New epigenetic inhibitors for Alzheimer's disease treatment

NIH RePORTER · NIH · R01 · $806,956 · view on reporter.nih.gov ↗

Abstract

Project Summary Alzheimer’s disease (AD) is a neurodegenerative disorder that is the primary cause of dementia, the mechanisms of AD have not been completely elucidated and there has yet to yield effective therapy that can prevent, stop or reverse cognitive deficits associated with AD. Epigenetics refers to functional modifications to the genome that do not involve a change in the DNA sequence. Epigenetics became an attractive field in recent years within the drug discovery research communities. Recent preclinical studies also provide robust evidence for the involvement of HDACs in various neurological diseases, and pharmacological treatment aimed at modulating epigenetic regulation. The goal of this application is to develop novel small molecules as HDAC11 selective inhibitor as potential AD therapeutics. In this proposal, we will explore SAR of our leading scaffold to develop a new generation of HDAC11-selective inhibitors (Aim 1), which will be tested to select top compounds with improved PK/PD properties and anti-AD effects using AD preclinical models (Aim 2). Towards the ultimate goal of developing novel HDAC11 inhibitors as effective AD treatments, the end-point of this application is to identify top candidates for Investigational New Drug (IND)-enabling studies.

Key facts

NIH application ID
10853200
Project number
1R01AG086433-01
Recipient
MASSACHUSETTS GENERAL HOSPITAL
Principal Investigator
Changning Wang
Activity code
R01
Funding institute
NIH
Fiscal year
2024
Award amount
$806,956
Award type
1
Project period
2024-06-15 → 2029-02-28