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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.

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).

(CNN) An experimental treatment for the rapidly progressive disease ALS, or amyotrophic lateral sclerosis, has been called potentially “game-changing.”

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.

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.

The team is developing a unique imaging biomarker to track TDP-43, a protein found in almost all cases of ALS.

MDA announces enrollment in the Phase I/II trial of SOD1 antisense oligonucleotides. Dr. Timothy Miller led the first trial of these SOD1 ASOs in ALS patients and is now the academic Principle Investigator for the Phase I/II trial, sponsored by Biogen Idec and Ionis Pharmaceuticals.

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.”

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.

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.