U.K. green lights study of pioneering gene therapy against cystic fibrosis

Researchers in the U.K. plan to launch a mid-stage clinical trial this month for an inhaled gene therapy to combat cystic fibrosis (CF). The U.K.'s National Institute for Health Research and Medical Research Council have ponied up £3.1 million ($4.9 million) in funding to get the trial off the ground.

U.K. Cystic Fibrosis Gene Therapy Consortium, a nonprofit research group, will coordinate the 130-patient trial, which the consortium called the largest of its kind and will include patients aged 12 and up. Patients in the double-blind, placebo-controlled study will get a dose of the gene therapy or a placebo once per month over the course of a year, with results expected in spring 2014, according to the group's release.

Cystic fibrosis is a genetic disease that causes a buildup of thick, sticky mucus in the lungs and leads to respiratory infections and other debilitating symptoms. In the U.K. the only available treatments address only the symptoms of the chronic condition, and CF patients have an average life expectancy of 41 years, the PharmaTimes reports. The consortium has been working for more than a decade on their gene therapy, which is inhaled into the lungs to deliver molecules of DNA into cells to replace the defective gene that causes CF.

The Medical Research Council has also come up with £1.2 million ($1.9 million) to fund research of a modified virus as a vehicle to provide improved delivery of the gene therapy.

CF patients are forced to take a battery of treatments to control the symptoms of their disease, yet in recent years, companies such as Vertex Pharmaceuticals ($VRTX) have advanced treatments that address the root genetic cause of the disease rather than just the symptoms. The FDA in January stamped an approval on Vertex's Kalydeco, a targeted drug for about 4% of CF patients in the U.S. who have the G551D mutation. And the drug developer is now working on other targeted approaches to address the root cause of the disease.

- get the consortium's release
- read the PharmaTimes article

Suggested Articles

Fifteen of the 22 patients in a gene therapy trial no longer needed transfusions, while the remainder needed fewer transfusions.

Argos Therapeutics is ending its kidney cancer trial and mulling options, including a merger or sale, to stay alive.

CNS Pharma says berubicin is the first anthracycline drug to cross the blood-brain barrier and could transform treatment of the highly invasive brain tumor.