MUSCULAR DYSTROPHY: Key Is Coenzyme Q - C&EN Global

Dec 7, 1970 - Some new fundamental information has been added to the slowly growing body of knowledge concerning muscular dystrophy and its possible ...
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THE CHEMICAL WORLD THIS WEEK

• Development of broader, more humanistic single-core cumculums for biology majors, and of new curriculums for training technical assistants. The report also calls for substantially increased support to medical students, possibly to be repaid by service in a national medical corps; establishment of a national institute of environmental studies; and increased federal support for natural science museums, marine biology stations, and scientific journals. It also recommends new programs for funding instrumentation, initially at a level of $25 million annually, funding of at least $50 million a year for construction of teaching and research facilities. Understanding life in molecular terms, the report concludes, is the unique basis for hope that man shall be able to cope with the major diseases to which he is subject.

RESEARCH:

Redirection of Effort Opinion is far from unanimous. But from some vantage points, the current research slowdown is seen more as a redirection of effort than a longterm decline, at least for contract research organizations. Speaking last week during a panel discussion on contract research at the American Institute of Chemical Engineers' annual meeting in Chicago, Dr. Edgar Manker, vice president of Garrett Research and Development Co., La Verne, Calif., said that Garrett's experience suggests a continuing demand for contract research. As long as an organization is able to identify significant research areas and carries out projects economically and professionally, it won't have difficulty getting research contracts, he says. A perhaps more representative evaluation of the current research situation came from Dr. Jack Bregman of Water Pollution Research and Applications, Inc., Washington, D.C. He finds contract research companies in trouble. The ones that seem to be doing well, he says, are those heavily involved in environmental areas. And there is a lack of skilled manpower, he points out, in those areas where funding is available. Dr. Bregman offers some recommendations to help alleviate problems caused by the slowdown. Contract research firms, he says, should go after more work in the industrial area. The money is not as great nor the projects as big, but projects can often begin one or two months following negotiations, compared to the longer lead

time in government work. Individual scientists and engineers, Dr. Bregman says, should take a hard look at their own futures and move to acquire the skills and knowledge needed for work in expanding research areas. Dr. Bregman feels that Government must take a hand in easing the situation by setting up a program to phase out current research projects as it phases in new ones. The relationship of scientists and engineers to Government has been as partners, not as grateful supplicants, he says. Thus, Dr. Bregman views Government as having an obligation to help in the transition period. If redirection is indeed the current phenomenon, some idea of the new directions for engineers can be gained from areas in the talking stage in the engineering division of the National Science Foundation. NSF's Lewis Mayfield points out that not all projects are in the same stage of formulation, but those being considered include enzyme engineering, superhard materials, earthquake engineering, wind engineering, communications systems, nonconventional battery materials, power systems, and metallurgy.

closely muscular dystrophy in mice correlates with the disease in man, the disease in mice closely resembles the clinical, histological, and physiological characteristics of progressive muscular dystrophy in humans. Dystrophic mice are identified at birth and are characterized by progressing signs of muscular weakness and a reduced lifespan. In humans the disease probably begins at infancy and progresses for Rye to 20 years. The Austin scientists now plan to use small-tissue enzyme microanalysis techniques to analyze human tissue samples from dystrophic patients to determine whether a coenzyme Q deficiency also exists in human beings with the disease. At the same time

MUSCULAR DYSTROPHY:

Key Is Coenzyme Q Some new fundamental information has been added to the slowly growing body of knowledge concerning muscular dystrophy and its possible future treatment as a result of a twopronged effort at the University of Texas, Austin. Key to the research (spearheaded by Dr. Karl Folkers, director of UT Institute for Biomedical Research, and Dr. Jean Scholler, director of UT laboratory for comparative pharmacology) is coenzyme Q. Coenzyme Q is a vitaminlike quinone that plays a part in biochemical mechanisms of electron transport and oxidative phosphorylation. Dr. Folkers, Dr. Scholler, postdoctoral fellow Gian Littarru, and graduate student David Jones find that mice with hereditary muscular dystrophy have a deficiency of coenzyme Q in their heart and hind leg muscles. The deficiency is more marked as the disease becomes more severe. Evidence for the deficiency comes from analyses of the succinate dehydrogenase-coenzyme Q reductase enzyme system in tissue of dystrophic mice. In a related study, the Texas team administered coenzyme Q therapeutically to mice in advanced stages of the disease. Treatment produced physical improvement of the mice and "a significantly prolonged lifespan," Dr. Folkers says. Although it isn't known just how

Folkers (left) with team Role of vitaminlike quinone

they will continue their research in mice. A prophylactic study will be carried out in which coenzyme Q will be administered to newborn dystrophic mice. Dr. Folkers and Dr. Scholler point out that the advanced disease probably can't be halted completely or reversed because of irreparable damage in muscle that begins at birth. But they are hopeful that early therapy in mice may be more successful in slowing deterioration. Details of the research, supported in part by the Robert A. Welch Foundation, Houston, and the Muscular Dystrophy Associations of America, Inc., will be disclosed in the forthcoming issue of Biochem. Biophys. Res. Commun. [41, 1298 (1970)]. DEC. 7, 1970 C&EN 19