see solutions. That started as a small child. I left my family when I was 14. I wanted to be a horse trainer and I put my first deal together to live with a family that had horses. By the time I was 15, I was traveling to major horse shows and was riding other people’s horses. By the time I was 18, I was determined to be a horse trainer and only went to school for two years for accounting.

Fast forward - I was 23 years old and I had a nice life in Southern New Hampshire, a farm, and show horses. I will never forget the day a friend of mine said, “Have you read this?” and handed me a Newsweek magazine. It was about HIV. I put down that magazine and I thought, “I must have this virus. I will be dead in a few years. You can’t test for it and there’s nothing you can do.” And the whole world changed. And so I did what everyone else does: I became a patient advocate.

A community started to come together. We started to have meetings, we started to create awareness, and we started to tell people that there was something out there but we didn’t know what it was. And you literally couldn’t do anything about it. You just knew something was happening and people were dying and there was nothing you could do. So it became about awareness first and telling the story and convening. That happened in home meetings, that happened in public settings, in the communities, and we started to educate people that this existed and we didn’t know what was happening.

And at the same time around the country, people started to create ACT UP. I wasn’t on the frontline of ACT UP, but I was behind the scenes. We all started fundraising and getting involved with the patient advocacy programs.

In 1987, I think the first assay was developed so you could get tested. This is what it was like. You went to a family planning center, they gave you a secret number – the number came on a piece of paper, and you never gave your name because you could be denied health insurance and the world discriminated against people at that point with HIV. They sent your blood away to a secret place and three weeks later, they gave you a phone call and they said, “Come in.” You sat down and they’d match your number to the number on the piece of paper. Then, they told you whether you were positive or negative.

And I will never forget that day, because one friend had already died and I learned I was negative. The first thing I did after I got over the shock was to question how could this be? “We all did the same thing as kids, right? Why not me and why them?” And that’s also important, because it started with me asking and thinking about that. We’re not all the same. Disease is not the same. Why do some people get it and why do some people not get it? And so that’s really what started this journey.

Fast forward to 1990 – in seven years, some of my close friends had died and many were HIV positive and getting sick. At that time, there was a big push for hospice care; there was no place for these people to die. Research was moving forward. A lot of the ACT UP community was out demanding solutions, fighting for rights, fighting for access to whatever therapies existed.

When I went to Boston, I went to get involved with the HIV/AIDS community and was very interested in research. I met my partner in 1990. He was HIV positive and died in 1995, one year before the cocktail came up. So it went from being intimately involved with a disease that you don’t know anything about to a loved one who you’re trying to keep alive. I was fighting for every potential therapy to test to see if it would work. At the end of his life in 1995, this included microchips that had been available in France. They were using ganciclovir, a drug that had to be delivered by IV to deal with MAC. A nasty side effect was seizures. My partner had a grand mal seizure, and at the time, I wondered if there was a better way to deliver the drug. Turns out, there were microchips in Paris developed to deliver the drug in the eye. I said, “Can we get them to the US and use them on Nick?”

And that’s kind of my first experience with medical technology. Again, I never saw a wall when they said, “No, you can’t. You can’t do this or you can’t do that.” I kept saying, “Well, there has to be a solution. There has to be a way to solve this problem.” My partner, Nick, was the first person to have ganciclovir microchips in the US. The microchips worked and he passed away with his eyesight.

Then in 1996, the cocktail came and it was remarkable. This was just 13 years from 1983 to 1996, from an unknown disease to the cocktail going into the patient population. It was a disturbing time, as many patients were very sick and people were dying. I knew many people who had less than 100 T-cells, they had a viral load through the roof, and they had miserable diseases that came along with HIV. Patients were diagnosed with full blown AIDS when their T-cells dropped below 200, and we started to see pneumonia, PCP, Kaposi’s, MAC, and all of these funky diseases. These beautiful young people were dying. They got very thin and it was a horrible death. What was remarkable was when the cocktail came to market, these people started gaining weight and it all went away.

ADAM: It’s like when insulin came out in 1922…

DANA: It just stopped, right? And it’s unbelievable to think in 13 years, we went from a potential pandemic to a controlled chronic disease and most of those people are doing well today. I say this whenever I talk to someone, “If I had to choose today between HIV and type 1 diabetes, I would choose HIV because it’s two pills at bed time and you can live your life, no testing, monitoring blood sugars, or dosing insulin 24/7/365.”

KELLY: How do you look back at being really involved in raising money and advocating? How easy or hard was it to be raising money, given the discrimination and the stigma?

