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CHF5074 restores visual memory ability and pre-synaptic cortical acetylcholine release in pre-plaque Tg2576 mice

Bibliographic

Year of Publication:
2013
Contact PI Name:
Luca Nicola Ferraro
Contact PI Affiliation:
Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara, Italy
Co-Authors:
A. Giuliani, S. Beggiato, V.A. Baldassarro, C. Mangano, L. Giardino, B.P. Imbimbo, T. Antonelli, L. Calzà
Primary Reference (PubMED ID):
Funding Source:
Emilia-Romagna Region
Study Goal and Principal Findings:

1-(3',4'-Dichloro-2-fluoro[1,1'-biphenyl]-4-yl)-cyclopropanecarboxylic acid (CHF5074) is a gamma-secretase modulator, devoid of anticyclooxygenase (COX) and Notch-interfering activities in vitro, but still able to reduce microglia activation in vivo. In previous studies, using  AD mouse models, CHF5074 shown to prevent brain plaque deposition without affecting Notch processing.  In addition, CHF5074 was found to reverse different types of memory deficits in young, plaque free mice. The beneficial effects of a subchronic CHF5074 treatment in young transgenic mice occurred at a stage that precedes plaque formation and were associated with a reduction in intraneuronal APP/Abeta and hyperphosphorylated tau. While these data suggest that CHF5074 reduces early cognitive deficits in transgenic mouse models of AD, there is no evidence as to its efficacy on cholinergic neuron dysfunction, which can take place along with intraneuronal Abeta accumulation, well before plaque deposition. Thus, in this study, the authors investigated the effects of an acute or subacute CHF5074 treatment on in vivo cognitive performance and on [3 H] acetylcholine (ACh) and GABA release from isolated nerve terminals (synaptosomes) obtained from 7- month-old transgenic Tg2576 mice. The behavioral and neurochemical effects of CHF5074 were compared with those induced, under the same experimental conditions, by the gamma secretase inhibitor LY450139 (semagacestat).  Results showed that Vehicle-treated Tg2576 mice displayed an impairment of recognition memory compared with wild-type animals. This impairment was recovered in transgenic animals acutely treated with CHF5074 (30 mg/kg), while LY450139 (1, 3, 10 mg/kg) was ineffective. In frontal cortex synaptosomes from vehicle treated Tg2576 mice, K+ -evoked [3 H]ACh release was lower than that measured in wild-type mice. This reduction was absent in transgenic animals subacutely treated with CHF5074 (30 mg/kg daily for 8 days), while it was slightly, not significantly, amplified by LY450139 (3 mg/kg daily for 8 days). There were no differences between the groups on spontaneous [3 H] ACh release as well as spontaneous and K+ - evoked GABA release. These results suggest that CHF5074 has beneficial effects on visual memory and cortical cholinergic dysfunctions in pre-plaque Tg2576 mice. Together with previous findings, these data suggest that CHF5074 could be a possible candidate for early Alzheimer’s disease therapeutic regimens. 

Therapeutic Agent

Therapeutic Information:
Therapy Type:
Small Molecule
Therapeutic Agent:
CHF5074
Therapeutic Target:
gamma Secretase
Therapy Type:
Small Molecule
Therapeutic Agent:
LY-450139 (Semagacestat)
Therapeutic Target:
gamma Secretase

Animal Model

Model Information:
Species:
Mouse
Model Type:
APP
Strain/Genetic Background:
Not Reported

Experimental Design

Is the following information reported in the study?:
Power/Sample Size Calculation
Randomized into Groups
Blinded for Treatment
Blinded for Outcome Measures
Pharmacokinetic Measures
Pharmacodynamic Measures
Toxicology Measures
ADME Measures
Biomarkers
Dose
Formulation
Route of Delivery
Duration of Treatment
Frequency of Administration
Age of Animal at the Beginning of Treatment
Age of Animal at the End of Treatment
Sex as a Biological Variable
Study Balanced for Sex as a Biological Variable
Number of Premature Deaths
Number of Excluded Animals
Statistical Plan
Genetic Background
Inclusion/Exclusion Criteria Included
Conflict of Interest

Outcomes

Outcome Measured
Outcome Parameters
Behavioral
Novel Object Recognition Test (NORT)
Biochemical
Neurotransmitter Release
Acetylcholine Levels
GABA Levels
Outcomes Notes:
Spontaneous neurotransmitter release was measured for [3H]Ach, GABA, glutamate, and K+-evoked.