FDA Green Lights Trials of New Type 1 Diabetes Stem Cell Therapy

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The United States Food and Drug Administration (FDA) has given the green light for experimental trials of a truly new therapy for Type 1 diabetes.

The new therapy, named VX-880, is referred to in a press release as an “investigational stem cell-derived, fully differentiated pancreatic islet cell therapy to treat T1D.” In plain English, VX-880 uses laboratory stem cells that have been grown into insulin-producing pancreatic beta cells. Those cells are then transplanted into a patient with Type 1 diabetes, and act just as the patient’s own beta cells should, hopefully restoring the body’s ability to sense glucose levels and secrete insulin in response.

It’s an exciting moment for the treatment. Vertex, the business developing VX-880, has already run successful proof-of-concept animal testing. The therapy is ready for the next huge hurdle, tests in humans.

While diabetes technologies and therapies have improved at a rapid pace over recent decades, there has still be very little meaningful work done on reversing the root cause of diabetes: the autoimmune reaction that destroys the beta cells in the pancreas that secrete insulin. A therapy that could restore pancreatic function could potentially function as an actual cure, correcting the root dysfunction rather than just mitigating its effects.

For now, that’s still a dream— there is a very long road for the therapy to travel before we can realistically talk about it as an option for patients. People with diabetes can be understandably skeptical about potential cures, having heard about so many breakthroughs over the years that have thus far amounted to little or nothing. Only a thin minority of the drugs that begin Phase 1 clinical testing ever make it to the market. Even if it works, we don’t yet know if VX-880 could wholly restore beta cell function, or only partially restore it, necessitating the continued use of some exogenous insulin.

While practical application may be a long way away, the therapy has already come pretty far to get to this point. Its genesis began years ago with the work of Dr. Douglas Melton of the Harvard Stem Cell Institute. Melton founded a company, Semma Therapeutics, to work on the therapy. Last year, Semma Therapeutics was acquired by a larger biotech firm, Vertex, for nearly $1 billion. That’s an eye-opening bet on the technology.

Dr. Melton is extremely optimistic about his breakthrough. In a 2019 interview, he stated, “I’m convinced that those cells will cure the disease.”

Of course, even a Type 1 diabetes “cure” could come with strings attached. VX-880, for example, will require the “chronic administration of concomitant immunosuppressive therapy” in order to prevent the new islet cells from rejection by the body’s immune system. No word yet on how burdensome immunosuppressive therapy might be for patients. Vertex has also investigated other methods of protecting new islet cells from the immune system; another concept would implant new islet cells in a kind of porous “tea bag” that would allow both glucose and insulin to filter through, but block immune cells.

Initial trials will restrict VX-880 to some of the patients most in need of a radical therapy, those with hypo unawareness and a history of severe hypoglycemia. The company will recruit about 17 patients for the Phase 1 testing.

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