In 2002, a study revealed that three-fifths of melanoma patients have a specific gene mutation that encodes a protein known as B-RAF. The result is a cell signaling pathway that turbo-boosts the growth of cancer cells.
PLX4032 is a drug developed by California biotech company Plexxikon in conjunction with pharmaceutical giant Roche. According to recent research, the drug shrank tumors by at least 30% in 24 of 32 patients with advanced melanoma who had the B-RAF mutation. Two additional patients had their tumors disappear entirely.
The formulation of PLX4032 given to trial patients is a much higher dose than Plexxikon had initially counted on, as researchers learned that blocking the pathway requires greater concentrations of the drug. However, instead of causing severe side effects or significant toxicity, PLX4032 appears to be so specific that it binds to the mutated form of the protein to the near exclusion of all others.
Long-term effects are not known, and the drug needs to be tested in a much larger patient set. Nonetheless, these important issues aren't stopping some people from calling this cancer's 'penicillin moment'.
PLX4032 joins another drug in development that shows promise for advanced melanoma patients, Bristol-Myers Squibb's fully human monoclonal antibody ipilimumab (MDX-010).
CANCER TYPE(S)
Advanced melanoma
TREATMENT TYPE(S)
Targeted signaling pathway inhibitor
WHERE WAS THIS STUDY PUBLISHED?
Nature
Source
Nature News
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