RTD Success Stories - Better targeting of multiple sclerosis
Multiple sclerosis (MS) is a disabling disease of the central nervous system that affects nearly one in a thousand people, principally young women. A variety of treatments can improve patients' quality of life, but with variable results. There is no definitive cure and current therapies are only partially effective. Multiple sclerosis brings high costs – to sufferers, their families and to health systems. In the United Kingdom, for example, the lifetime cost per sufferer is estimated at over EUR1.5 million, and the total annual cost in the EU is around EUR 12.5 billion. thus, early and effective treatment, while not offering a cure, can reduce the very high social and economic costs of the disease – related to its early onset, long duration, loss of productivity and high treatment costs. The problem with available treatments is that they do not work for all patients. Given the high cost of these treatments, the ability to identify those patients who would benefit most, or would not suffer from adverse effects, would go a long way to improving the management of MS and ensuring earlier treatment and better outcomes. Searching for signposts "Despite the fact that the range of new drugs has increased considerably in recent times, there is no definitive cure for MS," explains Professor Koen Vandenbroeck of the Department of Neuroscience at the University of the Basque Country in Spain. "So there is a pressing need both for novel therapeutic targets and for approaches to increase the effectiveness of existing treatments. The research we are doing may help to identify biomarkers [biological indicators] that serve to delineate patient groups more likely to benefit from specific treatment options." Together with partners from the EU and Russia, Prof. Vandenbroeck is working in the EU-funded UEPHA-MS network, which is investigating why some MS patients respond better to therapies than others, and to identify new biomarkers. Pharmacogenomic biomarkers can be molecules, including special forms of DNA, genes, proteins, or immune cells. Critically, they can predict a patient's response to drugs. "There is no magic concoction against multiple sclerosis. We need to make sure that every patient can benefit from a treatment specifically tailored to his or her genes. The network's contribution is helping to put the partially blurred current picture of MS drug management into sharper focus," says Prof. Vandenbroeck. Towards the personal touch The UEPHA-MS team is sharing genetic and clinical data on MS and preparing the ground for treatment clinical trials which, in the best possible scenario, could take place in two or three years' time. The ultimate goal is to identify new therapeutic possibilities and pave the way for a cure for MS. Progress in recent years has been huge, explains Prof. Vandenbroeck, including identification of around 50 risk genes associated with MS. "This hints at a primordial immune-based mechanism underlying the disease. We are just now starting to understand something of the precise molecular make-ups that may make people vulnerable to MS and that may determine the individual efficacy of drug treatments," he continues. Among its successes, the team has identified new biomarkers for the likely effectiveness of interferon-B. UEPHA-MS researchers have also discovered that 'natural killer cells' can influence the effectiveness of certain drugs. "These findings may constitute an exciting starting point for the development of personalised medicine for treating multiple sclerosis," affirms Prof. Vandenbroeck. "Better targeting and timing of treatment would then lead to lower costs for patients and health-system providers as well as offering sufferers a better quality of life." - Project full name: United Europeans for the development of Pharmacogenomics in multiple sclerosis - Project acronym: UEPHA-MS - Project reference number: 212877 - UEPHA-MS Project Website - Name/country of project coordinator: Koen Vandenbroeck, University of the Basque Country, Spain - Total project cost: EUR 2 359 000 - EC contribution: EUR 2 359 000 - Project start/end: September 2008 to September 2012 - Other partner countries: Spain, Netherlands, Germany, France, Russia