Drug intelligence / Profile preview

melphalan + carboplatin + mannitol + sodium thiosulfate

Development stage
Unknown
Lead developer
OHSU Knight Cancer Institute
Modality
Small Molecules
Administration
Intra-arterial, Intravenous
01

Overview

This therapeutic regimen is a combination of chemotherapy and supportive agents administered via a specialized delivery method for the treatment of recurrent or progressive central nervous system (CNS) embryonal or germ cell tumors. The protocol involves the intra-arterial (IA) administration of **melphalan** and **carboplatin**, both of which are DNA alkylating agents that inhibit tumor cell division. To enhance the delivery of these drugs across the blood-brain barrier, **mannitol** is administered intra-arterially to induce osmotic blood-brain barrier disruption (BBBD). Additionally, **sodium thiosulfate** is administered intravenously as a delayed otoprotective agent to mitigate the risk of hearing loss, a common side effect of carboplatin therapy. This specific protocol is being investigated by the OHSU Knight Cancer Institute for high-risk pediatric and adult brain tumors including medulloblastoma and atypical teratoid rhabdoid tumors (ATRT).

Other names
IA Melphalan and Carboplatin with BBBD and Sodium ThiosulfateIntra-Arterial Melphalan and Carboplatin with Osmotic Blood-Brain Barrier Disruption
02

Targets

OCLN (Occludin)DNAFree carboplatin and circulating platinum species

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