SCIENTISTS have heralded a new therapy for one of the deadliest forms of cancer after trials showed a new drug can extend the lives of patients with advanced malignant melanoma.
Patients taking the new drug, which attacks a genetic mutation found in about half of all cases of the aggressive skin cancer, live significantly longer than those given standard treatment, a landmark study has found.
Preliminary results suggest that the drug, known as RG7204 or PLX4032, is the first melanoma therapy with a proven benefit on survival. Though the cancer is treatable if caught early, fewer than 10 per cent of patients survive for a year if it has spread.
The trial has been so successful that Roche, the drug company, has ended its control arm, so that participants who had received a placebo can now take the active drug.
While RG7204 is suitable for only the half of melanoma patients whose tumours carry the right mutation, and only 80 per cent of these respond, scientists said it would transform treatment of the disease.
Richard Marais, Professor of Molecular Oncology at the Institute of Cancer Research in London, said: “What this means is that now, for the first time in melanoma, we have a treatment that actually works for an appreciable proportion of patients.”
James Larkin, of the Royal Marsden Hospital in London, said: “This is an incredibly exciting breakthrough. Malignant melanoma is a very difficult disease to treat and with a growing incidence in younger people the results of this trial are very encouraging.”
The drug also marks a milestone in the development of personalised genetic medicine, as it was designed to target a genetic mutation that is found in about 50 per cent of melanoma patients. The mutated gene, called BRAF, was identified in 2002 by a British team led by Professor Mike Stratton, of the Wellcome Trust Sanger Institute near Cambridge.
The company is also developing a diagnostic test for the BRAF gene targeted by RG7204, to identify which patients can benefit. Such targeted treatment is considered to be the next frontier of cancer medicine.
In a separate study, published in the journal Nature, a team led by Dr Andy Futreal, of the Sanger Institute, has identified a gene that is mutated in one in three cases of kidney cancer.