allogeneic hematopoietic stem cell transplantation (Richard Burt protocol)

This investigational cellular therapy involves a non-myeloablative allogeneic hematopoietic stem cell transplantation (HSCT) protocol developed by Dr. Richard Burt at Northwestern University for the treatment of severe, refractory systemic sclerosis (scleroderma). Unlike the more common autologous HSCT, which uses the patient's own cells, this approach utilizes hematopoietic stem cells from a healthy, HLA-matched donor (typically a sibling). The procedure involves a conditioning regimen consisting of non-myeloablative doses of cyclophosphamide and fludarabine, combined with alemtuzumab (CAMPATH-1H) for T-cell depletion. The therapeutic intent is to achieve 'graft-versus-autoimmunity,' where the donor-derived immune system recognizes and eliminates the recipient's autoreactive lymphocytes, effectively resetting the immune system to a non-pathogenic state. This specific allogeneic approach remained experimental and did not progress beyond early-phase clinical investigation.

Other names

Non-myeloablative Allogeneic Hematopoietic Stem Cell TransplantationAllogeneic HSCTAllo-HSCT for Systemic Sclerosis