A Monoclonal Antibody Treatment For ALS Could Begin Human Trials By 2023, According To Pasithea

Pasithea Therapeutics Corp. KTTA, a biotech company looking for new treatments for psychiatric and neurological disorders, received an AU$1 million (US$694,000) grant to help fund research into its new drug candidate for amyotrophic lateral sclerosis (ALS).

Australia’s largest independent funder of ALS research FightMND awarded the grant to Pasithea after it added a novel monoclonal antibody (mAb) treatment for the disease to its pipeline as part of its acquisition of Alpha-5 integrin LLC in June.

Here’s why the company is exploring the potential of mAbs — and alpha-5/beta-1 integrin blockers in particular — as a potential treatment for ALS.

Have Researchers Only Scratched The Surface Of Monoclonal Antibody Treatment Potential?

The human body creates antibodies on its own to fight infection. These are proteins in the blood that are each uniquely designed to detect a particular antigen — a kind of “fingerprint” molecule found on pathogens that gives away their identity as foreign invaders.

The catch is that the immune system can’t make an antibody for an antigen it’s never seen. That’s where mAbs come in. First developed in 1975, these lab-made antibodies can be engineered to target the specific disease doctors are trying to treat. Once treated, a patient who’s never had something like COVID before will already have those COVID antibodies ready to go.

Unlike vaccines that mimic the antigens of a particular pathogen to trigger an immune response so the body will make its own antibodies, mAbs deliver those antibodies ready-made via a direct infusion. Moreover, because they’re engineered in a lab, developers can add extra weapons and features to make them even more powerful.

Rituximab, for example, was developed by Biogen Inc. BIIB in the 1990s to treat cancer by targeting the CD20 antigen and then delivering cytotoxins that kill the cancer cells as the drug finds them.

The Latest Research on ALS Points To Antibody-Based Treatments As A Possible Breakthrough

More recently, mAbs have been gaining relevance as a treatment for neurodegenerative diseases, including ALS. ALS is a devastating disease that typically kills patients within two to five years after diagnosis. 

As motor neurons throughout the brain begin dying at a rapid pace, the brain loses the ability to trigger muscle movements. Eventually, all movements from walking to breathing become impossible. 

Little is understood about how or why the disease develops or progresses as quickly as it does, which has made finding effective treatments extremely difficult. The reportedly most effective drug on the market is Riluzole, sold under the brand name Rilutek by Sanofi SA SNY. But even with this treatment, patients can only expect to add another two or three months to their prognosis. 

While the causes are not fully understood, researchers are gaining insights into the actual processes triggering that destruction, including oxidative stress, neuroinflammation and protein misfolding. 

That understanding is what’s spurred the recent interest in antibody-based therapies for ALS. The antibody-based therapies currently being investigated for ALS vary in specific targets and mechanisms, but the goal is to stop one or more of those cell destruction processes. 

Pasithea’s Alpha-5 Integrin Treatment Could Soon Reach Human Trials

One of the latest drugs to emerge in this space is Pasithea’s recently acquired drug candidate PAT103, a mAb targeting the alpha-5/beta-1 integrin. The alpha-5/beta-1 integrin is a protein on the surface of cells that plays a key role in the brain inflammatory cascade(among other tasks). 

Researchers have found high concentrations of alpha 5 integrins in areas in the brain where motor neurons are located, both in animal models and human brain samples.  Moreover, levels continue to increase as the disease progresses.

By blocking this integrin, PAT103 aims to lower their activity and reduce the inflammatory response in the brain.

Preclinical research of the anti-alpha-5 integrin treatment has already yielded promising results. In animal models of ALS, mice treated with the drug candidate survived longer than controls and experienced improvement in motor function. These studies have been conducted both at Stanford University and in an independent laboratory.  Reproducing results is an important step in the scientific process. 

Based on these data, Pasithea says it plans to run toxicology studies and submit an investigational new drug (IND) application next year, aiming to begin human clinical trials by the end of 2023. 

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