The ALS Association
FOR IMMEDIATE RELEASE
Washington, D.C., (January 15, 2016) — The ALS Association is pleased to announce a new collaboration between the Gladstone Institutes in San Francisco, which is a member of The Association-funded Neuro Collaborative and the biotechnology company Biogen in Cambridge, Mass., to discover novel drug targets for the treatment of amyotrophic lateral sclerosis (ALS).
The Neuro Collaborative is an innovative partnership among three California research labs focused on development of new ALS therapies: The Svendsen lab at Cedars-Sinai in Los Angeles, site of the groups’ stem cell core; the Cleveland lab at the University of California San Diego, engaged in development of novel therapeutics; and the Finkbeiner lab at the Gladstone Institutes, which is affiliated with the University of California at San Francisco.
The Neuro Collaborative is funded by The ALS Association with a $5 million grant stemming from the funds raised through the Ice Bucket Challenge. Part of the Neuro Collaborative’s mission is to develop potential therapies that can be delivered to pharmaceutical companies for clinical trials.
Gladstone Senior Investigator Steven Finkbeiner, MD, Ph.D. and his group, invented and use a novel robotic microscopy system, called “Brain Bot,” which allows longitudinal imaging of individual neurons in culture, making target identification and drug discovery faster and more efficient. The lab has identified protein homeostasis as an important factor underlying neurodegeneration. To this end, they are leveraging their microscopy system to develop novel small molecule therapeutics that can clear toxic proteins from neurons and extend their lifespan. In addition, the lab is carrying out screens in search of novel drug targets that could be used to design new therapeutics that mitigate neurodegeneration.
Biogen has carried out genetic screens for genes that affect Drosophila ALS models and identified numerous potential targets that improve ALS-related phenotypes in these models. To evaluate and further validate potential targets for the development of new medicines, Biogen is interested in carrying out secondary screens in mammalian neurons. The new collaboration between the Finkbeiner lab and Biogen will greatly accelerate this effort.
“We are deeply gratified to see the emergence of this new collaboration,” said Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The ALS Association. “We believe the fastest route to new therapies for ALS is through forging partnerships such as this one, which combines the expertise of a major research laboratory with that of a major biotechnology company, both of which have proven their ability and commitment to advancing the discovery of new ALS treatments.”
“This collaboration offers us the opportunity to bring our state-of-the-art imaging technology fully to bear on the critical challenge for discovery of new ALS drug targets,” Dr. Finkbeiner said.
ALS is a significant focus of drug discovery and development at Biogen, a research-driven biotechnology company that has developed multiple medicines for neurologic conditions. This collaboration is expected to lead to better understanding of disease mechanisms, identification of new drug targets, and accelerate development of new treatments for ALS.
“Biogen is committed to ALS research and collaborating with the world’s best scientists to tackle this devastating disease,” said Spyros Artavanis-Tsakonas, Ph.D., Chief Scientific Officer at Biogen. “We are excited to combine our capabilities in pathway discovery and target validation with the screening technologies and imaging expertise of the Gladstone Institutes’ Neuro Collaborative to find new answers to ALS.”
The Gladstone-Biogen collaboration is scheduled to start in the first quarter of 2016 and will be led by Dr. Finkbeiner and Dr. Ashkan Javaherian at the Gladstone Institutes along with Dr. Mark Kankel and Dr. Anindya Sen from the Pathway Discovery research group at Biogen. The collaboration will include developing novel cellular models of ALS and building an infrastructure for high-throughput screening to identify new drug targets and develop small molecule therapeutics for ALS.
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.
About the Gladstone Institutes
To ensure our work does the greatest good, the Gladstone Institutes focuses on conditions with profound medical, economic, and social impact—unsolved diseases of the brain, the heart, and the immune system. Affiliated with the University of California, San Francisco, Gladstone is an independent, nonprofit life science research organization that uses visionary science and technology to overcome disease. Find more information at gladstone.org.
Through cutting-edge science and medicine, Biogen discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, hematologic conditions and autoimmune disorders. Founded in 1978, Biogen is one of the world’s oldest independent biotechnology companies and patients worldwide benefit from its leading multiple sclerosis and innovative hemophilia therapies. For product labeling, press releases and additional information about the company, please visit www.biogen.com.
About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.alsa.org.