Vertex Pharmaceuticals has decided to pause clinical trials of its celebrated type 1 diabetes therapy, VX-880, after two participants receiving the treatment died. In a statement, Vertex clarified that the 2 deaths were “unrelated” to its experimental therapy. Nevertheless, the biotech firm has placed the study “on a protocol-specified pause, pending review of the totality of the information by the independent data monitoring committee and global regulators.”
Miriam Tucker of Medscape was the primary to report the news.
Within the race to search out a cure for type 1 diabetes, VX-880 stands out as the world’s most promising lead. The therapy involves a transplant of recent pancreatic islet cells which have been grown in a laboratory from pluripotent stem cells. These latest lab-grown cells can sense blood sugar concentrations and secrete insulin, similar to healthy natural islet cells, replacing the cells which can be lost within the autoimmune attack that defines type 1 diabetes.
VX-880 remains to be in an early phase of experimentation — thus far, only 14 patients have received the treatment. But the outcomes have been spectacular. In June, when Vertex shared its latest data, we learned that two VX-880 patients had achieved true insulin independence, and each other patient had enjoyed significant improvements, including lower A1C, higher time-in-range, reduced insulin requirements, and freedom from severe hypoglycemia.
Considered one of the 2 patients to realize insulin independence, nonetheless, has now died. His name was Brian Shelton, and he was the face of Vertex’s innovation. Shelton, an Ohio resident originally from Brooklyn, was the primary patient to receive VX-880 and the primary to realize insulin independence with it.
In late 2021, the Recent York Times declared that Shelton had been “stands out as the first person cured of the disease” with the brand new therapy. Many individuals within the diabetes world disagreed with this assessment — Shelton still required immunosuppressive drugs to guard his latest islet cells, which introduces potentially significant unwanted side effects — but his results were nonetheless remarkable and well value celebrating. In an interview with Good Morning America, Shelton stated that he considered himself “cured.”
Shelton’s obituary, which notes that he remained “independent of insulin injections until his death,” asks for any memorial contributions to be sent to the pioneering type 1 diabetes charity JDRF. Many individuals from the diabetes community have left comments on the page, thanking Shelton for his bravery and selflessness in offering to undergo this vital experiment. One woman stated, “I cannot thank you adequate for giving me a reason to hope.”
Vertex is just now starting Phase 1 trials of a related therapy named VX-264. This therapy will use the identical lab-grown islet cells as VX-880, which have already proven so successful, but will encapsulate them in a physical device to shield them from the immune system. Ideally, the capsule would remove the necessity for immunosuppressive drugs, which not only cause unwanted side effects but will also be toxic to the implanted islet cells. If VX-264 succeeds — prior efforts to encapsulate islet cells have failed — some experts may consider it to be an authentic cure for type 1 diabetes. The trial remains to be recruiting volunteers in the US, Canada, England, and the Netherlands.