DANA: Whether it’s type 1 diabetes, HIV, or any other disease, there’s the same psychological process that people go through and there are stigmas. There’s the initial shock of the diagnosis and then it’s somehow trying to learn everything you can about how you’re going to deal with it. You eventually get through the process to acceptance. You manage disease.

I think you have to really appreciate that the person who gets a disease is still that person, whether you’re a professional runner or a pianist or an engineer. You just also happen to have HIV or cancer or type 1 diabetes and you learn to accept it. I think most people get to acceptance, but you also have to have hope – I think hope is part of medicine. It helps keep you going. It gives you the ability on those down days to hopefully find a place that says, “There’s people working on this problem and I can be part of the solution.”

Although there was clearly fear and stigma about HIV/AIDS, there was also an engaged patient and supporter community where people were really committed. They gave their time, they gave their money, they quit their jobs, they lit things on fire, they marched on the NIH, they demanded better solutions, and they took care of each other. And it was tough because there was a lot of shame, there was a lot of guilt, and there was a lot of anxiety about the disease – who had it and who didn’t have it. There was a time when someone suddenly had the “look” – he became very thin. You didn’t have to guess anymore. You suddenly went from a person on the street to someone with AIDS and it was no longer a secret. As time went on, it became easier to raise money and once the face of AIDS was in the public and it went from an AIDS patient to a son dying with the disease, the community was raising a lot of money. I guess the personalization of the disease and the impact to the family is really when awareness and funding improved.

What Can Diabetes Advocates Learn From the HIV/AIDS Movement?

KELLY: What do you think that all of us as diabetes advocates can learn from the HIV/AIDS movement?

DANA: With HIV, it’s easy to romanticize about something that happened a long time ago. I think it was a once-in-a-lifetime phenomenon. Gay men were just starting to be accepted. They no longer feared losing their jobs as much as in the early 1980s. There were a couple gay characters on television. The gay pride movement was just starting, but growing quickly. It wasn’t the days of getting your head bashed or you died because you were gay – at least in most urban areas. The community was really starting to come together and there was more acceptance and less fear. It was an important time in the history of the gay community. The disease came and they got really mad. It was also gay men – they didn’t have children and they didn’t have families. This disease came and started killing them at a critical time when suddenly they thought the future was very bright. They were well-educated, they were successful people, and they got really angry and they didn’t have to worry about family constraints; there were no children and they were freer to get involved and demand action. I think getting involved and demanding action is the common thread for diabetes advocates – type 1 diabetes can’t be a spectator disease.

KELLY: So was the AIDS movement just unique? Or are there any lessons to be learned that we can apply to diabetes? People say to me, “Why aren’t you guys marching on Washington?” In reality, it’s a lot tougher. It’s really hard just to get people to sign petitions. We are so far from that!

DANA: It was the 1980s and 1990s. It really was a unique situation. I mean, if it happened a decade later, I don’t think there would have been as big a response. It was just the perfect storm – acceptance was slowly increasing and the gay community saw that this was going to push them back in the closet. They said, “This is not okay. We need to deal with this and no one is going to do this but us.” With diabetes, there are other people that kind of do things for the community. I’m afraid our constituents may think advocacy is someone else’s job. The world has changed a lot since the 1980s. I think it was about survival as a community and the disease became the mechanism to activate the community.

KELLY: I almost think that there’s that urgency happening in diabetes, but it’s just behind closed doors. Children are dying, adults are dying, and we have government institutions that think diabetes is not a dangerous disease. It is a dangerous disease, not exactly in the same way, but maybe there are some parallels. If we need some urgency, perhaps it makes sense to share data from the T1D Exchange on things like death from severe hypoglycemia.

DANA: I don’t think that the severe hypoglycemia death data is enough alone. You can’t compare HIV/AIDS and death without therapy to anything, including diabetes. It’s impossible. With AIDS, it was about a community going backwards. It was also an important time in the history of the gay community – Rock Hudson and celebrities had the virus and they suddenly came out. The government didn’t care about this population. The country didn’t care about the population. No one was helping them, so they lit the world on fire. Once we had drugs, we started to put them into people. When we started to see there was efficacy, we kept fighting. We were relentless about getting them moving forward, getting the companies involved, and getting the NIH involved to support the studies and test the therapies. Eventually, we ended up with a cocktail.

