New York, NY, October 29, 2014
— JDRF, the leading research and advocacy organization funding type 1
diabetes (T1D) research, announced today that JDRF-funded partner,
ViaCyte, Inc., a leading regenerative medicine company, has for the
first time ever implanted a person with type 1 diabetes (T1D) with an
experimental encapsulated cell therapy product candidate, called
VC-01TM, which is being developed for the treatment of T1D. This
individual, and others to follow, is participating in a trial to
evaluate the safety and efficacy of the VC-01 product candidate, a
potential replacement source of insulin-producing cells.
“JDRF is excited about the historic research advance of this
encapsulated cell replacement therapy for type 1 diabetes,” said Derek
Rapp, JDRF president and CEO. “Encapsulated cell therapies have the
potential to fundamentally transform the lives of people with type 1
diabetes by restoring a person’s independence from insulin injections
and reducing concerns of the complications that arise from living with
the disease. We are enthusiastic about the progress of research in this
field, and we look forward to the full enrollment in the trial and to
results from this study which could move us one step closer to a new and
effective treatment for type 1 diabetes. This day would not have been
possible without the basic stem cell research and the insights learned
to protect insulin-producing cells from the autoimmune attack that JDRF
has been funding for years.”
The study of ViaCyte’s experimental therapy, VC-01, is initially
being conducted at the University of California at San Diego (UCSD). The
experimental product encapsulates immature human islets derived from a
stem cell source in an immune-protective device called the Encaptra®
drug delivery system. At first, a small group of people in the study
will be followed for several months to ensure the safety of the therapy.
After the initial evaluation of this first group of participants at the
UCSD site, it is expected that additional sites will be activated to
enroll more people in early 2015.
The primary goal of this first study is to evaluate the safety of the
VC-01 product candidate in people who have had T1D for at least 3
years; not to make them insulin independent. However, the study is also
designed to evaluate the effectiveness of the VC-01 product candidate in
replacing the lost insulin-production function that is central to T1D,
by measuring C-peptide levels as a marker of insulin production. This
study will provide researchers with critical information about the
functioning of the encapsulation device at the implantation site and
about the maturation and survival of the implanted cells.
Dr. Paul Laikind, President and CEO of ViaCyte, said, “Treating the
first patient with our stem cell-derived islet replacement product
candidate is an exciting next step in our quest to transform the way
patients with type 1 diabetes are impacted by the disease. Moving from a
promising idea to a new medicine is a long and challenging journey and
we are grateful to JDRF, and all its supporters, for the tremendous and
continued support they have provided. At ViaCyte we have been working
over a decade to reach this milestone, and JDRF has been there every
step of the way, providing critical funding, excellent advice, and
advocating on our behalf.”
People in the study will go about their daily lives with the
implanted product for up to two years. They will be closely monitored by
clinicians during this time. The encapsulated cell therapy here
involves putting cells with the potential to mature and produce insulin
in a protective barrier and implanting them in the body using a
minimally invasive procedure. Once matured these encapsulated immature
human islets might sense a person’s glucose levels and produce insulin
while the barrier shields them from the body’s T1D autoimmune attack as
well as foreign graft rejection. If they perform as they have in animal
studies, the cells will continuously assess the amount of glucose in the
blood and release the appropriate amount of insulin. Importantly,
encapsulated cell replacement therapies have the potential to overcome
the major obstacles that have limited the use of human islet
transplantation in people with T1D: limited donated islets and the need
for lifelong administration of immunosuppressive drugs to prevent
destruction of the newly introduced islets. Stem cell-derived islet
sources may represent an unlimited supply of replacement
insulin-producing cells and the protective devices eliminate the need
for immunosuppressive drugs.
JDRF is the leading global organization focused on type 1 diabetes (T1D) research. JDRF’s goal is to progressively remove the impact of T1D from people’s lives until we achieve a world without T1D. JDRF collaborates with a wide spectrum of partners and is the only organization with the scientific resources, regulatory influence and a working plan to bring life-changing therapies from the lab to the community. As the largest charitable supporter of T1D research, JDRF is currently sponsoring $568 million in charitable research in 17 countries. For more information, please visit www.jdrf.org
