EVMS research could lead to a treatment for ALS

Story Date: Wed, 10 Feb 2016 08:48:00 CST

When Edward Johnson, PhD, Professor of Microbiology and Molecular Cell Biology at EVMS, identified the Pur-alpha protein more than 20 years ago, he didn’t know that his discovery might eventually lead to a treatment for ALS. Fast-forward to 2016, and his findings have become instrumental in groundbreaking research involving this debilitating disease.

ALS, sometimes called Lou Gehrig’s disease, is a neurological disorder that causes motor nerve cells, or neurons, to degenerate or die, which means they stop sending messages to muscles. Most people with ALS die from respiratory failure in three to five years of the onset of symptoms. 

With the help of Dr. Johnson and his EVMS team, a group of researchers from several schools, including LSU Health New Orleans and the University of Pittsburgh, determined that the Pur-alpha protein could protect against the degeneration of motor neurons in diseases like ALS. 

The researchers found that cell protection is compromised in ALS patients because disease-causing mutations cause Pur-alpha to be mislocalized to the body of the cell, instead of the nucleus where it should be.

Their study determined that providing external Pur-alpha protein suppressed motor neuron degeneration, and they believe it could serve as a novel therapeutic target for developing a treatment for ALS patients. Their findings were published in the journal Acta Neurpathologica in January.

Dr. Johnson worked with Earl Godfrey, PhD, Professor of Pathology and Anatomy, and Dianne Daniel, PhD, Associate Professor of Microbiology and Molecular Cell Biology, to provide the researchers Pur-alpha plasmid clones and antibodies for the study. The EVMS investigators are now testing a synthetic version of the protein designed by Drs. Daniel and Johnson to duplicate the effects of Pur-alpha. 

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