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Biogen, Columbia to map ALS disease genes with ice bucket money

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(Bloomberg) — Biogen Inc. (Nasdaq:BIIB) and Columbia University Medical Center will map the genes and clinical traits of 1,500 people with ALS, also known as Lou Gehrig’s disease, in an attempt to find a treatment for the devastating neurodegenerative disorder.

The effort is being financed in part out of the $115 million the ALS Association raised last year in a viral fundraising campaign that had people dump buckets of ice water over their heads and challenge others to do the same. The ALS Association is giving $3.5 million to the project.

A genetic map of the disease may help reveal the secrets of a disorder that’s not well understood, including how much a person’s genes contribute to the likelihood of developing ALS, said Tim Harris, Biogen’s senior vice president of precision medicine.

“The expectation is we’ll be able to find genes involved in phenotypes other than the presence or absence of the diseases, affecting things like how fast the onset was, or how quickly it progresses,” he said. Phenotypes refer to patients’ physical characteristics, everything from eye color to how they experience the symptoms of a disease. Phenotypes may be driven by genetics.

ALS is a progressive disorder that affects nerve cells in the brain and the spinal cord. Patients in later stages may become paralyzed.

About 30,000 Americans have the disease, according to the ALS Association. There are no approved treatments that can alter the course of the disease, only medications to help with the symptoms.

Challenging disease

Biogen, based in Cambridge, Mass., has been working on ALS for years, and one experimental treatment failed in the final stage of testing in 2013. The biotechnology company has gone back to the lab bench and plans to start human trials of a new drug candidate by the end of the year, Donald Johns, Biogen’s head of ALS research, said in an e-mailed statement.

The ALS project is part of an ongoing collaboration in genetics research with Columbia University, to which Biogen has committed $30 million.

The ALS Association, a nonprofit organization based in Washington, estimates that about $77 million of the Ice Bucket Challenge funds raised last year will be used for researching drugs, according to the group’s website. It has given to both academic researchers and companies, including a $1.5 million contribution to Cytokinetics Inc. (Nasdaq:CYTK), which is starting a final-stage trial for its drug tirasemtiv.

The association plans to distribute funds broadly in early-stage research, while holding back some dollars to help propel any promising treatment through clinical trials, said Lucie Bruijn, the association’s chief scientist.


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