HDFTY – A Huntington’s disease study to repurpose FTY720

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First announcement 14th Mar 2024.

HDFTY – A Huntington’s disease study to repurpose FTY720 – funding aquisition
Funding organisations: we are seeking funding from private and state organisations
Proposed start: 2024

Prof. Jens Pahnke, University of Oslo (UiO) & Oslo University Hospital (OUS), Oslo, Norway
Prof. Tatyana Gurevich, National Huntington’s Disease Center at Sourasky Medical Center, Tel Aviv, Israel
Interested, pls send email to us !

We have investigated novel treatment options for Huntington’s disease since a few years based on our previous work on Alzheimer’s disease and ABC transporters.
Recently, we have discovered an effective treatment to modulate disease onset and progress in a sex-dependent manner in zQ175dn mice (Wu et al. 2022) based on a known molecular signalling mechanism involving ceramides and sphingomyelin metabolism. We have tested a repurposed drug used in Mutiple sclerosis treatment, fingolimod also known as FTY720, in a cross-over design study involving 128 mice until an age of 57 weeks and could confirm disease preventing effects in female mice and disease delaying effects in male mice. We could also determine the rescue of organ atrophy due to HD in the mice (testis, brain) and improvement of essential motor funtions.

The data are prepared for publication. A preprint of the article can be downloaded here: OSF.io – Open access HD projects

Keywords: Huntington’s disease; drug repurposing; neurodegenerative diseases; Sphingomyelin signalling; Fingolimod; FTY720

Resources / Links:
A7HD project application to JPND 2024 – read more….