-Craig Idlebrook and the staff at T1D Exchange
On September 3rd, Vertex Pharmaceuticals, a biotechnology company, announced it will purchase Semma Therapeutics, which is developing an innovative cellular therapy to treat type 1 diabetes. The price paid will be $950 million.
Semma Therapeutics has been building on the work of molecular biologist Douglas Melton, PhD, of the Harvard Stem Cell Institute. In 2014, Melton made headlines when he announced that he and his research team had discovered a way to vastly improve the laboratory production of insulin-producing beta cells. These cells had been notoriously difficult to grow in a lab setting, and previous efforts at beta cell replacement therapy largely involved harvesting cells from donors and trying to coax their maturation in petri dishes.
Melton and his team devised a clever two-step way to identify and collect beta-like cells that had undergone this maturation process, thereby enriching mixes of cells that could produce insulin. The first step involved sequencing the DNA of each cell and pinpointing those cells with the right genetic signature to mark its beta cell qualities. The second involved using a protein made only by the mature cells as a hook by which to fish beta cells out of a cellular mix.
Collaborating scientists at Semma Therapeutics later added in a second method for further enriching beta cells. They took advantage of the beta cells’ propensity to cluster together once mature. The combination of these two innovative practices helped increase the yield of beta cells from a donor sample from 30 percent to 80 percent, according to a report in The Harvard Gazette. This work was significantly supported by grants and philanthropy focused on advancing type 1 diabetes, according to the report.
The mass production of insulin-producing beta cells is just one half of what’s needed to make beta cell transplant therapy successful; the transplanted cells also must be shielded from the immune system of a person with type 1 diabetes, which would otherwise attack and kill the cells, damaging the body’s blood sugar management. It appears that Semma’s scientists have devised a way to successfully encapsulate the cells to protect them once implanted, according to Vertex’s announcement.
The therapy is in the preclinical stage of development, and it has successfully restored insulin secretion in animal models. While promising news, this therapy still must prove itself in human clinical trials before it will be ready for submission to the FDA.
Our friends at JDRF New England honored Semma Therapeutics two years ago with the Nancy Jones Diabetes Champion Award recognizing them for their vision and innovative approach toward curing type 1 diabetes. Congratulations to the team!
To watch a video of Doug Melton’s work, see below: