One year after Osiris Therapeutics in partnership with JDRF started the Phase 2 trial evaluating Prochymal, a formulation of adult mesenchymal stem cells (MSCs), in newly diagnosed type 1 diabetes patients, the company has released a statement providing an update on this groundbreaking trial. This first-of-its kind trial is testing MSCs from unrelated adult donors in 63 pediatric and adult patients to assess the safety of MSCs in this population and whether the treatment shows signs of slowing progression of the disease.
Prochymal is designed to provide therapeutic benefit by controlling inflammation, promoting tissue regeneration, and preventing scar formation. In type 1 diabetes, the patient’s own immune system attacks and destroys insulin-producing islet cells in the pancreas resulting in the loss of blood-sugar control. Currently, there are no approved treatments for altering the rate of destruction of these critical islet cells, called beta cells.
The participants in the trial were randomly assigned to receive either Prochymal or placebo, and both the physicians and patients remain blinded as to which patients received stem cells.
The interim assessment at one year showed that systemic infusions of Prochymal were well-tolerated in this unique population. There were no differences in adverse event rates between the Prochymal and placebo groups. Importantly, no patients experienced a reaction to the infusions despite the cells being unrelated to the recipient, unmatched, and used without immunosuppression. No significant differences in the rates of disease progression, as measured by stimulated C-peptide levels at the one year time point, have been observed. However, there was a trend towards fewer hypoglycemic events for patients treated with Prochymal as compared to controls. The patients will be followed for another year (for a total of two years), after which time a complete analysis of the data will be conducted.