Motor neurone disease patients push US to fast-track promising new drug
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Version 0 of 1. A US biopharmaceutical company is seeking accelerated approval for a new drug for a type of motor neurone disease after thousands of patients signed a petition calling for the medicine to be expedited to market. The California-based Genervon Biopharmaceuticals said early clinical trials had proved successful in slowing down the disease, which affects almost half a million people worldwide, and now wants the US authorities to fast-track the drug, called GM6 or GM604. Accelerated approval is rare, but the US Food and Drug Administration did fast-track an HIV/Aids medicine in 1992, as portrayed in the Oscar-winning film Dallas Buyers Club. Until now, dozens of companies have conducted clinical trials on treatments for the fatal amyotrophic lateral sclerosis (ALS), but all have apparently failed. The ALS Therapy Development Institute in Cambridge, Massachusetts, estimates the number of patients at more than 30,000 in the US and at least 450,000 worldwide. One person is diagnosed with the disease every 90 minutes, it says. Most people with ALS, also known as Charcot or Lou Gehrig’s disease, live between two and five years after diagnosis. Professor Stephen Hawking, who is perhaps the most famous person affected, has lived with the disease for 52 years but is an exception. Even though only 12 ALS patients took part in Genervon’s phase 2A trial for GM6, a change.org petition urging the FDA to authorise the drug immediately has gathered more than 137,000 signatures since it was launched on 21 January. “If the FDA does not grant accelerated approval, it will likely be three more years before patients are able to access this drug – meaning that most people living with ALS will not live to see it reach market,” says the man behind the petition, 54-year-old Nicholas Grillo, who lives in San Francisco and was diagnosed with ALS in October 2011. Genervon’s CEO, Winston Ko, is to meet FDA officials in the coming days to ask for accelerated approval. “At the same time as for ALS, we also conducted highly successful trials with the GM6 drug on Parkinson’s disease,” he said. The only ALS drug now on the market, Riluzole, prolongs life by two or three months, and costs between $1,000 and $2,000 a month in the US. “That word ‘treatment’ is laughable, because a few months added to life expectancy is a terrible joke,” 29-year-old Angelina Fanous said in a New York Times blog last week. ALS sufferers say they have nothing to lose by taking GM604 at this stage. Stephen Myers, who is 58 and was diagnosed five years ago, says: “We all know the risks and are prepared to take them.” Sarah Coglianese wrote on her ALS blog, “If I’d been told there was a chance that living in the washing machine and eating Styrofoam would help me, I would have done it.” The FDA declined to comment on the request, citing drug review confidentiality, and the powerful Washington-based ALS Association is not supporting the early approval campaign. Last week the association posted an open letter signed by itself and 11 other organisations and scientists, saying “it is difficult to determine whether there would be any benefit to people with ALS if we were to repeat this in a larger study or the long-term impact on quality of life. (…) “Giving a medication broadly without a clinical trial exposes those with ALS to possible side-effects that may reduce their quality of life, risks making them worse, and pulls money, personnel, and effort away from finding the cure that all of us are working together to find.” But the association’s opposition seems to have backfired. Instead of calming supporters of early approval, it has angered them and speeded up the pace of signatures added to Grillo’s petition. The association was behind the ALS ice bucket challenge, which attracted Bill Gates and many other celebrities and raised $115m for research last summer. |