A breakthrough in the treatment of cystic fibrosis by researchers in Israel has been reported in an upcoming edition of The Lancet.
Dr Eitan Kerem and colleagues at Hadassah Hebrew University Hospital in Jerusalem found that a new drug, PTC124, is able to bypass the genetic defect in protein-making and improve the functioning of weakened cell membranes.
The research focussed on premature “stop” signals in genes responsible for the transmembrane conductance regulator (CFTR), which creates the protein channels crossing the cell membrane that permit chloride ion transport.
The premature signals are defects that result in dehydration of the mucus, encompassing the body’s epithelial cells. The new drug was created to permit the CFTR pathway to function normally, by allowing the protein-making machinery to avoid or bypass the stop signals.
Researchers studied 23 patients who received PTC124 in two cycles. Each cycle consisted of three PTC124 doses per day for two weeks followed by two weeks without treatment.
The dose in the first cycle was lower than the dose in the second. The researchers measured drug efficacy by measuring tiny voltage changes across the nasal epithelial cells, informing improvements in chloride ion transport through the cell membranes.
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