The long-term treatment of myasthenia gravis (MG) relies on drugs which suppress the immune system. I listed some of these in my previous post titled How is innovative neurology research energising myasthenia? Steroids are the established first line immune suppressing treatment for MG but because of their many nasty side effects, they cannot be used at effective doses for long periods. This is why neurologists treating MG use so-called steroid-sparing agents to reduce, or eliminate, the need for steroids.

Little red pills. Jon nagl on Flikr.
Little red pills. Jon nagl on Flikr.

Azathioprine has the best evidence of effectiveness as a steroid-sparing drug, and it is the acknowledged favourite of neurologists. Azathioprine may however fail or cause unacceptable side effects. It is also unsuitable for people who lack TPMT, the enzyme that breaks it down. It is in these situations that things become slightly tricky for the neurologist.

By NLM - NLM Pillbox,, Public Domain,
By NLM – NLM Pillbox,, Public Domain,

In theory, neurologists are spoilt for choice when they can’t use Azathioprine. Methotrexate is my favourite option in such cases because it has an easy weekly dosing regime and it is fairly well-tolerated. Alas, a recent paper in Neurology titled A randomized controlled trial of methotrexate for patients with generalized myasthenia gravis has unsettled me by suggesting that methotrexate is not living up to its top billing. The authors of the paper studied 50 people with myasthenia gravis who were already taking steroids. They put some of them on methotrexate, and the others on placebo. The outcome was surprising; methotrexate did very little to reduce the requirement for steroids, and it did nothing to improve the symptoms of MG.

By Fdardel - Own work, CC BY-SA 3.0,
By Fdardel – Own work, CC BY-SA 3.0,


This is clearly disappointing. Whilst waiting for further studies to confirm or refute this finding, I wonder how reliable the other steroid-sparing MG drugs are. How good are mycophenolate, ciclosporin, cyclophosphamide, tacrolimus, and rituximab? What really works in MG? To the rescue comes the International consensus guidance for management of myasthenia gravis, just hot off the press! Alas, the experts who drafted this guidance only compounded my woes. They made many treatment recommendations, but these came with as many caveats. They said the evidence for mycophenolate and tacrolimus in MG is rather thin, and the evidence-based ciclosporine and cyclophosphamide have potentially serious side effects. And they couldn’t agree on how promising rituximab, the new kid on the block, really is.

By Oguenther at de.wikipedia - Own work mit Jmol auf Basis RCSB PDB 2OSL., Public Domain,
By Oguenther at de.wikipedia – Own work mit Jmol auf Basis RCSB PDB 2OSL., Public Domain,

We are therefore back to the question, what to do when Azathioprine fails? The experts tell us to stick to the usual suspects, but they urge caution. Perhaps what we need are newer and safer alternatives such as Lefluonamide, so new to the MG arena that it did not get a mention in the expert guidance.



5 thoughts on “What is the startling research unsettling the treatment of myasthenia gravis?

  1. On my third immune suppressive drug, always have had to have them combined with steroids to manage symptoms. Cyclosporine currently seems to be working. Regular blood tests and side effects are a given.
    Glad to read about another potential treatment!


  2. I have myasthenis and rheumatoid arthritus, i was on cell cept and am now trying methyltrexate vecause of the rheumatoid can i still take cell cept. Donald drachman from johns hopkins did jy thymectomy in 1979 i was one of his first, can you help i am worried thanks judith kapp


  3. I am a female patient with Myasthenia Gravis for 30 years and I have suffered tremendously because of this disorder. I had a thymectomy twenty five years ago and still have symptoms of MG. I came across Best Health Herbal Centre website after so many years researched through the internet. I was so desperate to find a way to treat this disorder naturally. Thank God for leading me to Best Health Herbal Centre. I started using Best Health Herbal Centre MG herbal formula this year. After 10 weeks of usage, my MG was totally reversed, symptoms like muscle weakness, difficulty swallowing, double vision, fatigue, impaired voice, or shortness of breath disappeared completely.


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