ALS

Phase 1 Clinical Trial to Test BIIB067 in People with ALS Currently Recruiting Participants – Muscular Dystrophy Association

In this article, the MDA announces the initiation of 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.

Medical Research Roundup – St. Louis Post-Dispatch

The St. Louis Post-Dispatch recently recognized Dr. Tim Miller’s newly awarded $2.3 million research grant from the National Institutes of Health. “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 – ALSnewstoday.com

This news feature describes a new publication by Dr. Miller and other colleagues. Their research indicates that 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 – Futurity.org

An article from futurity.org 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. “These results let us move forward in the development of this treatment and also suggest that it’s time to think about applying this same approach to other mutated genes that cause central nervous system disorders,” says lead author Timothy Miller, assistant professor of neurology at Washington University in St. Louis.

Breakthrough ALS Research at Washington University – Fox2Now St. Louis

Dr. Miller is featured in this news article and video about his Phase I clinical trial of antisense oligonucleotides targeting the SOD1 protein in ALS patients with SOD1 mutations. “We can do go in a technique called antisense oligo nucleotides and  take that small mistake in those RNA instructions and like a small eraser and go in an erase that mistake and fix it.” said Dr. Timothy Miller.