Promising New Treatment for Type 1 Diabetes

The results of a phase 1 trial published in the November 25th online edition of the journal, Science Translational Medicine, offer insight into a possible new treatment for type 1 diabetes.

The underlying cause of type 1 diabetes is autoimmune attack on the insulin-secreting cells in the pancreas.  Many treatments are already directed at suppressing the immune system but cause grave potential side effects such as cancer and infections.

These treatments include various medications as well as bone marrow transplantation.

Immune cells in the body, called regulatory T cells (“Treg cells”), protect the insulin-producing cells in the pancreas from immune system attacks. Treg cells have been shown to be defective in autoimmune diseases. Thus, the researchers in this study hypothesized that replacing Treg cells in patients with type 1 diabetes could reverse autoimmunity and protect the remaining insulin-producing cells.

The 5-year trial followed 14 patients aged 18-43 with newly diagnosed Type 1 diabetes. Doctors extracted several million Treg cells in roughly 2 cups of blood and replicated the Treg cells to produce several billion copies of themselves.  The billions of Treg cells were then infused into the bloodstream in order to protect the pancreatic islet cells.

After 12 months, nearly a quarter of the infused Treg cells remained and, more importantly, continued to effectively protect the pancreatic islet cells without any serious side effects, such as infections or infusion reactions.

Ideally, early treatment with Treg cells could help avoid the need for daily insulin injections and thereby halt disease progression.

The treatment has possible implications for other autoimmune diseases such as rheumatoid arthritis and lupus.

Since Phase 1 trials typically only examine if a drug or intervention is safe without focusing on efficacy, the next phase of this research would be a trial to test the effectiveness of Treg cells as a treatment for type 1 diabetes.

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