University Researchers Take A Step Forward In Treating Neurological Diseases

Apr 12, 2017

In 2014 the 'Ice Bucket Challenge' was a viral phenomenon to raise awareness and money for A.L.S., or Lou Gehrig’s disease. New research published today from the University of Utah is providing an important step toward treating this class of degenerative brain diseases.

A.L.S. and ataxia are neurodegenerative diseases. People who have them often lose control of motor functions – they may not be able to walk well or their speech could be slurred and they often result in death.

"We don’t really know what causes neurodegenerative diseases. These are diseases where a previously well-functioning nerve cell starts to function abnormally and then eventually dies," says Stefan Pulst. 

Pulst is a professor and the Chair of the neurology department at the University of Utah.

While there are currently no treatments for diseases like ataxia or A.L.S., Pulst helped write two new papers in the journal Nature that offers a first step.

First, they were able to recreate A.L.S. and ataxia in mice. Then, by injecting them with engineered DNA, they were able to very precisely target and attack the disease-causing gene in both conditions.

"The therapy that we developed really destroys the expression of that gene," says Daniel Scoles. 

Scoles is an Associate Professor of Neurology at the University of Utah. He’s an author on one of the new papers.

With this targeted gene therapy the mice in their studies showed improved movement, balance and coordination.

The researchers describe this as proof that they can improve neurodegenerative diseases, at least in rodents. Pulst estimates it could be another five years before this research is expanded to human trials, but he says it has given him a renewed optimism untreatable conditions could someday be cured.