Israeli scientists have achieved a world-first milestone in ALS research, unveiling an RNA-based gene therapy that successfully halted — and even reversed — nerve cell deterioration in laboratory models. The breakthrough, led by Tel Aviv University in collaboration with Ben-Gurion University, the Weizmann Institute, and global partners, identifies a microRNA molecule capable of stopping the toxic chain reaction that destroys motor neurons.
Researchers discovered that in ALS patients, muscle cells produce too little of a critical microRNA known as microRNA-126. This molecule normally instructs nerve cells to regulate a protein called TDP-43. When microRNA-126 levels drop, TDP-43 accumulates, forming harmful clusters that attack mitochondria and cause the deadly degeneration of motor neurons.
By supplementing microRNA-126 in ALS tissues and genetically modified mice, nerve cells not only stopped deteriorating — they regenerated. Scientists say this is the clearest evidence yet that protecting communication between muscle and nerve cells is just as important as targeting the neurons themselves.
ALS is a rapidly progressive neurodegenerative disease that leads to full-body paralysis, with most patients surviving only three to five years after diagnosis. Israel has about 600 patients currently living with the illness.
The study’s authors believe this research lays the foundation for the first gene therapy that targets ALS at its root cause. Clinical trials are expected to be the next major step, offering hope for a disease long considered incurable.
