Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition

HL Narver, L Kong, BG Burnett, DW Choe… - Annals of …, 2008 - Wiley Online Library
HL Narver, L Kong, BG Burnett, DW Choe, M Bosch‐Marcé, AA Taye, MA Eckhaus…
Annals of neurology, 2008Wiley Online Library
Early treatment with the histone deacetylase inhibitor, trichostatin A, plus nutritional support
extended median survival of spinal muscular atrophy mice by 170%. Treated mice continued
to gain weight, maintained stable motor function, and retained intact neuromuscular
junctions long after trichostatin A was discontinued. In many cases, ultimate decline of mice
appeared to result from vascular necrosis, raising the possibility that vascular dysfunction is
part of the clinical spectrum of severe spinal muscular atrophy. Early spinal muscular …
Abstract
Early treatment with the histone deacetylase inhibitor, trichostatin A, plus nutritional support extended median survival of spinal muscular atrophy mice by 170%. Treated mice continued to gain weight, maintained stable motor function, and retained intact neuromuscular junctions long after trichostatin A was discontinued. In many cases, ultimate decline of mice appeared to result from vascular necrosis, raising the possibility that vascular dysfunction is part of the clinical spectrum of severe spinal muscular atrophy. Early spinal muscular atrophy disease detection and treatment initiation combined with aggressive ancillary care may be integral to the optimization of histone deacetylase inhibitor treatment in human patients. Ann Neurol 2008; 64:465–470
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