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Mechanistic plausibility

Clenbuterol 

March 11, 2026

Clenbuterol’s effects on neuroinflammation, muscle hypertrophy, and mitochondrial activity appear potentially relevant to ALS disease pathology. Data in murine models of motor neurondisease as well as two small flawed clinical trials in PALS are also somewhat promising but due to the small sample sizes, lack of randomization or blind-ing or placebo controls, these must be […]

Alpha Lipoic Acid

May 28, 2025

ALA has several plausible mechanisms for slowing ALS progression, including enhancing energy production, reducing oxidative stress as a potent antioxidant and anti inflammation. Preclinical studies demonstrated better motor function and improved survival. One open label study suggested improved QOL and fatigue when administered as a palladium lipoic acid complex, but motor function was not assessed. […]

Caffeine

June 16, 2023

Caffeine is inexpensive, reasonably safe at doses of under 400 mg daily, and has plausible mechanisms by which it could slow ALS progression. However, data from pre-clinical models are contradictory and a two cohort studies showed no clear relationship between caffeine intake and ALS progression. Based on all this, we cannot endorse caffeine as anALS […]

Astaxanthin

January 25, 2023

There are theoretical mechanisms supporting the potential role of astaxanthin in the treatment of ALS, however, there are no ALS specific preclinical data exploring this treatment. One verified “ALS reversal” occurred while taking astaxanthin in the setting of a cocktail of various other therapies an association that does not prove causality. There have been no […]

Ozone

November 21, 2022

Ozone therapy has possible mechanisms for treating ALS. A preclinical study in very small numbers of mTDP43 mice (which has yet to be peerreviewed) suggested benefits on motor function and survival (21,22); however, these benefits were not seen in mSOD1 mice (20). One verified “ALS reversal” occurred on a cocktail of alternative therapies including ozone […]

Butyrates

March 21, 2022

Butyrates have plausible mechanisms for slowing ALS progression and positive pre clinical studies. One trial suggests that sodium phenylbutyrate (NaPB) in combination with Tauroursodeoxycholic acid (TUDCA) can slow ALS progression and prolong survival, but the specific contribution of NaPB toward this effect is unclear. Butyrates appear reasonably safe for use in humans. Based on the […]

Melatonin

June 2, 2021

Melatonin has plausible mechanisms, some positive (and some negative) pre-clinical data, and two case reports in which it was part of a cocktail of treatments associated with recovery of lost motor function. As we have stated previously, there aremultiple possible explanations for cases like these. There was also a very small, flawed retrospective study suggesting […]

Tamoxifen

February 1, 2021

Tamoxifen is reasonably safe, has plausible mechanisms for treating ALS, and has at least one positive preclinical study. One case report and 2 small human trials suggested an association between tamoxifen (at higher doses) and slower ALS progression but this is not enough evidence to recommend this medication as an ALS treatment. Moving forward, we […]

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