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Multi-target action of the novel anti-Alzheimer compound CHF5074: in vivo study of long term treatment in Tg2576 mice

Bibliographic

Year of Publication:
2013
Contact PI Name:
Laura Calzà
Contact PI Affiliation:
Health Science and Technologies Interdepartmental Center for Industrial Research, University of Bologna, Bologna, Italy
Co-Authors:
S. Sivilia, L. Lorenzini, A. Giuliani, M. Gusciglio, M. Fernandez, V.A. Baldassarro, C. Mangano, L. Ferraro, V. Pietrini, M.F. Baroc, A.R. Viscomi, S. Ottonello, G. Villetti, B.P. Imbimbo, L. Giardino
Primary Reference (PubMED ID):
Funding Source:
Chiesi Farmaceutici Parma Italy
Study Goal and Principal Findings:

The study investigated effects of CHF5074 on AD neuropathology, spine density/morphology, cell cycle, microglia activity, and cognitive impairment in Tg2576 mice. CHF5074 is a nonsteroidal anti-inflammatory derivative devoid of cyclooxygenase inhibitory activity. In vitro, CHF5074 behaves as a γ secretase modulator preferentially inhibiting Aβ42 production.  CHF5074 has been shown to inhibit brain plaque deposition and to attenuate or reverse contextual and spatial memory deficit in different transgenic mouse models of AD, also reversing long-term potentiation deficit in the hippocampus. However, recent data have shown that CHF5074 is able to promote axon growth and astrocyte plasticity by modulating Rho-GTPase-dependent signaling. In this study, the effects of a long-term (13-month) treatment with CHF5074 on indicators of brain functionality and neurodegeneration in transgenic Tg2576 mice were assessed and compared with those induced by a prototypical γ-secretase inhibitor (DAPT).Tg2576 mice fed with standard diet displayed an impairment of recognition memory. This deficit was completely reverted by the higher dose of CHF5074, while no effects were observed in DAPT-treated mice. Similarly, amyloid plaque burden, microglia activation and aberrant cell cycle events were significantly affected by CHF5074, but not DAPT, treatment. Both CHF5074 and DAPT reduced intraneuronal Aβ content, also increasing Aβ40 and Aβ42 plasma levels. This comparative analysis revealed a profoundly diverse range of clinically relevant effects differentiating the multifunctional anti-inflammatory derivative CHF5074 from the γ-secretase inhibitor DAPT and highlighted unique mechanisms and potential targets that may be crucial for neuroprotection in mouse models of AD.

Therapeutic Agent

Therapeutic Information:
Therapy Type:
Small Molecule
Therapeutic Agent:
CHF5074
Therapeutic Target:
gamma Secretase
Therapy Type:
Small Molecule
Therapeutic Agent:
DAPT
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
Experiment Notes

The estimated ingested doses of CHF5074 were about 20 and 60 mg/kg/day (behaviourally effective dose when given chronically for 9 months); the estimated ingested dose of DAPT was about 60 mg/kg/day.

Outcomes

Outcome Measured
Outcome Parameters
Behavioral
Novel Object Recognition Test (NORT)
Histopathology
beta Amyloid Load
Biochemical
Brain-beta Amyloid Oligomers
Brain-beta Amyloid Peptide-Total
Plasma-beta Amyloid Peptide 40
Plasma-beta Amyloid Peptide 42
Immunochemistry
Activated Microglia
Activated Astrocytes
Intracellular beta Amyloid Peptide
Cyclin A
Mature Neurons
Microscopy
Dendritic Spine Density
Dendritic Spine Morphology
Biomarker
Plasma-beta Amyloid Peptide 40
Plasma-beta Amyloid Peptide 42
Toxicology
Mortality