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Mayo Clinic's autologous adipose-derived mesenchymal stromal cell (MSC) therapy is an investigational biologic treatment for amyotrophic lateral sclerosis (ALS). The process involves harvesting a patient's own adipose tissue via fat biopsy, followed by the isolation and ex vivo expansion of MSCs in the Mayo Clinic Human Cellular Therapy Laboratory. These expanded cells are then administered back to the patient via intrathecal injection into the cerebrospinal fluid. The therapeutic mechanism is hypothesized to be multifactorial, involving the paracrine secretion of neurotrophic factors to support motor neuron survival and the modulation of neuroinflammatory pathways to slow disease progression. Clinical studies, including Phase I safety trials, have demonstrated that the procedure is generally well-tolerated, with transient pain being the most common adverse effect, warranting further exploration in Phase II efficacy trials.
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