A breakthrough that could serve as the foundation for treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) has been made by Israeli researchers who identified a previously unknown mechanism involved in the development of the neurodegenerative disease.
The study highlights, for the first time, the importance of toxic muscle-secreting molecules in the pathology of ALS and focuses on a specific microRNA whose levels were found to decrease as a result of ALS-causing muscular mutations.
ALS is a lethal, progressive disease that destroys motor nerve cells in the brain and spinal cord, leading to atrophy, paralysis and eventually death due to failure of the respiratory muscles.
“While we are not claiming we have found the cure for ALS, we have certainly moved the field forward,” said Dr. Eran Perlson of Tel Aviv University’s Sackler Faculty of Medicine, who led the study in collaboration with TAU doctoral students Roy Maimon and Ariel Ionescu, and Dr. Oded Behar of Hadassah Medical Center in Jerusalem.
Their findings were recently published in the Journal of Neuroscience.
Recent work has demonstrated that the alteration of microRNAs (miRs) is involved in many neurodegenerative diseases, including ALS. The TAU study identifies a new mechanism related to the causes and effects of ALS, in which the muscle secretes toxic molecules that kill axons and neuromuscular junctions, leading to muscle atrophy. It also found reduced levels of one specific miR — miR-126-5p — in ALS models.
“We demonstrated in lab work and on mouse models that we can successfully ameliorate ALS symptoms using this miR as a potential drug,” Perlson said. “We further demonstrated that muscle tissue — not only motor neurons — is undoubtedly involved in the progression of ALS. This point is particularly important, as it contradicts other theories in the field.”
Further studies are planned.