Public Release: 

Trial of 'resistance-busting' skin cancer drug begins as first patient receives treatment

Institute of Cancer Research

A patient has become the first to receive a new 'resistance-busting' experimental skin cancer drug with the launch of a phase I clinical trial.

The patient has received a new panRAF inhibitor - a new type of drug under development to address the problem of drug resistance in advanced skin cancer and a number of other cancer types.

The trial is the culmination of a pioneering research programme to design, synthesise and develop the new drug class, led by scientists at The Institute of Cancer Research, London, and the Cancer Research UK Manchester Institute at The University of Manchester.

It is starting just three months after a major publication in the journal Cancer Cell described the potential of this new drug class, which is potentially able to treat melanomas - the most serious type of skin cancer - that do not respond or have become resistant to existing therapies.

The phase I trial of the drug - which is yet to be given a formal name and is currently known as BAL3833/CCT3833 - is sponsored by The Institute of Cancer Research (ICR) and The Royal Marsden NHS Foundation Trust.

The trial is funded by the Wellcome Trust, the NIHR Biomedical Research Centre at The Royal Marsden and the ICR, The Christie charity and the Cancer Research UK Manchester Institute.

The first patient began treatment at The Royal Marsden, with patients also to be treated at The Christie NHS Foundation Trust in Manchester.

Just last month, a new consortium was announced to develop this drug class for patients - following an agreement between academic organisations, funders and Swiss-based biopharmaceutical company Basilea Pharmaceutica International Ltd.

The trial will recruit around 25 patients with advanced, solid tumours - focusing on advanced melanoma - with the aim of establishing the safe maximum dose for a planned phase II clinical trial.

PanRAF inhibitors block several key cancer-causing proteins at once including BRAF, which drives about half of all melanomas. Existing BRAF inhibitors are designed to block that protein - but most patients develop resistance to them within a year.

This consortium - including the ICR, the Wellcome Trust, Cancer Research Technology (CRT) and the Cancer Research UK Manchester Institute/The University of Manchester - has granted Basilea exclusive worldwide rights to develop, manufacture and commercialise a series of novel panRAF inhibitors.

Basilea will assume full operational responsibility for the research programme after the phase I trial, and is also carrying out biomarker research along with the Cancer Research UK Manchester Institute.

Professor Caroline Springer, Professor of Biological Chemistry at The Institute of Cancer Research, London, and leader of the ICR's research programme on panRAF inhibitors, said:

"Our new inhibitors are an example of an exciting new approach to cancer treatment that knocks out several important cancer signals at once, in order to treat cancers that develop resistance to drugs targeted at just one cancer signal.

"It's very exciting to go from publication of our laboratory results on panRAF inhibitors to assessment of the new treatment in the first patient in just three months. It demonstrates our belief in the promise of this work, and our desire to attempt to deliver benefits for patients as quickly as possible."

Dr James Larkin, Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust, who is leading the clinical trial, said:

"The major problem with current targeted therapies is resistance to treatment. This drug has been developed in the laboratory specifically to tackle this problem and we are very excited to be treating the first patient in this clinical trial."

Professor Richard Marais, Director of the Cancer Research UK Manchester Institute and leader of its research programme on panRAF inhibitors, said:

"This trial is the culmination of over a decade of research. BRAF drugs can give valuable extra months of quality life to about half of melanoma patients, but sadly it is not a cure and most patients eventually develop resistance. These new drugs are engineered to get around this problem by shutting down the routes that tumours use to bypass BRAF drugs. They work very well in the laboratory and we look forward to now seeing if they also work well in patients."

Dr Paul Lorigan, Reader in Medical Oncology at The Christie NHS Foundation Trust, said:

"We are very excited that this new treatment, borne out of great innovation and collaboration, will potentially make a real difference for our patients with melanoma."


Notes to editors

For more information contact Henry French on 020 7153 5582 / For enquiries out of hours, please call 07595 963 613.

The Institute of Cancer Research, London, is one of the world's most influential cancer research institutes.

Scientists and clinicians at The Institute of Cancer Research (ICR) are working every day to make a real impact on cancer patients' lives. Through its unique partnership with The Royal Marsden NHS Foundation Trust 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 50,000 NHS and private 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, with the ICR, is the only National Institute for Health Research Biomedical Research Centre for Cancer. First awarded the status in 2006, it was re-awarded in 2011. A total of £62 million is being provided over five years, to support pioneering research work, and is being shared out over eight different cancer themes.

