Novel NIH program will advance fight against ALS, other neurodegenerative diseases
UC Irvine will receive $8 million from the National Institutes of Health to establish one of six national centers dedicated to creating a database of human cellular responses that will accelerate efforts to develop new therapies for many diseases.
Leslie M. Thompson, UCI professor of psychiatry & human behavior and neurobiology & behavior, will partner with researchers from Cedars-Sinai Medical Center's Regenerative Medicine Institute, the Gladstone Institute of Neurological Disease, UC San Francisco, Johns Hopkins University and the Massachusetts Institute of Technology.
They will study brain cell activity in motor neuron disorders including ALS and build a detailed archive of these disease "signatures" that identifies cell targets for new drug treatments. ALS, or amyotrophic lateral sclerosis, also called Lou Gehrig's disease, attacks motor neurons, cells that control the muscles.
Overall, the NIH is awarding $64 million to six research groups to establish centers that support the Library of Integrated Network-Based Cellular Signatures program. The UCI-based center will be called NeuroLINCS.
The goal of the LINCS program is to utilize the latest cutting-edge technology and scientific methods to catalog and analyze cellular function and molecular activity in response to perturbing agents – such as drugs and genetic factors – that have specific effects on cells. LINCS researchers will measure the cells' tiniest molecular and biochemical responses and use computer analyses to uncover common patterns – called signatures. LINCS data will be freely available to any scientist.
"Human brain cells are far less understood than other cells in the body," said Thompson, who's affiliated with the Sue & Bill Gross Stem Cell Research Center and UCI MIND. "The collective expertise of NeuroLINCS investigators provides a unique opportunity to increase our knowledge of what makes brain cells unique and what happens during neurodegenerative diseases – with a strong focus toward effective treatments. We feel this will have broad application to a number of human brain diseases."
She and her colleagues will study the effects, or signatures, of perturbing agents on induced pluripotent stem cell-derived neurons and glial cells from "unaffected" cells and those exhibiting the pathology of motor neuron diseases.
At UCI, Thompson will work closely with the UCI Genomics High-Throughput Facility to explore gene expression patterns in these brain cells, which is expected to yield novel insights into pathways and gene networks that guide the development of cell signatures.