Help Mouse with Lou Gehrig's Disease. Win $1 Million.

Add another group to the growing list of those who realize that if we wait around for the current way of doing biomedical science to produce cures for disease, those cures will forever be “10 years off,” as researchers in cancer, Alzheimer’s, Parkinson’s and just about everything else have assured me (and everyone else) for years and years.


This time it is a group focused on ALS (amyotrophic lateral sclerosis), or Lou Gehrig’s disease, that has decided the traditional way of finding cures just isn't working. So the non-profit group Prize4Life has taken a page from the X Prize Foundation: today it is announcing a $1 million prize for discovery of a drug that can extend life in lab animals with ALS.


Prize4Life, founded by a group of Harvard Business School students when their friend Avi Kremer was diagnosed with ALS at the age of 29 in 2004, is the first disease-oriented group to use a prize approach for biomedical research. But prizes to foster discovery and innovation have been gaining in popularity. The X Prize Foundation offers $10 million windfalls for a spacecraft able to fly a crew 60 miles up and back (it was won in 2004), for genomics (for development of technology to accurately sequence 100 human genomes at a cost of no more than $10,000 each), and for other achievements.


ALS, a rapidly progressing neurodegenerative disease, typically kills within two to five years of diagnosis. Most commonly striking people between 40 and 70, it affects men slightly more than women and is caused by the degeneration of motor neurons. About 30,000 Americans have it at any one time, and there is no known cure.


All of which makes ALS ripe for a prize approach, Prize4Life’s chief scientific officer and neuroscientist Melanie Leitner told me. It has only one FDA-approved treatment (Riluzole, which extends life only two or three months and costs $7,000 to $10,000 a year). U.S. foundations and other non-profits spent only $29 million on ALS research last year, and the National Institutes of Health is spending only $39 million this year. Even worse, the field has seen one clinical trial after another flame out, and there is concern that the mouse model of ALS has been leading scientists astray: mice thought to be genetically identical, Leitner explained, turn out to have different levels of expression of a key protein involved in ALS, called SOD1. High levels of expression are associated with shorter survival, and lower levels with longer survival. So when an experimental drug extended survival, scientists thought they were on the right track. But it turned out, in many cases, that they just happened to have given the drug to a mouse that, for genetic reasons, was destined to live longer anyway. No wonder that when the drug was tried in people it bombed. (The list includes Pfizer’s Celebrex, which failed in a 12-month double-blind, placebo-controlled efficacy study in 300 people; creatine, topiramate/Topamax and . . . .well, it is a sad list of failure.)


The first $1 million prize that Prize4Life dangled in front of scientists was for a biomarker of the disease—some measurable protein or other biochemical that scientists can measure and that indicates whether an experimental drug is having an effect on ALS. That should reduce the cost of clinical trials, “de-risking” them somewhat and thereby attracting more biotech or pharma companies to the field. It has attracted about 50 teams, Leitner says, including those pursuing “out of the box” ideas.


The prize announced today is for the discovery of a compound that extends life in mice with two different mouse versions of ALS by 25 percent. “An effective treatment for ALS is desperately needed, and the existing mouse model is the primary gateway to clinical trials. The identification of a treatment capable of meeting the high survival bar set forward in this prize should attract the attention of those with the resources necessary to move a potentially effective ALS therapy into the clinic," said Tom Maniatis, Professor of Molecular and Cellular Biology at Harvard, an ALS researcher and member of Prize4Life’s Scientific Advisory Board. “The Kremer prize will only be awarded for a therapy that makes a major difference in the disease, the kinds of therapies that ALS patients really need.”


Time will tell whether a prize approach can work in biomedicine. Something better.

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