Amyotrophic lateral sclerosis, also known as ALS or Lou Gherig’s Disease after the famous baseball player who was forced to retire after experiencing the disease, is a fatal neurodegenerative disease. The medical term for ALS accurately describes the effects the disease has on patients. Amyotrophic refers to three root terms: a meaning “no,” myo meaning “muscle,” and trophic meaning nourishment, all of which combine to indicate that the condition leads to no nourishment of muscle tissue and atrophy, or wasting away, of the affected tissue. Lateral indicates the upper and lower areas of the spinal cord that lead to muscle atrophy, and sclerosis refers to the hardening and scarring of the affected regions.

In ALS, motor neurons that provide an essential connection between the brain and spinal cord and the rest of the body degenerate or deteriorate rapidly. When the motor neurons eventually die, the brain can no longer cause or control muscle movement, leading to their eventual atrophy. Soon after, individuals can experience partial or total paralysis of voluntary muscles, leading to an inability to control muscle movement, speak, eat, and even breathe. The average life expectancy after receiving an ALS diagnosis is between three and five years.

Lack of Available Treatments

Currently, there is no cure for ALS. While familial, or inherited, ALS accounts for some cases of the disease, nearly 95% of cases occur sporadically, without any known genetic precursor. 

In an effort to find a potential preventive treatment for ALS and other neurodegenerative diseases, researchers at Boston University’s School of Medicine have turned to study biological models that simulate neurodegenerative conditions. As a result, scientists have identified a certain protein that appears to serve a unique, beneficial purpose in protecting the brain from the mechanisms of ALS.

Klotho Protein

In Boston University’s School of Medicine laboratory model, researchers identified an anti-aging protein called klotho protein that showed neuroprotective effects. In fact, increasing klotho protein levels reduced neurological deficits in experimental models of both Alzheimer’s Disease and multiple sclerosis (MS). Researchers then posited that klotho protein increases may aid in protecting the brain against other neurodegenerative diseases, as well.

In a subsequent study on a laboratory model, klotho protein provided similar neuroprotective effects against ALS. Klotho protein was shown to reduce ALS-associated neurological deficits, thus providing a potential decrease in the manifestation of symptoms. In conjunction with the presence of anti-inflammatory brain cells called microglia, klotho protein shows potential to protect the brain against inflammation, degeneration, and motor neuron loss.

Future Implications

Klotho protein therapy, along with other activities that increase klotho levels, have been shown to potentially prolong the life of an ALS patient by as much as 300 days. Further, increasing klotho levels appears to improve quality of life by reducing ALS symptoms in patients who have already discovered the disease.

While the klotho protein has long shown benefits for other neurodegenerative diseases, this new information provides hope and potential therapeutic applications for the thousands of patients currently suffering from ALS.

Resources:

https://www.eurekalert.org/pub_releases/2019-06/buso-pop062719.php
https://link.springer.com/article/10.1007/s12031-019-01356-2
https://www.als.org/understanding-als/what-is-als