Drug makers are edging closer to new treatments for cystic fibrosis that could stop the destructive impact of the disease, says an article in The Wall Street Journal today.
"We really believe with these new drugs that we should be able to add decades to the lives of patients in a very short period of time," Robert J. Beall, Ph.D., president and chief executive of the Cystic Fibrosis Foundation, says in the article.
The drug that's furthest along is a pill from Vertex Pharmaceuticals, which on Wednesday filed for regulatory approval of its drug Kalydeco (VX-770). If approved, it will be the first drug on the market that targets the underlying cause of CF.
The CF Foundation played a significant role in the development of the potential drug, which is for people age 6 and older who carry at least one copy of the G551D mutation of cystic fibrosis.
The story also highlights 8-year-old Capri Faulk, who has CF and participated in the Kalydeco clinical trials. Since starting on the medicine in 2009, she has added 24 pounds to her 49-pound frame, and can play outside with her brother and sister without getting exhausted, her mother Nicole Faulk says in the article.
"I had hopes and dreams for her, and I was scared they would be robbed from her and from me" until the new drug came along, she said.