Timothy Miller, MD, PhD

Timothy Miller, MD, PhD, former NEALS Co-Chair, has led the application of an antisense oligonucleotide (ASO) based therapeutic approach for neurodegenerative diseases. He pioneered cerebrospinal fluid (CSF) delivery of these ASO in animal models and then led the first in human trial for CSF ASOs in humans in amyotrophic lateral sclerosis (ALS). Dr. Miller’s work is the foundation for RNA-targeted therapies for multiple neurodegenerative diseases including ALS.

Recognizing the importance of miRNA in neurodegeneration, the Miller Lab has recently developed miRNA inhibitors and is working on moving these to human clinical trial. In addition to developing antisense oligonucleotides for neurodegeneration, he has focused on the other important aspects of novel therapeutics by demonstrating that SOD1 CSF protein is an appropriate pharmacodynamics marker and developing ways to measure SOD1 protein half-life in humans. The next SOD1 ASO trial for ALS is underway with Dr. Miller as the academic PI in collaboration with Ionis Pharmaceuticals and Biogen.

He is the David Clayson Professor of Neurology at Washington University School of Medicine in St. Louis.