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Cystic-fibrosis gene-therapy trial rescued

The UK government has awarded £3.1 million (US$4.9 million) to support a key gene-therapy trial for cystic fibrosis that was previously threatened by lack of funding. The trial, now scheduled for launch by the UK Cystic Fibrosis Gene Therapy Consortium this spring, is to be the largest of its kind.

Cystic fibrosis is a disease caused by mutations in the gene CFTR. Since CFTR was first sequenced in 1989, researchers have been chasing the hope of using gene therapy to treat the condition (see Human genetics: One gene, twenty years). Early attempts failed, however, and many in the field moved away from the approach, pursuing small-molecule drugs instead. The first of those drugs to correct the function of the CFTR protein was approved by regulators this year, but only works in a small fraction of patients with a specific kind of mutation (see Drug bests cystic-fibrosis mutation).

Meanwhile, the approximately 80 researchers in the UK consortium never gave up on the gene-therapy goal and developed a technique that uses liposomes (tiny vesicles made out of lipids) to shuttle CFTR into cells. Early clinical trials yielded promising safety and molecular data, but the fate of the approach seemed in question last summer when the Cystic Fibrosis Trust, a UK charity that had long supported the project, announced that it could not raise enough money to fuel the next phase of the trial.

Today, the consortium announced a new grant of £3.1 million from the UK Medical Research Council and the National Institute for Health Research. The money will be adequate to support a trial in 130 people with cystic fibrosis. Patients will receive their first doses this May, and results are expected in the spring of 2014.

Relying on liposomes means that the CFTR gene will not be integrated into the patient’s genome. Although that may ease some safety concerns, some in the field are concerned that the liposome method may also reduce efficacy.  It also means the treatment will need to be repeatedly administered. Patients in the consortium’s trial will receive monthly doses for one year, but patients with cystic fibrosis don’t blink an eye at the thought of repeated doses, says Eric Alton, the consortium’s coordinator and a gene-therapy researcher at Imperial College London. Many spend hours a day taking medicines, breathing inhalers, and wearing special chest-thumping vests to loosen the mucus in their lungs (shown above). “When we tell them this treatment is once a month, they just laugh,” he says.

The Cystic Fibrosis Trust is not the only charity that has struggled to maintain support for research: at the end of last year, Cancer Research UK announced that it would cut its research budget by about 10% over the next three years as a result of the global financial crisis (see UK cancer charity slashes research budget). For Alton the experience was a lesson in the need to be creative in seeking alternative funding. “We no longer think this is sustainable on philanthropy alone,” he says.

The consortium has approached venture capitalists with the idea of forming a biotechnology company around the intellectual property that the project has generated over the past decade. Even with the new government grants, that idea is still very much on the table, says Alton. “The government warmly encouraged us to find industrial support,” he said. “And we are absolutely considering that.”

Image: courtesy of User:ImGz via Wikimedia.

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