Progress in Clinical Neurologic Trials, vol 1, Amyotrophic Lateral Sclerosis.

This book on amyotrophic lateral sclerosis (ALS) is the first in a series of proposed monographs on clinical trials of neurological disorders. It consists of 21 presentations given by scientists at a meeting in June 1988, under ,the auspices of the Scientific Advisory Committee of the ALSMND Research Foundation. The conference was devoted entirely to consideration of methodological problems underlying therapeutic trials for patients with ALS. The authors are investigators from a number of countries, the majority residing in England and the United States.

According to the editor, the possibility of stopping the disease process and progressive course of ALS is unlikely for the near future; however, the search continues for a cure. This volume is an attempt to establish methods of advancing the progress of these studies in order to understand and assess claims of beneficial drug therapy.

Chapter contents include nomenclature, definition and classification of motor neuron disease, aims and assessment, methods in trial design, selection of patients in therapeutic trials, the influence of compliance and other human factors in treatment trials, bulbar dysfunction, and assessment of dysphagia.

Throughout the book, authors emphasize the importance of accurate assessment of motor power and disease progress in the clinical trials. Methods of measurement that are analyzed and evaluated are manual muscle testing, functional evaluation, discussion of the value and practicality of various functional scales, pulmonary functional testing, electromyographic assessment, evaluation of motor dysphagia, and a general critique of assessment methodology in ALS. An extensive paper on computer modeling offers this form of theoretical analysis as a helpful methods in planning ALS therapeutic trials. In my opinion, the discussions of testing tend to be weakened by the lack of emphasis on the role of physical therapists as specialists who are essential to the use and analysis of assessment methods.

Two English physical therapists discuss their special problems for trials in motor neuron disease. They consider the obstacles encountered in evaluating physical therapy in such controlled experiments. These obstacles center on problems of objective testing, difficulty in finding a representative patient group, difficulty in standardizing individual physical therapy, lack of availability of the service, and cost/benefit judgments. The many difficulties described for trials to evaluate physical therapy in ALS appear to preclude a scientific approach to such studies.

In well-written papers, the presenters approach the subject in a comprehensive, analytical manner. No conclusions are reached, however, as to the most productive form of clinical design or assessment method of a therapeutics agent. Ideas propounded reflect the experience of individul scientists and are offered as items for discussion (and perhaps argument) for the conference participants.

References at the end of each chapter document the content. Illustrations consist mostly of tables, graphs, and a few line drawings. Appendixes are added where appropriate. A subject index provides reference to particular topics in the text. A list of contributors, with their degrees and employment addresses, helps the reader to asess the experience and professional viewpoint of each contributing scientist.

Despite the discouraging outlook for the disease, some authors express hope for the future of AlS clinical trials. They suggest that developing recombinant techniques that produce a host of molecules relevant to the nervous system could be helpful in this regard. Improvement in measurement techniques, computer modeling, and general improvement in trial design also offer reasons to be optimistic for the future.

This volume meets its intended purpose of addressing methodological problems of clinical testing for ALS. It should be of value for those attempting to perform clinical trials and those who need to evaluate therapeutic claims of medication, including all physicians and supportive health personnel who deal with this condition. The text offers valuable reference material for physical therapists who are involved in treatment of patients with ALS or those active in researching the disease.

Mary C Singleton, PhD The University of North Carolina at Chapel Hill Chapel Hill, NC