Discover Magazine: CF Foundation’s Novel Strategy Leads to Life-Changing Therapy

“For the first time in over a decade, a striking silence fills the Cheevers' barn-style home,” begins a story in the September issue of the science magazine Discover.

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"The deep, rumbling cough that plagued sisters Laura, 14, and Cate, 12, every night of their lives, leaving them exhausted and weak, has finally stopped.”

The full-length article “Doorway to a Cure” tells the story of a new cystic fibrosis drug -- the first to address the underlying genetic cause of CF -- and the CF Foundation's role in bringing this life-changing treatment to those with the disease, including Laura and Cate Cheevers of North Andover, Mass. The drug treats about 4 percent of people with CF and was developed by Vertex Pharmaceuticals Inc. with support from the CF Foundation.

The search for more cutting-edge therapies that will benefit greater numbers of people with CF is ongoing, fueled by the science that led to the discovery of this breakthrough drug, according to the story.

“We're not going to settle for less than 100 percent of patients,” says Robert J. Beall, Ph.D., president and CEO of the CF Foundation, quoted in the story on the Foundation's focus on finding effective therapies and a cure for all people with CF.

The story describes the unusual strategy the Foundation took in 2000, investing $40 million in a research program with a for-profit company to help discover innovative approaches to help advance new CF therapies. “At the time it was unheard of for a nonprofit to take such a gamble,” the article says.

“Robert Beall deserves a lot of credit for placing a huge and expensive bet on an enterprise that could well have failed,” Francis Collins, M.D., Ph.D., director of the National Institutes of Health, comments in the article. The Foundation's drug development strategy is a promising model for attacking other genetic diseases and is now being adopted by other groups, Collins adds.

Discover is a leading science magazine written for the general public, with a circulation of about 500,000.

Read and download “Doorway to a Cure,” or look for the September issue of Discover on newsstands now.

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