JDRF Reaches New Milestones in Work to Drive Cures for Type 1 Diabetes and Improve Lives

Annual Report showcases year of advances, driven by $121.5 million in direct support and nearly $300 million in additional funding for T1D research

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JDRF

Feb 14, 2020, 17:12 ET

NEW YORK, Feb. 14, 2020 /PRNewswire/ -- JDRF, the leading global organization funding type 1 diabetes (T1D) research, funded $121.5 million directly and helped generate more than $400 million in total T1D research funds from nonprofits, government, and industry to propel a year of remarkable breakthroughs in 2019, JDRF announced today. JDRF research funding increased by 10 percent over the previous year.

"I am more excited about our progress than ever before, given the tremendous breakthroughs made in 2019," said Aaron J. Kowalski, Ph.D., president and CEO of JDRF. "We could not be driving this work forward without the support of so many dedicated individuals, partners and organizations all helping us deliver on our mission to cure T1D and improve lives."

JDRF funding supported more than 180 active T1D research grants and is funding about 70 clinical trials for drugs, biologic and devices to prevent or cure T1D. The organization also supported new investment through the JDRF T1D Fund in 15 T1D companies, including 11 focused on cures.

"We are focused on beta cell therapies and immune therapies to deliver cures for T1D," Kowalski said. "At the same time, we continue accelerating work in glucose control and complication therapies to improve the lives of those living with type 1 diabetes today."

Highlights of breakthroughs outlined in the 2019 Annual Report are:

Progress Toward Cures:

Drug delays T1D for more than 2 years: For the first time ever, an immune therapy, called teplizumab, was able to delay the onset of T1D for more than two years in those with a high risk of developing the disease. Teplizumab now is in a phase III clinical trial in recently diagnosed individuals. The Food and Drug Administration (FDA) also granted Breakthrough Therapy Designation to teplizumab, which is intended to expedite the development and review of the drug. JDRF funded one of the first clinical trials of teplizumab, and funds TrialNet, which conducted the study.
Better insulin-producing beta cells from stem cells: JDRF-funded researchers have created mature insulin-producing beta cells from stem cells that respond to blood sugar more like human beta cells. What's more, after transplantation, these beta cells were functional in a matter of days versus two to six weeks after transplantation.

Improving Lives:

Second artificial pancreas system: The 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. JDRF has been a leader in supporting artificial pancreas systems for more than 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.
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). Injectable glucagon has been approved in the United States for several decades, but this is the first non-injectable treatment. Administering nasal glucagon is much less invasive and is a simple process that can save time during an emergency. 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 glucagon drug that can be used in bi-hormonal pumps. 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. Results from the CONCEPTT trial played a major role in the most recent American Diabetes Association's clinical guidelines update.
Europe and Japan approve SGLT inhibitors for use in adults with T1D: In early 2019 the European Medicines Agency approved both Zynquista™ (sotagliflozin) and Forxiga® (dapagliflozin and marketed in the United States as Farxiga) for use by adults with T1D. The Japanese Ministry of Health, Labour, and Welfare also approved the use of Forxiga, in March 2019, as a second SGLT inhibitor option to Suglat (ipragliflozin), which was approved in late 2018. SGLT inhibitors have previously been used for the treatment of type 2 diabetes. The approval provides the international T1D community with another option to improve daily blood-sugar management.
Pharma company makes 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.
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 immunotherapies 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 in the development of preventive therapies.

Reducing Complications:

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. 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.

JDRF's Annual Report also cites progress in advocacy and community engagement, two areas aimed at both supporting research and the T1D community. "The combined strengths of research, advocacy and community engagement are helping us support more members of the T1D community and enabling more people to support our mission," Dr. Kowalski said. "JDRF works to build partnerships that advance research and build and sustain critical support for type 1 diabetes (T1D) research funded by the Federal Government."

Expanding Access to Coverage:

Coverage2Control Campaign: Knowing the significant impact of the rising cost of insulin and importance of pump choice to the T1D community, JDRF and its supporters continued to call on insurers, employers, drug companies and the government to provide affordability, choice and coverage to help people with T1D better control the disease. Specifically, using JDRF's campaign tools, more than 27,000 supporters sent over 145,000 messages to UnitedHealthcare leadership and shared their concerns on social media, urging them to change their policy that restricted pump choice for adults and children with T1D.
Advocating for the Special Diabetes Program: After advocates met with nearly every Congressional office as part of JDRF 2019 Children's Congress and Government Day, and sent countless emails, Congress provided nearly $100 million for the Special Diabetes Program (SDP) through May 22, 2020. JDRF and our advocates will continue to urge Congress to pass a multi-year renewal of the SDP to allow researchers to build on the exciting progress made to date, and to explore new opportunities without fear of interruption.

Learn more about JDRF's research and advocacy priorities at jdrf.org and read the health insurance guide JDRF has complied to help the T1D community navigate their healthcare and health insurance 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 five 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.

SOURCE JDRF