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This investigational cell therapy consists of autologous mesenchymal stem cells (MSCs) harvested from the patient and administered via intrathecal infusion. Developed by Hospital e Maternidade Dr. Christóvão da Gama in Brazil, the therapy is being evaluated for the treatment of amyotrophic lateral sclerosis (ALS). The mechanism of action relies on the therapeutic plasticity of MSCs, which are thought to provide neurotrophic support, reduce neuroinflammation, and potentially slow the degeneration of motor neurons. A Phase 1 clinical trial (NCT02987413) has investigated the safety and tolerability of escalated doses of these cells in ALS patients to determine the feasibility of this approach before proceeding to larger efficacy studies.
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