Testing a Mitochondria-Targeting Compound in Alzheimer's Disease

Principal Investigator

Mentors

  • Johan  Auwerx, MD, PhD

    Johan Auwerx, MD, PhD

Project Goals

The study aim is to assess whether a compound that supports healthy mitochondrial stress responses offers benefit in Alzheimer's disease.

Project Summary

Mitochondria are best known for packaging energy into a usable form for the cell, but they also are emerging as potential very early markers of Alzheimer's disease–related changes. These indispensable cell structures show these changes sometimes decades before dementia begins to manifest. 

Qi Wang, PhD, and her colleagues hypothesize that preventing or repairing this damage might in turn inhibit progression of Alzheimer's disease. To assess this idea, they plan to use a compound called 9-TB, which shows signs of supporting healthy stress responses in mitochondria. They will test the compound in a lab model of Alzheimer's disease, including both sexes to determine whether there are sex-based differences in responses. 

Following this set of studies, Dr. Wang and her colleagues will analyze how cells respond to 9-TB–related improvements in mitochondrial function. They will assess the cell response at several levels, from gene activity to protein production and location in the cell, known as a multiomics analysis. This work is expected to highlight candidate genes involved in these beneficial responses that Dr. Wang and her coworkers will further assess computer-based and molecular lab studies, including a simulation of how 9-TB might interact in humans to produce beneficial effects.

Dr. Wang and her colleagues ultimately aim to set the stage for phase I clinical studies of drug candidates that generate this healthy mitochondrial stress response. These effects likely would involve several factors in Alzheimer's disease development, and the group believes any candidates would be poised to be multi-targeting treatments for the condition.

Publications

First published on: September 05, 2023

Last modified on: April 15, 2024