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This experimental autologous mesenchymal stem cell (MSC) therapy is an Advanced Therapy Medicinal Product (ATMP) developed by Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico for the treatment of Progressive Supranuclear Palsy (PSP). The therapy involves harvesting mesenchymal stromal cells from a patient's own bone marrow, expanding them under Good Manufacturing Practice (GMP) conditions in the hospital's Cell Factory, and administering them via intra-arterial infusion through the internal carotid arteries. The mechanism of action is hypothesized to involve the secretion of neurotrophic factors, anti-apoptotic signals, and anti-inflammatory cytokines that support surviving neurons and modulate the neurodegenerative environment. A Phase 1 clinical trial (NCT01824121) evaluated the safety and preliminary efficacy of this approach, utilizing a randomized, double-blind, sham-controlled crossover design.
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