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Drug tested at Upstate shows marked improvement in some patients with cystic fibrosis

SYRACUSE, N.Y. — Upstate University Hospital patients who have the cystic fibrosis mutation, G55ID, are experiencing marked improvement in lung function and nutritional status through a recently approved FDA oral medication called Kalydeco.

Upstate served as one of the test sites for the Kalydeco three-year clinical trial. Findings from the national trial demonstrated that Kalydeco is the first medication of its kind to effectively address the underlying cause of cystic fibrosis (CF).

“Kalydeco has proven successful in improving the quality of life for our CF patients who have the G55ID mutation, which accounts for about four percent of the CF population in the United States,” says Ran Anbar, MD, who served as the principal investigator of Upstate’s Kalydeco trial.

“Breathing for patients on this medication is less labored and we are finding that they are less prone to infections and other complications resulting from CF,” said Anbar, professor of pediatrics and medicine, director of Pediatric Pulmonary Medicine and medical director of the Robert C. Schwartz Cystic Fibrosis Program at Upstate Medical University.

Cystic fibrosis is a rare, inherited chronic disease that affects the lungs and the digestive system. The FDA approved Kalydeco last January for use in people with the CF mutation, G55ID. Vertex Pharmaceuticals Inc. developed the drug, with scientific, clinical and significant funding support from the Cystic Fibrosis Foundation.

According to the Cystic Fibrosis Foundation, in people with the G551D mutation, a defective protein caused by CF moves to its proper place at the surface of the cell but does not function correctly. Instead, the defective protein acts like a locked gate, preventing the proper flow of salt and fluid in and out of the cell.

Kalydeco works by helping to unlock that gate and restore the function of the defective protein, dramatically improving lung function and helping patients gain weight — all key clinical indicators of CF. It is hopeful that this drug will prevent progression of the disease.

While Kalydeco represents a breakthrough for patients with this particular mutation, it also represents hope for all people with CF, according to Preston W. Campbell, III, MD, executive vice president for medical affairs for the Cystic Fibrosis Foundation. “The CF Foundation is committed to finding a cure for CF, and the approval of Kalydeco marks an important first step in making that mission a reality,” he said.

Nearly 90 percent of people in the United States with CF have the Delta F508 mutation. A national clinical trial is currently under way to test the effectiveness of Kalydeco, used in combination with another potential therapy, VX809, in people with the Delta508 mutation. Upstate is currently participating in another national clinical trial to test the effectiveness of Kalydeco in CF patients with the mutation R117H.