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This investigational cell therapy consists of allogeneic (donor-derived) adipose-derived mesenchymal stem cells (AD-MSCs) developed by the Royan Institute in Tehran, Iran, for the treatment of amyotrophic lateral sclerosis (ALS). The therapy is administered intravenously and leverages the multipotent properties of mesenchymal stromal cells, which are known for their immunoregulatory, anti-inflammatory, and neuroprotective capabilities. The biological mechanism involves the delivery of neurotrophic factors and immunomodulation to support the survival of upper and lower motor neurons, which are progressively destroyed in ALS. Clinical evaluation, including a Phase 1 study (NCT02492516), has primarily focused on assessing the safety and tolerability of a 2 million cells/kg dose in patients with sporadic ALS.
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