Timothy M. Miller, MD, PhD, the David Clayson Professor of Neurology at WashU Medicine, has been awarded the 2026 Potamkin Prize for Research in Pick’s, Alzheimer’s, and Related Diseases, one of the most prestigious international honors in dementia research. He received the award April 19 at the American Academy of Neurology’s annual meeting in Chicago.
An internationally recognized neurologist, Miller has devoted his career to understanding how neurodegenerative diseases develop and progress and to translating that knowledge into therapies for patients. His research focuses on the mechanisms that drive protein abnormalities in diseases such as ALS and dementia and on developing targeted therapies to counter those processes.
“The Potamkin Prize is a tremendous honor, and I am deeply grateful to my colleagues, collaborators and especially the participants and families involved in research,” Miller said.
Miller is a pioneer in the development of therapies based on antisense oligonucleotides (ASOs), a targeted approach to disease treatment that interferes with the production of harmful proteins. His work has helped lay the scientific and clinical groundwork for ASO therapies for neurological diseases. Miller led the international clinical trials that resulted in the U.S. Food and Drug Administration’s approval of tofersen, an ASO drug that blocks production of the toxic protein that causes a rare form of ALS and has been shown to slow or stabilize the degeneration typical of ALS.
Miller’s lab developed an ASO approach for targeting tau, a protein involved in Alzheimer’s disease and other dementias. The ASO based on this work is now in a large Phase 2 clinical trial in early Alzheimer’s Disease with results expected this year.
Highly regarded as one of the top prizes in Alzheimer’s and dementia research, the Potamkin Prize recognizes scientists who have made major contributions to understanding the causes of Pick’s, Alzheimer’s and related dementias, and who have advanced efforts to prevent, treat and cure such neurodegenerative diseases. The award is presented jointly by the American Academy of Neurology (AAN) and the American Brain Foundation and funded through the philanthropy of the Potamkin family.
Miller is one of 11 members of the WashU Medicine Department of Neurology selected to receive 2026 AAN awards. As part of the Potamkin award, Miller delivered a presentation on his work during a conference session titled “New Perspectives on Alzheimer’s Therapeutics.”