LONDON (Reuters) - A new kind of cystic fibrosis drug, designed to bypass a genetic defect to treat the disease, has produced promising results in mid-stage clinical trials, Israeli researchers said on Thursday.
The medicine, known as PTC124, is being developed by PTC Therapeutics, a privately owned biotechnology company in South Plainfield, New Jersey.
Cystic fibrosis — a disease in which the body produces a thicker-than-normal mucus that clogs the lungs and other organs — is caused by mutations in the cystic fibrosis transmembrane-conductance regulator (CFTR) gene.
PTC124 can bypass the defect in patients’ protein-making machinery and improve the functioning of cell membranes, Eitan Kerem and colleagues of Hadassah Hebrew University Hospital, Jerusalem, reported in an online edition of the Lancet journal.
They studied the drug in a Phase II trial involving 23 patients with cystic fibrosis and found convincing changes in cell membrane function, coupled with modest but statistically significant improvements in lung function.
Stephen Hyde and associates at the UK Cystic Fibrosis Gene Therapy Consortium in Oxford commented that the positive findings showed further larger-scale trials were warranted.
PTC124, which is given by mouth, is one of a number of experimental drugs in development to correct CFTR defects. Vertex Pharmaceuticals also has a compound called VX-770 in Phase II tests.
Cystic fibrosis affects about 70,000 children and adults worldwide, according to the U.S. Cystic Fibrosis Foundation.
Reporting by Ben Hirschler; Editing by Louise Ireland