by Jeoffry B. Gordon, MD, MPH
As a family doc in practice for over 30 years I have been always tried to conceptualize disease states beyond the standard text book parameters.
Thus I was elated by the prospect of discovering a new beneficial treatment for muscular dystrophy when 5 years ago I gave some Viagra to a 57-year old patient of mine with limb girdle muscular dystrophy for the usual purpose. In June, 2005 he returned to tell me that for 2 to 5 days after he took Viagra he felt substantially stronger in all his muscles.
He had better balance; he could get out of a chair easier; he could now do standing push-ups against a wall for exercise. Over 3 days this would gradually wear off and he would return to his base line state. This phenomenon was reproducible with additional doses.
Over the past years I have given all my PDE5 inhibitor samples to him and this helped to maintain his functional independence. Of course, since he is so gravely handicapped he is on Medicaid and it was impossible to get that insurance (or I suspect any other insurance) to pay for this (expensive) medicine for this indication.
Impressed as I was by this probable therapeutic advance, I repeatedly discussed this with the pharmaceutical reps who said they would report it to their superiors. I eventually spoke to a Pfizer medical director who seemed to have other things on his mind. A medical student and I wrote up a “case report” and submitted it to JAMA and Neurology to be rejected both times.
I presented the case to the local neurologist who worked with the local MDA affiliate and eventually to the national medical director of MDA. Both showed no interest in following up. I contacted a local university medical school pediatrician who worked on muscle diseases and a local neurologist who did clinical research and both refused even to give a Viagra to a sample patient out of legal concerns.
On February 20, 2008 the New York Times published an article “Lacking a cure, a new tack on a muscle disease” about frustration with progress toward improving the treatment of muscular dystrophy patients. I emailed every nationally renowned doctor mentioned in the article and got back only one tepid response.
Thus I was both surprised and pleased when my patient brought me a copy of the July-August 2010 edition of Quest, the Muscular Dystrophy Association’s research and health magazine, which reported on page 20 that the MDA was supporting research to test the effects of Tadalafil (Cialis) on men with Becker muscular dystrophy by Dr. Ronald Victor, MD a cardiovascular disease specialist at Cedars Sinai Medical Center in Los Angeles.
The article reported that MDA had supported 2 studies which showed that PDE5 type chemicals improved muscular and cardiac function in dystrophin deficient mice. When I spoke to Dr. Victor, he was surprised and excited to find an apparently successful clinical case which supported his future research and my patient was put in touch with him. He is, in fact, a cardiologist and came to this research through trying to improve cardiac muscle function with PDE5 inhibitors and was not a muscular dystrophy clinician.
In this case, as is so often the case in other scientific and clinical medicine insights, innovation has multiple contemporaneous independent sources. I am not writing this essay to claim priority of discovery, but to point out how frustrating it has been to bring this accidental (serendipitous) insight to the attention of the medical world and validate it. Has the rigidity of our rules (of evidence, publishing, academia, and law) stifled communication to the extent that clinical insights are being lost?
Jeoffry B. Gordon is a family physician.
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