AANS Neurosurgeon | Volume 27, Number 2, 2018


Potential New Therapeutic Target for ALS

A research team led by researchers from Louisiana State University Health Sciences Center recently discovered a protein called Pur-alpha that is a component of stress granules in cells and may protect against the toxic degeneration of cells in diseases such as amyotrophic lateral sclerosis (ALS). The researchers found that the Pur-alpha protein is essential for the formation of stress granules, confirmed by the observation that the depletion of Pur-alpha impairs the ability of cells to form them. This built-in protection is compromised in ALS patients because disease-causing mutations in the FUS gene protein cause it to become mislocalized to the body of the cell, rather than the nucleus, where it incorporates into the stress granules. The stress granules then stall out, trapping Pur-alpha inside and unable to function. The team also found that supplying external Pur-alpha protein helps in suppressing the motor neuron degeneration associated with FUS-carrying ALS-causing mutations by promoting stalled stress granule turn-over. The study suggests that Pur-alpha could serve as a novel therapeutic target for developing a treatment for ALS patients. “Our findings further suggest that defective RNA-metabolism strongly contributes to ALS pathogenesis and neurodegeneration. Also, I’m excited to see how these discoveries could shed light onto the basic biological pathways linking several familial forms of ALS,” said the study’s lead author. To read more about this study, click here

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