Tofersen Phase 3 Drug Results and Use with ALS

At the American Neurological Association (ANA) Annual Meeting, Dr. Timothy Miller presented VALOR study of Tofersen Phase 3 Drug Results and Use with ALS. Tofersen is an antisense drug being evaluated for the potential treatment of SOD1-ALS. For more information and to learn about Tofersen Phase 3 Drug Results please see links below. Biogen Announces Topline […]

Promising Results for Investigational Treatments for Inherited ALS (Links to an external site)

Two preliminary studies were published simultaneously in the New England Journal of Medicine, both aimed at reducing superoxide dismutase 1 (SOD1) protein levels in people who had amyotrophic lateral sclerosis (ALS) due to SOD1 mutations. In the first study, a phase I/II trial of intrathecal tofersen, Timothy Miller, MD, PhD, of Washington University in St. […]

Prospective natural history study of C9orf72ALS clinical characteristics and biomarkers

Prospective natural history study of C9orf72ALS clinical characteristics and biomarkers Our team’s hard work has been published in Neurology. This paper profiles the clinical features, such as age at disease onset, survival duration, and measures of disease progression, of ALS patients with mutations in the C9orf72 gene. By defining the natural history of this patient […]

New Diagnostic Test for Neurofilament

Biomarkers are measures reflective of biological processes that occur in the body. In the setting of disease, biomarkers may be used for diagnostic, prognostic or treatment monitoring purposes.

Experimental Drug Shows Promise for Genetic Form of ALS (Links to an external site)

An early stage trial of an investigational therapy for amyotrophic lateral sclerosis (ALS) suggests that people could tolerate the experimental drug and, in exploratory results, the experimental drug was linked to possible slower progression in people with a genetic form of the disease caused by mutations in a gene called superoxide dismutase 1 (SOD1).

Molecule Throws Roadblock in Alzheimer’s Path (Links to an external site)

An article by New Atlas describing Dr. Miller’s STM work states, “When it comes to the drama of Alzheimer’s disease, the compound in the starring role as the villain is tau. It’s a protein that normally helps our neurons function properly, but one that can clump together into plaques that damage brain cells. Now researchers have […]

Antisense Oligos Tango with Tau Transcripts to Reverse Tauopathy (Links to an external site)

In an article about Dr. Miller’s newest Science Translational Medicine publication, ALZFORUM writes, “In collaboration with the pharmaceutical company Ionis in Carlsbad, California, Timothy Miller and his colleagues at Washington University in St. Louis designed a potent ASO that shuts down human tau gene expression in transgenic mouse models.”

Publication by Mariah L. Hoye, PhD Candidate

Congratulations to Miller Lab member Mariah L. Hoye on her publication! Mariah is a PhD candidate who is studying microRNAs, which are small compounds that regulate a variety of processes in the body, and their role in ALS disease progression. Her manuscript that investigates microRNAs specific to motor neurons in ALS rodent models was recently […]

Publication by Kathleen Schoch, PhD

Congratulations to Miller Lab member Kathleen Schoch on her publication! Dr. Kathleen Schoch is a postdoctoral research assistant in the Miller Lab who focuses on dementias characterized by the pathological accumulation of the protein “tau”. She published a paper in Neuron that examined the potential of a unique tau-lowering therapeutic strategy to treat Alzheimer’s disease, […]

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models. 2018 (Links to an external site)

Mutations in superoxide dismutase 1 (SOD1) are responsible for 20% of familial ALS. Given the gain of toxic function in this dominantly inherited disease, lowering SOD1 mRNA and protein is predicted to provide therapeutic benefit. We have developed next-generation SOD1 ASOs that more potently reduce SOD1 mRNA and protein and extend survival in SOD1G93A mice. […]

Dr. Timothy Miller Wins Sheila Essey Award for ALS Research (Links to an external site)

The ALS Association, in partnership with the AAN and the American Brain Foundation, are awarding research funding to Timothy M. Miller, M.D., Ph.D., the David Clayson Professor of Neurology from the Washington University School of Medicine in St. Louis. The award recognizes significant research contributions in the search for the causes, prevention, and cure for amyotrophic lateral sclerosis (ALS). Since 1996, The ALS Association and the American Academy of Neurology have jointly chosen recipients of the award.

Ste. Genevieve Riverdogs Baseball Team Supports ALS Research

Prior to the final game of the season, the Ste. Genevieve Riverdogs baseball team surprised Coach Jeremy Hoog with a touching tribute as well as a donation to Dr. Timothy Miller’s ALS Research Lab in honor of Coach Hoog’s mother – and the Riverdogs’ biggest fan – Cheryl.

Prior to the final game of the season, the Ste. Genevieve Riverdogs baseball team surprised Coach Jeremy Hoog with a touching tribute as well as a donation to Dr. Timothy Miller’s ALS Research Lab in honor of Coach Hoog’s mother – and the Riverdogs’ biggest fan – Cheryl.

MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models

Hoye ML, Koval ED, Wegener AJ, Hyman TS, Yang C, O’Brien DR, Miller RL, Cole T, Schoch KM, Shen T, Kunikata T, Richard JP, Gutmann DH, Maragakis NJ, Kordasiewicz HB, Dougherty JD, Miller TM. MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models. J Neurosci. 2017 May 31;37(22):5574-5586. doi: 10.1523/JNEUROSCI.3582-16.2017. Epub 2017 Apr 17. PMID: 28416596; PMCID: PMC5452343.

Genetic Mutations Linked to Higher Proportion of ALS Cases Than Previously Believed (Links to an external site)

New research indicating genetic mutations may underlie more ALS cases than scientists originally thought. Dr. Miller’s close colleague Dr. Matthew Harms states, “To our surprise, we found that 26 percent of sporadic ALS patients had potential mutations in one of the known ALS genes we analyzed. This suggests that mutations may be contributing to significantly more ALS cases.”

Video: 2011 MDA Telethon ALS Research Update

MDA Telethon ALS Research Update

MDA presents an update regarding research on ALS, featuring Dr. Timothy M. Miller from Washington University in St. Louis. MDA is supporting a clinical trial of antisense therapy seeking to counteract the affects of a toxic version of the protein SOD1 in patients with an inherited form of ALS.