What drugs are being developed for the treatment of ME/CFS?

February 2, 2021

Commentary by Dr Charles Shepherd,
Hon Medical Adviser, ME Association

An interesting review of the latest research into new drug therapies that might be able to tackle the underlying disease process in ME/CFS, rather than just being used to treat symptoms, has been published by the American journal ‘Pharmacological Research‘.

Special attention has been paid as to how new treatments might impact on the immune systems of people with ME/CFS as well as any detected metabolic and mitochondrial dysfunction.

It is encouraging to note that one of the authors has links to the major pharmaceutical company Pfizer.  However, as noted in their conclusion: 

 “interest among pharmaceutical companies and biotech/venture investors appears to be almost non-existent”.

This is clearly something that needs to be addressed on both sides of the Atlantic. In the UK, the CFS/ME Research Collaborative is showing a clear signs of interest,

Given the clinical and pathological overlaps between ME/CFS and Long Covid, and the research initiatives looking at the treatment of Long Covid, this review should also be of interest to the Long Covid patient and medical community.

The review covers very similar ground in relation to treatments that have already been assessed in clinical trials to the information that is contained in the Treatment section in the MEA Purple Book.

The conclusions regarding safety and efficacy of drugs which have already been assessed as possible treatments for ME/CFS are also largely consistent with the findings of the extensive evidence-based review that was carried during the preparation of the new NICE guideline on ME/CFS.  

And good to see that they have noted the revised recommendations regarding CBT and GET in the new guideline. 


While ME/CFS remains a poorly understood disease, there is a growing understanding of its mechanistic roots. New research directions are emerging with the potential to generate better diagnostic tools, biomarkers and pharmacological treatments. 

Approaches to therapy under investigation are directed at specific molecular or cellular irregularities associated with ME/CFS such as autoantibodies, immune dysregulation (e.g. NK cell function) or mitochondrial dysfunction. 

Recent discoveries suggest the possibility of target-based drug discovery programs directed against specific proteins implicated in either the initiation or propagation of ME/CFS symptoms (e.g. PDHK). 

These developments are occurring at the same time as advances in the diagnosis and detection of ME/CFS. 

Investments in patient profiling and tool generation are starting to yield fruit, and we anticipate the availability of reliable clinical diagnostic tools in the near future. 

Despite its significant human cost and economic impact, ME/CFS research has yet to attract major financial support. 

Recognising this deficiency, the U.S. National Institutes of Health (NIH) have established a Trans-NIH ME/CFS working group to coordinate a multi-institute effort to learn about and treat this disease. 

However, at the current time, interest among pharmaceutical companies and biotech/venture investors appears to be almost non-existent. 

It is to be hoped that increased awareness of the basis and impact of ME/CFS will stimulate the investment in research that will be necessary for development of effective new therapies.

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