Israeli Scientists Halt ALS Progression with Groundbreaking RNA Gene Therapy
Tel Aviv – In a world first, researchers at Tel aviv University have successfully used an RNA-based gene therapy to halt the progression of Amyotrophic Lateral Sclerosis (ALS) in preclinical trials, offering a potential turning point in the fight against the devastating neurodegenerative disease. The findings,published today in The Lancet Digital Health,demonstrate the therapy’s ability to significantly slow,and in some cases reverse,motor neuron deterioration in animal models.
ALS, also known as Lou Gehrig’s disease, progressively destroys motor neurons, leading to paralysis and ultimately death. Currently, there is no cure for ALS, and existing treatments offer only modest benefits in slowing disease progression. This breakthrough offers a fundamentally new approach, targeting the root genetic cause of the disease rather than managing symptoms. The research team, led by Dr. Eran Perlson, anticipates initiating human clinical trials within the next two years.
The therapy utilizes a novel RNA-based technology to deliver genetic instructions directly to motor neurons, correcting a common genetic defect found in a notable percentage of ALS patients – a mutation in the SOD1 gene. Unlike conventional gene therapies that permanently alter DNA, this RNA-based approach offers a temporary and perhaps safer intervention, reducing the risk of unintended long-term consequences.
“We are essentially ‘re-educating’ the cells,” explained Dr. Perlson. “By delivering the correct RNA instructions, we can temporarily overcome the effects of the mutated gene and restore normal cellular function.”
In the study, mice genetically engineered to exhibit ALS symptoms showed a marked improvement after receiving the RNA therapy. Researchers observed a significant reduction in neuronal damage, improved muscle function, and a prolonged lifespan compared to untreated control groups. The therapy was administered directly into the spinal fluid of the animals.
The team is now focused on scaling up production of the RNA therapy and conducting further safety studies in planning for human trials.They are also exploring the potential of adapting the technology to target other genetic mutations associated with ALS, as well as other neurodegenerative diseases.
“This is a major step forward, but it’s just the beginning,” said Dr. Perlson. “We are hopeful that this therapy will eventually provide a meaningful treatment option for people living with ALS and thier families.”
