In January, hundreds of leaders from the JDRF community, our in-house researchers and esteemed guest speakers gathered at our internal, annual Mission Summit to discuss the path to a cure for type 1 diabetes (T1D). At JDRF, our mission is to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications. As International Chair of the Board of Directors Mark Fischer-Colbrie drove home in his welcoming remarks, “With this group of people and our fabulous researchers, we are going to find a cure.” To track progress toward this goal, we heard from leading scientists attempting to answer critical questions around the causes of T1D, how we might prevent it, improve health outcomes, and ultimately, create a world where T1D no longer exists.
Unraveling an immensely complex disease like T1D requires a multi-faceted approach. So JDRF works with many partners to catalyze knowledge creation and translate that knowledge to new therapies and products that our community can access. We attract researchers to the T1D field and also recruit new researchers through our training grants. In 2017 alone, we funded 56 post-doctoral fellowships for the next generation of research leaders and created a new psychology program to better meet behavioral health needs.
But our biggest focus is the funding JDRF provides in six priority research areas that are critical to the success of our mission: artificial pancreas, beta cell replacement, glucose control, prevention, restoration and complications.
Our Work to Cure T1D
JDRF funds important basic research that helps us gain a better understanding of T1D—and is essential if we’re going to cure this disease. Take, for instance, the JDRF Network for Pancreatic Organ Donors with Diabetes (nPOD). Similar to what tissue banks have done for cancer, nPOD has collected more than 40,000 tissue samples, shared samples with researchers in 19 countries, and resulted in 179 peer-reviewed papers that expand how we look at the progression of diabetes in humans. We also analyze data from longitudinal studies like TrialNet, TEDDY, ASK and Fr1Da to examine progression, onset and life with T1D.
While JDRF doesn’t have laboratories, our scientists facilitate collaboration and convene investigators to focus on common areas of interest to ensure the very best minds are focused on T1D.
This involves learning from different diseases and applying that knowledge to T1D. In the Mission Summit keynote, Jeffrey Bluestone, Ph.D., President and CEO of the Parker Institute for Cancer Immunotherapy and the A.W. and Mary Margaret Clausen Distinguished Professor at the University of California, San Francisco (UCSF), challenged us to think about a “hack” for diabetes through the lens of cancer research. As a leading immunologist, JDRF grantee and former director of the UCSF Diabetes Center, Dr. Bluestone explained how treatments that use the body’s immune system to fight cancer can improve our understanding of diabetes.
Dr. Bluestone told us to think of it this way: the immune system monitors the body’s gas pedal (the immune response) and the brakes that shut down the immune response in autoimmune diseases like T1D. Together, these intrinsic factors—the role of shutting down the immune response and controlling itself—work like an emergency brake. The observation that you could “pump” the brakes for immune suppression led to an explosion of new cancer drugs. Going further, by 2010, scientists discovered how to “turn off” the brake and engage the immune response directly. This re-education of the immune system so that it no longer attacked healthy cells—turning off the brake and turning on the gas—led to a paradigm change in cancer treatment known as immune therapy.
What does this have to do with T1D? As researchers started discovering the immune system’s on and off signals, they found that in the process, some cancer patients became autoimmune, and over time, some (but not all) patients were diagnosed with T1D. This was the first indication that immune therapy has implications outside of cancer, and gives us a chance to look at real-time disease development by following blood glucose over time. Researchers are actively studying how we can engage the immune system brake to shut down only the response we want, and discovering this connectivity gives us a new way to think about how we can cure the disease moving forward.
Our Work to Prevent T1D
At JDRF, we talk a lot about finding a cure for T1D. While a “cure” can mean different things to different people, most agree though that there are two things we need in order to cure T1D: 1) we need to stop the destruction of pancreatic beta cells, and 2) replace the beta cells that are lost, either through bringing cells into the body or through stimulating the body’s own production of such cells, to produce a sufficient amount of insulin.
We also see the importance of data in prevention. To do so, we’ve started a relationship with IBM that allows us to take big data and turn it into the first precision medicine effort to identify the risk and onset of T1D. IBM’s analysis of the existing data could open the door to better understanding the risk factors for T1D, and is another case of innovation speeding progress toward a cure.
Our Work to Treat T1D and its Complications
Of course, as we work to measure drivers of the disease and alter the course of T1D, we are simultaneously working to accelerate the research development pipeline to create new products that make a tremendous difference in the lives of people with T1D. Commercially available artificial pancreas technology is one example (with many systems in clinical testing). Other examples include our very early funding for beta cell encapsulation several decades ago, glucose-responsive insulin more than a decade ago, and currently, numerous immunology approaches to preserve beta cells. We are able to accelerate progress through pipeline by providing the advocacy muscle to fund additional research, push regulators to approve products, and make sure insurers provide reasonable and affordable access.
As Anne Peters, M.D., a professor of medicine at the University of Southern California’s (USC) Keck School of Medicine and director of the USC Westside Center for Diabetes, eloquently described, our ultimate goal is a cure, but in the meantime, we’re motivated by outcomes—and more specifically, better ways to define clinically meaningful T1D outcomes beyond hemoglobin A1c. We want regulators to understand that A1c alone is not the sole measure of health in a person with T1D, and that time in range and frequency of severe lows matter as well. JDRF is working to help the FDA understand T1D clinical outcomes in other ways to accelerate the approval of new therapies.
Looking to the Future
This idea that we can’t change the outcome of T1D without first changing our outlook was a prevalent theme at Mission Summit, and best highlighted in a conversation on how to envision the future with David Panzirer, trustee of The Leona M. and Harry B. Helmsley Charitable Trust.
JDRF and the Helmsley Charitable Trust are the two largest private sector funders of T1D, and together have supported multiple research grants that are both high-risk and high-reward to encourage bold thinking and new approaches. As Mr. Panzirer described, if you want to have an impact, you have to stay laser focused on mission, a philosophy that has led to strategic investments in primary prevention and tools to ease the burden of T1D, as well as environments that foster collaboration and data-sharing.
We can’t change the outcome of T1D without first changing our outlook.
JDRF’s T1D Fund fills an important gap between philanthropy and the business community, and is another example of supporting projects that have a clear impact on the lives of people with T1D. The reality is that diabetes is a billion-dollar problem, and convincing venture capitalists that T1D is a great place to make a difference is one solution. In the last year, the Fund made investments in eight companies and between the contributions from the foundation and individual donors—including a recent commitment of $5 million from the Helmsley Charitable Trust—has more than $65 million in investable assets. These funds support the T1D Fund’s goal of identifying and advancing the highest impact commercial opportunities in active partnership with venture and industry capital sources.
This work is compounded by the fact that for every $1 JDRF invests in research, an additional $2.50 is brought into the field through our efforts. To date, our direct funding, partnerships and advocacy have led to nearly $5 billion invested in T1D research and community support.
Together, our community is the perfect combination of mission and heart–comprising more than 1 million members actively involved with JDRF today. As we keep working together, we are confident that can create a world without T1D.
To continue tracking our headway, read our latest annual report here.