Smaller Biotechs May Pave Way For New Generation Of Therapeutics For Hard-to-Treat Neurological Disorders

Smaller Biotechs May Pave Way For New Generation Of Therapeutics For Hard-to-Treat Neurological Disorders

An estimated 200 million Americans — more than 60% of the population — suffer from at least one neurological disorder. That includes dementia, strokes, migraines and other diseases or infections that affect the nervous system.

However, some smaller biotechs such as Pasithea Therapeutics Corp. KTTA, reports a robust pipeline of preclinical drug candidates for a range of hard-to-treat psychiatric and neurological disorders. 

The Central Nervous System Plays A Central Role In So Many Debilitating Disorders

The central nervous (CNS) — the brain and spinal cord — and the peripheral nervous system (PNS) — all the nerves that branch off from the CNS into the rest of the body — work together to monitor and maintain the state of the entire body by detecting stimuli and controlling voluntary and involuntary muscle movements.

In short, it’s because of the nervous system that anyone can think, feel, learn, remember and speak. The nervous system also enables the body to perform basic motor functions like walking and breathing. Behind the scenes, that system is keeping the heart beating, digestive system working, hormones in balance and even making sure pupils dilate and contract in response to light levels. 

Damage to the CNS is often systemic and widespread, resulting in loss of mobility, motor control and other functions depending on the specific kind of damage.

Multiple sclerosis (MS), dementia, Parkinson’s disease and amyotrophic lateral sclerosis (ALS) are all examples of disorders linked to CNS damage. The progressive diseases impair cognitive and motor functions that, when left untreated, eventually render the person incapable of functioning without 24/7 care and expensive medical interventions.

These neurological disorders — disorders located in the nervous system — affect hundreds of millions of people around the world and cause roughly 90 million deaths worldwide each year, making them the second-leading cause of death after heart disease.

But along with fatalities, neurological disorders rob sufferers of their ability to lead independent, fulfilling lives. According to the Pan American Health Organization (PAHO), neurological disorders account for 8.2 million years lived with disability across the Americas — a metric used to measure the quality of life and disability-free life expectancy. 

The Rise And Fall And Rise Of CNS Drug Development

As the importance of the CNS in so many diseases became clear, many pharmaceuticals in the early 2000s began investing heavily in drug development research targeting the CNS. But challenges in designing treatments that can successfully pass through the blood-brain barrier caused many to lose motivation.

The slow progress in overcoming this challenge led major companies like Novartis International AG NVS, Pfizer Inc. PFE, Merck & Co. Inc. MRK and AstraZeneca plc AZN to cut back on funding or completely close research programs into neurological diseases in the early 2010s. 

In the past couple of years, however, a resurgence in development efforts spearheaded by smaller pharmaceuticals and biotechs has seemingly brought new hope to the space. As smaller companies make headway, big pharma is once again taking an interest in CNS drug research.

Pasithea Targets Neuroinflammation To Treat CNS Disorders 

One of the reportedly most promising avenues of CNS disorder research is in the role of neuroinflammation, or the inflammation in and around brain cells. While the data doesn’t yet prove that neuroinflammation is a cause of CNS disorders, a growing body of evidence suggests that it is a key contributing factor to how debilitating a disease is and how rapidly it progresses. 

That contributing role makes sense when you consider that inflammation can cause cellular damage and death. With chronic inflammation, neurons can end up dying or becoming damaged at a faster rate than the body can repair and reproduce them.

As neurons are impaired, that all-important communication network gets weaker, and the body’s ability to modulate cognitive and motor functions decrease. 

Because of this connection, biotechs like Pasithea Therapeutics say they are exploring drugs that can stop or slow that neuroinflammation, in hopes of stopping or slowing the progression of these debilitating CNS disorders. 

The biotech’s pipeline includes treatments for MS and ALS as well as psychiatric disorders like schizophrenia and bipolar disorder.

In October, Pasithea partnered with Evotec SE EVO to initiate a new chemical entity (NCE) development program that would focus on small-molecule therapeutics that target neuroinflammatory pathways that have been linked to schizophrenia and depression.

It launched another NCE program in February to develop a tolerizing vaccine for MS. 

This month, Pasithea received a grant to help fund its research into a monoclonal antibody (mAb) treatment for ALS. The mAb aims to slow the progression of the disease by blocking inflammatory signals. 

In the past, some companies that have developed treatments for unmet needs have gone on to generate significant revenue and returns to shareholders — Pasithea seems to be looking to take a similar approach with a reported dual focus on creating life-changing therapeutics, and long standing value for itself and its constituents.

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Featured photo by Jesse Orrico on Unsplash

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