Glimmer of hope as drug shows signs of fighting skin cancer

For the melanoma patients who signed on to try a drug known as PLX4032,the clinical trial was a last resort.

For the melanoma patients who signed on to try a drug known as PLX4032,the clinical trial was a last resort. Their bodies were riddled with tumors,leaving them with months to live. But a weeks after taking their first dose,nearly all of them began to recover.

Lee Reyes,30,who had begun using a feeding tube because of a growth pressing at his throat,bit into a roll. Nothing,ever tasted as good. Rita Quigley,grateful to find herself breathing since learning she had skin cancer,went shopping.

It was a sweet moment,in 2008,for Dr Keith Flaherty,the University of Pennsylvania oncologist leading the drugs first clinical trial. A new kind of cancer therapy,it was tailored to a genetic mutation that was driving the disease,and after six years of disappointments,his faith in such a targeted approach finally seemed borne out. His collaborators at five other cancer centers,who had tested dozens of therapies with no success,were equally elated.

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The six doctors sent each other before and after CT scans of patients. One was of Mark Bunting,52. His initial scan showed the cancer in his bones. After two months on the drug,it had all but disappeared.

The only two patients on the trial whose tumors continued to grow were the ones who did not have the particular gene mutation for which the drug had been designed. They were removed from the trial. By late December,tumors in the 11 patients who did have the mutation had shrunk.

Urged on by Flaherty,the companies that owned the drug had spent months devising a new formulation that could be absorbed at higher doses. In December,the doctors prepared to convince the FDA that the drug should be tested in a larger Phase 2 trial. The agency required a summary of side effects there had been only a few and any deaths; there had been none since the drug was reformulated.

The side effects struck at the 1,120 mg dose. Many patients had been taking the reformulated drug for five months without relapsing. The doctors had hoped by pushing up the dose they could shut down the cancer.

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Kerri Adams,in Oklahoma City,woke up one morning covered in a rash. Another womans hand swelled. Maybe the drug,designed to turn off the defective B-RAF protein,was,at high doses,affecting its role in healthy cells.

When the dose was reduced,side effects subsided. In May,before he was to present the trials data,Flaherty received a message. The first patient to respond in the trial,Elmer Bucksbaum,had been admitted to the hospital. The cancer had spread to his brain. But had the drug held off the cancer elsewhere in his body? Or would other patients,too,relapse? Bucksbaum died days later.