UB, Women and Children's Hospital Played Key Role in Development of new Cystic Fibrosis Drug Approved by FDA

BUFFALO, N.Y. -- The Cystic Fibrosis Therapeutics Development Center of the University at Buffalo and Women and 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 today 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 Western New York who has been on this drug longer than anyone in the world," says Drucy S. Borowitz, MD, clinical professor of pediatrics in the UB School of Medicine and Biomedical Sciences and director of the CF Therapeutics Development Center of UB and Women and Children's Hospital of Buffalo.

The Buffalo center 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 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 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, says Borowitz.

"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 Borowitz.

That treatment, says Borowitz, is currently in a Phase 2 clinical trial and is several years away from FDA 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 ," she says.

The University at Buffalo is a premier research-intensive public university, a flagship institution in the State University of New York system and its largest and most comprehensive campus. UB's more than 28,000 students pursue their academic interests through more than 300 undergraduate, graduate and professional degree programs. Founded in 1846, the University at Buffalo is a member of the Association of American Universities.