Researchers Show How Lou Gehrig’s Disease Progression Could Be Delayed - NEUROSCIENCE

A team of biomedical scientists has identified a molecule that targets a gene known to play a critical role in the rapid progression of amyotrophic lateral sclerosis (ALS), sometimes known as Lou Gehrig’s disease, the neurodegenerative disease that affects motor neurons — nerve cells in the brain and spinal cord that link the nervous system to the voluntary muscles of the body.

The research, done on a mouse model of ALS, aims at blocking the gene, thus providing an important stepping stone for the development of novel treatments to delay the progression of ALS and, potentially, other human diseases.

Specifically, the team, led by Maurizio Pellecchia, a professor of biomedical sciences in the School of Medicine at the University of California, Riverside, reports in the journal Cell Chemical Biology on the design of 123C4, a molecule the lab developed that targets the EphA4 receptor, a gene in animal models and in humans that is efficacious in delaying the progression of ALS.

Importantly, the expression of EphA4 is associated not only with the progression of motor neuron disease, but also with other conditions including abnormal blood clotting, spinal cord and brain injury, Alzheimer’s disease, as well as gastric and pancreatic cancers.

“Research in assessing the therapeutic value of EphA4 for these diseases has been hampered, however, by the lack of suitable pharmacological EphA4-inhibitors,” said Pellecchia, who holds the Daniel Hays Endowed Chair in Cancer Research and is the director of the Center for Molecular and Translational Medicine. “While the exact mechanism responsible for the therapeutic efficacy of our agent, 123C4, is still to be fully understood, we are confident that 123C4 – or its derivatives – will find wide application in preclinical studies as well as human clinical trials for the treatment of ALS and potentially other human disorders.”

Source and further reading:https://ucrtoday.ucr.edu/44253

Journal article:http://www.cell.com/cell-chemical-biology/fulltext/S2451-9456(17)30025-9
Source: Corina Marinescu