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In terms of economic loss to the community and the country, although no exact figures have been compiled, the many considerations involved in muscular dystrophy would certainly point to the disease as a severe drain on American resources. Of the adults stricken by muscular dystrophy, only a few are able to maintain jobs which would make them self-supporting. Since the disease makes people progressively less active, those afflicted find themselves increasingly incapacitated as far as finding work they can do, and are increasingly dependent on aid from members of their families or outside people for their everyday needs. In the case of children, parents who might otherwise be more gainfully employed, often have to give up all or part of their work to take care of the needs of their helpless children. And, in families where more than one person is afflicted, the economic status often reaches complete destitution. Cost of wheelchairs, braces, physical therapy, and other measures are items of extreme cost to individuals and their families.
The only reason that costs to Government agencies have not been higher is that heretofore such agencies have not fully assumed obligations to these patients, preferring to use their limited means on diseases for which the prognosis was not so serious as with muscular dystrophy. For example, if muscular dystrophy patients were given adequate physical therapy, the minimum cost for 200,000 patients would be estimated at $2,080,000 per year.
There is a great need for medical research to seek means of alleviating the crippling and fatal effects of muscular dystrophy but limited funds have prohibited any extensive research program until very recently. Basic research has only been started in the last 3 years, as a result of the voluntary contributions to the Muscular Dystrophy Associations of America. Apart from a small number of Government-supported projects, through the National Institute for Neurological Diseases and Blindness, all major work in the study of muscular dystrophy is being supported by this association which has, in the 3 years of its existence, disbursed $682,370 for the establishment and maintenance of 35 separate research projects. (See appendixes A, list of research projects, B, financial report, for specific expenditures.)
Among the preliminary findings revealed so far as a result of patient-history studies, are the characteristics of transmission of muscular dystrophy by heredity. It has been found, for example, that the most common form of such transmission is from a healthy mother to her sons. Second most common is from an afflicted parent to a child of either sex. Most important is the finding that new "dystrophy bearing” families are constantly arising by mutation.
Basic studies on protein constituents and on the chemistry of muscle have yielded fundamental contributions to knowledge of normal muscle. These findings are now beginning to be applied to the study of diseased muscle in muscular dystrophy. Analysis of dystrophic muscle has already shown the presence of defects in important metabolic processes. Whether these are causes or effects of muscular dystrophy must still be investigated.
Muscular dystrophy has been produced in several species of animals by experimental means (by cortisone overdosage, and by vitamin-E deficiency), and investigations are now being made to determine the chemical abnormalities of these forms of the disease to devise effective methods of treatment and to determine in what way these findings may be applied to the disease in human patients.
While the Muscular Dystrophy Associations of America have been interested in every facet of the muscular dystrophy problem, limited means have made it necessary to restrict investigations to the field of chemical analysis of muscle, in which field trained technicians are available to work on a modest basis. Increased funds would be necessary not only to support work in related fields, but also to train personnel for investigation in other areas not yet studied, such as the role of the various hormones in growth functions and maintenance of muscle relation of liver to muscle structure through its synthesis of protein, role of the various elements in muscle integrity, the factors of metabolism of sugar in muscle, of contracture in muscle tendon units, and many other related problems which may hold at least a partial answer to the search for a cure for muscular dystrophy.
Although public contributions to muscular dystrophy research through the muscular dystrophy associations are answering some of the research needs, in the long run, research on a scope wide enough to make a conclusive contribution to the discovery of an effective treatment and cure for muscular dystrophy would be dependent on increased Government aid. Since the voluntary muscles constitute the largest organ in the body and since many of the chemical reactions which occur in muscle occur also in other tissues, it may reasonably be expected that any fundamental knowledge of muscular dystrophy will lead to significant findings about other parts of the body—and to findings significant to other diseases. The effect of such information on the alleviation of human suffering and economic loss caused not only by muscular dystrophy, but by other conditions as well, is incalculable.
The following research programs are being financially supported by the Muscular Dystrophy Associations of America, Inc., as of August 1, 1953:
Title of research and investigator
allocated by MDAA since commencement of project
New York Hospital, Cornell Med- Investigation into the Pathogenesis of Progressive $290, 358 ical College, New York.
8,856 Degeneration in Cortisone-Treated Rabbits.
Dr. John Taylor Ellis. University of Paris, France.. Studies on the Biochemistry of Muscle in Progressive 18,500
Dr. Georges Schapira.
