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Mefloquine in the treatment of progressive multifocal leukoencephalopathy
  1. T E Gofton1,
  2. A Al-Khotani1,
  3. B O'Farrell1,
  4. L C Ang2,
  5. R S McLachlan1
  1. 1Department of Clinical Neurological Sciences, University of Western Ontario, London, Ontario, Canada
  2. 2Department of Pathology (Neuropathology), University of Western Ontario, London, Ontario, Canada
  1. Correspondence to Dr Richard S McLachlan, Department of Clinical Neurological Sciences, University Hospital, London Health Sciences Centre (LHSC), 339 Windermere Road, London, Ontario, N6A 5A5, Canada; rsmcl{at}


Mefloquine, an antimalarial medication with efficacy against JC virus, was used to treat progressive multifocal leukoencephalopathy. A 54-year-old woman with sarcoidosis presented with a progressive cerebellar syndrome. MRI showed lesions affecting the right cerebellum that progressed over time to the brainstem. JC virus was found in the cerebrospinal fluid (CSF), and brain biopsy confirmed the diagnosis of progressive multifocal leukoencephalopathy. Mefloquine 1000 mg/week was initiated 6 months after symptom onset. Clinical progression stopped immediately, and JC virus became undetectable in the CSF. No clinical or imaging evidence of disease progression has occurred over 20 months of follow-up. This is the first report of successful treatment of progressive multifocal leukoencephalopathy with mefloquine.

  • Progressive multifocal leukoencephalopathy
  • mefloquine
  • JC virus
  • leukoencephalopathy

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  • Study Sponsorship No external funding was provided for this project.

  • Disclosures The authors have no financial interest in the products discussed within this case report.

  • Competing interests None.

  • Patient consent Obtained.

  • Ethics approval This study was conducted with the approval of The University of Western Ontario Research Ethics Board.

  • Provenance and peer review Not commissioned; externally peer reviewed.