Mycophenolate treatment for MS; why not?

M
Could an old transplant drug be the answer for MSers without insurance? #MSBlog #MSResearch

Epub: Michel et al. Mycophenolate mofetil in multiple sclerosis: a multicentre retrospective study on 344 patients.J Neurol Neurosurg Psychiatry. 2013 May 23.

OBJECTIVES: Mycophenolate mofetil (MMF) is an immunosuppressive agent, sometimes used as a disease-modifying therapy for MS. Several studies have reported the relative safety of this treatment but, to date, its efficacy has rarely been described. This group performed a retrospective study to assess the safety and efficacy of MMF in MSers.

METHODOLOGY: Three French MS centres included all of their MSers treated by MMF. The main outcome criterion was annualised relapse rate (ARR) in the 1 year period after onset of MMF compared with the 1 year control period. Treatment with another immunosuppressive drug, such as mitoxantrone or cyclophosphamide, in the 2 years preceding initiation of MMF was included in a subgroup analysis. MMF safety and progression of the Expanded Disability Status Scale (EDSS) score were also assessed.

RESULTS: 344 MSers were included; 149 MSers were previously treated with another immunosuppressant (IS group). Mean MMF treatment duration was 25.3±1.1 months. During the 1 year control period, ARR was 1.11±0.08, and for the 1 year treatment period, ARR was reduced significantly to 0.35±0.05 (p<0.0001, Wilcoxon paired test). Adverse events (occurring in 11% of patients) were mainly digestive disorders, benign infections, asthenia and transitory lymphopenia. Concerning the progression of disability, in the subgroup of MSers without previous immunosuppressant treatment, EDSS remained stable between initiation and 1 year after the beginning of MMF.

INTERPRETATION: These results suggest that MMF can improve or stabilise MSers and can be used as an alternative therapy.



“The problem with this data is that it is not controlled; i.e. there were no placebo-treated MSers to compare with. I have little doubt that MMF is probably an effective DMT, but as its patent is about to expire (Sept, 2013) who will do the necessary trials to show that it has a favourable risk:benefit ratio? MMF, in combination with other drugs, has transformed the solid-organ transplant field. Could we prescribe if for MS? Individual clinicians may get away with treating the occasional MSer with MMF, but at higher level this would not be legal. I have covered off-label prescribing in a previous post; you may be interested to read what I had to say about it then.” 

03 Jan 2013
It is not illegal as suggested to prescribe off-label where other licensed drugs are available. It is potentially (the Avastin litigation has not been resolved) ‘illegal’ for NHS trusts to pay for off-licence drugs in those circumstances .
16 Dec 2012
Henry V. Off-label prescribing. Legal implications. J Leg Med. 1999 Sep;20(3):365-83. Some excerpts: ….. The FDA has an official policy to provide physicians the ability to prescribe approved drugs for unapproved uses called 
12 Sep 2012
It is not illegal as suggested to prescribe off-label where other licensed drugs are available. It is potentially (the Avastin litigation has not been resolved) ‘illegal’ for NHS trusts to pay for off-licence drugs in those circumstances 

About the author

Prof G

Professor of Neurology, Barts & The London. MS & Preventive Neurology thinker, blogger, runner, vegetable gardener, husband, father, cook and wine & food lover.

1 comment

  • The use of CellCept to treat MS, while not exactly common, is not all that unusual here in the US. I gave it a whirl several years ago, but saw no benefit whatsoever. Then again, none of the immunosuppressives have done me any good, so the result wasn't surprising…

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