Drug intelligence / Profile preview

alemtuzumab + fludarabine + melphalan (Washington University School of Medicine)

Development stage
Unknown
Lead developer
Washington University School of Medicine
Modality
Monoclonal Antibodies → Antibody-Based Therapeutics, Small Molecules
Administration
Intravenous
01

Overview

This reduced-intensity conditioning (RIC) regimen was developed by the Washington University School of Medicine for children with severe thalassemia major undergoing unrelated donor hematopoietic cell transplantation (HCT). The regimen specifically combines alemtuzumab (a CD52-directed monoclonal antibody), fludarabine (a purine analog), and melphalan (an alkylating agent), often supplemented with thiotepa and hydroxyurea as part of the URTH trial protocol. The goal of this combination is to provide sufficient host immunosuppression to allow for stable engraftment of donor cells while minimizing the toxicities associated with traditional myeloablative conditioning. Alemtuzumab serves to deplete host T-cells to prevent graft rejection and graft-versus-host disease (GVHD), fludarabine provides lymphotoxicity and inhibits DNA synthesis, and melphalan acts as a cytoreductive agent to create space in the bone marrow.

Other names
URTH Trial conditioning regimenReduced Intensity Conditioning for Thalassemia
02

Targets

DNADNA polymerase familyCD52 (CD52 Antigen)

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