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Op-Ed: A ‘Cure’ for Diabetes? The Last Miles

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Op-Ed: A ‘Cure’ for Diabetes? The Last Miles

“How did we get here and where are we going?”

That’s what headlines across the nation and around the world recently proclaimed after clinical trial results showed that the first human subject of a stem cell-derived replacement therapy needed 91% less insulin after just a half dose of the therapy.

As a scientist, head of the largest global nonprofit funder of type 1 diabetes (T1D) research, and a person who has lived with type 1 diabetes for over 35 years, I applaud the brilliant researchers that made this advance possible. JDRF is proud that we started funding research studies over 20 years ago that contributed to this breakthrough. The challenge and the opportunity today is to see this exciting advancement reach the millions of people who live with T1D around the world. To accelerate cures for type 1 diabetes and other life-threatening diseases, we need strategic collaborations, which bring together patients, researchers, policymakers, and company leaders.

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This exciting diabetes advancement began in the late 1990s, when JDRF led efforts in Congress and prior administrations, along with other patient and health advocacy organizations and research institutions, to create and expand federal policies on stem cell research.

We used our international network to initiate funding partnerships with governmental agencies outside the United States, launched research into stem cells worldwide, and were invited to be a founding member — the only foundation — of the International Stem Cell Forum, all before 2004.

In funding, JDRF awarded numerous grants that are just now reaching clinical trials — offering a light at the end of the tunnel for people burdened by type 1 diabetes and the management and complications that it brings.

In August 2000, JDRF funded Douglas Melton, PhD, with a 10-year grant to make beta cells from stem cells, funding facility construction and research that brought about the first new human embryonic stem cell lines in 2004.

In 2014 and 2015, Dr. Melton published a paper that outlines how to make stem cells into beta cells and founded Semma Therapeutics. Shortly thereafter, the JDRF T1D Fund — JDRF’s innovative venture philanthropy fund — made one of its first investments in the company. Two years later, Semma was acquired by Vertex for almost $1 billion.

Vertex started a clinical trial of VX-880 — the stem-cell derived therapy recently covered extensively by news media outlets.

This very important milestone is built upon years of work by many committed stakeholders. It took actions by JDRF, the federal government, leading academic research teams, and multiple companies like The Leona M. and Harry B. Helmsley Charitable Trust to get us this far. JDRF funding has totaled $150 million in direct funding and millions more in indirect funding so far — and advocacy efforts. The payoff when successful will be much greater. Eliminating the need for people with diabetes to take insulin, significantly reducing both the human and economic costs of this devastating disease.

Stem cell-derived islets could provide an unlimited source of insulin cells that, in theory, could be available to all people with type 1 diabetes. Now is the time to double down to ensure that we accelerate through these last miles and see the first cures for people with type 1 diabetes become available.

It’s been 20-plus years of research that has brought us to a place where stem cell-derived islets could be tested in people. And once we have proven, approved therapies, it will take more time to ensure everyone who needs them has access to them.

JDRF is in it for the long haul, and we believe that competition drives innovation and that “multiple shots on goal” is a good thing. JDRF and our JDRF T1D Fund will continue to support the research, companies, and policies required to see these advancements into the hands of people around the world. We believe that someday, you’ll read about type 1 diabetes not in your medical books, but in your history books. We believe that someday, type 1 diabetes will be a thing of the past, and stem cell-based therapies will be part of the cures for this disease. And it will be because of science — and critical collaborations that bring those breakthroughs to people who need them.

Want to learn more about the new stem-cell-based therapy? Read “Vertex: New Stem Cell Based Type 1 Diabetes Treatment Shown Effective in First Patient.”

Aaron J. Kowalski, PhD

Aaron J. Kowalski, PhD

Aaron J. Kowalski, PhD on social media

Kowalski is President and Chief Executive Officer of JDRF.

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