Stem Cell Therapy For Diabetes: UCSF and Novocell Receive a Huge New Grant

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I just got word that a company named Novocell and a team of researchers from the University of California San Francisco (UCSF), headed by Jeffrey Bluestone, have received a huge grant for work on a cellular therapy for diabetes. How big? $20 million. That has to make for a good morning.

The money comes from the California Institute for Regenerative Medicine, which was created in 2004 after California voters passed Proposition 71, the California Stem Cell Research and Cures Act. (Remember that one? It set aside $3 billion — yes, billion — for stem cell research at California universities and research institutions. Who cares that our infrastructure is literally falling apart? We’re leading the pack when it comes to stem cells.)

The goal of this particular grant is to help Novocell and the UCSF team develop a “first-in-kind cellular therapy for the treatment of diabetes.” Translation: they’re trying to figure out a way to develop a renewable source of replacements for the insulin-producing cells that are lost when you develop Type 1 diabetes. (Novocell also hopes these cells will be useful for people with Type 2.)

This line of research is hugely exciting but it does come with important caveats. First of all, any transplant that’s not from your own body carries a risk of rejection — which means that much like a donor kidney, an islet transplant requires you to take immunosuppressive drugs to stop your body from rejecting it. And second, when it comes to islet transplants, people with Type 1 are doubly screwed:  the fact that Type 1 is an auto-immune disease means that something has triggered our immune systems to want to attack our own insulin-producing cells. In other words, even if we managed to make the new islets look like they came from our own bodies (sidestepping the rejection issues faced by a non-diabetic person), we would still be at risk: the same part of our immune system that killed off our original islet cells could decide to strike again.

The goal is to figure out a way to achieve tolerance — current approaches include either encapsulating the islets so that our immune systems can’t get to them, or “reprogramming” our immune systems into not attacking the transplanted cells in the first place. It’s an enormous challenge, but it’s one that I know Bluestone is particularly well qualified to face. He’s been working for some time on trials involving something called an anti-cd3 monoclonal antibody, which aims to stop the immune systems of newly diagnosed diabetics before they can kill off all the pancreas’s insulin-producing cells  — ideally stopping the disease in its tracks. (As a participant in a 2001 trial of that drug, I can personally vouch for it success — it’s been nearly 9 years since I received the treatment and I still produce a measurable amount of insulin.)  Bluestone also made headlines again last year when he found that two popular anti-cancer drugs can reverse type 1 diabetes in mice.

Bluestone himself is quick to point out that the results of the cancer-drug study came with many caveats, including the fact that drugs, while approved for use in people with cancer, have not yet been tested in diabetic humans. Nonetheless, I’m excited — both about that particular project, and about the potential for this new grant. Bluestone is at the top of the field in diabetes research. Novocell’s work looks promising. And while a stem-cell-based cure for diabetes is still likely a long way off, I’m thrilled to hear that this project received such great funding. Like countless other diabetics, I wish them enormous success.

For more information on this grant, check out the press release, or this article about CIRM from the New York Times.

Some additional information about Novocell (from the company’s press release):

Novocell is a preclinical therapeutic company focused on diabetes. Our therapy is based on the encapsulation and implantation of pancreatic beta cell progenitors (Pro-Islet-1) derived from human stem cells which secrete insulin in patients. Our goal is that Pro-Islet-1 can free both Type 1 and Type 2 diabetic patients from insulin dependence on a long term basis, while reducing or eliminating hypoglycemic, microvasculature and weight-mediated cardiovascular complications. Novocell is a private company headquartered in San Diego, California, with additional operations in Athens, Georgia.

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