Main Menu

New ‘Smart’ Pill Shown to Extend Lives of Patients with Most Aggressive Skin Cancer



Monday 6 June 2011



A new pill that precision targets the activity of a faulty gene present in half of terminally ill metastatic melanoma patients has been shown to significantly extend overall survival compared to standard chemotherapy. Data from the BRIM3 study shows that patients with the mutated BRAF gene who were given the twice daily vemurafenib pills were significantly more likely to be alive at 6 months than those receiving dacarbazine.


The Institute of Cancer Research (ICR) was responsible for key research on the BRAF gene, which paved the way for the development of this first-of-its-kind personalised treatment for advanced melanoma.


Based on the significance of the data, the trial was halted early as it was deemed unethical for patients to continue receiving standard chemotherapy and not vemurafenib.


Vemurafenib, a new generation tailored treatment, precision targets the faulty BRAF gene jamming the signal that causes melanomas to grow uncontrollably. Blocking the activity of this gene can cause cancer cells to die and tumours to shrink. All cancers are different in their genetic properties and therefore patients respond differently to treatment. Vemurafenib presents an opportunity to personalise the treatment approach for metastatic melanoma.


Vemurafenib is not currently licensed and the diagnostic test for BRAF mutation is not currently commercially available. The pill, previously known as PLX4032 or RG7204, was recently filed with both the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) who will review it over the coming months for licensing.


Dr James Larkin, UK Principal Investigator for BRIM 3 from The Royal Marsden, said: “Without question, the results of this trial represent a turning point in the treatment of this disease. Vemurafenib targets the faulty BRAF protein which is present in 50% of melanomas and this trial has shown that vemurafenib significantly prolongs life in comparison with chemotherapy”.


Over the past twenty-five years, rates of malignant melanoma in Britain have risen faster than any other common cancer. It is now the second most common cancer in young adults (aged 15-34) in the UK, affecting almost twice as many young women as young men, although men are more likely to die from it.


Professor Richard Marais, whose work at the ICR demonstrated the importance of BRAF in melanoma, said: “This is the biggest breakthrough in melanoma treatment in more than 30 years. The results demonstrate for the first time that a targeted therapy can work in melanoma and will change our approach to treating this disease. It is an enormous advance in the field.”


Further trials are underway exploring the use of vemurafenib in a variety of other cancers including ovarian, thyroid and colorectal cancer. In conjunction Roche and partner company Plexxikon have developed a diagnostic test to determine whether patients have the faulty BRAF gene, which could deliver results in a matter of days.


The most frequent grade 3 adverse events with vemurafenib were skin related and included cutaneous squamous cell carcinoma, a common skin cancer treated by local excision. Additionally, generally mild and reversible increases in liver enzymes (GGT, ALT, AST, alkaline phosphatase, and bilirubin) were observed in some patients.


The most common adverse events were rash, photosensitivity, joint pain, hair loss and fatigue.


Media Contact: Hannah Rhind from Roche on 01707 367 845 or 07884 116922 or ICR Science Communications Manager Jane Bunce on 0207 153 5106 or after hours 077217 47900

Notes to Editors:


Advanced Melanoma and BRAF


Advanced melanoma is the deadliest and most aggressive form of skin cancer. A person with advanced melanoma typically has a short life expectancy that is measured in months. There are an estimated 2000 deaths annually in the UK from malignant melanoma, with young people disproportionately affected by the disease. Over the last twenty-five years, rates of malignant melanoma in Britain have risen faster than any other common cancer. If current trends continue, it is anticipated that there will be around 15,500 cases of malignant melanoma diagnosed per year within the next 15 years. Until recently there has been no major advance in treatment for 30 years and patients with advanced melanoma have had very few treatment options.


The BRAF protein is a key component of the RAS-RAF pathway involved in normal cell growth and survival. Activating mutations in the BRAF gene cause this pathway to be overactive, which may lead to excessive growth and cancer. Mutations in the BRAF protein are found in about 50 percent of melanomas and it is estimated that approximately eight percent of all solid tumours contain BRAF mutations.




Vemurafenib is an investigational, oral medicine that is designed to selectively inhibit a cancer-causing mutated form of the BRAF protein. Vemurafenib is being co-developed under a 2006 licence and collaboration agreement between Roche and Plexxikon. A polymerase chain reaction-based companion diagnostic, the cobas® 4800 BRAF V600 Mutation Test, is being co-developed by Roche Molecular Diagnostics and Plexxikon in parallel to identify people whose tumours carry the BRAF V600E mutation. Vemurafenib is not currently licensed for the treatment of BRAF-mutated, advanced melanoma.


Roche in the UK

Roche aims to improve people’s health and quality of life with innovative products and services for the early detection, prevention, diagnosis and treatment of disease. Part of one of the world’s leading healthcare groups, Roche in the UK employs nearly 2,000 people in pharmaceuticals and diagnostics. Globally Roche is the leader in diagnostics, and a major supplier of medicines for the treatment of cancer, transplantation, virology, bone and rheumatology, obesity and renal anaemia. Further information at:

The Institute of Cancer Research (ICR)

  • The ICR is Europe’s leading cancer research centre
  • The ICR has been ranked the UK’s top academic research centre, based on the results of the Higher Education Funding Council’s Research Assessment Exercise
  • The ICR works closely with partner The Royal Marsden NHS Foundation Trust to ensure patients immediately benefit from new research. Together the two organisations form the largest comprehensive cancer centre in Europe
  • The ICR has charitable status and relies on voluntary income, spending 90 pence in every pound of total income directly on research
  • As a college of the University of London, the ICR also provides postgraduate higher education of international distinction
  • Over its 100-year history, the ICR’s achievements include identifying the potential link between smoking and lung cancer which was subsequently confirmed, discovering that DNA damage is the basic cause of cancer and isolating more cancer-related genes than any other organisation in the world

For more information visit


The Royal Marsden Hospital
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. For more information, visit or contact Belinda Payne  on 020 7808 2107 or [email protected]


comments powered by Disqus