Vertex Early-Stage Diabetes Treatment Wows Patients and Clinicians

 In October, Vertex Pharmaceuticals announced stunning results in a type 1 diabetes patient who had been dosed with the company’s experimental fully differentiated pancreatic islet cell replacement therapy. After an infusion of VX-880, the patient began to produce his own insulin.

Over the holiday weekend, The New York Times profiled the patient’s success story. Brian Shelton was a former postal delivery carrier forced into early retirement due to diabetes. On multiple occasions, his blood sugar fell and he lost consciousness. Several of the incidents occurred while he was driving. Shelton was enrolled in the Vertex Phase I study and became the first to be infused with the company’s experimental treatment.

Six months after he was treated, Shelton’s body is now producing its own insulin—something he had never been able to do. When interviewed by the Times, Shelton likened the results he has seen to a miracle.

“It’s a whole new life,” he told the newspaper.

Shelton was first diagnosed with type 1 diabetes more than 40 years ago. He was insulin-dependent. According to Vertex, before enrolling in the Phase I study, Shelton took 34 units of insulin per day and had undetectable fasting and stimulated C-peptide levels—a signal that he could not produce his own insulin. Ninety days after treatment with the Vertex asset, Shelton’s glucose-responsive insulin production had been restored.

While Shelton is experiencing a new lease on life thanks to Vertex, there is still a long way to go before the Boston-based company will be able to potentially bring VX-880 to market. The Phase I/II study that Shelton took part in is a five-year study and will include 17 patients with severe cases of type 1 diabetes.

Diabetes researchers outside of Vertex and its clinical associates have called the results seen in Shelton remarkable. Peter Butler, M.D., a research scientist at UCLA, expressed excitement about the potential of VX-880.

“It is a remarkable result,” Butler told the Times. “To be able to reverse diabetes by giving them back the cells they are missing is comparable to the miracle when insulin was first available 100 years ago.” 

VX-880 was initially developed by Harvard University biologist Doug Melton, Ph.D., who began seeking a potential therapy for his children who had been diagnosed with diabetes.

Type 1 diabetes is an autoimmune disease, which is historically difficult to manage. The body’s immune system attacks and destroys insulin-producing cells, known as beta cells, in the pancreas until the pancreas stop producing insulin. There are currently no cures or preventative measures for type 1 diabetes.

Currently, there are more treatment options for type 2 diabetes, but there are multiple companies aiming at type 1 diabetes. The Salk Institute is developing an insulin-producing pancreatic cell cluster to treat type 1 diabetes. Although still in the preclinical stages, the data has shown promise. As BioSpace previously reported, pancreatic cell clusters were developed with stem cell technology. Earlier this year, vTv Therapeutics, announced positive results from its lead diabetes candidate, TTP399. The N.C.-based company data suggested that TTP399 can lower blood glucose in patients without increasing the risks of diabetic ketoacidosis, which is a serious complication of the disease. TTP399, a novel, oral, once-daily glucokinase activator, is being developed as adjunctive therapy to insulin for type 1 diabetes. vTv plans to initiate a late-stage study of TTP399, which was previously granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA).

https://www.biospace.com/article/type-1-diabetes-patient-calls-results-of-vertex-s-early-stage-treatment-a-miracle-/