Made famous in the tech industry, moonshots are ambition ventures with the promise of near-term profitability. In the pharmaceutical industry, Vertex Pharmaceuticals, known for its robust cystic fibrosis treatments (CF), is currently undertaking a moonshot of its own, the treatment of Type 1 Diabetes.
Type 1 Diabetes afflicts 1.5 million Americans and requires a rigorous routine of insulin maintenance. If patients do not monitor their insulin levels, they can lose limbs, become blind, or die. In fact, diabetes is the leading cause of blindness in the U.S. Short of a transplant of the cells in the pancreas that produce insulin, there is no cure for Type 1 Diabetes.
Nevertheless, Vertex Pharmaceuticals recently made headlines for curing a man of Type 1 Diabetes. According to the New York Times, “Mr. Shelton, now 64, may be the first person cured of the disease with a new treatment that has experts daring to hope that help may be coming for many of the 1.5 million Americans suffering from Type 1 Diabetes.” The Times article explores how Mr. Shelton, a retired postal worker, enrolled in a Vertex study that infused enrollees with insulin-producing cells grown from stem cells.
Once infused, Mr. Shelton has to take immunosuppressant drugs to prevent his body from rejecting the infused cells. Ostensibly, the treatment appears to have worked because Mr. Shelton has not experienced a rapid rise or reduction in his insulin levels.
Experts caution that more time and data is needed in order to prove efficacy. Key caveats to the treatment include the length of time the infusion cells last, the long-term effects of the infusion, and the effects of the immunosuppressant drugs.
Despite these caveats, the treatment has granted Mr. Shelton a whole new way of life. In fact, Vertex’s treatment has the potential to improve hundreds of thousands of patients’ lives and radically upend the insulin market.