Timothy Miller, MD, PhD

During my PhD studies, I focused on understanding the molecular details of nerve cell degeneration. I continued this interest clinically, with residency training in neurology, which propelled a research track focused on developing a novel antisense oligonucleotide (ASO) therapeutic for amyotrophic lateral sclerosis (ALS). This research launched a clinical trial in ALS, which I had the opportunity to lead. At Washington University, my clinical experience, combined with my research efforts in ASO therapeutics, allowed me to broaden my research focus to include studies of proteins related to dementia. Dr. Kathleen Schoch joins me in this endeavor and our earlier work using ASOs to lower levels of tau was supported by a Paul B. Beeson award, with Drs. David Holtzman and Alison Goate as mentors. With their support and our continued research, the tau-targeted ASOs are now in early clinical trial for Alzheimer’s disease. I am encouraged by the success ASOs have shown in the treatment of neurodegenerative disease and am enthusiastic about our current study supported by BrightFocus to investigate antisense-mediated lowering of TREM2 to better understand and treat Alzheimer’s disease.