DAVID: I agree that there isn’t a sense of urgency amongst those with type 1 diabetes. It seems like those with the sense of urgency are the parents of children living with type 1. I agree with what Dana says about comparing type 1 to HIV/AIDS, but the fact is we need to do more about the perception that type 1 is a safe and managed disease – because it is not. We have a large population of people who have type 1 diabetes who are no longer engaged in their own disease. I can’t tell you how many adults with type 1 say they were told there would be a cure in five years. This kind of hype has caused people to tune things out, and in my opinion, to their own detriment. A lot of people just do what they do to get by, paying very little attention to new tools and devices that could make their lives easier. We need to figure out how to re-engage these people and get them to try a pump or CGM or smart pen, which will ultimately drive more dollars to fund more innovative solutions.

KELLY: If you had the power, how would you change diabetes advocacy as you see it today?

DANA: One of the most important components of the successful HIV/AIDS effort was that everyone was on the same team. There was one message, and there was a leading group that was highly organized, highly educated, highly strategic, and highly effective. Most of it was driven by ACT UP.

Contrast that with diabetes – two different primary public charities. JDRF is handling type 1 and ADA is predominately working on type 2. None of us are surprised when we hear of the type 1 diagnosis and people say, “Oh, so you just have to stop eating sugar?” This country has failed at educating our citizens on what diabetes really is – there is an acute chronic form of diabetes and a chronic form of diabetes. And that’s really where it starts. If I was king of the world, I would bring everyone together and create one big national campaign that says, “This is diabetes.” I would educate people about the differences between type 1 and type 2 and about the importance of testing and learning about prediabetes.

What if we talked about blood sugars? What if every single person knew what their blood sugar was – what if elite athletes knew their number? I think I know when I’m going a little bit low even though I don’t have diabetes – I’m shaky, I’m cranky, my head’s foggy, I don’t write as well, I can’t work as fast, and I don’t feel good. And if I eat too much sugar, I know I’m shaky and I’m miserable and I’m going to rip your head off.

What if the community could think of a different story? What if every single person was really aware in a nonjudgmental way about these two forms of diabetes – one that is an acute and one that’s non-acute. What if everyone wore a CGM? You’d actually get to know what happens with your body. That’s why I’m such a big supporter of CGM. It’s almost impossible to have a conversation with someone about something they can’t see.

KELLY: In terms of stigma, HIV/AIDS probably has more in common with type 2 than type 1. However, I think parents often have guilt with type 1 diabetes: “I’m not doing nearly enough.”

DANA: In HIV/AIDS we had a lot of guilt and shame; patient guilt, survivor guilt and even family guilt. Shame in type 1 may be that “my A1c is not good enough.” We have all these measures and we have all these metrics that we’re supposed to meet. I worry that often in type 1 diabetes, once the shock is over and the management starts, the person can disappear and it becomes about managing their diabetes. Whether it’s a spouse or a parent or a caregiver or friends, suddenly it’s “Kelly, what’s your number? Kelly, where’s your meter? Kelly, what are you eating?” And I see it with parents and kids. When the kid walks through the door, the first thing isn’t, “How was your day, honey? How was sports and how was everything?” You’re not my child anymore; you’re my diabetic child, “Did you test? What was your blood sugar?”

DAVID: As a parent, I think finding the balance of helping and being too involved is the hardest thing. As my wife Karen told me, “This is Morgan’s disease and our job is to help her and equip her with all of the knowledge so that when she leaves our house she can make the right decisions.” This concept is very tough to accept from a parent perspective, but as with any child, they take their cues from those closest to them. If I set a good example for my children by taking care of myself – by working out, eating right, etc. – the chances increase that my children will do the same.

KELLY: What are successful disease-related campaigns you can think of?

DANA: Stand Up to Cancer has celebrities and an awesome brand in a crowded space. It is raising very respectable money. You see it everywhere. It’s in the World Series right behind home base. And quite uniquely, that campaign used celebrities that don’t have the disease – it’s a great strategy to create awareness and the most creative thing I’ve seen done recently. The Susan G. Komen brand also comes to mind. She was one woman that passed away, and her sister created this brand that now raises lots of money.

Dana's Background at the Lee Iacocca Family Foundation

KELLY: Dana, how did you transition from working in AIDS to type 1 diabetes?

DANA: After 1996, I took a break from what I was doing in HIV/AIDS and it really was a chance meeting when I met the Iacocca family. It was a time in the Iacocca Foundation when Lee was taking a much more active role and was thinking about how to use the Foundation’s resources. I had the opportunity to join the foundation when there was very little infrastructure and started by focusing on operations and understanding how to best direct Iacocca funding. They were primarily funding Joslin at the time through the creation of five strategic endowments totaling approximately $20 million, but they had also started supporting investigator-initiated projects. It was a fantastic opportunity for me to work with Lee; he was asking all the right questions and raising awareness: “How are we curing this disease? How are we going to develop a plan? What are the gaps?”

