PersonalizedMed_Graphic (2)The goal of precision medicine is to foster a new healthcare approach where patients receive a tailored system based on their individual disease. This takes medicine away from the broad spectrum blockbuster drug approach, towards designing a specific treatment regimen to patient’s unique characteristics. It is hoped that this approach will be more likely to be effective for patients and improve their treatment outcomes. Rebecca Cummings of Cancer Research UK and Hannah Murfet of Horizon Discovery examine the potential of personalised medicine and recent collaborations in this exciting area.

Clinical trials for new treatments are typically tested on large patient populations to assess safety and efficacy. Individual tissues from individual patients show different properties; therefore studying treatments in large populations may not show how or where a treatment could be most effective. Where there is no or limited response to treatment this has a profound impact on the patient and the whole healthcare process, from losing precious time to the funding spent on ineffective treatment. Notable successes for precision medicine are increasing on an exponential rate; this includes lung cancer, breast cancer, chronic myeloid leukaemia and colorectal cancer. Enabling new designs for trials to support this research is crucial.

One programme that is looking to assist with taking precision medicine to the standard of care in the UK is Cancer Research UK’s Stratified Medicine Programme 2 (SMP2). The programme aims to change the way we deliver innovative targeted therapies to late-stage lung cancer patients in the UK by using the specific genetic profile of each patient’s individual lung tumour. Through this research it is hoped that it will be possible to identify groups of patients who are more likely to benefit from a number of different targeted drugs.

SMP2 will take up to 2,000 non-small cell lung cancer patients through innovative Next Generation Sequencing (NGS) testing. NGS allows the patients genetic biomarkers to be determined and matched to a treatment regimen under the National Lung MATRIX trial (MATRIX). The trial is being led by Birmingham’s CRCTU and Prof Gary Middleton, and is a multiple biomarker/multi-drug trial (multiple drugs reviewed against multiple genetic markers). This co-ordinated approach aims to assist the UK in establishing the infrastructure for precision medicine and drive forward the understanding on the relationship between biomarker and treatment.

Precision medicine is having a profound impact on both healthcare and regulatory infrastructure. New concepts must be considered from the volume of data, to new biomarkers of unknown clinical significance, to challenges over accuracy and consistency. One notable challenge has been observations in proficiency testing studies of Next Generation Sequencing; this has shown that variation in biomarkers detection can depend on the workflow approach undertaken.

To ensure consistency of results in the SMP2, Cancer Research UK will use Horizon Discovery’s reference materials. These reference materials contain the cancer specific genetic biomarkers and can be screened by NGS to confirm agreement across instruments and laboratories and to show consistency. The value of these reference materials is that they are cell-line derived and thus provide a sustainable common reference point. The programme relies on a community effort to embed precision medicine into UK healthcare. Collaborations between Cancer Research UK and organisations such as Horizon Discovery are contributing to this success.

In a world of precision medicine, the cost of treatment will be reduced in the long term, by removing expensive and ineffective approaches to diagnosis and treatment. It is a really exciting time for the pharmaceutical and medical device fields. It’s time to make medicine personal.