Author Topic: Superpill could treat 2,000 genetic diseases (Read 161 times)

Primo · « **on:** April 24, 2007, 06:27:39 PM »

Source:
http://www.dailymail.co.uk/pages/ live/articles/health/healthmain.html?in_ article_id= 450072&in_page_id=1774

'Superpill' could treat 2,000 genetic diseases
Last updated at 10:19am on 24th April 2007

A radical new pill that could treat almost 2000 inherited diseases including some forms of cystic fibrosis and muscular dystrophy could be available within three years, scientists have said.

The drug is able to repair faulty genes in the body that lead to crippling illness, and is already in the early stages of human trials after promising results in mice. Scientists say that if the trials go well, it could be licensed as early as 2009.

The drug is unique because it can correct a mutation that leads to thousands of diseases.

Known as PTC124, it has already had encouraging results in patients with Duchenne muscular dystrophy and cystic fibrosis.

The pill works by effectively forcing the body to ignore genetic mutations, and to produce normal proteins, rather than the mutated versions which lead to disease. Patients would have to take it throughout their lives.

"There are literally thousands of genetic diseases that could benefit from this approach," Lee Sweeney, of the University of Pennsylvania, who is leading the research, said. "What's unique about this drug is it doesn't just target one mutation that causes disease, but a whole class of mutations."

Made by PTC Therapeutics, the pill has been staggeringly successful in animal models. A study published today in Nature magazine shows that in mice with a mutation that causes Duchenne muscular dystrophy, the drug starts dystrophin production and restores their muscles to health.

The research also suggests it should also work against more than 1,800 other genetic illnesses.

Experts today hailed the "wonder pill" as a major breakthrough.

Dr Marita Pohlschmidt, director of research at the Muscular Dystrophy Campaign, said the results were "very encouraging".

She said: "We look forward to the publication of the full results of these clinical trials so that we can see more clearly what the prospects and possible timescales are for treatment."