Up-regulating BDNF with an ampakine rescues synaptic plasticity and memory in Huntington's disease knockin mice

DA Simmons, CS Rex, L Palmer… - Proceedings of the …, 2009 - National Acad Sciences
DA Simmons, CS Rex, L Palmer, V Pandyarajan, V Fedulov, CM Gall, G Lynch
Proceedings of the National Academy of Sciences, 2009National Acad Sciences
Cognitive problems occur in asymptomatic gene carriers of Huntington's disease (HD), and
mouse models of the disease exhibit impaired learning and substantial deficits in the
cytoskeletal changes that stabilize long-term potentiation (LTP). The latter effects may be
related to the decreased production of brain-derived neurotrophic factor (BDNF) associated
with the HD mutation. This study asked whether up-regulating endogenous BDNF levels
with an ampakine, a positive modulator of AMPA-type glutamate receptors, rescues plasticity …
Cognitive problems occur in asymptomatic gene carriers of Huntington's disease (HD), and mouse models of the disease exhibit impaired learning and substantial deficits in the cytoskeletal changes that stabilize long-term potentiation (LTP). The latter effects may be related to the decreased production of brain-derived neurotrophic factor (BDNF) associated with the HD mutation. This study asked whether up-regulating endogenous BDNF levels with an ampakine, a positive modulator of AMPA-type glutamate receptors, rescues plasticity and reduces learning problems in HD (CAG140) mice. Twice-daily injections of a short half-life ampakine normalized BDNF levels, activity-driven actin polymerization in dendritic spines, and LTP stabilization in 8-week-old mutants. Comparable results were obtained in 16-week-old HD mice with more severe LTP deficits. Ampakine treatments had no measurable effect on the decreased locomotor activity observed in the mutants but offset their impairments in long-term memory. Given that ampakines are well tolerated in clinical trials and were effective in this study after brief exposures, these results suggest a novel strategy for chronic treatment of the cognitive difficulties that occur in the early stages of HD.
National Acad Sciences