Vemurafenib is taken in pill form

In 2002, a paper appeared in top science journal, Nature, called “Mutations of the BRAF gene in human cancer”.

It described how scientists at The Institute of Cancer Research and the Wellcome Trust Sanger Centre had made a breakthrough.

They’d discovered that a gene called BRAF was faulty in about seven out of 10 cases of melanoma – the most serious form of skin cancer.

The researchers had also isolated a particular fault in the gene, which they dubbed V600E, which was responsible for about five out of 10 melanoma cases.

We’re extremely proud to have helped support this work, which you can read more about in Kat’s post from a couple of years ago. And over the years we helped support research to map out how this gene causes cancer, and how it might be targeted with drugs.

Today, a decade later, pharmaceutical giants Roche have launched a new drug called vemurafenib, or Zelboraf, in the UK. Building on the fundamental work of our researchers, the drug targets melanomas caused by the BRAF V600E mutation.

We’ve tracked vemurafenib’s development on this blog, from its early days as a chemical called PLX4032 to the excitement it generated at last years’ ASCO cancer conference in the US.

But while it’s always great to see new drugs emerge – particularly for conditions like advanced melanoma, which has seen little progress for decades – we need to temper this excitement with a few caveats.

Not for everyone

Firstly, vemurafenib doesn’t seem to work in people who don’t have the BRAF V600E mutation in their melanoma – which is about half of all patients.

It’s also not licensed for people whose melanoma, thankfully, hasn’t begun to spread.

And some people will be too poorly to receive the drug.

Given these facts, we’ve estimated (very roughly) that it would probably only be suitable for between 500 and 1,000 of the 11,000 people diagnosed with melanoma each year.

Not a cure

Secondly, vemurafenib doesn’t cure advanced melanoma. It can prolong life by months, relieve the symptoms of the disease, and shows more clinical effect than almost every other treatment for advanced melanoma. But, sadly and inevitably, the cancer develops resistance to the drug and comes back.

The image behind this link, which is not for the faint-hearted, encapsulates the drug’s power, but also cancer’s devastating ability to overcome it.

Vemurafenib also has significant side-effects, including a second, less serious form of skin cancer called basal cell carcinoma, which is easy to treat.

Researchers – including some we fund – are currently working out what causes drug resistance and these second cancers.

Not approved by NICE

Finally, although it’s now licensed for sale in this country, the drug hasn’t yet been approved by NICE or the Scottish Medicines Consortium (SMC) for routine NHS use. NICE is currently assessing the clinical evidence that Roche has provided, and – according to their website – expects to make a decision by October.

Until that time, patients in England can ask their clinician to request the drug via the Cancer Drugs Fund; those living in the rest of the UK can apply to Exceptional Case Committees or related bodies.

And here’s the final point to make – vemurafenib, like all new, targeted treatments, is likely to be expensive.

As we told the media this morning, we want Roche to make sure the price they initially offer the NHS is affordable, so that the drug can be approved as speedily as possible.

We recognise that everyone – even pharma giants – are feeling the effects of the recession. We do understand that the price of a drug reflects not just the cost of the trials to develop it, but the cost of failed trials of other drugs (only about 10 per cent of drugs initially tested, make it through to routine use). And we recognise the vital role the pharmaceutical industry plays in shouldering the costs – and the risks – of large clinical trials.

But too often in recent years, price has been a stumbling block in getting effective drugs to patients that need them.

It would be deeply frustrating – for patients, for researchers, and for supporters who donate to charities like ours – to see this unhappy story being told yet again.


Image via Wikimedia Commons