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FDA approves drug to treat cystic fibrosis

Updated: 2/3/2012

Women & Children’s Hospital of Buffalo and University at Buffalo researcher, patients played key role in trials

The Cystic Fibrosis Therapeutics Development Center of the University at Buffalo and Women & Children’s Hospital of Buffalo played a critical role in the development of the new breakthrough drug called Kalydeco, which the U.S. Food and Drug Administration approved this week for the treatment of a less-common mutation of cystic fibrosis.

“We enrolled the first patient in this Phase 3 study, which began in 2009, so there is a patient in WNY who has been on this drug longer than anyone in the world,” says Drucy S. Borowitz, MD, Clinical Professor of Pediatrics, UB School of Medicine and Biomedical Sciences and Director, CF Therapeutics Development Center of UB and Women & Children’s Hospital of Buffalo.

Buffalo was the first site in the world to begin recruiting patients for the Phase 3 clinical trial. 

The drug repairs a specific genetic defect that affects approximately 4 percent of CF patients; this mutation happens to be more common among patients of Irish descent, explains Dr. Borowitz.

“This drug demonstrates the promise of the human genome project because it proves that you can use high throughput screening to develop drugs for genetic diseases,” says Dr. Borowitz.

She says that Kalydeco, developed by Vertex Pharmaceuticals, is the first of its class, a small molecule that “repairs” mutated proteins so that they function normally.

The drug provides the critical “proof of concept” for another drug in clinical trials that promises to treat the majority of patients with CF.  This concept for drug development may also benefit people with diseases other than cystic fibrosis.

“At our CF center, we are actively involved in the development of another treatment which combines Kalydeco with a second drug to make the most common CF mutation function more normally,” says Dr. Borowitz. 

That treatment is currently in a Phase 2 clinical trial and several years from approval.

“We believe that that treatment, if successful, will change the course of CF from a disease that people die from to a condition that people live with,” she says.  The success of Kalydeco demonstrates the power of truly translational research, she adds. A key factor, she says, is the willingness of Western New Yorkers to participate in clinical trials.

“We can’t advance the science without people,” she says, “our patients are willing to put themselves on the line. Knowledge of basic science and the integration of our patients into clinical research is really what translational research should be. The path we are taking here in Buffalo for drug development is part of an incredible story of American innovation and compassion and international cooperation to change the course of disease”.

“I am incredibly proud of our CF research team here in Buffalo led by Dr. Borowitz.  Their dedication to serving the needs of our patients and families participating in research is second to none, “said Dr. Daniel W. Sheehan, PhD, MD, Chief, Division of Pulmonolgy.  “This dedication to family-centered care is why our center was the first site to enroll for the Kalydeco Phase 3 trial.  And why earlier this month, we were able to enroll the first infant in the nation for the Baby Observational and Nutrition Study (BONUS), which will evaluate factors that contribute to poor growth in babies with CF.   Additional kudos are due to Dr. Jack Sharp, who was honored last weekend with a “Commitment to a Cure Award” from the Western New York Chapter of the Cystic Fibrosis Foundation.”

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