Drug intelligence / Profile preview

cyclophosphamide + fludarabine + thiotepa + melphalan + muromonab-CD3

Development stage
Unknown
Lead developer
St. Jude Children's Research Hospital
Modality
Monoclonal Antibodies → Antibody-Based Therapeutics, Small Molecules
Administration
Intravenous
01

Overview

This multi-agent conditioning regimen was developed by St. Jude Children's Research Hospital for use in haploidentical hematopoietic stem cell transplantation (HSCT) in infants and children under two years of age with high-risk hematologic malignancies. The regimen consists of a combination of chemotherapy agents—cyclophosphamide, fludarabine, thiotepa, and melphalan—alongside the monoclonal antibody muromonab-CD3 (OKT3). It is designed to provide intensive cytoreduction and profound immunosuppression to facilitate the engraftment of T-lymphocyte-depleted haploidentical grafts (often selected using the CliniMACS system) and subsequent natural killer (NK) cell infusions. The primary goal of this specific combination is to minimize regimen-related toxicity and graft-versus-host disease (GVHD) while maximizing anti-leukemic effects in a vulnerable pediatric population.

Other names
Chemotherapy and antibodiesSt. Jude haploidentical transplant conditioning regimenOKT3-based conditioning regimenOKT-3-based conditioning regimenOKT 3-based conditioning regimen
02

Targets

CD3 (T-cell surface glycoprotein CD3)DNAPOLA1 (DNA polymerase alpha)POLD1 (DNA polymerase delta)DNA-directed primase/polymerase protein (PrimPol)RNR (Ribonucleotide reductase)DNA polymerase family

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