Muscle Fibers and in Actomyosin to Muscular Con-
Dr. Albert Szent-Gyorgyi.
and 5th Ave. Hospital, New York. Dr. Arthur S. Abramson.
mal and Degenerating Muscle.
Dr. H. Stanley Bennett.
3, 700 J. Meyer Memorial Hospital, surement of Diaphragmatic Strength in Health and Buffalo, NY.
in the Presence of Muscle Disease.
Dr. Howard G. Dayman.
4,331 Boston, Mass.
Dr. John Gergely.
Dr. Edwin F. Gildea.
18, 387 Wis.
Mitochondrial Systems of Normal and Dystrophic
Dr. John W. Harman. Albama Polytechnic Institute.... Relation of Diet to the Production and Cure of Muscu- 10, 280
lar Dystrophy in Laboratory Animals.
Dr. Edwin L. Hove. Tairersity of Arkansas.
The Role of Nucleic Acid in Muscular Dystrophy.-
Dr. William K, Jordan.
8,164 able Radioactive Potassium Determinaion.
Dr. A. S. Rose.
and Rehabilitation of Muscular Dystrophy Pa
Dr. Jerome S. Tobis. toiversity of Texas. The Study of Metabolic Patterns of Muscular Dys
1,700 trophy Patients.
Dr. Roger J. Williams. l'niversity of Utah.. Preparation of Laboratory Space for the Study of
8,000 Hereditary and Metabolic Disorders.
Dr. Max M. W'introbe.
11,880 Anserine and their Possible Physiological Roles in
Dr. Theodore Winnick and Dr. Harry M. Hines. Worcester Foundation for experi- The Effect of Steroid Hormones on the Incuration of 10, 190 Tental Biology, Massachusetts. Tocopherol with Skeletal Muscle.
Dr. Harris Rosenkrantz,
Chemical Analysis of Developing Muscle Tissue------ 8, 343
7, 722 lationships under Normal and Pathologic Condi
Dr. A. R. Buchanan and Dr. Verne Van Breemen. I This is by far the most extensive research program and includes a metabolism ward, dietary kitchen, ert site laboratory facilities, psychiatrist, physical chemist, and a large staff of investigators and techDs.
Title of research and investigator
Total funds allocated by MDAA since commencement of project
Western Reserve University, Cleve- Study of the Muscle Enzymes in Progressive Muscular land, Ohio.
Dr. Paul J. Vignos, Jr. University of I ittsburgh..
Synthesis and Metabolism of Glycocyamine...
Dr. Robert E. Olson.
Trials of Purified Growth Hormone.
Dr. T. S. Danowski.
Dr. Teru Hayashi.
Dr. David Machmansohn.
Repair of Skeletal Muscle in Experimental Muscu
Dr. Karl E. Mason.
Diseases and Blindness, Washing- Metabolic Changes and Endocrinological Studies
on a Series of Neuromuscular Cases. Johns Hopkins Hospital, Baltimore, Relation of Vitamin E to Glycolysis in Skeletal Muscle. Md.
Dr. Kenneth L. Zierler.
The following projects, which received our financial support in the past, have now been completed:
Title of research and investigator
allocated by MDAA since como mencement of project
Duke University, Durham, North
University of Colorado...
Micromethods in the Investigation of Muscular
gen in Muscular Dystrophy.
Experimental uscular Dystrophy, an Pro
tein Fractions of Dystrophic Muscle. Dr. Karl E. Mason.
Studies in Physical Therapy as it
may be applied to Muscular Dystrophy, under the direction of Dr. A. S. Abramson, chief of physical medicine rehabilitation service, Veterans' Administration Hos
pital, Bronx, N. Y. Fellowship:
Cornell University Medical Col.
lege, New York.
Fellowship known as Muscular Dystrophy Associa
tions of America, Inc., Fellowship for the study of
Statement of income and disbursements for the fiscal years ended Mar. 31, 1952
Dr. BAILEY. May I interrupt a moment, Mr. Chairman, and include the work being done in muscular dystrophy at the National Institute of Neurological Diseases and Blindness? We have a rather large project in muscular dystrophy at the present time. Would you like for that to be included in the list for the record ?
