Promising New Treatment for ALS: Stabilizing the SOD1 Protein

Amyotrophic lateral sclerosis (ALS), a deadly neurodegenerative disease, affects about 30,000 Americans at any given time. Scientists have been tirelessly working to find effective treatments and, more significantly, a cure. A recent breakthrough suggests that a new treatment, based on a study in mice, has shown promise in stabilizing the SOD1 protein, which is linked to the disease.

Targeting and Stabilizing the SOD1 Enzyme

Published in the journal PLOS Biology, the study focused on targeting and stabilizing the SOD1 enzyme, which protects cells from toxic byproducts associated with consuming food and breathing oxygen. The SOD1 enzyme is involved in many cases of ALS, and dysfunction of this enzyme leads to the disease's progression.

The Promise of the S-XL6 Molecular Stabilizer

A 'molecular stabilizer' called S-XL6 was discovered that successfully restored the SOD1 protein's function and halted its toxic effects in mice, rats, and dogs. S-XL6 stabilized 90% of SOD1 proteins in blood cells and 60-70% in brain cells in mice. An investor has already purchased the rights to a patent for the treatment, indicating the promising future of this novel approach.

Co-treatment Possibility with Biogen's Qalsody

If the treatment proves successful during clinical trials in humans, it could potentially become a co-treatment for Biogen's Qalsody, a breakthrough regimen for ALS that received accelerated FDA approval in 2023. The combined use of Qalsody and S-XL6 could provide a more comprehensive treatment strategy, potentially slowing the disease's progression and improving patients' quality of life.

Other Promising Developments: Desloratadine and IMS-088

Apart from S-XL6, other compounds have also shown promise in combating ALS. Desloratadine, a selective 5HTR2A antagonist, was found to alleviate ALS-like pathology in hSOD1G93A mice. The study showed that DLT administration significantly delayed ALS symptom onset time, prolonged lifespan, and ameliorated movement disorders, gastrocnemius injury, and spinal motor neuronal loss.

Another study found that blocking certain inflammatory signaling pathways using the NF-kappa B inhibitor IMS-088, derived from the naturally occurring Ashwagandha plant, could reverse nerve cell alterations and improve motor and cognitive function. This study underscores the key role of neuroinflammation in ALS disease processes, with possible implications for other related diseases, including frontotemporal dementia (FTD) and other inflammatory neurodegenerative conditions.

Moving Towards Clinical Trials

With these promising results from animal studies, researchers are hopeful to move the S-XL6 molecule to clinical trials in humans soon. The potential of these treatments gives hope to those suffering from ALS and their loved ones. With continued research and innovation, we are moving closer to slowing down, stopping, or even reversing this devastating disease.