The drug, PLX4032, targets and destroys cells with the BRAF mutation, which is implicated in 60 per cent of melanomas.
The incidence of melanoma has quadrupled over the past 30 years. It affects 10,400 new patients annually, most commonly women in their twenties. Melanoma is easily treated in its early stages but less than 2 per cent of patients survive beyond two years with metastatic disease.
What is the research?
Scientists at the University of Pennsylvania gave PLX4032 to 16 patients with BRAF-positive melanoma.
Participants lived for a median of six months without disease progression and more than half experienced shrinkage of their tumours by, on average, 30 per cent. This included patients whose cancer had already spread to the liver, lung and bone.
Some mild and more serious adverse events were reported, but the safety profile was judged 'favourable' for this population. Larger trials are planned for later in the year.
What do the researchers say?
Dr Keith Flaherty of the University of Pennsylvania, principal investigator for the study said: 'PLX4032 has shown both tumour shrinkage and delay in tumour progression in patients whose tumours harbour a BRAF mutation, as well as improved quality of life for symptomatic patients.
'In addition to a new and important chapter in the story of targeted therapy development in cancer, we are especially excited for our melanoma patients for whom there are few treatment options.'
What do other researchers say?
Dr Jodie Moffat, Cancer Research UK's senior health information officer, said: 'This trial looked at a potential new drug for melanoma on just 16 patients who also had a specific genetic mutation.
'While these results are interesting, they need to be followed up in much larger studies before we know if this is a suitable treatment for people with advanced melanoma.'
She added: 'When melanoma is diagnosed early, treatment is often simpler and more likely to be effective.'
- A drug in development for melanoma shrank tumours in 60 per cent of susceptible patients.
- The drug has been tested in 16 patients, with larger trials due later this year.
- Melanoma is easily treated in its early stages and changes in moles or other patches of skin should be examined without delay.