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Clearing of Protein Paves Way for Neuron Survival in ALS, Study Says

The ALS Association reports in a recent news release that researchers have demonstrated increasing the clearance of a misfolded protein from neurons improves their survival; a finding that emphasizes the importance of the autophagy pathway in maintaining motor neuron health.

The release notes that in both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), a protein known as TDP-43 accumulates and clumps together, however researchers say it has been unclear whether the accumulation was toxic and whether clearing it from the cell could be therapeutic.

During the current study, researchers used a robotically controlled microscope in order to track individual motor neurons derived from ALS patients, and they observed the results as TDP-43 accumulated and cleared.

Sami Barmada, MD, PhD, Gladstone Institutes, San Francisco, led the research, the release says, under the direction of associate director and senior investigator Steven Finkbeiner, MD, PhD, who is also a professor of neurology and physiology at the University of California, San Francisco.

According to the release, the researchers found that motor neurons were highly sensitive to the level of accumulated TDP-43 and individuals cells varied in their ability to clear it. The researchers also state that they used novel compounds to increase that clearance by stimulating a cell protein recycling program called autophagy. The results suggest that stimulation of autophagy increased survival of motor neurons, and of astrocytes.

Lucie Bruijn, PhD, MBA, chief scientist for The ALS Association, explains that the study “shows us the importance of the autophagy pathway in maintaining motor neuron health. These findings will allow us to focus more on this pathway as we search for new therapeutic targets.”

The association adds that the research was funded in part by its Golden West Chapter.

The study appears in the journal Nature Chemical Biology.

Source: The ALS Association