New Research on Lou Gehrig’s Disease

UCR School of Medicine
Maurizio Pellecchia

A team of biomedical scientists have identified a molecule that targets a gene known to play a critical role in the rapid progression of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

The team is led by Maurizio Pellecchia, UCR professor of biomedical sciences in the School of Medicine, the Daniel Hays Endowed Chair in Cancer Research, and the director of the Center for Molecular and Translational Medicine.

In the journal Cell Chemical Biology, Pellecchia reports on the design of 123C4, a molecule the lab developed that targets the EphA4 receptor, a gene successful in delaying the progression of ALS.

“As in any preclinical study, we must acknowledge that several obstacles are still in the way of translating agents like 123C4 into viable therapeutics,” Pellecchia said. “In collaboration with [biotech firm] Iron Horse Therapeutics, we hope these additional studies will further facilitate the translation of these agents into novel treatments.”

The research was funded by grants from the National Institutes of Health as well as a UCR Collaborative Seed Grant.


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