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autologous hematopoietic stem cells-Northwestern University-Stiff-Person Syndrome
- Development stage
- Unknown
- Lead developer
- Northwestern University
- Modality
- Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
This investigational cell therapy approach involves autologous hematopoietic stem cell transplantation (HSCT) specifically developed and studied by Northwestern University for the treatment of severe, treatment-refractory Stiff-Person Syndrome (SPS) and its variants, such as Progressive Encephalomyelitis with Rigidity and Myoclonus (PERM). The therapy utilizes a patient's own hematopoietic stem cells, which are collected and then reinfused following a non-myeloablative conditioning regimen. This regimen typically consists of high-dose immunosuppression using cyclophosphamide, anti-thymocyte globulin (ATG), and rituximab to deplete the existing dysfunctional immune system. The goal of the procedure is to "reset" the immune system, thereby halting the autoimmune attack on glutamic acid decarboxylase (GAD) and other neural targets. Clinical results from Phase 1/2 trials have demonstrated sustained clinical improvement in stiffness, ambulation, and functional status in patients with anti-GAD65 antibodies.
- Other names
- Autologous Hematopoietic Stem Cell TransplantationHSCT for Stiff Person SyndromeNon-myeloablative HSCTAutologous HSCT
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