Translating the advances in cystic fibrosis (CF) gene-therapy research into the clinic hasn’t been successful. In fact, during the 21 years since the gene responsible for causing cystic fibrosis was discovered, and despite the early excitement about the possibilities of gene therapy, no gene therapy is even close to emerging.
Right now, one thing cystic fibrosis patients can do to improve their health is to exercise.
That point is hardly lost on noted gene-therapy researcher James Wilson, M.D., Ph.D. To help patients now, he formed Team CF, an elite mountain biking team composed primarily of women, as well as Club Team members spread throughout the U.S. The goal, as member Matt Holloway says, is to make “CF” stand for “Cure Found.”
The immediate goal, however, “is to use Team CF to promote fitness as beneficial to those with CF,” emphasizes Dr. Wilson, who is professor of pathology and laboratory medicine, gene therapy program, University of Pennsylvania School of Medicine. He is also editor-in-chief of Human Gene Therapy, a peer-review journal published by Mary Ann Liebert, Inc.
The youngest of the elite mountain bike racers, Kaitlyn Rose Broadhurst, has cystic fibrosis herself. A cross-country runner at Neumann University, the 21-year old was persuaded to compete in mountain biking during an internship for the Cystic Fibrosis Foundation. Elite team members trained her. She has competed in two races and won them both.
After each race, she says, “I’ve been approached by several people for information about the team or about joining it. A lot of people are surprised I’m even doing this.”
At Kaitlyn’s first race last July, Mallory, a CF patient and five-year old daughter of Matt Holloway, watched her closely.
“I told Mallory that Kaitlyn raced and won in her first-ever mountain bike race,” Holloway says. “She looked up at me and said, ‘That means that I could race mountain bikes.’ I said ‘yes,’ and then she said ‘and I could win medals too, Daddy!’ I said ‘You sure can!’
“To me, this is a two-part inspiration,” Holloway continues. “First, it gives my daughter a phenomenal role model, the knowledge that she can do things that are harder for her to do and be successful. Second, as a parent of someone with CF, it is the most encouraging thing to see another person exercising at a competitive level and be so healthy, all due to the fact that she exercises regularly and works just as hard as the healthy person next to her.”