: Amyotrophic Lateral Sclerosis (ALS) is one of the most devastating neurodegenerative disorders, leading to death 3-5 years after symptom onset. Approved since 1996, Riluzole is the only drug available for ALS, but its benefit is limited. Clinical trials of all other potential ALS drugs in the past decade have failed. Current rapid progress in translational research is likely to make an increasing number of novel therapies available for clinical trials in ALS. Thus, ALS clinical trialists must be prepared to conduct much more effective and efficient clinical trials in the near future. We propose to hold a scientific meeting on ALS clinical trials at Tarrytown House, Westchester, New York, on June 13-15, 2003. The goals of this meeting are: 1) expanding the knowledge base in regard to clinical trials for both experienced and future clinical trialists in ALS; 2) generating new clinical trial designs through discussion of controversies, new ideas, and new directions; and 3) evaluating the current collaborative study groups and examining new ideas for a national collaborative study group. On Day 1, we will focus on the educational component, discussing translational research that is producing novel therapies, current issues in clinical trials, in general, and lessons from trials in diseases other than ALS. Day 2 will focus on 6 topics crucial for developing effective and efficient future ALS trials: 1) problems in defining the natural history of ALS; 2) selecting primary outcome measures; 3) protecting patients and preserving trial quality; 4) study designs and aims; 5) emerging surrogate markers; and 6) drug combination trials. Day 3 will focus on funding issues, the issues of the clinical trial community, and the pros and cons of national and regional study groups as effective collaborative structures. The participants will include ALS clinical trialists, basic research scientists, young clinical investigators/fellows, funding agencies, regulatory agencies, drug companies, and anyone who has a significant interest in ALS clinical trials. The proposed meeting will be the first NIH-supported scientific meeting in ALS clinical trials, providing an unprecedented opportunity to discuss issues that must be resolved so that clinical trialists can conduct more effective and sophisticated trials that make the best use of advances in translational research, and create the greatest opportunities to identify effective new drugs in ALS.
