New Treatment for CF Shows Promise
The results of early tests involving a new treatment for cystic fibrosis
(CF), is being reported as promising. The treatment involves a drug with
the chemical name PCT124. According to news releases, PCT124, when
given to a person with CF, appears to bypass a specific genetic defect
that causes CF. Researchers indicate that the presence of PCT124 allows
functional proteins to form in the body which produce a thin, freely
flowing mucus in contrast to the thick, sticky mucus that builds up and
clogs the lungs. Recently researchers at the Hadassah Hebrew University
Hospital, Jerusalem, reported that in their study of 23 individuals with
CF, those who received PCT124 experienced positive changes in cell
membrane function and modest and yet significant changes in lung
function.
CF is a life-threatening genetic disorder that affects approximately
30,000 children and adults in the United States. Currently all the drugs
approved for this condition are designed to alleviate CF symptoms.
PCT124 represents a new, and hopefully important advancement in the
way CF will be treated in the future.