Drug intelligence / Profile preview

Alemtuzumab + Cyclophosphamide + Busulfan

Development stage
Unknown
Lead developer
Masonic Cancer Center
Modality
Small Molecules, Stem Cell Therapies → Cell Therapies, Monoclonal Antibodies → Antibody-Based Therapeutics
Administration
Intravenous
01

Overview

This myeloablative preparative regimen is a combination conditioning therapy used prior to allogeneic hematopoietic stem cell transplantation (HSCT). Developed and evaluated by the Masonic Cancer Center at the University of Minnesota, specifically in the MT2014-10C clinical trial, the regimen consists of the monoclonal antibody alemtuzumab and the alkylating agents cyclophosphamide and busulfan. It is designed for patients with high-risk hemoglobinopathies and other non-malignant red cell transfusion-dependent disorders, including sickle cell disease, thalassemia, and Diamond-Blackfan anemia. Alemtuzumab serves as a potent lymphodepleting agent to prevent graft rejection and graft-versus-host disease (GVHD), while cyclophosphamide and busulfan provide intensive myeloablation to clear the recipient's bone marrow, facilitating the engraftment of donor stem cells.

Other names
Myeloablative Preparative Regimen-Masonic Cancer Center, University of Minnesota-sickle cell disease-thalassemia-Diamond-Blackfan anemia-paroxysmal nocturnal hemoglobinuria-Glanzmann's thrombasthenia-severe congenital neutropenia-Shwachman-Diamond syndromeMyeloablative Preparative Regimen (University of Minnesota)Alemtuzumab/Cyclophosphamide/Busulfan conditioning regimen
02

Targets

DNACD52 (CD52 Antigen)

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