Pasithea's Latest Drug Candidate Could Bring New Hope To ALS Patients Without Effective Treatment Options

Amyotrophic lateral sclerosis (ALS) is a rare but painful and fatal disease that slowly shuts down all voluntary movements in the body, from walking to breathing. Roughly 5,000 new cases are diagnosed each year in the United States — and once diagnosed, patients have a life expectancy of just three to five years.

With so many questions still unanswered about the causes of the disease, drug developers have struggled to create treatments that offer much relief for these patients. Pasithea Therapeutics Corp. KTTA, a developmental-stage biotech, says it is hoping to bring new hope to those suffering from ALS with the recent addition of a novel drug candidate to its pipeline.

What Is Amyotrophic Lateral Sclerosis?

ALS is a progressive neurodegenerative disease that causes nerve cells throughout the body to stop working and die. Without those nerve cells, the body loses the ability to trigger voluntary muscle movements, starting with mild weakness or stiffness and eventually leading to paralysis.

For the roughly 25,000 Americans suffering from the disease, that means they will progressively lose the ability to speak, eat, move and even breathe without the assistance of machines.

What makes the debilitating disease even more troubling is that it’s so hard to predict who will get it. While being the offspring of a parent with ALS puts you at high risk of developing it yourself, about 90% of cases have no known family history of the disease. So, it’s not strictly genetic.

Research into environmental risk factors — like exposure to toxins or poor diet and lifestyle — has also yielded few answers. While some data suggests military veterans are twice as likely to be diagnosed than the general public — and men slightly more likely than women — it’s still not known why or what factors may be at play.

One clue researchers have is the age of onset. Symptoms typically appear between the ages of 55 and 75, which suggests that it might be an age-related disease.

The Limited Treatment Options for ALS Patients

With so little understanding of the causes, developing treatments for the disease has been reportedly challenging. Two drugs that have been approved by the Food and Drug Administration (FDA) for ALS so far are Rilutek, sold by Sanofi SA SNY and Radicava, manufactured by Mitsubishi Tanabe Pharma America Inc.

The most effective is Rilutek, known as Riluzole in generic form, which works by blocking the release of glutamic acid into the central nervous system. Because toxic levels of glutamate may be linked to ALS, blocking glutamic acid is thought to keep those levels closer to normal. But even with it being the most effective drug so far, it can only prolong tracheostomy-free survival — living without a surgically implanted breathing tube — by about two to three months on average.

The mechanism of action for Radicava, or Edaravone, is less clear, but it may be providing antioxidants that protect against damage and death in all cells, including the nerve cells that are dying at rapid rates in ALS patients. While some data shows that the antioxidant may improve symptoms over the short term, it doesn’t prolong survival at all.

Pasithea Reports Targeting Inflammation in Hopes of Slowing ALS Disease Progression

Pasithea says it is hoping to change that dismal treatment landscape with its latest drug candidate. Recently acquired as part of the acquisition of Alpha-5 integrin LLC,  the company is developing a monoclonal antibody for the treatment of ALS and other neuroinflammatory disorders by blocking the Alpha-5/Beta-1 integrin.

Integrins are a kind of receptor on the surface of the cell. They can bind to other structures, trigger cell migration and activate processes like cell growth and reproduction. The Alpha-5/Beta-1 integrin, or fibronectin receptor, that Pasithea  is targeting is specifically responsible for regulating several neuroinflammatory cells.

By blocking the expression of this particular integrin, this novel drug aims to reduce neuroinflammation, which would then hopefully slow the rate of damage and death of motor neurons. 

Inflammation has become a focus of some ALS research because the immune response can cause serious damage if prolonged or over-activated. Its main role in the body is to target and destroy infected cells and harmful foreign substances — but its lethal powers can leave no shortage of casualties in its wake. 

The damage from chronic or frequent inflammation builds up over time, which has made it the focus of research on many age-related or progressive diseases like Alzheimer’s disease and multiple sclerosis. 

Because ALS may be an age-related disease and because its symptoms are linked to the progressive death of nerve cells, targeting inflammation to reduce cell damage and death may help slow the progression of the debilitating disease.

With the new acquisition, Pasithea is planning to move this drug into ALS clinical trials as soon as possible. 

“Our plan is to file an Alpha-5 investigational new drug application (IND) with an orphan drug designation by the end of 2023,” Dr. Tiago Reis Marques, CEO of Pasithea, said in a statement on the acquisition.

This post contains sponsored advertising content. This content is for informational purposes only and is not intended to be investing advice.

Featured photo by Jon Tyson on Unsplash

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