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| Cross-sectional schematic illustration of ViaCyte device |
It’s not often a promising new approach to one of the biggest health problems in the US today gets called “the holy grail” of treatments, but that’s what one independent expert called the type 1 diabetes therapy that ViaCyte is developing.
So, it’s no wonder that ViaCyte’s therapy is not only generating interest, it’s also generating capital. The Juvenile Diabetes Research Foundation has just announced that it is providing an additional $3 million in funding to help ViaCyte move the treatment out of the lab and into clinical trials in people.
This matches the $3m in supplementary funding that our Governing Board approved for the company back in December of last year. Like JDRF we have been funding ViaCyte’s product for several years, through several stages of development. The reason why we are both willing to continue to fund what is, for now at least, called VC-01 is because it’s such a promising therapy.
VC-01 is a small implantable device that contains cells, derived from a human embryonic stem cell line, that can generate insulin. The genius of the device is that it allows the cells to monitor the body’s blood glucose levels and so make insulin when it’s needed, but because it is in a capsule it is isolated from the body’s immune system, which would otherwise try to destroy it.
In a news release announcing their additional funding Julia Greenstein, PhD, JDRF’s vice president of cure therapies called the research “very promising.”
The ability to encapsulate and thereby protect implanted insulin-producing cells has been a focus for JDRF because of its potential to solve multiple problems at once. ViaCyte is currently at the forefront of developing this technology, making this a very attractive research opportunity for us.
It’s not just patients with type 1 diabetes who could benefit from this therapy. Patients with insulin-dependent type 2 diabetes could as well. Diabetes costs California a whopping $24 billion every year, so being able to cut that even by a small amount would be hugely important.
Here’s our video about this CIRM and JDRF supported Viacyte project:
K.M.
The Stem Cellar 
ALS Community Tells FDA ‘We Have No Time To Waste’; Urges Changes in Trial Design and Review Process
Because of the relentless effects of ALS, a diverse group of ALS voices — patients, families, organizations and the community — came together to speak about their experiences with ALS and to urge the FDA to work with them to overcome some of the obstacles faced in ALS research, clinical trials and standards of care.
Among the strongest and most poignant voices in the room — some of them computer-generated — came from people with ALS, whose common themes included weighing the benefits versus risks in participating in early-phase clinical trials.
“When a trial is going well, those with ALS should be able to keep taking the drugs,” said Robert Anderson, an individual living with ALS. “What is the risk — you think it might kill him sooner? It does not matter, if you may be dying in a year from ALS anyway. You need to step beyond that in your heads.”
Physical therapist Sara M. Feldman, who works at the MDA/ALS Center of Hope in Philadelphia, underscored Anderson's comments.
“The people we work with that come into these clinical trials are truly heroes,” she said. “They're doing this not because they think they're going to benefit from it, but because they are hoping that someone will benefit in the future.”
Throughout the day, the panel heard similar comments urging the FDA to think differently about its clinical trial and drug approval process.
“Regulatory barriers inhibit treatment, based on a traditional model that I believe should be updated,” said Jonathan Glass, a physician conducting a phase 2 clinical trial at Emory University in Atlanta involving the injection of neural stem cells into the spines of people with ALS. “A major impediment to developing new treatments is adherence to standardized paths that are counterproductive and do not address the complexities of this disease.”
The ALS community made it clear it wants to work with the FDA to address and fast-track solutions to its unique needs.
http://mda.org/media/press-releases/als-community-tells-fda-we-have-no-time-waste-urges-changes-trial-design-and