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 £100 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.

About The Christie

  • The Christie opened in 1901 and is now one of Europe's leading cancer centres and the largest single-site centre in Europe
  • Because of its specialist nature, 26% patients are referred to The Christie from outside the Greater Manchester and Cheshire area
  • It has one of the largest radiotherapy departments in the world as well as centres in Oldham and Salford. It also houses the UK's largest brachytherapy service
  • The Christie delivers chemotherapy treatment through the largest chemotherapy unit in the UK, as well as via 10 other sites, its new mobile chemotherapy unit and in patients' homes
  • The Christie's NIHR Clinical Research Facility is a large, high quality, dedicated clinical research environment where our patients can participate in complex and early phase clinical trials. Around 400 clinical trials may be taking place at any one time
  • Its charity, which is one of the largest in the UK, provides enhanced services over and above what the NHS funds. It has over 30,000 supporters, who helped raise a record breaking £14.8m last year, with 83p in every pound going directly to patients
  • New developments include:
    • a new £28.5 million Manchester Cancer Research Centre (MCRC) opening this winter. The MCRC is a partnership between The Christie, The University of Manchester and Cancer Research UK
    • a new Maggie's Centre, expected to open on our site in 2016, providing free practical, emotional and social support for patients, their family, friends and carers
    • the UK's first high energy proton beam therapy service, due to start treating patients in 2018. The Christie was selected to deliver this specialist treatment, along with University College London Hospitals NHS Foundation Trust. Currently patients have to travel to America for this treatment
  • The Christie is one of seven partners in the Manchester Academic Health Science Centre, one of only six health science centres in the country
  • The Christie's School of Oncology provides undergraduate education, clinical professional and medical education - the first of its kind in the UK
  • Hear from Christie staff, patients, volunteers and fundraisers via the weekly Christie blog -

About Cancer Research UK

  • Cancer Research UK is the world's leading cancer charity dedicated to saving lives through research.
  • Cancer Research UK's pioneering work into the prevention, diagnosis and treatment of cancer has helped save millions of lives.
  • Cancer Research UK receives no government funding for its life-saving research. Every step it makes towards beating cancer relies on every pound donated.
  • Cancer Research UK has been at the heart of the progress that has already seen survival rates in the UK double in the last forty years.
  • Today, 2 in 4 people survive cancer. Cancer Research UK's ambition is to accelerate progress so that 3 in 4 people will survive cancer within the next 20 years.
  • Cancer Research UK supports research into all aspects of cancer through the work of over 4,000 scientists, doctors and nurses.
  • Together with its partners and supporters, Cancer Research UK's vision is to bring forward the day when all cancers are cured.

For further information about Cancer Research UK's work or to find out how to support the charity, please call 0300 123 1022 or visit Follow us on Twitter and Facebook.

About The University of Manchester

The University of Manchester, a member of the prestigious Russell Group of British universities, is the largest and most popular university in the UK. It has 20 academic schools and hundreds of specialist research groups undertaking pioneering multi-disciplinary teaching and research of worldwide significance.

The University of Manchester is one of the country's major research institutions, rated fifth in the UK in terms of 'research power' (REF 2014), and has had no fewer than 25 Nobel laureates either work or study there. The University had an annual income of £886 million in 2013/14.

Cancer is one of The University of Manchester's research beacons - examples of pioneering discoveries, interdisciplinary collaboration and cross-sector partnerships that are tackling some of the biggest questions facing the planet.

The Wellcome Trust is a global charitable foundation dedicated to improving health. We provide more than £700 million a year to support bright minds in science, the humanities and the social sciences, as well as education, public engagement and the application of research to medicine.

Our £18 billion investment portfolio gives us the independence to support such transformative work as the sequencing and understanding of the human genome, research that established front-line drugs for malaria, and Wellcome Collection, our free venue for the incurably curious that explores medicine, life and art.

About Basilea

Basilea Pharmaceutica Ltd. is a biopharmaceutical company developing products that address increasing resistance and non-response to current treatment options in the therapeutic areas of bacterial infections, fungal infections and cancer. The company uses the integrated research, development and commercial operations of its subsidiary Basilea Pharmaceutica International Ltd. to develop and commercialize innovative pharmaceutical products to meet the medical needs of patients with serious and potentially life-threatening conditions. Basilea Pharmaceutica Ltd. is headquartered in Basel, Switzerland and listed on the SIX Swiss Exchange (SIX: BSLN). Additional information can be found at Basilea's website

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