Sept. 3, 1999

ON GENE THERAPY NEWS AND TELETHON EXCITEMENT

This Report was written from Telethon headquarters in Hollywood some 48 hours prior to showtime for the 1999 Jerry Lewis MDA Telethon, which airs at 9 p.m. EDT on Sunday, Sept. 5.

The weeks preceding the Labor Day weekend broadcast of our annual Jerry Lewis MDA Telethon are always hectic, given the fact that many hundreds of hours of preparation are required to ensure that the 21 1/2-hour event will proceed according to plan, or at least as close to plan as possible.

This year, even given the "unpredictibility factor" that's part and parcel of every MDA Telethon, the level of stress and uncertainty was unusually high. This was due in large part to Jerry Lewis' recent bout with viral meningitis, an illness that had its onset in early August while Jerry was on a performing tour in Australia. Jerry has been under constant doctors' supervision ever since, trying to conserve energy when possible while pushing forward with plans to host our 1999 Telethon.

It's a pleasure to report the glad news that Jerry stands ready and eager to assume his customary duties here at CBS Television City in Hollywood. It's clearly a testament to our national chairman's extraordinary dedication to MDA's cause, as well as to his remarkable recuperative powers.

Meanwhile, Jerry and all of us at MDA waited with considerable apprehension to learn the outcome of a much anticipated meeting of the Recombinant DNA Advisory Committee, a subcommittee of the National Institutes of Health.

The purpose of the Sept. 2 meeting in Maryland was to determine whether proposed human trials of gene therapy as a potential treatment in limb-girdle muscular dystrophy (LGMD) could proceed under the direction of MDA-funded researchers. Years of painstaking work were riding on this meeting. So were the hopes of thousands of families served by MDA.

Word on the committee's ruling came to us in the early afternoon of Sept. 2 at Telethon headquarters in Los Angeles, and the word was "go."

Within hours, MDA-funded investigators in Ohio, acting on the committee's positive recommendation, implemented the first step in testing gene therapy in human beings. They administered a gene therapy injection into a foot muscle of Donavon Decker of Huron, S.D., who has LGMD.

It was a historic moment, perhaps the most significant moment in neuromuscular disease research since the momentous 1986 discovery of the gene that, when defective, causes Duchenne and Becker muscular dystrophies.

The injection is the initial stage in a study to determine the safety of using gene therapy in humans with LGMD. The study is being conducted under the supervision of Dr. Jerry Mendell of Ohio State University in Columbus and Dr. James Wilson of the University of Pennsylvania in Philadelphia. Five other universities are participating in this collaborative MDA research initiative.

If successful, this study will pave the way for testing of gene therapy's effectiveness in correcting the faulty instructions provided to muscle cells by a flawed LGMD gene.

In another sense, this is also an important first step in MDA's larger strategy to develop gene therapy to treat all of the genetic diseases targeted by our Association. Duchenne muscular dystrophy remains a paramount priority. Because the Duchenne/Becker gene is so large in comparison to other genes, MDA-funded researchers are still tackling the challenge of developing a reliable "transport" system to get the gene inside cells.

In the meantime, our gene therapy efforts with LGMD have advanced to this critical juncture.

This milestone development would have been unthinkable without the leadership and inspiration provided by Jerry Lewis. For more than three decades, Jerry has used the platform of our nationally broadcast MDA Telethon to galvanize the American populace in support of MDA's efforts to conquer neuromuscular diseases. His tireless efforts have earned the recognition of the American Medical Association, which bestowed upon Jerry and MDA a Lifetime Achievement Award "for significant and lasting contributions to the health and welfare of humanity."

I find myself reflecting on other legendary figures from show business, some such as Frank Sinatra, Sammy Davis Jr. and Dean Martin who are no longer with us, who made crucial contributions to MDA's research efforts through their participation in our Telethon. By answering Jerry's call and contributing their talent to help make our Telethon an unbeatable entertainment event, they helped draw attention to MDA's urgent cause and made the pledge phones ring time after time. The result was a constant acceleration over the years of MDA's research progress.

Those beloved performers will always have our gratitude. So will the many celebrities and others who are giving of themselves to make our 1999 Telethon another extraordinary television event. It's wonderful to know we have so many friends who understand the direct relationship between our Telethon's success and MDA's continued ability to foster new research studies seeking treatments and cures.

Elsewhere on our Web site you'll find a press release and FAQ with more information on the new MDA-funded LGMD gene therapy development, plus a comprehensive series of articles in the current Quest magazine providing background on our gene therapy efforts. Also in Quest is a special preview of Jerry's 1999 Telethon.

Please remember that, at any time before, during or after our 1999 Jerry Lewis MDA Telethon, you can make a pledge to support MDA's life-enhancing programs in research, clinic services and summer camps for "Jerry's kids." To donate, simply click here, or have your credit card ready and call 800-FIGHTMD (800-344-4863). As always, on behalf of Jerry Lewis and all of us at MDA, thank you.

With every best wish . . .

The Ross Report Archive