The CHAIRMAN. Yes, sir; we would be glad to have it. (The materials follow :)
NATIONAL INSTITUTE OF NEUROLOGICAL DISEASES AND BLINDNESS-STATEMENT ON
RESEARCH PROGRAM IN MUSCULAR DYSTROPHY
The National Institute of Neurological Diseases and Blindness has concen. trated its medical neurological research program during the fiscal year 1954 on that group of disorders generally known as neuromuscular. Such disorders are usually grouped together in neurologic research, inasmuch as a nerve cell in the spinal cord and its nerve, and the muscle fibers that are attached to this nerve are generally considered as a single structural and functional unit which carries the name "lower motor neuron.” Thus, a disorder anywhere along this chain, wbether in the nerve cell, the nerve, or the muscle, may cause a wasting or weakness of muscle, and it is only by understanding the functional anatomy of the entire unit that one can differentiate and investigate the various disorders.
This group of neuromuscular disorders comprises muscular dystrophy, myositis and dermatomyositis, myotonia, myasthenia gravis and amyotrophic lateral sclerosis. The most promising research in these disorders lies in understanding the pathophysiological and chemical factors underlying the disease. This insti. tute has approached this problem by organizing a clinical research team consisting of scientists qualified in neuromuscular chemistry, radioactive isotope techniques and personnel trained in electronic nerve and muscle conduction, and finally, personnel trained in the application of pharmacological agents.
Chemical studies will be performed on the primary muscle proteins, and their formation, particularly myosin and actin, which, when reacting with a chemical called adenosine-triphosphate, forms one of the bases of neuromuscular contraction. Studies of the effect of potassium on such mechanisms will be analyzed by the use of radioactive potassium (K_42). All of these studies will be correlated with electrical responses, and the pathology of the disorder will be controlled by adequate examination of biopsied muscle.
Though many of the investigations outlined above may be done in basic laboratories, the clinical implication of the findings necessitates a research team in which clinical neurologists with laboratory training collaborate with laboratory researchers. It is only by such cross-fertilization that the importance of such findings as might be obtained can be correlated rapidly with the diseases of the neuromuscular system.
The CHAIRMAN. In connection with what I have said about one organization, my attention is called to the fact that the fraternal Order of Eagles has contributed over $400,000 to the Damon Runyon Fund, and the National Junior Chamber of Commerce, or units throughout the Nation, are also engaged in raising funds for that cancer activity, under the name of the Damon Runyon Fund, all of which illustrates that there is a possibility of increasing public interest in these matters to a point where it can prove most helpful to you who are engaged in research and in special studies.
My attention has now been called to the fact that the New York State Federation of Women's Clubs has adopted multiple sclerosis as a public health project of that organization for the year 1953–54.
All of this is very gratifying, indeed, and it goes to illustrate what I have visualized as a possibility if we can get the thing moving the way we would like.
Dr. Traeger, what else do you have to offer?
Dr. TRAEGER. I would like to ask a question, Mr. Chairman, about the plans for the hearings. The agenda is still incomplete and there are other people who want to be heard and who should be heard. May I be informed as to your plans? Shall we continue this afternoon or try to finish this morning?
The CHAIRMAN. How many witnesses do you have in mind ?
The CHAIRMAN. Will you give me the subject on which they will appear, as well as their names?
Dr. TREAGER. Well, there will be Dr. Brooks and Dr. Van Meter from the cerebral palsy group; Mr. Owen and myself from multiple sclerosis, and then the other neurological disorders which will be taken up by Dr. Bailey, and the statement of the research and training program of the national institute by Dr. Bailey.
The CHAIRMAN. During the noon recess I would appreciate it if you would sort of get together and get the different witnesses lined up, so to speak, in a way that will conserve our time for the reason that we have many witnesses appearing today also on the subject of blindness and the subject of diabetes. We should like to finish our program today. So, if you can help in conserving the time of the committee, it will enable us to cover the whole field that we have in mind today, and I will ask the discussion leaders on the program of blindniess, one of whom I understand will be Dr. Johnson of the University Hospital at Cleveland, Ohio, to also have this in mind and also the doctors who will appear in the discussion for the American Diabetes Association.
We have a rather ambitious program today, larger than usual, and it will require that we give a lot of thought as to how to proceed.
We will adjourn for an hour and I would appreciate it if everyone can be here as promptly as possible so we will lose no time.
(At the expiration of the recess, at 1:45 p. m., the committee reconvened.)
The CHAIRMAN. The committee will come to order.
Dr. Traeger, I wonder if it would be possible for you to agree upon a period of time in view of the fact that our schedule is so full today,