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 would like to see a larger human ALS clinical trial of tamoxifen at 80mg daily. Interestingly, one study suggests that tamoxifen may decrease a person’s risk for getting ALS. We hope to see this independently replicated.
Patient trials
Antiretrovirals
Antiretrovirals are a group of diverse drugs developed for HIV infections that vary widely in theoretical efficacy against HERVs, side effect profiles, and cost. HERV expression is apparently increased in some PALS; however, it is unknown if this is a
beneficial, neutral, or pathological process. Furthermore, it is not clear if ARV-targeted mechanisms such as cell infection and viral replication are taking place in PALS. Based on the lack of evidence for use of ARVs in PALS who test negative for HIV and HTLV, we cannot recommend them as a treatment for ALS. We look forward to the results of the two ongoing trials of ARVs in PALS.
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