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March 2012

Above & Beyond

Roe Van Epps, 41, says that participating in a Phase 3 clinical trial for the new CF drug now known as Kalydeco has changed her life.
Roe Van Epps, 41, says that
participating in a Phase 3 clinical trial
for the new CF drug now known as Kalydeco has changed her life.

For years, Roe Van Epps had been carrying laundry baskets up three flights of stairs from the washer and dryer in the basement of her Connecticut home. After each set of stairs, she would pause to rest, use her inhaler and then resume. It was one of countless daily routines she had become accustomed to as part of life with cystic fibrosis, a disease the 41-year-old has fought since she was diagnosed as an infant.  

Then one day last winter, Roe found herself zipping up the stairs, not pausing at all. “Oh, my God,” Roe thought. Something was up.

Finding Relief in a New Treatment for CF

A few weeks before, Roe had begun participating in a Phase 3 clinical trial for Kalydeco (formerly known as VX-770). The drug was discovered in a collaboration between Vertex Pharmaceuticals and the Cystic Fibrosis Foundation, which provided significant scientific, financial and clinical support throughout the development process.

It was the fifth drug trial that Roe had participated in, and initially she was not optimistic. She had learned not to get her hopes up, since so many potential drugs never make it to patients. Still, she felt a responsibility to the larger CF community, and participating in drug trials was, she knew, crucial to the fight against the disease.

Now, as she looked back down the stairs, she had what she thought was clear reason for optimism. Nevertheless, she was cautious. “Maybe I’m just having one amazing day,” she remembers telling herself. 

For decades, Roe had known only a never-ending cycle of small victories followed by inevitable sickness and setbacks — no matter how well she ate or how faithfully she followed her daily treatment regimen, which usually took five hours every day.

“You learn to think short-term,” she says, “to focus on staying strong through this winter or this year. When I first started to feel better, I hardly told anyone. I thought if I said anything, it wouldn’t be true.”

But in the weeks following, even she couldn’t deny the evidence. Kalydeco was working. Her husband watched amazed as she shoveled snow for the first time. Co-workers mentioned that she wasn’t coughing as much. Her friends and family noticed she was no longer missing birthdays and other get-togethers. After not seeing her for a month, her hairdresser exclaimed, “Honey, even your skin looks better!”   

Advocating for Continued Advances in CF Treatments

Roe has continued to improve. Even though Kalydeco has opened new possibilities for her, Roe understands that she still has CF, and she continues to follow her daily treatment regimen faithfully.

She is also actively working to adjust to a new world of long-term planning and unexplored life choices, from learning to snow-shoe or ski to becoming the friend and family member who takes care of others, who visits others in the hospital — including those who once took care of her.

This January, the U.S. Food and Drug Administration approved Kalydeco for people with CF ages 6 and older who have the G551D mutation of CF. It is the first drug on the market that targets the underlying cause of CF.

Roe is well aware that Kalydeco is only effective for about four percent of the more than 30,000 people in the United States with CF, who have the G551D mutation — yet she also knows that the science behind Kalydeco has paved the way for research that could one day lead to a cure for all people living with the disease.

“We’ve done it once,” Roe says. “We can do it again. The science is really getting there. We just have to keep fighting, keep participating in trials. I really believe there’s a future for all of us.”

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