A new era in the treatment of skin cancer was heralded by doctors yesterday as they unveiled what has been called the biggest breakthrough in 30 years.
A twice-a-day pill that targets a faulty gene present in melanoma, the deadliest form of skin cancer, has been shown to reduce the risk of death by almost two-thirds in patients with the advanced form of the disease.
James Larkin of the Royal Marsden Hospital in London, the principal investigator in the UK, said: "This is a pivotal moment. Without question, the results of this trial represent a turning point in the treatment of this disease."
Professor Richard Marais of the Institute of Cancer Research, who first demonstrated the importance of the gene in melanoma, said: "This is the biggest breakthrough in melanoma treatment in more than 30 years. The results demonstrate for the first time that a targeted therapy can work and will change our approach to treating this disease. It is an enormous advance in the field."
The drug is the first "personalised" treatment for melanoma, designed to target cases of the disease which carry the faulty gene, called a BRAF mutation, which account for about half of all cases. As such, it marks a milestone in the transformation of cancer medicines from blunderbuss treatments for everybody to designer drugs tailored to individual cases.
Melanoma has been the fastest rising cancer in the UK over the past 25 years, with more than 10,000 cases a year and 2,000 deaths. It is the second most common cancer in young adults aged 15 to 34 and affects twice as many women as men, although more men die of it.
A typical victim is the pale-skinned office worker who spends two weeks broiling on a Mediterranean beach until their skin is red and blistered. Covering up in the midday sun and using high-factor sun cream is the best defence against the cancer.
The international drug trial involved 670 patients in the US, Canada, Europe and Australia, including 50 in the UK. It showed survival rates at six months increased from 64 per cent for those on standard chemotherapy to 84 per cent on the new drug, called vemurafenib.
The improvement was so dramatic that three months into the trial participants receiving standard chemotherapy were offered the chance to switch to vemurafenib.
The findings were published yesterday by the New England Journal of Medicine and were presented at the annual meeting of the American Society for Clinical Oncology in Chicago.
Dr Larkin said: "For the last few decades we have treated melanoma with chemotherapy and it didn't work very well. Now that we understand the biology of the disease we can target the genes responsible. We know the BRAF mutation drives the disease."
Roche, the Swiss multinational, developed the drug with its partner Plexxikon, a biotech company which is also developing a test to screen patients for the BRAF mutation.
The companies are likely to apply for a licence for vemurafenib later this year. It is expected to cost tens of thousands of pounds per patient per year.
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