Michael Rose

Session two: Translational research in adult NMDs

Translational research beyond the laboratory

Michael Rose - King's College, London

The emergence of translational research arose from the recognition that basic science research often seemed far removed from the goal of finding effective treatment that could be used in medical practice to improve meaningful health outcomes. Thus far most of the effort has been in the development of therapeutic products suitable for clinical trials. However this on its own will not achieve the goal. In many muscle diseases a large amount of groundwork needed to be ready for effective clinical trials. Even after treatment is found to be efficacious further work is required to gauge its effectiveness and ensure that such treatment becomes part of standard care. Additional studies may be required to appreciate the impact of interventions over a longer period in terms of their resource implications and their effects on public health and society. To date work on the laboratory phase of translational research has attracted far more effort and 90% of the funding while the work required for subsequent phases has been under-appreciated and underfunded. Much of the translational research beyond the laboratory requires widespread collaboration amongst a range of stakeholders on a scale and scope yet to be achieved. The tasks required in the translational pathway beyond the laboratory may take some years and until these are pursued they will be a major barrier to success.

Michael Rose

26 Oct 2011