First Potential Targeted Drug Identified for Rare Brain Cancer

Brain tumors are one of the deadliest forms of cancer mainly because of their sensitive location, and complex surgery involved to remove them. In a breakthrough development, a new research carried out by researchers at UC, San Francisco have led to much needed treatment options for patients for whom removing tumors through surgery is not viable.

Using tumor genome sequencing, the team identified the first targeted drug for the treatment of chordoid gliomas. The condition which is an uncommon type of brain cancer develops in the third ventricle, which is a fluid-filled pocket to provide cushion to the brain. While these tumors develop slowly, their proximity to the hypothalamus make them difficult to remove and can be fatal.

Study Reveals Single Mutation of Distinct Gene Common across Few Testing Samples

Published recently in the Nature Communications, the study involved sequencing the genomes of 13 chordoid glioma tumors. What was found to be common across all 13 samples was PRKCA to be the only gene to witness single mutation. PRKCA is an element of a key growth pathway MAP kinase, which does not perform in normal brain cells. However, mutation in PRKCA leads to activation of MAP kinase pathway to result in reckless division of cells developing into tumor.

Although PRKCA is so far not known to be mutated in any other cancer, the research lead revealed most human tumors activate the same growth pathway through some genetic mechanism. As MAP kinase pathway is commonly implicated in cancer, the targeted drugs are commercially available and approved by FDA. Trametinib- a drug used to treat melanoma was tested by researchers on brain cells with PRKCA mutation.