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