Experimental ALS drug could offer new hope for patients if approved, researchers say

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New hope could be on the horizon for ALS patients in the form of a "breakthrough" drug, researchers say.

Neuvivo, a California biopharmaceutical company that develops therapies for neurodegenerative diseases, is seeking approval for a new medication for ALS (amyotrophic lateral sclerosis).

ALS is a progressive neurodegenerative disease that gradually impairs muscle function, eventually robbing the patient of the ability to walk, talk, eat and even breathe, per the ALS Association.

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This month, Neuvivo submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for NP001 (sodium chlorite infusion).

New hope could be on the horizon for ALS patients in the form of a "breakthrough" drug, researchers say. (iStock)

The drug has been granted orphan drug and fast track designations by the FDA and is eligible for Accelerated Approval and Priority Review, which could shorten the time to a potential approval, according to a press release.

"If approved, NP001 would be the first immunotherapy for ALS, and the first ALS treatment shown to preserve lung function," Matthew W. Davis, MD, chief medical officer of Neuvivo, told Fox News Digital.

An urgent need

There are currently no FDA-approved treatments for ALS that preserve breathing function or extend life by more than two or three months, the company noted.

The leading cause of death in patients with ALS is loss of lung function (respiratory failure).

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Approximately 30,000 adults in the U.S. are currently living with ALS, and one in 300 people will be diagnosed in their lifetime.

The average life expectancy after a patient is diagnosed with ALS is 18 months.

While existing drugs combat ALS through the nervous system, NP001 would be the first therapy that works by controlling inflammation in the immune system. (iStock)

"Half of the people living with ALS today will unfortunately not likely be living in three years," Davis said.

Dr. Earnest Lee Murray, a board-certified neurologist at Jackson-Madison County General Hospital in Jackson, Tennessee, confirmed that current ALS treatments are "very limited" in their effectiveness. 

A different kind of drug

While existing drugs combat ALS through the nervous system, NP001 — if approved — would be the first therapy that works by controlling inflammation in the immune system.

"NP001 would be indicated for all patients with ALS, although our data supports that patients with inflammation are likely to have significantly greater benefit," Davis said.

"Half of the people living with ALS today will unfortunately not likely be living in three years."

Murray, who is not affiliated with Neuvivo, noted that excessive inflammation has been a known factor in the progression of muscle weakness in ALS. 

While initial studies of NP001 did not show any "statistically significant" improvement, the neurologist said, "reanalysis of the previous data looked at certain subgroups of patients with particularly high inflammatory markers."

ALS is a progressive neurodegenerative disease that gradually impairs muscle function, eventually robbing the patient of the ability to walk, talk, eat and even breathe, per the ALS Association. (iStock)

"This data suggests that NP001 can slow progressive loss of muscle function, especially when it comes to a patient’s ability to breathe, by targeting a specific immune system cell called a macrophage," Murray told Fox News Digital.

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"Halting the activation of macrophages oftentimes can have an effect on other immune cells, which will hopefully alter the progression of ALS."

During the first month of treatment, NP001 is given as a 30-minute infusion each day for five consecutive days. (iStock)

During the first month of treatment, NP001 is given as a 30-minute infusion each day for five consecutive days, according to Davis.

Every month thereafter, the medication is given for three consecutive days in the same way.

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NP001 was "generally well-tolerated" in clinical trials, Davis said.

The most commonly reported side effect was pain, redness and swelling where the drug was infused, which were "generally transitory and mild."

"This data suggests that NP001 can slow progressive loss of muscle function."

Murray echoed that early studies have demonstrated that NP001 is safe and well-tolerated. 

"I am cautiously optimistic that as NP001 moves though the FDA process of clinical trials, it will show true improvement in ALS patients and offer some hope to patients and families dealing with this devastating disease," he said.

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As the drug undergoes FDA review, Neuvivo will continue to post updates for patients and caregivers on its website, according to Davis.