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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.

Henry

Image via Wikimedia Commons

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Comments

Philly Morris April 10, 2012

Well done to CRUK for all this work on Melanoma which in it’s self is about to become one of the biggest killers of young women and men across the world. If this research work goes on to bring a cure for the cancer then we all must get behind CRUK. Hopefully a drug can be made that prolongs life for years at a far cheaper price. I do agree with Laura’s comments. Its shocking that donations to CRUK are funding research by the brilliant teams there but the drug companies then make it to expensive for general use. This needs to me addressed. Surely CRUK could start its own none profit company to make the drugs for the less well off?

Henry Scowcroft March 16, 2012

Hi Laura,

You raise a good point. We are very concerned about the high prices being offered on important new drugs such as vemurafenib and the prostate cancer drug abiraterone, which you may also have heard about recently. In these two cases, we helped fund research that laid the foundations for the discovery and development of the drugs, so we are understandably concerned that they will be made available for cancer patients on the NHS.

To explain a little more, about 40 per cent of the research we fund is what’s known as ‘basic’ research – laboratory investigations to find out what drives cancer cells to grow and spread. This kind of research underpins virtually all the progress that we’re making in cancer today, from new treatments and diagnostic techniques to understanding the molecular ‘nuts and bolts’ that underpin the disease. This is often at the very earliest stages of the development of a treatment – for example, identifying a new target for therapy, or testing chemicals that might one day be developed into drugs.

When scientists find something new, they publish their results so other researchers can use the findings to help their work. This speeds things up so that everyone can benefit from the fruits of others’ labours (be they government or charity researchers, or scientists working for pharma companies) and get bright ideas turned into new treatments to benefit patients as quickly as possible.

In the case of vemurafenib, the initial discovery of the BRAF mutation we talked about above (which our supporters helped fund) sparked international interest in developing drugs to exploit it. Much of this subsequent work – involving significant amounts of drug development and lab testing, as well as large trials in lots of patients – was paid for and carried out by pharmaceutical companies, and this new drug is the result.

Large, international trials are the only way we can really tell whether a new cancer drug is effective at treating the disease. But such trials are hugely expensive, costing many millions of pounds, and Cancer Research UK simply can’t afford to run them. So we need pharma companies to do this, and they are financially able to both run the trials and to shoulder the risk of them not working – and, frustratingly, nine out of 10 of them don’t.

In some cases, when our laboratory researchers discover a chemical that might one day become a drug, we can ‘license’ it to a pharma company through our tech transfer arm (a company called Cancer Research Technology). This means we stand to earn money if and when the drug comes to market, which is then reinvested in our research. And we now try to make sure that with these deals, the resulting drug should be sensibly priced.

Although we don’t have any direct financial involvement with vemurafenib, we do with the prostate cancer drug abiraterone, which was in the news this week. This is currently deemed too expensive for the NHS, and we’ve been quite vocal in calling for the drugs company to lower the price so the NHS can afford it. We hope this will happen, and that the drug will be available to men with advanced prostate cancer as soon as possible.

Finally, with regard to vemurafenib, NICE hasn’t decided whether this is cost-effective for the NHS yet. But as we said above, we hope the manufacturers offer it to the NHS at a price it can afford, so NICE can recommend it to the NHS. If this doesn’t happen, we and others will be fighting hard on behalf of patients to try to persuade everyone to come to an agreement.

Henry
Cancer Research UK

laura newland March 14, 2012

Are the drug companies using money from CR to research drugs then make them to expensive to use by the people needing them who have donated to CR?

I really hope not as i would make one hell of a fuss!