Research scientists at Cedars-Sinai Medical Center in Los Angeles have found that immune cells in the brain play a direct role in the development of amyotrophic lateral sclerosis, or ALS, offering hope for new therapies to target the neurodegenerative disease that gradually leads to paralysis and death.
The findings will appear in the journal Science on March 18.
The researchers focused on a genetic mutation that causes ALS, also known as Lou Gehrig’s disease, and frontotemporal dementia, another neurological disorder that typically leads to changes in personality, behavior and language.
The investigators developed two genetic strains in mice lacking the gene, known as C9orf72, which they found is important for the function of the immune system in the brain.
Instead of developing ALS, mice without the gene unexpectedly suffered immune system abnormalities. Structures within immune cells – known as lysosomes – that normally dispose of unwanted cellular material stopped functioning properly without the C9orf72 gene.
excerpt © 2016 Cedars-Sinai