REACTION TO "DATELINE NBC" SEGMENT ON PETER LAW'S MYOBLAST TRANSFER TREATMENT BY CHRISTOPHER J. ROSA, DIRECTOR OF SPECIAL SERVICES FOR STUDENTS WITH DISABILITIES, QUEENS COLLEGE (CUNY) AND CHAIR OF THE SUBCOMMITTEE ON EMPLOYEE DISABILITY CONCERNS, THE PRESIDENT'S COMMITTEE ON EMPLOYMENT OF PEOPLE WITH DISABILITIES
I am writing a counterpoint opinion in response to the 12/28/98 Dateline NBC segment regarding myoblast transfer experiments conducted by Peter Law, Ph.D. at the Cell Therapy Research Foundation in Memphis, TN. This report featured the personal account of David Plemmons, a man said to have Becker muscular dystrophy who claims that myoblast transfer therapy (MTT) has restored his ability to walk. I write to add a perspective that was conspicuously absent from the Dateline dialogue -- the voices of the literally thousands of people affected by Becker and Duchenne muscular dystrophy who have chosen not to apply for participation in Law's MTT experiments.
As someone who is the same age as the gentleman featured in the Dateline segment and who has a definitive diagnosis of Becker muscular dystrophy, I stand to gain as much as anyone from the success of MTT or any other treatment for muscular dystrophy. Indeed, as I get older, as my neuromuscular disease progresses, as I lose more of my physical abilities and encounter greater respiratory problems, I feel an urgency that makes accounts like those offered by Mr. Plemmons more compelling to me than at any time in my life. And yet, despite Mr. Plemmons' very moving account, I choose not to apply to participate in MTT experiments.
Like most individuals affected by neuromuscular disease, I follow progress in neuromuscular disease research with keen interest. Those of us who closely follow these developments remember that in the early 1990's MTT represented the most hopeful approach to treatment of Duchenne and Becker MD. However, after six independent research teams found MTT to be ineffective in the mid 1990's, the scientific community moved away from MTT and towards other promising approaches, such as gene therapy.
Dr. Law claims that his refinements in MTT technology enable him to employ this approach more effectively than top scientists and leading medical researchers. While no one hopes that this is true more than me, I am concerned that he has not shared his data with the scientific community so that one may assess the effectiveness of his therapy. I am troubled by the fact that there is no validated scientific evidence upon which to base our decisions to mortgage our futures in order to participate in his experimental therapy. Indeed, despite years of requests from dozens of world-renowned investigators to evaluate claims regarding the effects of injecting healthy, immature muscle cells as a potential therapy for muscular dystrophy, The Cell Therapy Research Foundation has not presented the medical community with adequate scientific evidence to show that this experimental and very costly procedure provides any measurable health benefits. Dr. Law steadfastly refused to permit independent investigators to objectively review his data. It causes me concern that, as far as I know, no major medical center in this country, or indeed the world, is treating patients with MTT.
As it is, we are left to base our decision regarding participation in MTT on only one account of an individual. While the words and images of Mr. Plemmons standing with assistance and taking a few halting, supported steps are stirring, Dr. Law maintains that some 119 other individuals have participated in these trials. It worries me that we have heard nothing about or from these other participants in the trials.
Mr. Plemmons feels as though his participation in MTT has significantly improved his strength; I pray that it has. However, even though I haven't moved my legs like Mr. Plemmons in three years, if I were sufficiently braced for support, if I devoted a similar amount of time and effort to physical conditioning, if I had people propping me up for support and balance, I am convinced that I would still be able to "walk" as Mr. Plemmons did -- even without myoblast transfer therapy. Granted, no two experiences with Becker MD are exactly alike and we have no idea of Mr. Plemmons' physical abilities prior to his participation in MTT.
Families affected by neuromuscular disease have been given no validated scientific information by The Cell Therapy Research Foundation upon which to base their decisions regarding whether or not to participate in this costly, experimental therapy. One cannot put a price on treatments or cures for neuromuscular disease. Most families I know affected by neuromuscular disease would do anything necessary to fund effective treatments for muscular dystrophy. However, $150,000 per patient -- or a total of some $18 million -- is a lot to invest in an experimental therapy for which no validated evidence exists to support the notion that it is effective. A family or individual could use that same $150,000 in ways that have proven to dramatically enhance the independence and quality of life of people with neuromuscular diseases; it could fund modifications to make homes completely accessible to people with neuromuscular disabilities; it could buy assistive technology that allows us to live and work independently; it could purchase personal assistant services that would allow us to live fully integrated in our communities.
For those families considering MTT, I implore you to be very cautious, to demand hard evidence of the therapeutic effects of this approach, and to guard scrupulously our most precious gift -- our hope that someday no one will ever again endure the harmful affects of neuromuscular disease.
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