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Experimental ALS drug may be more effective than existing ones

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When cortical cultures are mixed in P3, UMNs have eGFP signaling in the UCHL1-eGFP receptor mice, and express proteins that cause pathogenesis in myotrophic lateral sclerosis. (ALS) (ac) Generation of hSOD1G93A-UeGFP and WT-UeGFP littermate controlled mice. (de) UMNs in the Layer 5 of the first cortex motor express eGFP at birth P3 of WT-UeGFP mice. (e) B8H10 antibody detects missing SOD1 protein in low-density hSOD1G93A-UeGFP mice, but does not control WT-UeGFP mice in P3. Scale bars = 200 µm in small magn, 50 am in large magn. Credit: Barış Genç et al, Scientific Report (2022)

New research on NU-9 experimental drugs, developed and developed by two Northwestern University scientists to treat ALS (amyotrophic lateral sclerosis), has been shown to be more effective than FDA-approved drugs for disease.

Most importantly, NU-9 has an effective effect when combined with these drugs, riluzole and edaravone. The drug was developed by Richard B. Silverman, Professor Patrick G. Ryan / Aon in the Northwest, and was conducted by P. Hande Ozdinler, a professor of veterinary medicine at the Northwest Feinberg School of Medicine.

The study, published recently in Scientific Report, showed that NU-9 extends the axon of small molecules in the SOD1 ALS mouse model. This new study on the extension of the axon of patients’ nerves further illustrates the benefits of NU-9.

The axon is the part of the motor neuron that connects the brain to the brain backbone and causes the corticospinal segment, which is depleted in ALS patients. Disruption axon they contribute to the rapid aging of ALS patients.

“For treatment to be effective, it is important for this treatment to improve axon growth and axon health,” said co-author of the study Ozdinler. “This is very important to connect the brain to the spinal cord and regenerate neurons that are depleted in patients.”

In ALS, the nerve endings in the brain (upper extremities) and the nerve endings in the spinal cord (lower extremities) die.

In a study published last year, Northwest scientists showed NU-9 improving two key factors that lead to upper motor neurons to be patient in ALS: protein breakdown and protein accumulation in the cell. All of these factors are toxic to the neuron and are common in patients with ALS and neurodegeneration in general. This study showed that the NU-9 environment inhibited microbial pathogens from damage so that viruses became similar to healthy viruses after 60 days of treatment in two different ALS mice.

NU-9 is moving to a clinical trial

NU-9 is moving in clinical trials. The company, AKAVA Therapeutics, which was launched last year by Silverman, is conducting a veterinary study required for the drug (now called AKV9 in the company) to receive FDA approval to become a New Research Factor. These studies include determining bone level and toxic effects.

“If all goes well, we hope to start with healthy volunteers in the Phase 1 clinical trial as early as 2023,” said study lead author Silverman.

Depending on the FDA’s response to the Phase 1 results, the Phase II trial to provide treatment for ALS patients may begin as early as 2024.

“NU-9 has a new operating system, and needs to be tested in humans for its effectiveness in ALS treatment,” Silverman said.

“It takes a long time – perhaps 10 to 12 years – to identify and bring a new drug to market,” Silverman said. “But this treatment is very exciting and hopeful about its potential to improve the lives of ALS patients, who have no hope for a long time.”

Other North West writers include Barış Genç, Mukesh Gautam, Benjamin Helmold, Nuran Koçak, Aksu Günay and Gashaw Goshu.


Damage to the ALS neuron is reversed with new field


Learn more:
Barış Genç et al, NU-9 promotes the safety of hSOD1G93A mouse and micro-organisms in vitro, especially in combination with riluzole or edaravone, Scientific Report (2022). DOI: 10.1038 / s41598-022-09332-4

hint: ALS treatment test may be more effective than existing ones (2022, May 17) recovered May 17, 2022 from https://medicalxpress.com/news/2022-05-experimental-als-drug-effective .html

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