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Targeted skin cancer drug launches in UK



Wednesday 14 March 2012



A life-extending pill for advanced melanoma that The Institute of Cancer Research (ICR) and The Royal Marsden Hospital helped develop is now available in the UK.

Vemurafenib, marketed by Roche as Zelboraf, is the first personalised treatment that has been shown in clinical trials to increase survival time for patients with a specific BRAF gene mutation. The European Commission has licensed the drug for adults with this mutation whose cancer is inoperable or which has spread.

New treatments for metastatic melanoma

The drug is based on research at the ICR* showing how the mutated BRAF gene is driving cancer development in around 50 per cent of malignant melanomas. Vemurafenib, which is taken as four pills twice a day, has been designed to block this cancer-causing form of the BRAF gene. A test is available to identify patients who have the BRAF V600 predictive biomarker and are therefore suitable for treatment.

In clinical trials led in the UK by The Royal Marsden Hospital, patients treated with vemurafenib lived an average of 13.2 months compared with 9.6 months for patients who received standard chemotherapy. These patients, who had BRAF-mutant advanced cancers, were almost nine times more likely to respond to vemurafenib than to standard chemotherapy (48.4 per cent versus 5.5 per cent).

The incidence of malignant melanoma is increasing in the UK, with around 10,000 people diagnosed and 2,300 deaths a year. Malignant melanoma disproportionately affects young people and is now the second most common cancer in people aged 15-34 in the UK. The disease is considered advanced if it has spread to other organs, and in this form it is difficult to treat and life-expectancy is short. Vemurafenib and a second drug launched last year called ipilimumab, an antibody treatment, represent the first major advances in treatment for 30 years for patients with advanced melanoma. 

Chief Executive of The Institute of Cancer Research, Professor Alan Ashworth, says: “Advanced melanoma is a devastating disease and new treatments are desperately needed, so it’s especially pleasing that patients will now be able to benefit from a drug that we helped develop. The success of vemurafenib demonstrates the importance of our approach to developing personalised medicines for cancer. Understanding the genetic and molecular causes of cancer helps us to create new, targeted therapies that - as this drug shows - can prove extremely effective.”

Chief Executive of The Royal Marsden Cally Palmer says: “This is an important and significant step forward for the treatment of patients with advanced melanoma. We are delighted that our patients were among the first in the UK to benefit from this clinical trial, led by Dr James Larkin at The Royal Marsden.”


- ENDS -

Media Contact: ICR Science Communications Manager Jane Bunce on 0207 153 5106 or after hours 077217 47900 or Senior Press Officer at The Royal Marsden Belinda Payne on 0207 808 2605


Notes to editors:


* Mechanism of Activation of the RAF-ERK Signaling Pathway by Oncogenic Mutations of B-RAF with corresponding authors David Barford and Richard Marais published in Cell Volume 116, Issue 6, 855-867, 19 March 2004

B-V599E-RAF is an oncogene in melanocytes with corresponding author Richard Marais published in Cancer Research Volume 64, Issue 7, p 2338-2442, 1 April 2004.

B-RAF is a therapeutic target in melanoma
with corresponding author Richard Marais published in Oncogene Volume 23, Issue 37 p 6292-6298, 19 August 2004.

The Institute of Cancer Research (ICR) is one of the world’s most influential cancer research institutes.

Scientists and clinicians at the ICR are working every day to make a real impact on cancer patients’ lives. Through its unique partnership with The Royal Marsden Hospital and ‘bench-to-bedside’ approach, the ICR is able to create and deliver results in a way that other institutions cannot. Together the two organisations are rated in the top four cancer centres globally.

The ICR has an outstanding record of achievement dating back more than 100 years. It provided the first convincing evidence that DNA damage is the basic cause of cancer, laying the foundation for the now universally accepted idea that cancer is a genetic disease. Today it leads the world at isolating cancer-related genes and discovering new targeted drugs for personalised cancer treatment. 

As a college of the University of London, the ICR provides postgraduate higher education of international distinction. It has charitable status and relies on support from partner organisations, charities and the general public.

The ICR’s mission is to make the discoveries that defeat cancer. For more information visit

The Royal Marsden NHS Foundation Trust

The Royal Marsden opened its doors in 1851 as the world’s first hospital dedicated to cancer diagnosis, treatment, research and education.

Today, together with its academic partner, The Institute of Cancer Research (ICR), it is the largest and most comprehensive cancer centre in Europe treating over 44,000 patients every year.  It is a centre of excellence with an international reputation for groundbreaking research and pioneering the very latest in cancer treatments and technologies. The Royal Marsden also provides community services in the London boroughs of Sutton and Merton and in June 2010, along with the ICR, the Trust launched a new academic partnership with Mount Vernon Cancer Centre in Middlesex. 

Since 2004, the hospital’s charity, The Royal Marsden Cancer Charity, has helped raise over £50 million to build theatres, diagnostic centres, and drug development units. Prince William became President of The Royal Marsden in 2007, following a long royal connection with the hospital.


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