Industry, Academic Labs Detailing ALS Treatment Actions

(Kevin Phillips, Pixabay)

27 Mar. 2023. Researchers from a developer of neurodegenerative disease therapies and university medical center are investigating the precise workings of a promising ALS treatment. Early findings from the collaboration between NeuroSense Therapeutics Ltd. and Massachusetts General Hospital are scheduled for presentation this week at the 2023 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders in Gothenburg, Sweden.

NeuroSense Therapeutics in Cambridge, Mass. discovers and develops treatments for degenerative diseases of the nervous system including amyotrophic lateral sclerosis or ALS, Alzheimer’s disease, and Parkinson’s disease. The lead product of the six year-old biotechnology enterprise is called PrimeC, designed to slow progression of ALS in patients. ALS, also known as Lou Gehrig’s disease, occurs when motor neurons in the brain and spinal cord affecting voluntary movements like speaking and walking begin wasting away, with the disease getting progressively worse over time. People with ALS generally lose their ability to stand, walk, chew, and swallow, thus requiring constant attention and support. Most people with ALS die from respiratory failure.

ALS currently has no cures, with only a few treatments to slow progression of the disease. NeuroSense says PrimeC is a new, unique formulation of two currently approved drugs Ciprofloxacin and Celecoxib, for limiting ALS progression. The company says PrimeC works by blocking cyclooxygenase or COX proteins associated with inflammation and pain. On their own Ciprofloxacin is an antibiotic for bacterial infections, while Celecoxib is a nonsteroidal anti-inflammatory medication for pain and swelling from three forms of arthritis. Science & Enterprise in Sept. 2022 reported on an early clinical trial of PrimeC with 16 ALS patients in Israel, showing recipients with chemical changes indicating the desired chemical actions taking place, and the drug not causing severe adverse effects.

Test on post-mortem brain tissue samples

In the new collaboration, the NeuroEpigenetics Lab at Mass. General is examining details of PrimeC’s mechanisms of action, to better understand its effects on patients. The NeuroEpigenetics Lab, led by neuroscientist Ghazaleh Sadri-Vakili, studies biomarker changes in cellular pathways affecting the nervous system that result in neurodegenerative diseases, using human cell and tissue samples and lab animals as models. Mass. General is a research and teaching hospital in Boston, affiliated with Harvard Medical School.

NeuroSense says its researchers are working with Sadri-Vakili and colleagues to evaluate PrimeC on post-mortem brain tissue samples from ALS patients, used by the lab in studies of neuroinflammation and oxidative stress. In the new study, the team is looking particularly for accumulations of TAR DNA-binding protein 43 or TDP-43 associated with neurodegenerative diseases, autophagy or cellular self-destruction defects, dysfunctions in mitochondria or cells’ energy centers, and oxidative stress.

“Our laboratory prides itself on partnering with industry collaborators,” says  Sadri-Vakili  in a NeuroSense statement, “to assess the neuroprotective efficacy of new treatments with the hopes of identifying novel therapies for ALS. Our in vitro model may serve as an effective tool to increase our understanding of PrimeC’s neurotherapeutic mechanism of action.”

NeuroSense is moving ahead with a mid-stage clinical trial, enrolling 69 participants with ALS to test PrimeC against a placebo, with the placebo group then receiving PrimeC after the test period. The company plans to correlate findings from the Mass. General study with the new trial results.

More from Science & Enterprise:

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• AI Analytics Find New ALS Genetic Drug Targets
• FDA Issues Action Plan for Rare Neuro Diseases
• ALS Therapy Start-Up Gains $5.1M in Seed Funds
• Preclinical Test Advances Synthetic Bio ALS Treatment

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