WEDNESDAY, Feb. 22 (HealthDay News) -- Melanoma that has spread
to other areas of the body is almost always fatal, but a new drug
appears to double survival for those with a certain type of this
skin cancer, researchers report.
A mutation in the BRAF protein occurs in about half the people
who develop melanoma. Researchers say Zelboraf (vemurafenib), a
drug approved by the U.S. Food and Drug Administration in 2011,
blocks that mutation, thereby killing the cancer cells.
"We demonstrated in a large group of patients overall survival approached 16 months, which is far beyond what we have seen in other trials," said lead researcher Dr. Jeffrey Sosman, a professor of medicine at the Vanderbilt-Ingram Cancer Center in Nashville, Tenn.
Survival for people with advanced melanoma is usually six to 10
months. Some new drugs have extended that to maybe 11 months, "but
this is the first time we have shown such a long extension in
survival," he said.
All of the patients had late-stage (stage 4) melanoma. Sosman
said it's hoped that Zelboraf will work even better when given
"If we use it in an earlier stage, then we are hoping to cure patients who would not be cured otherwise," he said.
Results of this phase 2 trial -- undertaken to assess the
effectiveness and safety of the drug -- are published in the Feb.
23 issue of the
New England Journal of Medicine.
For more than a year, Sosman's team followed 132 patients with
previously treated metastatic melanoma, meaning the cancer had
spread and was considered inoperable.
Zelboraf is a pill that patients take twice a day, and more than
half who took the drug responded to it, the researchers said.
A response meant more than a 30 percent reduction in the size of
tumors. Only 14 percent of patients failed to show any response to
Zelboraf whatsoever, meaning their disease continued to
Previously, no more than 10 percent of patients responded to any
of the other available treatments, the researchers said.
The main limitation with Zelboraf is that tumors eventually
Side effects include joint and muscle aches, which are fairly
easy to manage in most patients, Sosman said.
Some patients also experienced skin changes, including sun
sensitivity and rashes. "Patients develop incredible
photosensitivity so patients have to be very careful about direct
sunlight and use sunscreen as well as just staying out of the
bright sun," Sosman said.
Also, the drug indirectly causes squamous cell cancers of the
skin in some patients. "These generally never spread and are easy
to remove, and patients who have gotten them can remain on
treatment," Sosman said.
The drug costs around $10,000 a month, Sosman noted. "It's
comparable to other cancer drugs that have shown improvement in
survival, but like a lot of cancer drugs, it's expensive," he said.
"You probably need to take the drug for an indefinite period of
Drug maker Hoffmann-La Roche funded the study.
About 70,000 Americans are diagnosed with melanoma each year. Of
these, about 8,000 will die from the disease.
Dr. Michele Green, a dermatologist at Lenox Hill Hospital in New
York City, said the study is a leap forward. "This finding is
significant in that there is nothing right now for this kind of
cancer. It's basically a death sentence," she said.
That the researchers showed improved survival is clinically
significant, she said, even though "the survival wasn't that much
If the drug were given for early-stage melanoma, it's possible
it would improve survival even more, Green said. Eventually, she
said the research will lead to a cure for melanoma.
"There's new hope every single day for melanoma," she said. "I think in our lifetime we are going to see cures for this cancer."
For more information on melanoma, visit the
U.S. National Library of Medicine.
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