Amyotrophic Lateral Sclerosis Medicine: Study Suggests to Focus on the Brain Not Just Spinal Cord for ALS Therapy

Northwestern Medicine scientists have discovered that the brain is a target for treating amyotrophic lateral sclerosis (ALS). It changes a long-standing belief that it starts with the degradation of spinal motor neurons and that the spinal cord should be the key focus.

In the study titled, "Upper Motor Neurons Are a Target for Gene Therapy and UCHL1 Is Necessary and Sufficient to Improve Cellular Integrity of Diseased Upper Motor Neurons," published in the journal Gene Therapy, scientists wrote that the degeneration of brain motor neurons is not just a byproduct of spinal motor neuron degeneration other than what was previously thought.

Stephen Hawking Gives Origin Of The Universe Lecture In Jerusalem — British scientist Prof. Stephen Hawking gave his "The Origin of the Universe" lecture to a packed hall on December 14, 2006, at the Hebrew University of Jerusalem, Israel. Hawking has ALS (Amyotrophic Lateral Sclerosis or Lou Gehrig's disease), which has rendered him quadriplegic and is able to speak only via a computerized voice synthesizer which is operated by batting his eyelids. (Photo by David Silverman/Getty Images) Getty Images

Amyotrophic Lateral Sclerosis (ALS)

According to Mayo Clinic, ALS is a progressive nervous system disease that affects the central nervous system and motor neurons, which causes loss of muscle control. It is also known as Lou Gehrig's disease from the baseball player diagnosed with it. Doctors do not always answer what caused it, although some cases are hereditary.

Signs and symptoms of ALS could vary from one person to another, depending on which neurons are affected. But usually, it includes difficulty in walking or in doing everyday tasks, tripping and falling, weakness on feet, legs, and ankles, hand weakness, clumsiness, muscle cramps and twitching of the arms, inappropriate crying, laughing or yawning, and changes in cognition and behavior.

ALS often begins with the arms, feet, or limbs and spread to other parts of the body as the disease progresses. The more neurons are destroyed, the weaker the muscles get, which eventually affect chewing, swallowing, speaking, and breathing. On the other hand, ALS does not typically affect bladder control or the senses and there is no pain felt in the early and late stages of the disease.

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Brain Should Be the Primary Target of ALS Treatment

ALS, hereditary spastic paraplegia, primary lateral scoliosis, and other upper motor neuron disease affect over 250,000 people in the US alone, Science Daily reported. Unfortunately, there is no cure or long-term treatment option yet for people suffering from these kinds of diseases.

But this study may open the doors for developing treatments as it is the first to reveal that the brain motor neuron degenerations are not a consequence of the decay of spinal motor neurons.

Furthermore, the study is the first to show that the UCHL1 gene plays a significant role in maintaining the health of brain motor neurons. A diseased UCHL1 gene is caused by the accretion of badly folded proteins and the assemblage of sticky protein clumps in the cells. More than 90% of ALS and other upper motor neuron diseases patients have these problems.

Researchers noted that the study reveals the first target gene that can revitalize the neurons, a huge medical breakthrough. News Medical Life Sciences reported that scientists have also previously identified an NU-9 compound that eliminates the ongoing degeneration of upper motor neurons.

The team plans to determine the best dose and site of injection to improve movement and reduce disease conditions in at least two ALS disease models. Then, they plan to translate these results into clinical trials that will likely take several years.