A new cloud-based data resource provides the research community with a comprehensive set of tools to help identify new subtypes of amyotrophic lateral sclerosis (ALS), a fatal neurological disorder.
The web-based tool is part of a collaborative effort with more than 100 scientists called Answer ALS, which includes biological and clinical data from more than 1,000 ALS patients. The information is intended to help investigators across the globe better understand ALS, also known as Lou Gehrig’s disease. Details of the resource are described in the peer-reviewed journal Nature Neurosciences.
«This is one of the largest resources for ALS samples in the world,» said Clive Svendsen, PhD, executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and a co-author of the paper and co-director of the Answer ALS program. «It’s a critical step forward in finding new treatments for a very complex disease that has really no effective treatments available.»
ALS is a progressive neurodegenerative disease that damages nerve cells known as motor neurons in the brain and spinal cord, leading to the loss of muscle control. With no known cure, it is usually fatal within five years of diagnosis.
Discovering new subtypes of ALS can provide clues to how an individual may respond to treatment. This information is useful for guiding disease management and can help lead to the development of new drugs that target specific cells and pathways that may only be found in certain subgroups.
«We don’t think ALS is one disease,» said Svendsen, who is also a professor of Biomedical Sciences and Medicine. «It’s very complex and we think there are different subtypes that can be targeted differently. We just need to uncover them.»
To successfully build the database, scientists first had to create a model of the disease that could be used to help study how ALS develops. Creating models of neurodegenerative diseases has been notoriously challenging because of the lack of reliable animal models or patient samples at early disease stages.
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Materials provided by Cedars-Sinai Medical Center. Note: Content may be edited for style and length.