Hadassah researcher Prof Eitan Kerem. He hopes the drug will be able to treat other diseases caused by the nonsense mutation.Seminal research led by an Israeli team from Hadassah Hospital in Jerusalem may yield dramatic relief for cystic fibrosis (CF) patients in the next few years. It also opens up new opportunities and hope for treating genetic diseases such as muscular dystrophy.
The promising new oral drug, PTC124, is being developed by US biopharmaceutical company PTC Therapeutics, located in New Jersey, in conjunction with Hadassah’s researchers Prof. Eitan Kerem and Dr. Michael Wilschanski. It has already passed a milestone in drug development.
This month, the researchers announced positive results from their Phase II clinical trials in humans, and published their findings in the prestigious medical journal The Lancet. The orally administered pill helped 16 out of 23 Israeli study patients improve their chloride (salt) channel functioning.
A treatment for other diseases
“We hope the drug will be able to treat other diseases caused by the mechanism responsible for this mutation,” says Kerem, head of pediatrics at Hadassah University Medical Center on Jerusalem’s Mount Scopus.
