Vertex Pharmaceuticals Will Launch a Clinical Trial of Stem Cell-Derived Beta Cells

Last week’s breakthrough news—that Vertex Pharmaceuticals will launch a clinical trial of VX-880, a stem cell-derived beta cell therapy in type 1 diabetes (T1D)—had its start in 2000.

That’s when JDRF funded Douglas Melton, Ph.D., to make beta cells from stem cells. In 2014, he did it. Recognized by Science magazine as one of the top discoveries of 2014, this advancement was a result of decades of JDRF funding aimed at overcoming cell shortages as a significant obstacle to beta cell replacement therapy.

In 2015, Dr. Melton founded Semma Therapeutics to develop these stem cell-derived beta cells into a potentially curative treatment for T1D.

In early 2017, the JDRF T1D Fund—JDRF’s innovative venture philanthropy fund—took notice, and made a catalytic investment in Semma, to bridge the company to its next large venture round, which took place at the end of that year.

In September 2019, Vertex made headlines when it acquired Semma for almost $1 billion, the largest transaction ever in a T1D cure-based therapeutic program and one of the largest transactions ever for a biotech company not yet in human clinical trials.

Now, Vertex announced FDA-clearance of their Investigational New Drug (IND) application for VX-880, which uses stem cell-derived beta cells to try to restore the body’s ability to produce insulin, in combination with immunosuppressive therapy to protect the cells from rejection. They will initiate a phase I/II clinical trial in the first half of 2021 in people with T1D with severe low blood-sugar (called hypoglycemia) events and those who struggle to perceive the onset of hypoglycemia.

If the results of the clinical trial are successful, we will be moving ever closer to a world without this disease.