By Rachel Barron
Legendary Yankee baseball player Lou Gehrig earned eternal fame for two things: First for his ability to play 2,130 consecutive games, and second, for what would eventually force him to leave the field forever. He had the disabling and fatal neuromuscular disease amyotrophic lateral sclerosis, or ALS for short. Today, folks diagnosed with what is also called Lou Gehrig’s disease have only one prospect when it comes to drug treatment.
But two non-profits have joined forces to boost that single number of ALS medications on the market. The Muscular Dystrophy Association and the ALS Therapy Development Institute said Tuesday they have entered into a $36 million research collaboration.
The deal calls for the association to dole out at least $6 million a year for the next three years, and for the institute give $6 million of its annual budget to the project.
Funding will go to further research, like drug discovery, at the institute’s 16,000 square-foot facility in Cambridge.
CambridgeMore specifically, the institute will use the financing in part to go back to basics, like identifying physiological pathways of disease progression.
The institute has already focused heavily on evaluating previously discovered pathways, notably those in published research. The process also involved testing drugs approved to treat other diseases.
“To the extent that the existing body of literature leads to one drugabble target, we’ve exhausted it,” said Sean Scott, president for ALS Therapy Development Institute. So far, the institute has evaluated 800 drugs.
But that’s not to say there hasn’t been some success. Currently, the institute has three drugs in early stage trials. Among them includes Abbott Laboratories’ HIV drug Ritonavir, and Bristol-Myers Squibb’ leukemia drug Hydroxyurea.
As well, the institute spun off the ALS drug developing company ALSGEN in 2004. The company has so far raised $1.75 million from private venture philanthropists.
About 30,000 people in the United States and 300,000 internationally suffer form ALS. This progressive neurodegenerative disease leads to paralysis. The disorder that causes muscle weakness and atrophy eventually leaves patients trapped in their bodies unable to speak, eat, or breathe on their own. Symptoms commonly appear in middle to late adulthood, with death in two to five years.
United StatesCurrently there is no known cure. So far, only one drug, Aventis Pharmaceuticals’ Riluzole, has been federally approved to treat the disease. But the therapeutic effects are modest, offering patients three more months on average to their life.
Aventis“When you are the relative of the patent and look around at who is in charge of curing your loved one. The answer is no one,” Mr. Scott says, which in part it why the collaboration has been spurred.
A big goal of the collaboration will be to see the institute license intellectual property and drug leads to biotech and pharma companies for further development.
As Mr. Scott sees it, the private sector hasn’t been as eager to come on board to develop ALS drugs due to the risk involved. “In a disease with no validated target, you’re up against a world of unknowns,” he said. But the day that changes, Mr. Scott anticipates big interest by the private sector. If a company can develop an effective ALS drug, he believed it will also tap a potential market worth $9.5 billion.