Researchers have made a discovery that may serve as a basis for treatment of the amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease.
Reduced levels of the microRNA miR-126-5 were linked to toxicity in patients with Lou Gehrig’s disease. Manipulating the molecule can improve ALS symptoms and slow down the neuron degeneration process.
ALS
The neurodegenerative disease attacks the cells in the brain and spinal cord that keep the muscles moving, which leads to muscle atrophy and permanent disability in patients.
There are a little over 6,000 people in the United States diagnosed with ALS each year. Sixty percent of people with ALS are men. The average age people get diagnosed of ALS is 55.
No cure is currently available for the condition and those diagnosed with ALS typically have between two and five years of life expectancy after diagnosis.
“Half of all people affected with ALS live at least three or more years after diagnosis. Twenty percent live five years or more; up to ten percent will live more than ten years,” the ALS Association said.
Researchers from Tel Aviv University in Israel however, have made a step closer to finding a cure for the condition with the discovery of a new ALS mechanism.
Toxicity And MicroRNA miR-126-5p In ALS Patients
In the study published in the Journal of Neuroscience, Eran Perlson of the Department of Physiology and Pharmacology at TAU, and colleagues found that in people with ALS, the muscles secrete toxins that damage the motor neurons.
They also proposed an approach to delay the disease through a molecule that paralyzes the genes responsible for the secretion of the toxin.
The researchers found that toxicity level tends to be higher in people with ALS and the higher toxicity is linked to reduced levels of the microRNA miR-126-5p. MicroRNAs are small molecules that play a role in regulating the translation of protein and have important role in other cellular processes.
Experiments conducted on mice showed that the symptoms of the disease improved when miR-126-5p is manipulated.
By modeling human environment using silicon chips, Perlson and colleagues also discovered that genetic manipulation of this particular microRNA can significantly slow down the neuron degeneration process.
“Overexpressing miR126-5p is sufficient to transiently rescue axon degeneration and neuromuscular junctions (NMJs) disruption both in vitro and in vivo,” the researchers wrote.
“We demonstrate a novel mechanism underlying ALS pathology, in which alterations in miR126-5p facilitate a non-cell-autonomous mechanism of motor neuron degeneration in ALS.”
Hope For A Cure For ALS
The researchers said that the microRNA may one day be used to treat ALS.
“While we are not claiming we have found the cure for ALS, we have certainly moved the field forward,” Perlson said. “The findings may be the basis of a future drug.”
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