New Drugs And Therapies Are Giving MS And ALS Sufferers New Hope

People suffering from incurable neurological diseases such as multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS) hope new therapies and drugs will arrive sooner than later. 
 

A chronic and inflammatory neurodegenerative disease, MS affects 400,000 people in the United States and 2.1 million people worldwide, according to the latest data from the National Institutes of Health (NIH). Affecting quality of life, employment, social relationships, and productivity, MS costs the U.S. healthcare system anywhere from $8,528 to $52,244 per patient per year. 
 

MS is a potentially disabling disease affecting the brain and spinal cord — the body’s central nervous system. The immune system of those with MS attacks the myelin, the protective sheath covering a person’s nerve fibers. It interrupts communication between the brain and the functions of the body. The end result for many people is permanent damage or deterioration of the nerves.
 

Signs and symptoms of MS vary widely and depend on the amount of nerve damage and which nerves are affected. Some people with severe MS may lose the ability to walk independently, while others may experience long periods of remission without new symptoms.

Recent ALS Research Spurs Optimism  

Although there is no recent data revealing the number of people in the U.S. affected by ALS, an estimated 31,000 people have the condition and an average of 5,000 new patients are diagnosed each year, according to 2017 statistics from the Centers for Disease Control and Prevention.

Age-related ALS mainly strikes people between 55 and 75 who live only two to five years after symptoms develop.  

Also called Lou Gehrig’s disease for the New York Yankee great who died of ALS in 1941, the disease belongs to a broader group of disorders known as motor neuron diseases which cause the gradual deterioration and death of motor neurons. 

Motor neurons are nerve cells extending from the brain to the spinal cord and muscles throughout the body. As motor neurons degenerate, they stop sending messages to the muscles, which gradually grow weaker, twitch and suffer from atrophy. Eventually, the brain loses its ability to initiate and control most voluntary movements such as walking, chewing, and talking. 

Merck & Co. Inc. MRK recently announced it’s paying $25 million to kick off a research partnership with biotech startup Cerevance, which analyzes donor brain tissue to find novel targets for new drugs for neurological disorders.

Cerevance’s best-known program is geared toward another incurable neurological disease — Parkinson’s — but Merck will determine whether the technology can help it discover and develop new drugs to treat Alzheimer’s disease.

Recent news in the MS research space touted Clene Inc.’s CLNN results from its Phase 2 VISIONARY-MS trial of CNM-Au8, an investigational gold nanocrystal suspension. The trial began before COVID-19 and ended prematurely, limiting enrollment to 73 of the 150 planned participants.

Pasithea Advances Trials For MS And ALS  

Pasithea Therapeutics Corp. KTTA, a biotechnology company focused on researching and discovering new and effective treatments for psychiatric and neurological disorders, announced its investigational MS vaccine PAS002. 

Trials of the vaccine have effectively delayed disease onset and reduced disease severity in a mouse model of the neurodegenerative condition, according to its preclinical study data. The vaccine, designed to promote immune tolerance to a specific myelin protein, also reduced the severity of relapses and helped the animals gain weight, indicating a better overall health status.

“Although early-stage data, we’re thrilled with the results of this study and the strong preclinical efficacy data of our tolerizing approach,” Pasithea CEO Dr. Tiago Reis said. 

Pasithea Therapeutics has also recently received a $700,000 grant to further its research into anti-integrin antibodies as potential treatments for ALS. 

The company is developing a monoclonal antibody (mAbs) for treating ALS and other neuroinflammatory disorders.

A particular integrin, called alpha-5/beta-1 or fibronectin receptor, has been found at high levels in certain immune cells in an animal model of ALS carrying mutations in the SOD1 gene. Mutations in this gene are estimated to account for up to 20% of familial ALS cases and up to 3% of sporadic ALS cases. 

The Alpha-5/beta 1 study found that integrin expression is significantly increased in ALS animal models and post-mortem human ALS and significantly upregulated with disease progression.

Pasithea has multiple monoclonal antibody candidates against the alpha-5/beta-1 integrin. Treatment with these antibodies has extended survival and improved motor function in the SOD1 mice model of ALS, suggesting a novel mechanism to treat patients with ALS.

For more information on Pasithea Therapeutics, visit www.pasithea.com. 

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