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Pennsylvania researchers reduce toxicity levels in ALS

Category: Musculoskeletal Diseases

Better known by its common name, Lou Gehrig's disease, amyotrophic lateral sclerosis is a fatal disease that destroys muscle strength and eventually leads to full-body paralysis. A new study conducted in animal models by researchers at the University of Pennsylvania has found a way to reduce the toxicity of ALS that slows deteriorated neurons and can possibly be attributed to cells in mammals.

Published in the journal Nature Genetics, the study's research team has possibly uncovered a new strategy for ALS treatment by working with a fruit fly model of the disease. Led by senior author Nancy Bonini, Ph.D., the scientists have made progress in understanding the way that ALS moves and attacks the nerve cells. In a lab test, the flies were genetically altered to express the human version of a gene that binds to RNA and has been found to be abnormal in patients diagnosed with ALS. In recent years, there has been a large increase in the understanding of ALS at the genetic level, which is why the team was examining this specific gene.

"There's been an explosion over the last five-plus years in the identification of genes that contribute to genetically inherited ALS," explained Bonini.

The flies that expressed symptoms that could be correlated to ALS in humans were injected with a compound that was meant to restore physical mobility and increase muscle strength, reversing the effects of degeneration. After injection, the scientists found that the flies were able to fly higher and climb faster, giving hope that the same progress could eventually arise in human models.

The results show advancement in the treatment strategy for ALS going forward, as well as the benefits of using simple animal models to shed light on human neurological diseases.

Important facts on ALS
Often called Lou Gehrig's disease, ALS affects nerve cells in the spinal cord and brain. As the motor neurons between the brain and spinal cord begin to deteriorate and die, a patient's ability to control muscle movement is lost. As a result, patients are almost fully paralyzed by the later stages of the disease.

Early symptoms of ALS typically include muscle weakness, specifically in the arms and legs, as well as in speech, swallowing or breathing. Without motor function, muscles begin to waste away and become thin over time.

Currently, there is no known cause behind ALS, but recent scientific research has brought doctors closer to understanding the disease. The U.S. Food and Drug Administration approved the usage of riluzole, which works to impede the progression of ALS, however, it only extends the average life span by a mere three months.

Hopefully, the lab tests conducted by the University of Pennsylvania team will help lead to the development of a new drug that can bring about a better quality of life for patients with ALS.