An orphan drug designation for a stem cell therapy (Prochymal; Osiris Therapeutics Inc.) in patients with newly diagnosed type 1 diabetes mellitus has recently been approved by the U.S. Food and Drug Administration.

The product is an intravenous formulation of adult mesenchymal stem cells (MSC) that are isolated from the bone marrow of healthy young adult donors, thereby avoiding the controversy associated with embryonic and fetal cell sources. Cell culture allows the large-scale production of thousands of doses from a single donation.

According to Osiris Therapeutics, MSCs are designed to provide therapeutic benefit by controlling inflammation, promoting tissue regeneration, and preventing scar formation. Preclinical studies indicate that MSCs may delay the progression of type 1 diabetes by preserving beta cell function.

A 62-patient, double-blind, placebo-controlled phase 2 study being conducted at 20 medical centers across the United States is evaluating the safety and efficacy of this stem cell therapy. In order to be eligible, patients must be aged 12-35 years and have been diagnosed 2 to 20 weeks before study entry.

The MSC therapy previously was granted FDA orphan drug status and expanded access approval for graft-versus-host disease, allowing its use in patients whose condition is life-threatening. Other potential indications under investigation include Crohn’s disease, myocardial infarction, and pulmonary disease.

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