In an effort to keep you apprised of the research activities here at the Center, I would like to share with you our plans for an ALS scientific conference next Spring. The goal of the conference is to bring together ALS experts and young investigators to brainstorm on the future of clinical trials in ALS.

As you are aware, clinical trials of all potential ALS drugs in the past decade, other than riluzole, have failed to show efficacy for the disease. Current rapid progress in translational research is likely to make an increasing number of novel therapies available for clinical trials in ALS in the near future. Thus, ALS clinical trialists must be prepared to conduct much more effective and efficient protocols. A scientific meeting would be the best way to comprehensively address the future of ALS trials.

The goals of the meeting are, first, to expand the knowledge base as regards clinical trials for both experienced and future clinical trialists in ALS; second, to generate new clinical trial designs through discussion of controversies, new ideas, and new directions; and third, to evaluate the current collaborative study groups and examine new ideas for a national collaborative study group for ALS.

Throughout the meeting we will focus on issues regarding translational research that is producing novel therapies, current issues in clinical trials in general, and lessons from trials in diseases other than ALS. Detailed sessions will be held to focus on 6 topics crucial for developing effective, 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. Finally, we will try to gain more understanding of the need or feasibility of national and regional research groups as effective collaborative structures.

The proposed meeting will be the first 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, to make the best use of advances in translational research, and to create the greatest opportunities to identify effective new drugs in ALS. The meeting will be sponsored in part by the MDA and will also involve Columbia’s celebration of Lou Gehrig’s 100th birthday.