Timothy Miller, MD, PhD, Clayson Professor and vice chair of research in the WashU Medicine Department of Neurology, has been selected as the 2026 recipient of the Carl and Gerty Cori Faculty Achievement Award. Awarded annually to one faculty member from WashU Medicine by the Faculty Senate Council and the Chancellor, the Cori Award recognizes faculty for their outstanding achievements as researchers and mentors, as well as service and dedication for the betterment of the university.
Miller is a world leader in the application of antisense oligonucleotides (ASOs) to treat neurodegenerative diseases. ASOs are small molecules that can target specific genes and change the production or behavior of certain proteins in the body. Miller’s team used ASOs to target mutations in the SOD1 gene, which can cause amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. His work, in collaboration with Don Cleveland, PhD (UC San Diego), Merit Cudkowicz, MD (Massachusetts General Hospital), and other leading scholars in the field led to the 2023 FDA approval of Tofersen. Continuing studies after FDA approval have shown that some SOD1 ALS patients are experiencing improvement in their strength and mobility, which is an unprecedented and remarkable outcome.
In addition to his research in the lab, Miller has built a world-class, comprehensive clinical care facility for ALS patients as co-director of the WashU Medicine ALS Center. From diagnostics and clinical trial access to individualized treatment plans and education for families, the ALS Center serves patients from all over the country and empowers them in their fight against a devastating disease.
Miller’s contributions extend to other neurodegenerative diseases as well. The research he previously conducted in collaboration with David M. Holtzman, MD, the Barbara Burton & Reuben M. Morriss III Distinguished Professor and director of the Knight Alzheimer Disease Research Center at WashU Medicine, led to clinical trials in early Alzheimer’s disease, where a tau-targeting ASO was shown to lower tau protein levels in cerebrospinal fluid and reverse the aggregation of tau tangles, which are responsible for the cognitive decline experienced by Alzheimer’s patients. Miller’s lab has continued to pursue new avenues for ASO applications, focusing on the precise genetic manipulation of proteins and cells to target specific disease mechanisms.
Miller has received several of the most prestigious awards for neurodegeneration research, including the American Academy of Neurology’s Sheila Essey Award for ALS Research, the Healey Center International Prize for Innovation in ALS, the Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research and the Potamkin Prize for Research in Pick’s, Alzheimer’s, and Related Diseases. He was elected to the National Academy of Medicine in 2023 and the American Association of Physicians in 2026.
In receiving the Cori Award, Miller joins an illustrious roster of WashU Medicine faculty, including three current and former Neurology faculty:
- John C. Morris, MD (2010)
- David M. Holtzman, MD (2015)
- Christina A. Gurnett, MD, PhD (2024)
“I am deeply honored to receive this award and extremely grateful to the many talented people who have helped conduct this work and to the many research participants for their dedication,” Miller said. “I also want to recognize the incredible research environment at Washington University that has enabled our studies.”
Miller will be honored with other faculty achievement awardees during the Founders Day celebrations on October 24.