DIABETES vs. THE CALIFORNIA STEM CELL PROGRAM: Disease-a-week Challenge #11
By Don C. Reed
Legend has it that the great Babe Ruth once predicted his own home run, pointing to the distant fence—before clouting that ball clean out of Wrigley Park.
Imagine the impact of such a home run moment, not in sport, but in the field of stem cell research. What disease would you point to, ready for a game-changing breakthrough?
As the father of a paralyzed young man, Roman Reed, naturally I would want spinal cord injury to be cured first.
But my unbiased over-the-fence prediction? I would have to say Type 1 diabetes, and a company called ViaCyte, Inc. (I hasten to add that I have no connection, financial or otherwise, with ViaCyte or any other biomed company; I wish success for all.)
Why Type 1 diabetes? Certainly this miserable condition deserves to be cured. Beside the pain of endless injections and finger pricks, diabetes carries the threat of foot amputation, even death, as happened to members of my own family. Approximately 1.5 million Americans are afflicted with Type 1. The medical expenses they face are staggering: an estimated $14.9 billion last year.
With the strong financial assistance and scientific support of JDRF and the California stem cell program, ViaCyte , Inc. has come up with an invention which could be a functional cure for Type 1 diabetes.
ViaCyte is developing the VC-01 product candidate, a cell replacement therapy, said ViaCyte President and Chief Executive Officer Paul Laikind, Ph.D, in a recent interview.
The active component of the VC-01 product is composed of pancreatic progenitor cells (the “before cells” which become the ones you really want) derived from a single embryonic stem cell line that was established by the company. These cells, called PEC-01 cells, are encapsulated in a device called the Encaptra drug delivery system. The Encaptra device currently being tested is about half the size of a credit card, and very thin. Tiny holes perforate it, like a superstrong teabag.
Type 1 diabetes happens when the body cannot process its sugar intake properly, because the insulin-producing beta cells (responsible for that job) are attacked by the immune system.
How might this be counteracted?
In the lab, embryonic stem cells are made into progenitor cells. When inside the Encaptra device, which is then put into the body (just under the skin), the progenitor cells turn into beta cells and other cells which regulate blood sugar levels. These cells produce insulin which goes out through the pores of the Encaptra device, and does its job.
Interestingly, the holes are small enough to keep the patient’s immune cells out (which might otherwise kill the implanted cells) but large enough to release insulin and other endocrine hormones, as well as letting in the needed nutrients.
The VC-01 product candidate (the special cell line combined with the Encaptra device) has cured experimentally-induced diabetes in hundreds of laboratory mice.
When will they be able to try it on people?
The Food and Drug Administration (FDA) has said yes to ViaCyte’s clinical trials on humans.
Already, several patients participating in the STEP ONE clinical trial have the VC-01 product in their bodies. These are all adults, chosen because without beta cells they have no way to make the insulin on their own. The first group of patients is receiving a low dose of cells in the devices, simply to evaluate the safety of the product candidate and establish the procedures for implanting it. If all goes well, additional patients will receive a higher dose of cells in the Encaptras that may provide therapeutic benefit.
But what if things go wrong, as sometimes happens with experimental treatments?
The device is designed to be easily removed.
Is it permanent? Probably not. As things stand now, replacements may have to periodically inserted, perhaps every couple of years. “How often?” is one of the questions the company hopes to determine with clinical testing.
With the VC-01 product, will the patient have to keep injecting insulin? Probably not. If it works as intended, it will be a huge improvement in their lives, much better than the current state of constant guesswork: trying to balance food intake and exercise, rest, and insulin injections.
How do we know this product will work? We don’t. It works in animals, but translating that to humans is always a challenge. That is what the clinical trials are designed to determine.
Who are the scientists involved? People like Kevin D’Amour, Howard Foyt, Allan Robins, Olivia Kelly, Evert Kroon, and many more.
If ViaCyte wins, will they make a lot of money? I certainly hope so. Medical product development is notoriously costly. If a major breakthrough can be shown to be financially worth the risk, other companies and corporations may be less likely to shy away. The long years of effort will pay off.
Is ViaCyte alone in its efforts? No. There are many worthy fighters in this battle: people like the tenacious and talented Doug Melton, backed by JDRF and the Harvard Stem Cell Institute.
And the California stem cell program, which began life as the citizen initiative Proposition 71? Investing almost eighty million dollars ($79,748,841) in diabetes cure research, the California agency supports not only ViaCyte, but also the work of other top scientists, including:
• Mark Anderson, Didier Stainer and Jeffrey Bluestone of UC San Francisco;
• David Tirrell of the California Institute of Technology;
• Roslyn Isseroff of UC Davis;
• Charles King, Maike Sander, Catriona Jamieson, and Yang Xu of UC San Diego.
Whoever wins, may they grow big as General Motors.
Because when that scientific home run happens, it will benefit every nation on this Earth.
Don C. Reed is the author of the forthcoming book, STEM CELL BATTLES: Proposition 71 and Beyond: How Ordinary People Can Fight Back Against the Crushing Burden of Chronic Disease.