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.
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