Download JDRF Research In The News Summary February 2014

JDRF Research
In the News
February 2014
JDRF Encapsulation Research
JDRF Supporting Upcoming Clinical Trial of ViaCyte's Encapsulated Cell Therapy for T1D
JDRF and ViaCyte, a leading regenerative medicine company, jointly announced that JDRF is providing additional milestonebased funding for the continued development of ViaCyte's VC-01™ encapsulated cell therapy product candidate for the
treatment of T1D. JDRF will fund up to $7 million to help ensure a rapid transition of the project into the clinical phase of
development once ViaCyte's investigational new drug application (IND) is filed with and accepted by the U.S. Food and Drug
Administration (FDA). This commitment builds on JDRF's previous support of ViaCyte's preclinical development program
focused on collecting the necessary animal safety and efficacy data to support introduction into clinical testing. ViaCyte’s
innovative VC-01 product candidate is a cell replacement therapy that could transform the way individuals with T1D manage
their disease by supplying an alternative source of insulin-producing cells with the potential to free individuals from a
dependence on external insulin use. For more information, please see JDRF’s Press Release.
Scientists Generate Insulin-Producing Cells in Mice
Researchers, funded in part by JDRF, have successfully turned mouse skin cells into insulin-producing beta cells. And when
those cells were transplanted by the scientists into a few dozen diabetic mice, their blood sugar levels returned almost to
normal. What's more, they noted, the technique used to transform these cells is safer than other methods that have been
used to transform one type of cell into another. One of the advantages of this approach is that by doing this type of
reprogramming in one step, you don't get the type of cells that have uncontrolled proliferation potential. This could be a
safety advantage. However, this is still early animal data and animal studies don't always translate to humans. The next step
needs to be to see if this happens in human cells and whether this technique can be adapted for humans. This research
could result in a new source of beta cells for encapsulation products.
JDRF Restoration Research: Immune Therapies
Study Tests Whether Drug AAT Can Reverse T1D
A clinical study is being run to test whether the anti-inflammatory drug Alpha 1 Antitrypsin (A1AT) can successfully change
the course of the disease in people newly diagnosed with T1D. Alpha 1 Antitrypsin is found in blood plasma and has an
ability to regulate the immune system. A1AT has been used to treat people with emphysema but research has also shown
promise for it to be a treatment for T1D and other autoimmune diseases, such as rheumatoid arthritis and asthma. A clinical
trial, funded in part by JDRF, is currently underway to test Alpha 1 Antitrypsin against people newly diagnosed with T1D. The
study, called the RETAIN (Research Trial of Aralast in New Onset Diabetes) I study. The RETAIN I trial involved only 16
participants and the study duration of 18 weeks was also relatively short. Results of the first trial showed promise with
participants able to take less insulin than they had needed to at the start of the trial. The RETAIN II study, will involve a larger
number of participants than RETAIN I and the total study duration will be approximately four years. Participants in the A1AT
1
treatment group will receive 12 weeks of the A1AT drug marketed under the brand name Aralast, while other participants
will receive a placebo. The goal of the treatment will be to observe a higher amount of C-peptide being produced by those in
the A1AT treatment group after 1 year. C-peptide is a marker of how much of the body's own insulin is being produced.
Network of Scientists Combat T1D
About two-and-a-half years ago, Toronto’s Sue Beamish was busy helping her son get ready to leave home for university
when they were blindsided with a T1D diagnosis. “The diagnosis came out of the blue,” Beamish said at a recent event
hosted by the JDRF Canadian Clinical Trials Network. “During this emotional and tumultuous time for our family, we saw very
quickly how positive a role technology has played in dealing with the day-to-day life with T1D. We have access to many
excellent monitors, pumps, and insulin that are available today, but having this technology, while great, is not enough. It’s
important to have the hope that someday soon there will be a cure.” Beamish was in Vancouver as a representative of the
WB Family Foundation, which recently donated $3 million to the JDRF CCTN. With the funding, the JDRF CCTN will expand
from Eastern Canada to the country’s West. The studies focus specifically on finding a cure for the disease that affects
millions of people around the world.
JDRF Artificial Pancreas Research
UVA’s Overnight Closed-Loop Makes For Great Dreams
There has been so much progress recently with automated insulin delivery systems (artificial pancreas systems), so readers
are always excited to learn about new prospects and choices for people with T1D. So, when a reporter (with T1D) heard
about the University of Virginia (UVA) JDRF-funded study testing overnight closed-loop control, she jumped at the
opportunity to participate! Over five days, the team at UVA let her wear the study’s DiAs (short for “diabetes assistant”)
system at night. DiAs is an artificial pancreas system that doses insulin automatically, based on continuous glucose readings.
The 'hardware' consists of a Roche Combo pump, two Dexcom CGMs [the second CGM is used as a backup at the FDA’s
request], and a control algorithm running on an Android cell phone. A Dexcom Share is used to transfer the CGM data from
the receiver to the DiAs system, although it is expected that this intermediate device won’t be necessary when truly wireless
sensors come out (there’s been lots of progress recently on that front too). For five straight days, she woke up at 7 am with a
blood glucose of 120 mg/dl, having spent most of the last eight hours at that level. It felt incredible – and it felt different
than just waking up at 120 mg/dl, which is possible when hearing the Dexcom alarms at night and adjust them appropriately.
Here, it was all about the machine doing the work.
JDRF Prevention Research
Strong Evidence Uncovered For Link Between Viral Infections and T1D
Researchers from the University of Cambridge, supported by the JDRF and The Wellcome Trust, have identified evidence
which suggests a link exists between viral infections and the onset of T1D. A crucial part of finding the link was that
researchers already knew that a link existed between T1D and a form of immune response genes known as anti-viral type I
interferon (type I IFN). The researchers found that the type I IFN signature they were looking out for was increased,
temporarily, in the children susceptible to T1D prior to the development of the autoantibodies which attack the insulin
producing cells in the pancreas. By contrast, no increased type I IFN signature was present in children that were healthy or
already had T1D. Researchers reviewed evidence for events preceding the temporary increase in the type I IFN signature and
found it to be associated with a recent history of upper respiratory tract infections, which include flu and the common cold.
T1D: Vitamin D Deficiency Occurs in an Early Stage
Low levels of vitamin D are commonly found in people with T1D. But even children who have multiple positive islet
autoantibodies without clinically diagnosed T1D have lower levels of vitamin D in their blood. This does not appear,
however, to influence the progression of the disease from pre-diabetes to diabetes, according to a recently published report
from scientists at the Helmholtz Zentrum München and the Technical University of Munich with support from JDRF. Vitamin
D is known as a major regulator of calcium levels and bone metabolism. Furthermore, it also influences the immune system.
Previous studies have shown that patients with recently diagnosed T1D have significantly lower vitamin D levels. Within the
group of children with positive autoantibodies, a few children quickly developed T1D – however this was independent from
their vitamin D levels. “Vitamin D deficiency precedes the onset of T1D but this may be a consequence of an immune
2
response,” Professor Ziegler says. “In the case of prediabetic children, we must therefore be mindful of the higher risk of
vitamin D deficiency and consider evaluating the need for vitamin D supplementation at an early stage of T1D.”
3