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Expert review advises against azathioprine for ALS treatment

Original source

AzathioprineALSUntangled · 20 August 2020 (opens in a new tab)

ALS Untangled advises against using azathioprine (AZA) as a treatment for amyotrophic lateral sclerosis (ALS). The review says two clinical trials did not support its effectiveness, while two reported cases of ALS improvement could not establish that AZA caused the changes.

Why this matters

Azathioprine carries potentially fatal short- and long-term risks and requires medical monitoring. The available evidence does not show that those risks are justified by a demonstrated ALS benefit. [S1]

Limitations and context

This is an independent expert review, not a new clinical trial. The review describes two published cases and two clinical trials, but the supplied source does not provide trial details. The cases were difficult to interpret because one person also had myasthenia gravis and the other used multiple medicines and supplements.

Summarised by Compass 10 August 2026

This summary was generated by AI from the source listed above. It is not medical advice, so read the original source for anything that affects your care.

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