20 July, 2025
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INDIANAPOLIS – Researchers at the Indiana University School of Medicine have unveiled a promising portfolio of novel drug targets that could accelerate the development of treatments for Alzheimer’s disease. This breakthrough comes as part of a long-standing initiative led by the TREAT-AD drug discovery center, which has been at the forefront of Alzheimer’s research for over five years.

The TREAT-AD center, short for Target Enablement to Accelerate Therapy Development for Alzheimer’s Disease, is a collaborative effort involving the IU School of Medicine, Purdue University, the Indiana Biosciences Research Institute, Lgenia Inc., and the University of Pittsburgh. Together, these institutions aim to rejuvenate the pipeline of potential Alzheimer’s therapeutics.

Collaborative Efforts and Leadership

Avi Benitah, a postdoctoral fellow at the IU School of Medicine, is among the many researchers contributing to this effort from the Indiana Biosciences Research Institute lab. The institute plays a crucial role in the TREAT-AD center’s mission to discover and study new therapeutic targets for Alzheimer’s disease.

“What has been collectively accomplished across the IU School of Medicine over the past several years has begun to solidify a true center of excellence that investigates the basic science of Alzheimer’s disease, translates it to therapeutic opportunities, and develops a pathway for next-generation clinical studies,” said Alan Palkowitz, senior research professor of medicine at the IU School of Medicine.

Palkowitz leads the center alongside co-principal investigators Bruce T. Lamb, executive director of the Stark Neurosciences Research Institute, and Timothy Richardson, senior research professor of medicine. According to Lamb, the TREAT-AD Center is unique due to its combination of academic and pharmaceutical expertise, ranging from bioinformatics and structural biology to medicinal chemistry and clinical research.

Significant Funding and Research Progress

The National Institute on Aging recently renewed its support for TREAT-AD with a five-year, $41.6 million grant. The center, established in 2019, initially focused on hundreds of proteins related to microglia, the brain’s immune cells, narrowing down to 30 potential targets and further refining to five promising candidates for new therapies.

According to the Alzheimer’s Association, more than 7 million Americans are living with Alzheimer’s disease, a number expected to rise to nearly 13 million by 2050.

Among the most advanced targets is SHIP1, a gene associated with late-onset Alzheimer’s. The IU School of Medicine has secured an additional $11.3 million grant to advance a SHIP1 inhibitor into clinical trials.

“The success of TREAT-AD continues to bolster the IU School of Medicine’s reputation as one of the nation’s preeminent Alzheimer’s disease research programs,” said Dr. Jay L. Hess, dean of the IU School of Medicine. “We are honored that the NIH has renewed funding for this critical program.”

Future Directions and Broader Implications

Over the next five years, TREAT-AD plans to expand its research beyond the brain’s immune system to explore additional drivers of neuroinflammation and their connection to Alzheimer’s disease. The center will also focus on developing a broader range of drug targets, including antibodies and silencing RNAs.

Jeff Dage, senior research professor of neurology at the IU School of Medicine, is leading efforts to enhance the use of biomarkers—biological indicators of Alzheimer’s pathology. His work on phosphorylated tau 217 as a novel blood biomarker has already contributed to the FDA’s clearance of the first blood test for Alzheimer’s.

“We envision biomarkers as an important part to enable the study of new targets and potential new drug molecules, not only for understanding an individual patient’s disease but for monitoring the effectiveness of these novel treatments,” Palkowitz explained.

The TREAT-AD center’s ongoing research and collaboration with regional institutions continue to position it as a leader in Alzheimer’s drug discovery, offering hope for millions affected by this debilitating disease.