Melanoma Drug Shrinks Tumors, Study Finds

A new drug aimed at treating advanced melanoma has been found to shrink tumors in 80 percent of patients in an early trial, according to research done at the Memorial Sloan-Kettering Cancer Center in New York City and other cancer centers. The findings were published on Aug. 26 in the New England Journal of Medicine.

The drug is a form a targeted therapy, which differs from chemotherapy drugs in that targeted therapies are designed to affect a specific protein in cancer cells, thereby minimizing side effects in the body.

The melanoma drug tested works by inhibiting a protein called BRAF. The gene for BRAF is mutated in about half of melanomas, study researcher Dr. Paul Chapman of Memorial Sloan-Kettering said in a statement. By inhibiting BRAF, tumors are shut off.

"We have seen many tumors shrink rapidly and, in some patients, quality of life improved dramatically," Chapman said.

Melanoma is a serious form of skin cancer that is linked to exposure to ultraviolet radiation, according to the Mayo Clinic. There were more than 68,000 new cases of melanoma in the United States last year, and more than 8,600 Americans died from the disease, according to the National Cancer Institute.

The drug, called PLX4032 and also known as RG7204, is an oral medication taken twice daily that blocks the BRAF protein on a cellular level. Two phases of the trial were completed with separate groups of melanoma patients, and a phase III trial which will test the effectiveness of the drug in a larger group of patients, is currently underway, according to the researchers.

However, there is not yet research on the long-lasting effects of the drug or whether it improves the survival rate of melanoma.

Side effects of the drug include rash, nausea, photosensitivity, fatigue and squamous cell carcinoma of the keratoacanthoma type, which are low-grade skin tumors that are easily removed.

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