Healey ALS Platform Trial receives “May Proceed” notice for three drugs

The Sean M. Healey & AMG Center for ALS at Mass General has received approval from the U.S. Food and Drug Administration (FDA) to proceed with administering three proposed drug regimens in the HEALEY ALS Platform Trial – the first trial of its kind for amyotrophic lateral sclerosis (ALS).

Miller receives international innovation prize

Recognized for developing experimental treatment for ALS Timothy Miller, MD, PhD, the David Clayson Professor of Neurology at Washington University School of Medicine in St. Louis, and a group of his colleagues have received the inaugural Healey Center International Prize for innovation in amyotrophic lateral sclerosis (ALS) research from the Sean M. Healey & AMG […]

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 population, this […]

Healey Center International Prize for Innovation in ALS Award

The award goes to Timothy Miller, MD, PhD, of Washington University School of Medicine in St. Louis, Don Cleveland, PhD, of the Ludwig Institute at the University of California at San Diego, Richard Smith, MD, of the Center of Neurological Study in La Jolla, California, Toby Ferguson, MD, PhD, for Biogen and Frank Bennett, PhD, […]

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

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).

Dr. Timothy Miller Wins Sheila Essey Award for ALS Research

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.

Medical Research Roundup: Dr. Tim Miller Awarded Research Grant from NIH

The Miller Lab has found differences between healthy people and people with amyotrophic lateral sclerosis (ALS) in biomolecules known as microRNAs. This project seeks to understand the microRNA differences and the effect of adjusting them to try to develop new diagnostic tests or treatments for ALS.

Genetic Mutations Linked to Higher Proportion of ALS Cases Than Previously Believed

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.”

ALS Trial Shows Novel Therapy is Safe

Highlights the exciting new results from Dr. Miller’s first Phase I trial of SOD1-targeting ASOs, stating that the investigational treatment for inherited forms of ALS has passed an early clinical trial for safety.

Breakthrough ALS Research at Washington University

Dr. Miller featured in news article and video about his Phase I clinical trial of antisense oligonucleotides targeting the SOD1 protein in ALS patients with SOD1 mutations.