UT Southwestern Medical Center researchers have found that a cancer drug could be used to treat a common form of dementia.
“Suberoylanilide hydroxamic acid (SAHA) holds promise as a first-generation drug for the prevention and treatment of familial frontotemporal dementia (FTD), a progressive, inherited neurodegenerative disease for which there is no treatment,” said Joachim Herz, professor of molecular genetics and neuroscience at UT Southwestern and the study’s senior author.
SAHA is in a class of drugs called histone deacetylase inhibitors, and is approved for use in a cancer called cutaneous T-cell lymphoma.
Dementia
“SAHA is already approved for clinical use in an unrelated condition, which should make it easier to move quickly to human trials,” said Herz.
The UT Southwestern researchers showed that SAHA increased the cell-signalling protein progranulin (GRN) levels in a dose-dependent way in cultured mouse cells and also demonstrated that it restored near-normal GRN production in cells from human subjects with FTD.
“We found a drug that can overcome the chemical deficiency associated with the condition, and we showed that it worked in cells taken from humans with FTD,” said Gang Yu, co-senior author of the study.
The study has been published in the current issue of the Journal of Biological Chemistry.
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