Meeting the unique challenges of drug discovery for neurodegenerative diseases

Abstract

There is a mounting need for therapeutics to effectively treat neurodegenerative diseases. Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, and multiple sclerosis almost all share pathological hallmarks of accumulated misfolded protein, ultimately leading to cellular degeneration and death [1]. There is much to be learned by the successes and failures of drug discovery efforts for these respective diseases. Exciting and novel ideas from academia often fail to reach drug discovery platforms and pharmaceutical companies have had little success in their neurodegenerative disease programs thus far; currently, only symptomatic treatments are available for the majority of these diseases [2,3]. While these diseases present unique challenges in terms of drug discovery, they also offer many opportunities to change the way academics and industry work together to efficiently develop new drugs. To bring new drugs into clinical practice for neurodegenerative diseases, efforts to translate academic discoveries into drug discovery and development efforts can be expanded and partnering between academic biologists, medicinal chemists and industry researchers encouraged. Cross-fertilization of ideas between these different neurodegenerative diseases as well as between academia and industry will foster novel developments and hopefully bring us closer to developing effective treatments for these diseases. These proceedings highlight new approaches to address and overcome the specific challenges of drug discovery for neurodegenerative diseases that were discussed at the 3rd Drug Discovery for Neurodegenerative Conference (held in Washington DC on 2–3 February 2009). This conference was hosted by the Alzheimer's Drug Discovery Foundation, in partnership with the National Institutes of Health, to advance drug discovery for neurodegenerative diseases by educating scientists on the process of translating basic research into novel therapies. Over the two day conference, speakers presented lectures and case studies on Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, multiple sclerosis, as well as orphan neurological diseases. All of these diseases share common challenges and require a broad and coordinated, multi-disciplinary approach to progress novel discoveries into effective therapeutics.