Researchers Huddle in Frigid New York
7 March 2007. New York felt like the windy city on February 5 and 6, when scientists gathered there for the conference on Drug Discovery for Neurodegenerative Disease. The 150 troupers who left their dens despite the bitter cold were rewarded with a warm, cozy venue and a full slate of speakers. Sponsored by the Alzheimer’s Disease Drug Discovery Foundation, the meeting brought together academic and pharmaceutical industry researchers to talk about finding new treatments for Alzheimer disease, with a goal of educating and engaging more academic researchers in the search. Meeting host Howard Fillit directs the ADDF, and was a founding director of its parent organization, the Institute for the Study on Aging. He called neurodegenerative diseases the greatest unmet medical need today, as the number of affected people is expected to triple in the next generation.

AD drug development comes with special risks and challenges, said keynote speaker Barry Greenberg of Neurochem, Inc. in Laval, Quebec, Canada. The hurdles line up to make a formidable obstacle course: Long, slow-moving disease, difficult diagnosis, lack of surrogate biomarkers for progression, poor preclinical animal models, challenging trial design, and an evolving regulatory scene. In addition, drugs need to act in the central nervous system, which raises special problems of delivery and toxicity. But the rewards will be great as well, as any reasonable disease-modifying drug will be a blockbuster, Greenberg said.

The meeting aimed to educate academic researchers about work on the other side of the fence. Drug discovery is a distinct discipline from academic science, and quite foreign to many academic researchers. Representatives from industry sketched out the process, explaining the path a potential therapeutic takes from screening, through hits to leads, then on to candidates that undergo pharmacology, toxicology, and preclinical testing in animal models. The hope is that by understanding how the pipeline flows, academicians see where their efforts can best feed into the venture.

An important take-home message was that drug development is a risky business—the pipeline looks more like a funnel than a river. For every drug that reaches the market, dozens of compounds and projects fail, most at early stages. At every step in the process, companies are looking for ways to minimize their risk, and appreciating that mind-set seemed the key to understanding what industry wants from academia. How, then, can academic researchers aid drug discovery? The theme that emerged was “complement, don’t compete.” Rather than try to duplicate pharma’s efforts, academicians could look for ways that their research can help reduce the risks of carrying programs forward. That can mean more targets, better assays, promising compounds, new animal models, or just about anything that offers new information on which to base decisions.

The talks ran the gamut from target identification and screening compound libraries to animal models, drug delivery, licensing and patents, and even some thoughts on starting your own company. Our coverage offers details on all. As always, we welcome reader comments and contributions from others who attended the conference.—Pat McCaffrey.