People were predominantly giving their money, as they do today, to the public charities in the space: ADA, JDRF or DRI. Lee coined this great phrase back in 2003 in a press release we did: “What I’ve learned through all of my research is that if you’re a mouse, I got you covered. And I understand I own thousands of mice through all of my research. But I want to move away from basic research. I want to see something helping people living with type 1 diabetes in my lifetime.” As soon as he said “in my lifetime,” it became very urgent.

The story I brought to the partnership was the HIV/AIDS story. We had to understand human disease, and I thought this would be very simple when I got into type 1: what’s the cause of the disease, let’s get targets, mechanisms of action, let’s do biomarker work, let’s screen for drugs, and let’s see what we can do. This didn’t seem, like an incredibly complicated problem to me.

KELLY: When you reviewed the space and starting investigating what others were doing, what did you find?

DANA: It was an important time from 2000 to 2008, because we saw islet transplantation end up as an intervention therapy for a small population. In the early 2000s, the buzz in the community was that this could be a mainstream therapy for everyone with type 1 diabetes. We also saw the birth of stem cell research. There was also some autoimmunity work, and devices were still fairly early. One thing we learned was that the low number of career scientists continued to be an area of unmet need. We also found that it was important to really understand patients, the cause of the disease, studying humans, and looking at potential ways to stop autoimmunity.

We found leaders in the field, and it was all about talents that had the right tools and the right technologies to do projects. The Lee Iacocca Foundation funded Dr. Denise Faustman, and Dr. Jerry Nadler who was working on similar study approaches designed to suppress the autoimmune response and see if insulin secretion could be restored. We were also an early investor in Bayhill and many other type 1 programs and projects. We were going after intervention studies, both phase 1 and phase 2a, that we thought could potentially move forward. I took Lee’s desire to see something in his lifetime very serious and still do.

KELLY: What made Lee want to do his own thing as opposed to donating to JDRF?

DANA: I think a lot of private charities and private foundations think of their resources as some of the most flexible and most powerful, because it’s their money and not donors. So rather than invest in an organization like JDRF, the Iacocca Foundation tried to fund the highest-risk, highest-reward projects. We also tried to support young scientists and give them the opportunity to work under a mentor.

KELLY: What’s the legacy from all of the work at Lee Iacocca foundation? Obviously so much learning about what doesn’t work is what drives us.

DANA: I think the legacy is the power of a family’s dedication to turn the loss of Lee’s wife and his children’s mother, Mary K. Iacocca into an organization that is dedicated to improving the lives of people with type 1 diabetes. Lee’s business approach to philanthropy was innovative and part of the venture philanthropy movement. I think what we learned was that investing in early scientists can be successful. We were a big supporter of Dr. Matthias Hebrok’s career. Through the Joslin endowments the foundation supported Dr. Amy Wagers, now at the Harvard Stem Cell Institute. Supporting the careers of very talented young scientists that have grown up into serious scientists doing brilliant research is something that has substantial return on investment.

HANNAH: How do we get more non-diabetes researchers into the field, especially in technology? There are all these scientists currently working with the government on drones and other advanced projects. If we could just get them to spend half a year on diabetes, taking what they know from other places, that could make a real difference.

DANA: Yes. That’s a question we’re still working on solving. How do you find the talent? And it’s something we struggle with all the time – how do you crack into these networks of people outside of diabetes and incentivize them to think about our problem. We want the people who have solved problems in traffic and other unrelated data-driven issues to think about diabetes. I always say to David, “We can’t keep sticking our heads down the same rabbit hole and expecting different results. We can’t keep going to the same people and asking them to develop a solution and expecting different results. We need to find new talent. We need to find new resources and talent to work with the people that have the historical knowledge of diabetes. This combination has the potential to develop new solutions.

KELLY: Are you optimistic about that?

DANA: I think our network of talent and partners gets better every year. It takes a long time to build those relationships and find them. It’s like breaking through a dam. Once in a while, you find someone that has a link to a network of brilliant people that have solved problems in other sectors or other areas. Once you find them, you have to invite them in and incentivize them to look at our problem.

 

--by Adam Brown, Hannah Deming, and Kelly Close

with additional editing and research help from Jessica Dong, Andrew Foley, Hannah Martin, and Manu Venkat