Biogen Idec will get early look at gene data from ALS collaboration
This article was originally published in Scrip
Biogen Idec will get a sneak peek at data from its project with two research institutions and several scientists to sequence the genomes of up to 1,000 amyotrophic lateral sclerosis (ALS) patients to inform its search for new drug therapies to treat the neurodegenerative disorder.
Biogen will fund millions of dollars worth of sequencing – less than $10 million during the next two years – conducted by the laboratories of Dr David Goldstein, director of the Center for Human Genome Variation at Duke University in Durham, North Carolina, and Dr Richard Myers, president and director of the HudsonAlpha Institute for Biotechnology in Huntsville, Alabama.
"Not all of the genes [associated with ALS] have been uncovered. There are more to be found and the best way to find them is with high-throughput screening," said Biogen senior vice president of translational medicine and biochemistry Tim Harris.
The company will get an early look at the data for its own drug development purposes, but Dr Harris said the intention is to make genetic information about the fundamental causes of ALS widely available as soon as possible.
Dr Goldstein's and Dr Myers' labs will attempt to sequence the genomes of 500 ALS patients in the first two years and sequence 1,000 genomes within five years. The information could offer a better understanding of the genetic factors behind ALS and new therapeutic targets.
Biogen has one drug candidate in development for ALS. The novel oral neuroprotective therapy dexpramipexole is in Phase III clinical testing under a licensing agreement with Knopp Biosciences. In Phase II testing, the drug showed a clinically significant effect on function and mortality (scripintelligence.com, 22 November 2011).
Through the ALS gene sequencing collaboration, Dr Harris said, "We're hoping to find other genes that will help us to understand the pathology of the disease better, so that we will have a better understanding of ALS or targets or where they converge."
The cause of ALS is not known, but approximately two people per 100,000 globally are diagnosed with disease. Only one approved drug treats ALS, adding two to three months to survival times, on average. Life expectancy generally is three to five years after diagnosis.
"Identifying the mutations that can lead to neurodegenerative disease provides a key foothold for developing new therapies and a framework for understanding variation in how patients progress and respond to treatment," Dr Goldstein said in a statement from Biogen.
Researchers at Duke and HudsonAlpha will work with several North American colleagues with a deep understanding of ALS and the genes associated with the disease: Dr Robert Brown, a neurologist at the University of Massachusetts Medical School; Dr Aaron Gitler, a geneticist at Stanford University in California; Dr Tom Maniatis, a molecular biologist at Columbia University in New York; Dr Guy Rouleau, a neuro-geneticist at the University of Montreal; and Dr Neil Shneider, a neurologist and neuroscientist in the Motor Neuron Center at Columbia University.
Dr Harris said the genetic fundamentals that Biogen and its collaborators are trying to uncover would have been discovered at some point without the company's investment, but Biogen hopes its investment will