Drug Targets BRAF Cancer Gene Shows Promise Against Metastatic Melanoma

A phase 1 clinical trial using a new formulation of an experimental drug that targets the BRAF cancer gene, has shown early promise in treating melanoma in patients with a mutated form of the gene and whose skin cancer has progressed to the metastatic stage.
A paper on the trial appears in the 26 August issue of the New England Journal of Medicine, NEJM.
Lead and corresponding author, Dr Keith Flaherty, director of Developmental Therapeutics at the Massachusetts General Hospital (MGH) Cancer Center in Boston, told the press that:
"Metastatic melanoma has a devastating prognosis and is one of the top causes of cancer death in young patients."
In the US, the current prognosis for surviving metastatic melanoma, where the skin cancer has spread to other parts of the body, is about 9 months. About 9,000 Americans die of the disease every year.
Many melanomas have a mutation of BRAF that activates a protein called serine-threonine kinase (B-RAF or BRAF) that drives the growth of cancer cells. The mutation is called V600E. These mutations of BRAF also occur in other cancers.
In this phase 1 trial, Flaherty and colleagues from other research centers in the US and Australia, found that a new formulation of PLX4032 (an earlier formulation had not succeeded in a previous trial), inhibited the V600E mutation of BRAF and 26 of the 32 patients they treated with it (81 per cent) showed a partial or complete response that lasted at least 19 months.
Read More Experimental Drug That Targets BRAF Cancer Gene Shows Promise Against Metastatic Melanoma

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