Development of measurement techniques

In the past few years we have witnessed an unexpected and unprecedented increase in the number of therapeutic trials in ALS, and it is reasonable to anticipate that this activity will continue, if not accelerate, in the future. Almost every major neurologic center in the United States and Europe is participating in an ALS trial. The evaluation of one drug alone involved over 60 centers and more than 1,500 patients. ALS conferences to review progress and plan for the future, which typically convened every 4 years, now occur every few months. At a recent American Academy of Neurology annual meeting, attendees to the sessions on ALS overflowed into the hallway.

Several interrelated events appear responsible for this intensified activity:

1. The revolution in molecular biology has led to a number of promising new drugs produced by recombinant methodology that have potential major commercial value.

2. Molecular genetic research has provided new insight into the pathogenesis, if not the etiology, of some familial forms of this enigmatic disease.

3. There have been promising new data about the possible role of glutamate neurotoxicity in the sporadic forms.

4. The natural history of ALS has been defined in quantitative and reproducible terms.

5. There have been significant improvements in the construction of therapeutic trials, resulting in trials that are more reproducible and theoretically more efficient.

6. Although ALS is a relatively uncommon disease, there is reason to suspect that any therapeutic efficacy demonstrated in ALS might lead to the treatment of other late-life degenerative diseases, such as Alzheimer's and Parkinson's diseases.

7. Patients and their families, as well as the voluntary health organizations representing them, have become increasingly active in promoting and supporting efforts to find effective treatment.

Over the past decade, the Neuromuscular Research Unit at Tufts-New England Medical Center has had …