JDRF celebrates top ten advances that make progress toward cures and improve the lives of people with T1D

New York, December 20, 2019—JDRF, the leading global organization funding type 1 diabetes (T1D) research, today shared a list of the top 10 advances made this year in curing T1D and improving lives of those living with the disease today.

“In 2019, our community saw tremendous progress in in every area of T1D research,” said Aaron J. Kowalski, Ph.D., president and CEO of JDRF. “This is a race being run by researchers around the globe, dozens of institutions and some of the most innovative companies in the world. With the backing of millions of supporters, together, we will cure this disease. “

The list reflects advances across T1D research and driven by a powerful network collaborators. Many were funded by JDRF or supported by the JDRF T1D Fund. Others were advanced by the National Institutes of Health’s Special Diabetes Program, the Leona M. and Harry B. Helmsley Charitable Trust, or by the private sector that made T1D a priority in their research and development programs.

Progress Toward Cures:

• Drug delays T1D for more than 2 years: For the first time ever, an immune therapy treatment, called teplizumab, was able to delay for more than two years the onset of T1D in those with a high risk of developing the disease. Teplizumab now is in a phase III clinical trial, being tested in people recently diagnosed with T1D by Provention Bio, a company with an investment from the JDRF T1D Fund. The FDA has also granted Breakthrough Therapy Designation to teplizumab in individuals at-risk of developing the disease. JDRF funded one of the first clinical trials of this drug, and funds TrialNet, which conducted the study.

• Pharma make landmark investment in T1D Research: Vertex Pharmaceuticals acquired T1D- focused Semma Therapeutics for $950 million. Semma was founded in 2015 with the goal of commercializing research to make beta cells from human-derived stem cells. The research team, led by Douglas Melton, Ph.D., had support from JDRF since 2000 and, in 2017, the JDRF T1D Fund provided an important investment in the company.

• Better insulin-producing beta cells from stem cells: JDRF-funded researchers have introduced an inventive step in the process of making mature insulin-producing beta cells from stem cells, with the resulting cells responding to blood sugar more like human beta cells. What’s more, after transplantation, these beta cells were functional in a matter of days. (Research thus far has insulin-producing beta cells responding to blood sugar challenges only 2 to 6 weeks after transplantation.) Not only does their discovery set the stage for cell therapy, since they can now make human beta cells in a dish, it opens avenues for finding novel drugs that can stimulate the expansion of beta cells.

• Collaboration with Cancer Researchers: In 2019 JDRF and The Leona M. and Harry B. Helmsley Charitable Trust teamed up with the Parker Institute for Cancer Immunotherapy on research that could help both diseases. New data show a subset of people with cancer who receive certain immune therapies go on to develop T1D or other autoimmune diseases. Understanding who is at risk and why some people go on to develop autoimmunity could help us develop preventive therapies.

New Therapies & Reducing Complications:

• FDA approves a second artificial pancreas system: The Food and Drug Administration (FDA) authorized an algorithm that enables the second artificial pancreas system: The Tandem Control-IQ™ advanced hybrid closed loop technology. It’s the first algorithm authorized as an interoperable automated glycemic controller, which means the algorithm could be a component of any open protocol, or interoperable, artificial pancreas system. Data came from the International Diabetes Closed Loop Protocol-3 (iDCL) trial, funded by the Special Diabetes Program. The study met all of its primary and secondary endpoints, including time-in-range and HbA1c, with no fingerpricks and no severe low blood sugar events. JDRF has been a leader in artificial pancreas systems for 15 years, from developing a roadmap for artificial pancreas development and partnering with the Helmsley Charitable Trust and the FDA to create a regulatory pathway for approval and commercialization of this technology. Industry experts have said that JDRF’s involvement cut five years off the approval process for the Medtronic 670G artificial pancreas system in 2016, the first approved system.

• FDA approves two treatments for low blood sugar: The FDA approved Baqsimi, the first non-injectable emergency treatment for severe episodes of low blood sugar (hypoglycemia). Severe hypoglycemia means that another person has to administer treatment because the person with T1D has impaired or lost consciousness or may be having a seizure. Injectable glucagon has been approved in the United States for several decades, but this is the first non-injectable treatment. The FDA also recently approved a glucagon pre-filled syringe and auto-injector created by Xeris Pharmaceuticals, which is also funded by JDRF to develop a bi-hormonal pump with insulin and glucagon. The GVOKE line of products features the first pre-mixed, pre-filled liquid glucagon formulation on the market.

• Recommendation: Pregnant women with T1D use continuous glucose monitors (CGMs) to monitor blood sugar: The United States has joined Australia and the United Kingdom in recommending that CGMs be used to improve HbA1c outcomes in pregnant women with T1D. JDRF funded the CONCEPTT trial, which showed that using a CGM during and prior to pregnancy improves health outcomes for both mothers and babies. It was the CONCEPTT trial that made the American Diabetes Association revise its clinical guidelines.

• End-stage kidney disease: A possible therapeutic target for people with T1D: In the United States, diabetic kidney disease is responsible for more than half of all new cases of end-stage kidney disease. To help prevent this outcome, JDRF-funded researchers Monika Niewczas, M.D., Ph.D., M.P.H., and Andrzej S. Krolewski, M.D., Ph.D. are seeking biomarkers of progression that could accelerate preventive therapies. They found 17 inflammatory proteins that were strongly associated with progression for end-stage kidney disease building on the work published in 2012. Many of the proteins are currently being tested in clinical trials for other chronic inflammatory disorders. Further testing these compounds in diabetic kidney disease may offer hope to people living with T1D that they may thwart end-stage kidney disease altogether.

• European approval of two adjunct therapies: The European Commission has approved two medications that help in glucose management by preventing the kidneys from reabsorbing glucose back into the blood. The Commission approved SGLT inhibitors for T1D: Forxiga® (dapagliflozin and marketed in the United States as Farxiga) and Zynquista™ (sotagliflozin), when used in conjunction with insulin to improve glycemic control.

• Clinical trial results: TTP399 was able to reduce HbA1c by 0.7%: A protein, called glucokinase (or GK), acts as a key regulator of sugar levels in the body. If blood glucose levels are deemed too high, activation of GK in the liver causes the body to use more glucose, which in turn lowers glucose levels in the blood. vTv Therapeutics has developed a GK activator, called TTP399, and joined forces with JDRF in 2017, to test it in people with T1D. In a clinical trial, the TTP399 group had a significant and clinically meaningful reduction of 0.6% in their HbA1c, whereas those in the placebo group increased their HbA1c by 0.1%. These promising findings support advancing to phase II-part 2 to confirm the results in a larger and more diverse T1D population. To find out more on this trial, go here.

About JDRF

JDRF is the leading global organization funding type 1 diabetes (T1D) research. Our mission is to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.2 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally for efficiency and broader fundraising impact, and uniting on a national stage to pool resources, passion, and energy. We collaborate with academic institutions, policymakers, and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our six international affiliates are dedicated to advocacy, community engagement and our vision of a world without T1D. For more information, please visit jdrf.org or follow us on Twitter: @JDRF.