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This is the story about an Indianapolis neurologist, Charles Bonsett, M.D., who believes he discovered an effective treatment for Duchenne muscular dystrophy over 50 years ago. Unfortunately, his discovery remains virtually unrecognized by the scientific and medical communities.

Dr. Bonsett is 98 years old and fears he will die without his discovery being used to treat the patients to whom he dedicated his professional career. In addition to his private practice, Bonsett was a clinical professor at Indiana University School of Medicine, and among other university directorships, led the Muscular Dystrophy Clinic at Riley Hospital for 30 years.

I have known Dr. Bonsett my entire career as a man of great intelligence, generosity, and integrity. I have heard him tell the story about his muscular dystrophy (MD) research multiple times. The story he relates is always exactly the same concerning the steps taken to define the problem in this disease and to identify the treatment. Bonsett’s years of investigation of MD and his discoveries have been almost completely ignored by the world. Worse, his research has been mysteriously ridiculed and discredited.

MD is a fatal genetic disease resulting in weakness and death of muscle. Research has centered since the 1980’s on the deficiency of a structural protein element of muscle called dystrophin, which is essential for the proper function of muscle. Dystrophin researchers have always had the ears of funders, including the Muscular Dystrophy Association affiliated Jerry Lewis, whose research funding has always flowed to supporting ways of fixing dystrophin. These organizations refused to even talk to Dr. Bonsett who pursued a different line of research. Really? He was denied the necessary big research money.

Dr. Bonsett instead focused on a metabolic approach to the disease. He examined the steps in the Krebs Citric Acid Cycle, which he hypothesized was defective in MD. Through systematically adding metabolites to muscle tissue cultures and observing the effects, he was able to find the main metabolic problem. He identified adenylosuccinic acid (ASA) as the crucial metabolite that when added to the culture corrected the problem and potentially became the treatment. MD is a complex dysfunction of multiple metabolic pathways rather than a secondary result of the dystrophin insufficiency.

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Dr. Bonsett was able to secure limited local research funds from the Indiana MDA, United Way, and Community Hospitals Foundation in Indianapolis that lead to early stage FDA clinical trials of ASA. Fourteen children were given ASA and all but one (who died of an unrelated medical problem) had reversal of their disease, some markedly. But the funding finally fell apart in 1990, and the process of drug development and manufacturing of ASA ceased. Dr. Bonsett watched nearly all of the children slowly die.

Did this brilliant neurologist actually find a treatment for Duchenne muscular dystrophy? I am certainly no expert in MD, but I know his research was honest, of excellent quality, and very detailed.

Bonsett’s work was recently recognized by Australian researchers as very credible. His research should likewise be revisited by American medicine, muscular dystrophy advocate groups, and the pharmaceutical industry. Even the dietary supplement industry, more feasible from a financial and regulatory standpoint, could be a consideration. I am told that the future of MD research is gene therapy, but that will take many years to potentially unfold. Is there not anyone willing to listen?

Many roll their eyes at the mention of Bonsett’s research. But just maybe, he deserves a Noble Prize in medicine.

Richard Feldman, MD, is the former Indiana State Health commissioner. The opinions are the writer's.

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