Researchers from the University of Toronto (U of T) have uncovered information about amyotrophic lateral sclerosis (ALS), known as Lou Gehrig's disease, that could lead to major breakthroughs in the treatment of not only ALS, but also of dementia.
Many cases of ALS are believed to be caused by the toxic buildup of some proteins, which leads to the death of neurons in the spinal cord and brain. When this happens, patients suffer paralysis and suffocation. Most ALS patients die within five years of diagnosis.
The research team, led by Professor Peter St. George-Hyslop, found a new way ALS attacks and kills nerve cells.
"These are dreadful diseases - the more we know about how they work, the faster we'll find treatments or even a cure," St. George-Hyslop, director of U of T's Tanz Centre for Research in Neurodegenerative Diseases, said.
The research involved looking more closely at the protein accumulations, particularly the FUS protein, which the team found changes from liquid to gel, allowing it to collect other cellular components and make new proteins that are sent in concentrated form to the outer edges of neurons. The FUS protein normally plays a key role in the healthy functioning of neurons, which transmit nerve signals in the brain and spinal cord.
The U of T team found, though, that mutations can cause the FUS protein to become too dense, which affects its ability to remelt and ultimate release the cellular components it has collected.
"This kills the nerve by throttling it and preventing it from making new protein in the parts of the cell that desperately need it," St. George-Hyslop said. "The mutations force the gelling process to go further than it should have gone."
The researchers' findings were recently published in the medical journal Neuron.
