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Personalised medicine briefsheet Personalised medicine is about matching patients with the treatment that will work best for them. It could transform the lives of cancer patients in the UK – that’s why, at Cancer Research UK, we’ve made it one of our top priorities. What do we mean by personalised medicine? Personalised medicine is where doctors look at the molecular characteristics of each patient’s cancer to work out what treatment is most likely to benefit them. Why do we need to personalise cancer treatments? No two cancers are the same. This means that even patients with the same type of cancer will respond differently to treatment – not just to cancer drugs, but often radiotherapy and surgery too. Cancer develops when the DNA in our cells is damaged. We have made rapid progress in understanding the gene faults that lead to cancer, and how they influence the molecular landscape in cancer cells. These molecular changes vary from one patient to another. Next, we need to discover how these molecular changes affect the way a person’s cancer responds to treatment, and develop rapid tests to detect them. The ultimate aim is to treat every patient as an individual, but this vision is still some way off. What happens now? At the moment, most patients with the same cancer are given the same treatment, even though some patients are unlikely to benefit. This is because, at present, doctors often have no way of predicting who will or will not respond. But, thanks to research and the generosity of our supporters, we are moving in the right direction. Doctors and scientists are now beginning to identify subgroups of patients with the same type of cancer, but whose cancers have slightly different characteristics, and who need different treatments. Grouping patients together like this and treating them accordingly is known as ‘stratified medicine’ – and it’s the first step towards personalised medicine. October 2010 What difference will personalised medicine make? A patient’s treatment is more likely to be successful if it is tailored to their cancer. Otherwise, patients could get treatments that will not help. And it’s not just at the bedside where we expect to see benefits. This approach should also speed up the development of new cancer treatments by increasing the efficiency of clinical trials that test them. Increasingly, doctors are designing trials for subgroups of patients who are most likely to benefit. This makes it more likely that a trial will produce a clear result on whether or not a treatment is working. Did you know? Although personalised medicine is still at an early stage, there are already tailored treatments available for some types of cancer. Deborah’s story ‘I was diagnosed with breast cancer in 2004 when I found a lump in my breast whilst away on a business trip. My lump was large and had spread within my breast, so I needed to have a mastectomy to remove it. Doctors identified my breast cancer as HER2 positive, which meant I could be given the drug Herceptin. I responded really well to the drug and, thankfully, after a long battle, I’m now in good health again. I hope that more people in the future will be able to benefit from tailored treatment, as I have done.’ Cancer Research UK’s early work underpinned the development of Herceptin. This drug has made a real difference for a quarter of women whose breast cancer is likely to respond to this treatment. Our impact We currently spend over £14 million each year on research and infrastructure that will help deliver personalised medicine for cancer. In recent years, we’ve made many advances that have resulted in more tailored treatments: •Our research paved the way for the development of tamoxifen, which revolutionised the treatment of breast cancer, and later our work shaped the use of this drug. Tamoxifen targets a molecule on the surface of breast cancer cells called the oestrogen receptor. Two thirds of breast cancers are suitable for this drug and patients have their cancer tested before they can be prescribed it. •Our early lab work on a molecule called EGFR was the springboard for the development of the drug Tarceva for lung cancer. Around 20-30 per cent of lung cancer patients have faulty levels of EGFR and could benefit from Tarceva. This research provided the foundations for the development of Erbitux, which can benefit around 6 out of 10 people with advanced bowel cancer. Doctors will test a cancer sample for EGFR before starting treatment. •Our scientists laid the groundwork for a new generation of drugs called PARP inhibitors that work particularly well in breast and ovarian cancer patients who have inherited a faulty BRCA gene. These drugs are still in development, and in the future, it will be important to test patients to see if their cancer is suitable for this drug. ‘Molecular typing of cancer is going to become more and more part of standard practice for choosing treatment. I have no doubt that this is going to be a huge part of cancer medicine in the future.’ Prof Peter Johnson, Chief Clinician, Cancer Research UK Our progress towards personalised medicine Cancer Research UK is leading the way towards an era of personalised medicine. The fruits of our groundbreaking research will save many more lives from cancer in the future and improve quality of life for patients. None of this would be possible without the generosity of our supporters. Our research spans many areas of cancer – from hunting for faulty genes and other molecular markers to guide treatment decisions, through to developing new targeted treatments. We are also developing the use of the latest technologies to monitor how patients are responding to treatments. Looking for markers of cancer A key focus of our five-year strategy is to find new biomarkers and develop tests for them. We fund many world-leading scientists in this field. Biomarkers are molecules that doctors can test for in a person’s blood, urine, or cancer. One of the many uses of biomarkers is to provide a simple way to spot differences between patients and their cancers. They are key to making personalised medicine possible - they are the ‘sign posts’ that help doctors select the best treatment for their patient. At our Cambridge Research Institute, Dr James Brenton and his team are investigating why some women with ovarian cancer respond well to chemotherapy and others do not. They have already found several molecules which they hope doctors will be able to use as biomarkers to predict who will and won’t respond. This is just one of over a hundred projects we fund that are geared towards discovering new markers of cancer. How we process drugs Professor Roland Wolf in Dundee is studying how differences in our genetic make-up alter the way we process drugs. In the same way that our genes determine our hair and eye colour, they also affect how our bodies break down drugs. This is another reason why patients respond differently to treatment. Our genes affect how long a drug stays in the body or how much is needed, and in the future, this may also guide treatment decisions. Stratified Medicine Programme Cancer Research UK recognises the huge potential of personalised medicine to revolutionise the treatment of people with cancer. To bring this vision a step closer to Take cancer sample reality, we have launched our pioneering Stratified Medicine Programme, with our Test sample for partners, to examine how rapid testing of particular molecules cancer samples can best be rolled out across the NHS. This type of testing is vital if tailored treatment is to become a routine part of cancer care, helping Use samples doctors to make timely decisions about See which for lab research the best treatments for their patients. treatment is appropriate Our programme involves taking a sample of a person’s cancer when they are diagnosed, and analysing it for molecular Develop tests changes. Scientists will use this information to see which patients would Treat to build up a picture of the different benefit from cancer patterns of faults linked with specific Was which treatment patients cancers. As other areas of research treatment progress and more targeted treatments effective? become available, doctors will be able to prescribe the best treatment for their patient based on this information. Develop Initially, the scheme will focus on collecting more samples from patients with bowel, breast, tailored lung, prostate, skin and ovarian cancer. treatments Making drugs more personal Personalised medicine isn’t just about matching the right drug to the right patient – it’s also about having more targeted treatments to choose from. We currently fund nearly 100 different projects across the UK to discover new cancer drugs, based on the molecules that we know drive cancer. International expert Professor Paul Workman, at The Institute of Cancer Research in Sutton, leads the largest of our teams. They are using cutting-edge technology to design drugs against new molecular targets in cancer cells. Several of the drugs they have designed are already being tested in clinical trials with patients. Cancer Research UK Tel: +44 (0)20 7242 0200 www.cancerresearchuk.org Registered charity in England and Wales (1089464) and Scotland (SC041666) Using scans to tailor treatment As part of our five-year strategy, we are also boosting research into imaging techniques. These could indicate early on whether cancer drugs are working. We have set up four new imaging centres to drive forward this work. At the University of Birmingham, Dr Andrew Peet is investigating new ways of visualising tumours to see how they are responding to treatment in ‘real time’. Doctors can use this information to help choose the best treatment option for their patients. ‘We’re still a long way from individual treatments for individual patients, but new technologies Dr such as those in cancer Andrew imaging are helping to Peet pave the way.’