LA JOLLA, Calif., Dec. 9 (UPI) -- U.S scientists say they have restored partial function to lung cells from cystic fibrosis patients, possibly opening the door to a new class of therapies.
However, medical researchers led by Scripps Research Institute scientists caution much work must be done before the therapy can be tested in humans.
"We are very excited by these results," said Scripps Professor William Balch. "Because we came at the problem of restoring cell function from a new perspective -- using biology to correct biology -- these findings have the potential to be game-changing."
The study, performed in collaboration with cystic fibrosis investigators across the United States and Canada, showed a compound called suberoylanilide hydroxamic acid, known as SAHA, can restore about 28 percent of normal function to lung surface cells with the most common, but most severe, cystic fibrosis mutation, researchers said. SAHA is already approved by the U.S. Food and Drug Administration as a treatment for lymphoma.
"The results are very promising," said Balch. "We know that cystic fibrosis individuals with 15 to 30 percent of normal cellular function, as can occur with certain mutations, have milder cases of the disease and a more normal lifestyle than patients carrying a severe mutation. The added degree of function conveyed by SAHA or a compound like SAHA could make a tremendous difference to patients with acute disease."
The study appears in the early online edition of the journal Nature Chemical Biology.
