They're not cures, but two novel drugs produced unprecedented gains in survival in separate studies of people with melanoma, the deadliest form of skin cancer, doctors reported Sunday.
In one study, an experimental drug showed so much benefit so quickly in people with advanced disease that those getting a comparison drug were allowed to switch after just a few months.
The drug, vemurafenib, targets B-RAF, a gene mutation found in about half of all melanomas. The drug is being developed by Genentech, part of Swiss-based Roche, and Plexxikon Inc., part of the Daiichi Sankyo Group of Japan.
The second study tested Bristol-Myers Squibb Co.'s Yervoy, a just-approved medicine for newly diagnosed melanoma patients, and found it nearly doubled the number who survived at least three years.
The new studies were presented Sunday at the oncology group's annual meeting in Chicago and published online by the New England Journal of Medicine.
"This is really an unprecedented time of celebration for our patients," said Dr. Lynn Schuchter, of the University of Pennsylvania's Abramson Cancer Center.
Melanoma is on the rise. There were 68,000 new cases and 8,700 deaths from it in the United States last year, the American Cancer Society estimates. Only two drugs had been approved to treat it, with limited effectiveness, until Yervoy, an immune-system therapy, won approval in March.
The experimental drug, vemurafenib, (vem-yoo-RAF-eh-nib), is aimed at a specific gene mutation, making it the first so-called targeted therapy for the disease. The drug got attention when a whopping 70 percent of those with the mutation responded to it in early safety testing.
In January, Action News profiled Corky Corcoran, of Franklinville, New Jersey, who went into the trial through the University of Pennsylvania after being diagnosed with Stage 4 melanoma.
Within months, 3 of the tumors inside his body were gone.
His oncologist, Dr. Ravi Amaravadi, says 80 per cent of patients have some response to vemurafenib.
Dr. Schucter says of hers, "Within 72 hours, their symptoms improve, pain medicines can be reduced."
The new study, involved 675 patients around the world with inoperable, advanced melanoma and the gene mutation. They received vemurafenib pills twice a day or infusions every three weeks of the chemotherapy drug dacarbazine.
After six months, 84 percent of people on vemurafenib were alive versus 64 percent of the others.
Less than 10 percent on the drug suffered serious side effects - mostly skin rashes, joint pain, fatigue, diarrhea and hair loss. About 18 percent of patients, including Corcoran, developed the less serious, squamous cell cancers. More than a third needed their dose adjusted because of side effects.
The study was sponsored by the drug's makers, and many of the researchers consult or work for them. The companies are seeking approval to sell the drug and a companion test for the gene mutation in the U.S. and Europe. A Genentech spokeswoman said the price has not yet been determined.
The other new drug, Yervoy, is not a chemotherapy but a treatment to stimulate the immune system to fight cancer.
About 502 patients in the study received dacarbazine and half also got Yervoy. After one year, 47 percent of those on Yervoy were alive versus 36 percent of the others. At three years, survival was 21 percent with Yervoy versus 12 percent for chemotherapy alone.
Side effects included diarrhea, rash and fatigue. More than half on the new drug had major side effects versus one quarter of those on chemotherapy alone.
Bristol-Myers Squibb paid for the study and many researchers consult or work for the company. Treatment with Yervoy includes four infusions over three months and costs $30,000 per infusion.
