The 6th Drug Discovery for Neurodegeneration Conference: An Intensive Course on Translating Research into Drugs

Abstract

Neurodegenerative disorders are rapidly becoming one of the greatest unmet health needs. This annual workshop facilitates innovation and progress in neurodegenerative disease drug discovery by convening stakeholders from charitable foundations, government, academia and industry who introduce scientists to the drug development and approval process. New to the 2012 workshop were candid discussions about re-visiting the CNS therapeutic development process. The continuing challenge is partly due to the poor forecasting potential of models of CNS diseases, as well as the lack of reproducibility of published studies, and greater need to increase focus on pharmacodynamic end points. Significant discussion centered on how to improve discovery approaches using examples of recent successes in the field. For example, the idea of combining reductionist, single-target strategies with functional-approach logic was suggested by several speakers, and widely discussed in the workshop. The didactic aspects of the workshop highlighted underlying concepts, best practices and trends that have characterized successful campaigns. The technical and scientific guidance was complemented by discussions of practical ways to approach the major funding gaps required for translation of projects from basic science to clinical investigations. This workshop has evolved to serve a critical educational need, with a wide range of investigator participation.