As an immunosuppressant drug, AZA has a plausible mechanism for slowing the progression of ALS. However, there is no pre-clinical data to support its use and two clinical trials did not support efficacy. There are 2 published cases in which
ALS reversals occurred on AZA, but it is not clear to us that the AZA actually contributed to the ALS improvements. One of these patients also had myasthenia gravis, which is known to cause reversible weakness and therefore complicates the measurement of ALS. The other patient was taking many different medications and supplements along with AZA. AZA has very serious, potentially fatal, both short and long-term risks associated with its use and requires medical monitoring. Based on the
available data, we do not advise the use of AZA as an ALS treatment
Patient case reports
Vitamin E
Vitamin E (a-tocopherol) is perhaps the most studied supplement in the history of ALS and was taken by one of the most famous ALS patients. Vitamin E has mechanistic potential in ALS as an antioxidant but appears in the SOD1 mutant mouse model to only have an effect on delaying disease onset. This bears out in human populations as large prospective cohorts show that long-duration vitamin E supplementation may decrease the risk of ALS, but randomized clinical trials show that even high dose vitamin E does not benefit the disease once ALS has been diagnosed. Although it is inexpensive and safe, we do not recommend vitamin E to slow, stop or reverse ALS
based on the lack of efficacy in clinical trials.
Glutathione
As an ALS treatment, glutathione and cysteine-containing supplements that increase glutathione appear reasonably safe, and they have a plausible mechanism, positive preclinical data and 2 interesting case reports. Unfortunately small clinical trials of glutathione itself and of acetylcysteine showed no significant benefit. Given these negative clinical trials, we do not advise PALS to take glutathione or cysteine-containing supplements for their ALS at this time.
Click here to download the complete review.
Gluten-Free Diet
Theoretically, gluten-induced autoimmunity could trigger ALS. However, the data supporting this link are weak, consisting of two association studies and a single case-report. Further studies are needed to confirm the relationship between GRDs and ALS, and the utility of the GFD in patients with both conditions. In spite of the fact that GFD is reasonably safe, it is a complex undertaking and is more expensive than a standard diet. While we wait for better data, it would be reasonable to screen PALS who have GI symptoms, iron-deficiency anemia, or an abnormal brain MRI for the antibodies associated with GFDs. Those with elevated antibodies could be referred to a gastroenterologist for further work-up, and if this is consistent with a GRD, then GFD could be tried under the guidance and monitoring of a dietician.
Click here to download the complete review.
Acupuncture
Acupuncture is reasonably safe, and has potential mechanisms of action, pre-clinical studies and case reports suggesting that it could be a useful treatment for ALS. However, before it can be endorsed even as a candidate for a phase II trial, the studies described above need to be independently replicated using more clearly verified diagnoses and more rigorous designs, including appropriate controls and validated ALS outcome measures.