Picture: JDRF CEO Aaron Kowalski at the 2019 JDRF One Conference.
On November 19, 2019, Aaron Kowalski delivered his 2019 State of the Foundation address to JDRF staff and volunteers at the JDRF One Conference in Minnesota. It was the first time a State of the Foundation address was delivered by a sitting JDRF CEO with type 1 diabetes.
The speech covered a broad range of topics, addressing the burdens of living with T1D, technological advances in T1D, and government and healthcare advocacy around T1D issues. Dr. Kowalski dedicated roughly 1/3 of his time to talk about cures for type 1 diabetes. He stated bluntly: “we need to fund more research.” We agree, and we hope Dr. Kowalski will take action to restore JDRF’s annual research grant expenditure from its ten-year low.
Cure Research for T1D
During the address, Dr. Kowalski identified two T1D Cure research pathways on which the JDRF is now focusing: 1) beta cell therapies and 2) immunotherapies. In introducing these topics, he made the exciting claims that “we are closer than ever to beating this disease” and that “we can now make the cells— enough cells to potentially cure everyone of T1D.” However, nothing in this presentation provided evidence that a cure is imminent.
Dr. Kowalski’s address, which can be found here, is discussed in greater detail below.
- A recent discussion with Dr. James Shapiro, author of the seminal Edmonton Protocol that was published in 2001, was presented to demonstrate that some people who received islet cell transplants were ‘cured’ (no need for external insulin; normal HbA1C levels). However, cell supply remains limited (sourced from cadavers) and aggressive immunosuppression is required daily, for life.
- Doug Melton’s research at Harvard and Semma Therapeutics was presented as a demonstration of our relatively new ability to expand islet cell supply by training embryonic stem cells. Dr. Kowalski noted that Semma’s recent sale to Vertex (for $950 Million) is proof of the viability of this research. It is worth noting that Semma has not yet started any human trials.
- Dr. Kowalski assured the audience that researchers are making progress at protecting beta cells from the auto-immune attack because of JDRF, stating that “we are in multiple human trials”, but declined to provide any specific examples. In fact, out of 494 grants funded by JDRF in 2018, only eight projects were testing a solution, or part of a solution, to protect beta cells from the autoimmune attack in humans.
- The main example Dr. Kowalski provided regarding immunology advances was a T1D prevention trial, not a cure. Teplizumab, which was tested in patients at a high-risk to develop T1D, posted successful results from a small trial this summer.
- JDRF T1D Fund, which makes investments in T1D companies in part for a profit return, was referenced often enough that it could be considered the main driver of current achievements at JDRF (both Semma and Teplizumab are T1D Fund investments).
Time for a Dedicated Practical Cure Initiative
Two of the cure pathways noted in the speech are the same ones that will drive a Practical Cure. Over the past decade, we have written about these pathways, often in the shorthand of cell supply and cell protection. We applaud the JDRF narrowing its focus, but we have not yet seen a clear definition of success, a roadmap, or commitment of resources towards a Practical Cure objective.
Dr. Kowalski referenced during the speech the impact of his paper on the artificial pancreas that pointed the way and provided direction for JDRF’s work in that field. It was a clear roadmap that defined the end game, pointed out challenges, and served as a guide for resource allocation. It is time for the same approach toward a Practical Cure.
If JDRF was to define a Practical Cure pathway and increase dedicated funding for projects— like beta cell or immune system modification therapies— that could deliver a Practical Cure for those people living with established T1D, Dr. Kowalski could bring us even closer to “beating this disease” during his tenure as JDRF President and CEO.