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PTC-MSC is an investigational allogeneic, third-party bone marrow-derived mesenchymal stromal cell (MSC) therapy developed by Azienda Ospedaliera San Gerardo di Monza in Italy. The therapy consists of MSCs expanded ex vivo in platelet lysate, designed to treat severe (grade II–IV) steroid-resistant acute graft-versus-host disease (GVHD) and acute phases of chronic GVHD following allogeneic hematopoietic stem cell transplantation. Its mechanism of action involves the secretion of immunomodulatory proteins, transfer of mitochondria, and release of exosomes or microvesicles, which collectively promote an anti-inflammatory environment and regulate the host's immune response. The product was evaluated in a Phase I clinical trial (NCT01764100) to assess feasibility, safety, and preliminary efficacy in patients who failed conventional immunosuppressive therapy.
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