A landmark in the search for an ALS drug
A US neurologist has won a 1 million prizereportedly the largest ever for developing a way to track the advance of a deadly neuromuscular ailment.
Tracking the inexorable advance of amyotrophic lateral sclerosis,the deadly neuromuscular ailment better known as Lou Gehrigs disease or ALS,has long been an inexact sciencea matter of monitoring weakness and fatigue,making crude measurements of the strength of various muscles.
This imprecision has hindered the search for drugs that could slow or block the diseases progress. But now a neurologist at Beth Israel Deaconess Medical Center in Boston has won a 1 million prizereportedly the largest ever for meeting a specific challenge in medical researchfor developing a reliable way to quantify the small muscular changes that signal progressive deterioration.
The winner,Dr Seward Rutkove,showed that his method could halve the cost of clinical trials to screen potential drugs for the disease,said Melanie Leitner,chief scientific officer of Prize4Life,the nonprofit group that created the competition.
The method does not provide a target in the body at which to aim drugs,nor will it help doctors better diagnose the disease. But Dr Merit Cudkowicz,a professor of neurology at Massachusetts General Hospital and a chairwoman of the Northeast ALS Consortium,compared Rutkoves discovery to the way magnetic resonance imaging expedited the development of drugs for multiple sclerosis.
You can use this as a tool to screen drugs to see if they will affect survival, she said,but added,The ultimate prize is finding a drug that works for ALS.
Rutkove,46,who has been treating patients with neuromuscular disease for 16 years,took advantage of the way our muscle fibres change electrical currents. With a hand-held device hooked up to electrodes on the patients skin,a doctor can send a painless electrical current into a given muscle,then measure the voltage that results.
As ALS spreads,motor neurons die off,causing muscles to atrophy. The deteriorating muscles behave differently from healthy ones,resisting the current more. In studies of humans as well as rats,Rutkove showed these variations were closely correlated with disease progression and length of survival. Its not like its the fanciest technology, he said. But I believe it will help people.
Each year,doctors diagnose about 5,000 new cases of ALS in the United States,according to the National Institutes of Health. Despite decades of clinical trials,the diagnosis remains a death sentence. It paralyses and suffocates patients while their minds remain intact. A few patients live for decadesthe physicist Stephen Hawking is the best knownbut most survive only three to five years after symptoms appear. And riluzole,the only ALS drug approved by the Food and Drug Administration,costs about 10,000 a year and extends life by just a few months. The high cost of clinical trials limits drug companies ability to test potential treatments. Researchers must recruit hundreds of patients and run trials that last as long as two years just to eliminate a drug from the running. One executive told us,For the cost of one ALS drug I can develop two multiple sclerosis drugs,so obviously I go with MS, wrote Avi Kremer,the 35-year-old founder of Prize4Life. Kremer,who has the disease himself,cannot speak or type. He made the remark during a Skype video chat from his apartment in Israel,using a sensor that tracks his forehead as he lifts his eyebrows.
Dr Doug Kerr,associate director of experimental neurology at Biogen Idec,which is working on an ALS drug,said more sensitive testing methods will allow us to test more drugs,and get an answer earlier.
Researchers say the 1 million prize,to be presented to Rutkove in June in New York,is the largest ever for solving a prescribed challenge in medical research. This kind of prize is hardly new. In the 18th century,such a challenge spurred a solution to Newtons famous problem of how to determine longitude at sea. A clockmaker,John Harrison,won the competition by inventing the marine chronometre.
Now these sorts of challenges are coming back into fashion. In December,the US Congress passed a law authorising federal agencies to use prize competitions as a complement to grants and contracts. Competitions can draw new eyes to old problems. The danger of a prize competition,though,is that if you make the wrong choices,you might be leading people in the wrong direction,said Paul A. Wilson,a professor at the Mailman School of Public Health at Columbia University.
Rutkove said his work had been under way,and supported by public financing,before he heard of the prize. But added that the challenge turned his focus toward reducing the cost of clinical trials and sped up his analysis.BINA VENKATARAMAN
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