Long-term hydromethylthionine treatment is associated with delayed clinical onset and slowing of cerebral atrophy in a pre-symptomatic P301S MAPT mutation carrier

Peter Bentham, Roger T. Staff, Bjoern O. Schelter, Helen Shiells, Charles R. Harrington, Claude M. Wischik*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

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Abstract

One of the mutations in the microtubule-associated protein tau, P301S, is causative for dominantly inherited frontotemporal dementia characterized by extensive tau pathology for which no licensed treatment is available. Hydromethylthionine is a potent tau aggregation inhibitor. We report treatment of an asymptomatic carrier of the P301S mutation using hydromethylthionine over a 5-year period beginning at the mean age of onset of clinical decline in the family. During the period of treatment, the rates of progression of cerebral atrophy were reduced by 61%-66% in frontal and temporal lobes, and the patient remained clinically asymptomatic.

Original languageEnglish
Pages (from-to)1017-1023
Number of pages7
JournalJournal of Alzheimer's Disease
Volume83
Issue number3
Early online date28 Sept 2021
DOIs
Publication statusPublished - 28 Sept 2021

Bibliographical note

ACKNOWLEDGMENTS
We thank the patient for permitting publication of the present report.
Authors’ disclosures available online (https://www.j-alz.com/manuscript-disclosures/21-0390r1).

Keywords

  • Frontotemporal dementia
  • hydromethylthionine
  • leucomethylthioninium
  • microtubule-associated protein tau
  • P301S

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