Drug intelligence / Profile preview

4-hydroperoxycyclophosphamide + amifostine

Development stage
Discontinued
Lead developer
Galera Therapeutics
Modality
Small Molecules
Administration
Ex Vivo, Intravenous
01

Overview

This is a combination of two agents used primarily in the context of hematopoietic stem cell transplantation and high-dose chemotherapy regimens. - **4-hydroperoxycyclophosphamide** (also known as perfosfamide) is an oxazaphosphorine alkylating agent and an active derivative of cyclophosphamide. It was developed for ex vivo purging of bone marrow or hematopoietic stem cells to eliminate malignant cells prior to autologous transplantation in patients with blood cancers such as acute leukemia and metastatic breast cancer. It acts by cross-linking DNA strands, leading to cell death in rapidly dividing malignant cells[6][8][9]. - **Amifostine** is a cytoprotective prodrug that is converted in tissues to an active thiol metabolite capable of scavenging free radicals and protecting normal tissues from the toxic effects of chemotherapy and radiation therapy without reducing antitumor efficacy. Amifostine has been shown to reduce cumulative toxicities such as myelosuppression, nephrotoxicity, neurotoxicity, mucositis, and taste disturbances associated with alkylating agents like cyclophosphamide[1][3][4]. In the context of high-dose chemotherapy or bone marrow transplantation using purged grafts (e.g., with 4-HC), amifostine may be administered systemically before chemotherapy or transplant infusion to protect normal host tissues from toxicity while not interfering with the anti-cancer effect[10].

Brand names
PergamidEthyol
Other names
perfosfamide + amifostine
02

Targets

DNA

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