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autologous hematopoietic stem cell transplantation (Burt Stem Cell Institute) (Richard Burt)
- Development stage
- Unknown
- Lead developer
- Northwestern University
- Modality
- Stem Cell Therapies → Cell Therapies, Small Molecules, Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
This therapeutic procedure involves autologous hematopoietic stem cell transplantation (HSCT) for the treatment of refractory autoimmune bullous skin disorders, specifically pemphigus vulgaris and pemphigus foliaceus. Pioneered by Dr. Richard K. Burt at Northwestern University, the protocol employs a conditioning regimen consisting of high-dose cyclophosphamide (a DNA-alkylating chemotherapy agent) and anti-thymocyte globulin (ATG, a polyclonal antibody biologic) to eliminate the patient's autoreactive immune cells. Following this lymphodepletion, previously harvested autologous hematopoietic stem cells are re-infused to restore blood production and reset the immune system, aiming to re-establish long-term immune tolerance and achieve treatment-free remission in patients who have failed conventional immunosuppressive therapies.
- Other names
- HSCTautologous HSCTBurt protocolhigh-dose cyclophosphamide and ATG with hematopoietic stem cell support
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