Skin cancer drug may prolong survival in advanced cases: Study

According to an international study a new treatment for advanced skin cancer almost doubles survival times.

Researchers say 132 patients in the U.S. and Australia who were given the drug vemurafenib gained several extra months of life. The study published in the New England Journal of Medicine found that those on the drug lived an average of 16 months, compared with nine months on conventional treatment.

Vemurafenib (Zelboraf) has been approved for use in Europe. According to the European Medicines Agency, the drug has been recommended for approval in Europe, pending final authorization by the European Commission.

Last year, the FDA fast-tracked approval for Zelboraf, a pill that targets a specific mutation in the BRAF gene that’s present in about half of all melanomas. At the time it was approved, studies showed the drug was shrinking tumors in most patients who took it, but researchers had not been following patients long enough to know how it might affect long-term survival.

The treatment is one of two drugs for late-stage melanoma, approved on fast-track in the US last year, which offer hope for patients with advanced melanoma. Vemurafenib is suitable for about half of patients with advanced melanoma as it targets tumors that express a certain gene mutation. Before that, there had been no new drugs for the cancer for more than a decade.

Dr Antoni Ribas, a professor of haematology/oncology and a researcher at the Jonsson Cancer Center at the University of California-Los Angeles, said, “This study shows that Zelboraf changes the natural history of this disease. This data is beyond what I would have expected. We're seeing a significant number of patients with durable responses to the drug, and that the whole group of treated patients is living longer. These results tell us that this drug is having a very big impact, and this changes the way we treat metastatic melanoma.”

“There’s a feeling that patients respond for a very short time and then relapse. Obviously that’s true, there are patients like that. But there is a group of patients who respond for much longer periods of time, and a number of patients are still alive at a year or two years. And I think we didn’t appreciate those numbers before,” said researcher Jeffrey A. Sosman, director of the melanoma and tumor immunotherapy program at the Vanderbilt-Ingram Cancer Center in Nashville, Tenn.

Along with those benefits, the drug does come with some significant side effects. “The side effects are certainly reasonable for a cancer drug. They’re not trivial,” Sosman said to WebMD. “A number of patients needed to have their drug held for a short time or needed to have their dose lowered, but almost all patients continued on the drug. Few stopped it.” The most problems seen in patients taking the drug were joint pain, rash, skin that easily burns in the sun, fatigue, and hair loss. More than a quarter of patients who take the drug also develop new squamous-cell skin cancers, although those are thought to be less dangerous than the melanoma. “These patients should be followed closely by a dermatologist, which is probably a good idea anyway,” Sosman said.

“We get some great responses initially, at least, so we can talk to patients with a smile compared to two years ago; it was a pretty much pessimistic view of the outcomes,” said researcher Kevin B. Kim, an associate professor of melanoma medical oncology at The University of Texas MD Anderson Cancer Center in Houston. For patients with large tumors who are suffering from pain and discomfort, “We can shrink tumors significantly, so the pain can be improved quite dramatically,” Kim said. “People feel stronger very quickly.”

Elizabeth Woolf, head of Cancer Research UK's information website Cancer Help UK, said, “This is an interesting, impressive but relatively small trial of a promising new-generation melanoma drug, which Cancer Research UK is proud to have played a role in developing.” But she said there were still questions that remain unanswered for example the cost.

“Everyone on the trial had the drug, so we cannot tell how large the benefits are, compared to people who didn't have it, or had another treatment. And because the drug targets a particular gene fault, only half of all melanoma patients are eligible. About half of those treated seem to benefit, so it could potentially help roughly a quarter of patients with advanced melanoma overall. Looking at these uncertainties, and now that the drug is available to UK cancer patients, it will be interesting to see what price the manufacturer charges so as not to place too great a strain on already scarce NHS resources,” she added.

Cancer Research UK said once the drug was licensed in Europe, patients would be able to discuss treatment options with their doctor. In England, patients will have to apply to the Cancer Drugs Fund, the charity said.

Kate Law, director of clinical and population research at Cancer Research UK, said the treatment was one of a new generation of cancer drugs targeted at patients with a specific genetic make-up. While it offered hope, she said, it was not a cure as the cancer eventually became resistant to the drug. She told the BBC, “This is not a cure - you're talking an extra six months of life. We're getting somewhere with these targeted drugs but we have a whole raft of research still to do to address the issue of resistance.”

About 76,250 people in the U.S. will be diagnosed with melanoma this year and about 9,200 are expected to die, according to the American Cancer Society. Metastatic melanoma is skin cancer that spreads to other parts of the body, such as to the lungs or bones. About half of patients with melanoma have a genetic mutation on the BRAF protein.

The study was paid for by Hoffmann-La Roche, the company that makes the drug.

Dr. Ananya Mandal

Written by

Dr. Ananya Mandal

Dr. Ananya Mandal is a doctor by profession, lecturer by vocation and a medical writer by passion. She specialized in Clinical Pharmacology after her bachelor's (MBBS). For her, health communication is not just writing complicated reviews for professionals but making medical knowledge understandable and available to the general public as well.


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