Drug intelligence / Profile preview

cyclophosphamide + anti-thymocyte globulin + autologous stem cell transplant

Development stage
Unknown
Lead developer
Northwestern Memorial Hospital
Modality
Stem Cell Therapies → Cell Therapies, Small Molecules, Antibody-Based Therapeutics
Administration
Intravenous
01

Overview

This refers to an intensive immunoablative conditioning regimen followed by autologous hematopoietic stem cell transplantation (HSCT) developed at Northwestern Memorial Hospital for the treatment of severe, refractory systemic lupus erythematosus (SLE). The protocol involves mobilizing peripheral blood stem cells using low-dose cyclophosphamide and G-CSF, followed by CD34+ cell selection to deplete lymphocytes. The conditioning phase consists of high-dose cyclophosphamide (200 mg/kg) and anti-thymocyte globulin (ATG), which aims to eliminate the pathogenic self-reactive immune system. Finally, the purified CD34+ stem cells are reinfused to facilitate the reconstitution of a new, potentially self-tolerant immune system. This Phase I study targets patients with life-threatening or refractory lupus manifestations, such as glomerulonephritis or vasculitis, who have failed standard therapies.

Other names
Immune Ablation and CD34+ PBSC Support in SLEHigh-dose cyclophosphamide and ATG with ASCT
02

Targets

DNA

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