TREAT-AD Case Study

The number of people living with Alzheimer’s disease is expected to nearly double over the next quarter century, creating an urgent need for new treatments that are able to slow or reverse its progression. Yet researchers still do not have a full picture of how this complex neurodegenerative disease progresses, and what can be done to stop it.

The Target Enablement to Accelerate Therapy Development of Alzheimer’s Disease (TREAT-AD) initiative was launched by the National Institute on Aging (NIA), part of the larger National Institutes of Health (NIH), to address this challenge. The program’s mission is to expand the pipeline of potential therapeutic targets by systematically identifying previously unrecognized contributors to Alzheimer’s and providing the scientific community with the tools needed to pursue them.

IBRI was chosen as a TREAT-AD partner because of its unique blend of capabilities in peptide design, small-molecule chemistry, iPSC-based pharmacology, and translational assay development. IBRI scientists advance prioritized targets emerging from TREAT-AD and drive them forward by designing and synthesizing candidate molecules and evaluating them in human cell-based systems.

TREAT-AD research has focused on a class of cells known as microglia, which are a key component of the brain’s immune system and play a role in both the inflammatory processes that drive Alzheimer’s as well as in clearing the cellular debris that the disease creates. To date, TREAT-AD has created candidate compounds to interact with four different aspects of microglia biology. One of these, the protein SHIP1, is a signaling protein that plays a role in microglial regulation. In December 2024, IBRI joined with Indiana University School of Medicine and Purdue University in securing a five-year, $11.3 million NIH grant to accelerate development of SHIP1 inhibitors as potential Alzheimer’s therapeutics.