Experimental drug could target melanoma that spreads to brain

In collaboration with the Press Association

A new drug could be effective for the treatment of melanoma that has spread to the brain, according to an early-stage clinical trial.

Patients from the US and Australia with advanced melanoma - the most serious form of skin cancer - were treated with an experimental drug called dabrafenib.

The drug substantially reduced the size of tumours that had spread to the brain.

Dabrafenib has been developed to treat melanoma in patients whose cancer is caused by faults in a gene called BRAF - first linked to cancer by UK researchers in 2002.

An altered form of this gene is present in about half of people with advanced melanoma and in several other types of cancer including some thyroid, colorectal, ovarian and lung cancers.

The phase I trial - published in the Lancet and funded by drugs company GlaxoSmithKilne - was primarily designed to establish the safe dose of dabrafenib. It involved 184 patients with incurable tumours, 156 of whom had melanoma.

Dr Gerald Falchook from the University of Texas MD Anderson Cancer Centre conducted the study with colleagues at Melanoma Institute Australia and Westmead Hospital in Sydney.

He said: "Brain metastases in most [nine of 10] patients given dabrafenib reduced in size, with four patients' metastases completely resolving.

"Patients with melanoma and brain metastases typically survive for less than five months, yet in this study, all ten patients were alive at this stage One patient remains on treatment at 19 months."

A BRAF-targeting melanoma drug called vemurafenib has already been licensed in the UK.

Dabrafenib not only worked in patients with the type of BRAF fault that vemurafenib has been proven to be effective against, but also against different types of BRAF fault.

Professor Mark Middleton, director of Cancer Research UK's Experimental Cancer Medicine Centre at Oxford, said: "All indications are that dabrafenib could be as effective as the newly licensed melanoma drug vemurafenib, which is available to patients through the Cancer Drugs Fund in England.

"The fact that dabrafenib was also effective for some patients with melanoma that had spread to the brain is really encouraging. This is the first drug to have any activity against these advanced cancers, and this work holds out promise that the whole class of these drugs could be effective for melanoma that's spread to the brain."

"After years of little to shout about, we're finally making progress against this tough cancer."

Copyright Press Association 2012


  • Falchook G et al, Dabrafenib in patients with melanoma, untreated brain metastases, and other solid tumours: a phase 1 dose-escalation trial (2012) The Lancet.