A Question for Dr. Mendell

Buckeye Farm Beat

It’s not every day you get to play a round of golf with one the nation’s leading gene therapists.

Published on: November 15, 2010

Dr. Jerry Mendell doesn’t play much golf. In fact the round he played this fall at Pleasant Valley Golf Course near Lancaster was the first he had played since he participated in the same event a year ago. No doubt he won’t play again until next year.

When you are the director of the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and a professor of pediatrics and pathology at The Ohio State University College of Medicine, and co-director of the Muscular Dystrophy Association supported muscle disease clinic at OSU, you don’t have much time for golf.

I met Dr. Mendell and several of his associates while attending a fundraising event in support of Andrew Kilbarger, age 12. You see four years ago Andrew became the first person ever to receive gene therapy for MD. According to an article in the MDA publication “Research,” Andrew has a form of MD called Duchenne muscular dystrophy. It is a genetic disease that begins in early childhood, causes progressive loss of muscle strength and bulk, and usually leads to death from respiratory of cardiac muscle failure by the time the patients are in their 20s. DMD occurs when a gene on the X chromosome fails to make the essential muscle protein dystrophin. The therapy uses several advanced technologies, including a miniaturized replacement dystrophin gene and nano delivery technology called Biological Nano Particles. BNP’s are engineered specifically to target and carry the minidystrophin gene to muscle cells. Mendell’s treatment involved an injection of a product called Biostrophin, which contains a functional gene for the muscle protein dystrophin, into Andrew’s bicep. The trial will test six boys with DMD, each of whom will receive minidystrophin genes in a muscle of one arm and a placebo in the other arm. Neither the investigators nor the participants will know which muscle got the dystrophin genes.

“Dr. Mendell has been wonderful and we know how lucky we are to have Andrew be able to participate in this research,” says his mother Julie. She knows the treatment is just a first step in learning how to attack the disease. While it may improve the muscles in Andrew’s arm, it is not likely to reverse his condition. "We don’t hit the moon our first trip. Somebody has to step up. Somebody has to be the first one," she told the Columbus Dispatch on the day of his treatment.

The good news is Andrew was there in his own golf cart to cheer on Mendell and three of his associates as they drove and putted golf balls in his name. The event raised more than $10,000 with the Fairfield County Cattlemen providing steaks after the round. Through the annual outing and other events the Kilbargers have raised nearly $100,000 for the research.

Mendell came even though he had just returned from meetings in Dallas late the night before and was preparing to go to Washington to talk strategy with the National Institute of Health later that evening. “Golf is challenging, but there is no way I could ever take the time it requires to play it,” Mendell confided to me as we walked between tees.

Of course it is not everyday you get to speak informally with a pioneering geneticist and one of the nation’s leading neurologists. He told me of the frustrations that had occurred in the 20 years of research trials required to bring this treatment to young patients. I suspected he would prefer not to be quizzed by an ag reporter, but a nagging question came into my mind as we moved through the round.

“Dr. Mendell do you think there is any reason we need to be concerned about genetically modified food,” I finally asked as the round neared an end.

Without hesitation, he replied, “No. I don’t see any basis for concern. The genetic modifications that are being made with seeds are nothing different than we have done for centuries. Our techniques may be different, but I can see no reason it would not be safe.”

The way he spoke I was quickly assured this is a trivial point compared to the difficulties of youngsters like Andrew and heartbreak that parents of children suffering from illnesses face everyday. Congratulations to Dr. Mendell and all the scientists who have the courage and vision move the world forward with new discoveries. And special thanks for taking some time support the Kilbargers with a round of golf.