Scientists report that targeting autoimmune inflammation associated with amyotrophic lateral sclerosis (ALS) using two drugs, one of them already approved for multiple sclerosis, could be a promising approach for treatment. Their study “Therapy of autoimmune inflammation in sporadic amyotrophic lateral sclerosis: Dimethyl fumarate and H-151 downregulate inflammatory cytokines in the cGAS-STING pathway” appears in The FASEB Journal.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects the nerve cells in the brain and spinal cord. There are two types of ALS: familial ALS, which is passed down through families and accounts for about 5–10% of cases, and sporadic ALS, which occurs without any family history and makes up about 90–95% of cases.

The current study focused on sporadic ALS which has been identified as an inflammatory disease where certain types of immune cells like cytotoxic T cells, mast cells, and inflammatory macrophages, mistakenly attack the neurons in the brain and spinal cord.

To test potential treatments, researchers in UCLA’s department of integrative biology and physiology, led by Milan Fiala, MD, of the David Geffen School of Medicine, treated immune cells from sporadic ALS patients with two substances: dimethyl fumarate (DMF) and molecule H-151. DMF is a drug already approved for treating multiple sclerosis. H-151 has been shown to block autoimmunity in laboratory models.

They found both DMF and H-151 reduced the expression of cytokines and granzymes, which are involved in the inflammatory process. The researchers also found that the effect of DMF was enhanced when combined with epoxyeicosatrienoic acids, certain fatty acids that can be found in the diet.

“The eicosanoid epoxyeicosatrienoic acids (EET) from arachidonic acid was anti-inflammatory in synergy with DMF. H-151 and DMF are thus candidate drugs targeting the inflammation and autoimmunity in sALS via modulation of the NFκB and cGAS/STING pathways,” write the investigators, who plan to seek approval for a clinical trial after more investigation in additional ALS patients’ immune cells.

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