Development of ferroptosis inhibitors for Huntington Disease

NIH RePORTER · NIH · R33 · $389,574 · view on reporter.nih.gov ↗

Abstract

Project Summary Ferrostatin is a potent inhibitor of ferroptosis; which is a non-apoptotic form of cell death potentially involved in a number of degenerative diseases. We have shown the effectiveness of Fer-1 in brain slice models of Huntington's disease (HD) and glutamate toxicity. However, ferrostatin's pharmacokinetic/pharmacodynamic (PK/PD) properties make it unsuitable for use in animal models to further test the role of ferroptosis in these diseases. We have made a series of ferrostatin analogs to explore ferrostatin's structure-activity relationship (SAR) and to improve activity and metabolic and plasma stability. While we have made significant progress in creating metabolically stable analogs with good potency and plasma stability, additional improvements are envisioned that could result in highly potent, stable compounds that could be dosed in vivo. The result of this investigation will be a set of optimized compounds for in vivo use to study the role of lipid peroxidation in a mouse model of Huntington Disease. In the longer term, we will be able to use these optimized compounds to probe the role of lipid peroxidation and ferroptosis in a variety of degenerative diseases.

Key facts

NIH application ID
10445418
Project number
4R33NS109407-02
Recipient
COLUMBIA UNIV NEW YORK MORNINGSIDE
Principal Investigator
Brent R. Stockwell
Activity code
R33
Funding institute
NIH
Fiscal year
2021
Award amount
$389,574
Award type
4N
Project period
2021-08-15 → 2023-07-31