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July 2010

Advancing Hope: Taking New Therapies from the Test Tube to the Bedside

Advancing Hope - Connections - July 2010 - Cystic Fibrosis Foundation
The CF Foundation’s successful drug development model was highlighted by NIH Director Francis Collins; Margaret Anderson, executive director of FasterCures/The Center for Accelerating Medical Solutions; and Foundation President and CEO Robert Beall at a U.S. Senate briefing this May.

 

Currently, most research to turn scientific advances into new treatments is slow-moving: It takes 10-15 years, on average, for an experimental drug to travel from a lab to U.S. patients, according to the Tufts Center for the Study of Drug Development.

But for people with chronic illnesses like cystic fibrosis, that is time they don’t have to wait.

At a May 20 U.S. Senate briefing, National Institutes of Health (NIH) Director Francis S. Collins, M.D., Ph.D., highlighted the Cystic Fibrosis Foundation’s success at quickening the pace of research to create new therapies for CF.

“There is great progress being made in cystic fibrosis research,” said Collins, who noted how advancements in treatments and care have transformed CF from a pediatric illness to one where nearly half of people with the disease are age 18 or older. The Foundation’s model, he said, is “proof of principle of what we ought to do for many other diseases.”

Collins said his goal is to take the hope and promise of recent research developments in life-threatening diseases such as CF “and turn that into real results.”

The briefing focused on how federal funding for medical research at the NIH and other agencies can be leveraged to more quickly solve pressing medical challenges, create new therapies for patients and save lives.

Collins, a distinguished geneticist who led the international effort to map the human genome, is a co-discoverer of the CF gene. The NIH is the primary federal agency for conducting and supporting medical research.

How does the CF Foundation’s business model work?

Companies that develop drugs are primarily interested in diseases that affect large numbers of people so that once a new drug is available, a sizeable return on investment is possible. With only about 30,000 CF patients in the United States and 70,000 worldwide, CF is considered a rare or “orphan” disease.

The Foundation’s approach to drug development is based on offering drug companies incentives for taking on major CF drug development projects. The Foundation funds research at the very earliest and riskiest stages of development, a time when few other investors will step up to the plate.

Armed with this strategic, new approach focused on reducing risk, the Foundation began successfully enticing drug companies to put their considerable expertise and resources toward CF therapies in the late 1990s.

What are the results?

“We have more than two dozen drugs in various stages of clinical trials, several of which are directed at treating the basic defect of cystic fibrosis,” said Robert Beall, Ph.D., president and CEO of the CF Foundation, at the Senate briefing.

“These efforts have changed this disease from one of hopelessness and despair into one of hope, optimism and excitement among the entire CF community. We have created a roadmap of how to take promising therapies for a disease from the test tube to the bedside.”

Virtually every approved CF drug available today was made possible because of Foundation support. Since the 1980s, the Foundation and its nonprofit research affiliate—Cystic Fibrosis Foundation Therapeutics, Inc., (CFFT)—have played an integral role in the development of Pulmozyme, TOBI, azithromycin, hypertonic saline and Cayston for use as CF treatments.

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The Cystic Fibrosis Foundation is an accredited charity of the Better Business Bureau.