New Compound Discovered Bringing Scientists One Step Closer to Finding a Cure for ALS

Introduction to ALS

As many have become aware of following the 2014 ALS Ice Bucket Challenge, Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s Disease is a nervous system disease which there remains no cure and results in a slow and disheartening death. While the ALS Ice Bucket Challenge took social media by storm raising awareness and $115 billion in funds dedicated to ALS research, the latest breakthrough in the search for a cure has been brought about by Northwestern Scientists who have discovered a compound that can repair degenerating brain cells that cause much of the damage in the paralyzing disease. 

What is ALS?

Amyotrophic Lateral Sclerosis is a neurological disease in which nerve cells in the brain that control movement (upper motor neurons) as well as nerve cells in the spine which also control muscle movement (lower motor neurons) die. This occurs when proteins misfold and clump inside the cell, and since proteins that misfold become toxic to the neuron, they can no longer function properly. The effects of this protein degeneration are simple; paralysis, and eventually patients no longer have the muscle capacity to breathe, nor keep their hearts beating which results in death. When a patient is diagnosed with ASL one of the first effects is the degeneration of upper motor neurons which are responsible for the initiation of muscle movement. When these neurons degenerate the brain can no longer communicate with the spinal cord, thus causing paralysis in patients. The breakthrough that has been discovered by Northwestern scientists directly combats these degenerating upper motor neurons, which with further research can be expanded throughout the lower motor neurons as well.

The Compound: NU-9

The study conducted by Hande Ozdinler, with the assistance of Richard B. Silverman researching, as well as Baris Genc, Oge Gozutok, and Mukesh Gautam was released on February 23rd, 2021. With all the research and experiments first conducted on rats, the conclusion has been clear, the compound NU-9 which has abilities to reduce protein misfolding in criteria cell lines addresses the two main factors which cause muscle degeneration in ALS patients; protein misfolding and protein clumping. After administering NU-9 both the mitochondria and the endoplasmic reticulum regenerated so much, to the point where these degenerated neurons were similar to healthy neurons after only 60 days of treatment. With these upper-neurons being regenerated the brain and the spinal cord can maintain a connection which could essentially prevent paralysis for an expanded amount of time. 

The Study

While the discovery of NU-9 is not a permanent solution to the cure of Amyotrophic Lateral Sclerosis, it certainly is a breakthrough in the medical field which not only provides hope for those diagnosed with ALS but all patients with neurological diseases or paralyzing agents. The Northwestern scientists as part of the Chemistry of Life Process Institute have created history, and those who have funded the research including R01 AG061708 from the National Institute on Aging of the National Institutes of Health, NUCATS, Northwestern University, Les Turner ALS Foundation, A Long Swim and the ALSA TREAT ALS Award, are an accessory to a life-changing trial which maintains the capacity to save thousands of lives. 

Works Cited

Association, ALS. “ALS Ice Bucket Challenge Commitments.” The ALS Association, 2021, www.als.org/ice-bucket-challenge-spending.

Association, ALS. “What Is ALS?” The ALS Association, 2021, www.als.org/understanding-als/what-is-als.

Paul, Marla | By Marla Paul. ALS Neuron Damage Reversed with New Compound, Northwestern Now, 23 Feb. 2021, news.northwestern.edu/stories/2021/02/als-neuron-damage-reversed-with-new-compound/.