Category: ALS Center News
FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene (Links to an external site)
Investigational drug for genetic form of ALS improves disease’s molecular signs (Links to an external site)
The trial, sponsored by the pharmaceutical company Biogen, showed that an investigational drug, known as tofersen, reduced molecular signs of the fatal disease, but at six months did not improve motor control and muscle strength. However, Miller and colleagues found evidence that longer-term use of the drug may help stabilize muscle strength and control.
New 12-Month Tofersen Data Presented by Dr. Miller at ENCALS Meeting (Links to an external site)
Dr. Miller recently presented updated data for the Phase 3 Tofersen trial at the ENCALS Meeting in Edinburgh, Scotland. Slides from the presentation can be viewed here: https://investors.biogen.com/static-files/4e9393b8-0f67-47a3-a331-05ef3ac7fc63
Amylyx Pharmaceuticals Announces Outcome of FDA Advisory Committee Meeting on AMX0035 for the Treatment of ALS (Links to an external site)
Biogen and Ionis Announce Topline Phase 1 Study Results of Investigational Drug in C9orf72 Amyotrophic Lateral Sclerosis (Links to an external site)
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 News Release; […]
Dr. Timothy Miller’s Featured Physicians Interview at Washington University (Links to an external site)
Check out Dr. Timothy Miller’s Featured Physicians interview, where WashU offers a more in-depth look at the people behind the white coats. Timothy Miller, MD, PhD – Washington University Physicians (wustl.edu)
Parsing Science Podcast: Silencing an ALS Gene
How could a gene that causes one type of ALS be switched off? In episode 87, Tim Miller from the Washington University in St. Louis discusses his research into therapies that target the single strands of DNA or RNA which cause many cases of amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s disease. His article “Phase 1–2 […]
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. […]
Tofersen demonstrates positive results in Phase I/II trial for amyotrophic lateral sclerosis (Links to an external site)
Biogen (MA, USA) has announced positive results from a Phase I/II study of tofersen – an antisense oligonucleotide – for the potential treatment of SOD1 amyotrophic lateral sclerosis (ALS). These results have been published in The New England Journal of Medicine.
Experimental drug shows promising results for rare form of ALS (Links to an external site)
Researchers led by a Washington University neurologist found promising results for an experimental drug to treat a rare, inherited form of amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease.
Research in St. Louis leads to promise in the fight against ALS (Links to an external site)
ST. LOUIS – Research taking place at Washington University’s School of Medicine is giving hope to those affected by amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
Experimental drug shows early promise against inherited form of ALS, trial indicates (Links to an external site)
Phase 1/phase 2 trial shows evidence for safety, biological activity of tofersen; phase 3 trial underway
Update #2: Impact of COVID-19 on ALS Research
NEALS Educational Webinar. As the COVID-19 situation continues to evolve, we know it is of particular concern for the ALS community, both for clinical care and research trials. To help address some of your questions and concerns, representatives from the NEALS leadership team will be hosting a second webinar on the impact of COVID-19 on ALS clinical research.
Impact of COVID-19 on ALS Clinical Research
NEALS Educational Webinar. As the COVID-19 situation continues to evolve, we know it is of particular concern for the ALS community, both for clinical care and research trials.
Healey ALS Platform Trial receives “May Proceed” notice for three drugs (Links to an external site)
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 (Links to an external site)
Recognized for developing experimental treatment for ALS
Prospective natural history study of C9orf72 ALS 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.
Healey Center International Prize for Innovation in ALS Award (Links to an external site)
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.
Challenge Me to Find the Path to a Faster Diagnosis of ALS
With funds raised through the ALS Ice Bucket Challenge, The ALS Association has been able to invest significantly in the identification of biological indicators (or biomarkers) for ALS.
Antisense Drugs for Huntington’s, ALS and Prion Diseases Could Meet the Dire Need for Brain Treatments (Links to an external site)
A genetic therapy that increases or lowers levels of a protein raises hopes for a treatment for neurological disorders.
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).
Experimental treatment could be ‘game-changing’ for genetic ALS, experts say (Links to an external site)
(CNN) An experimental treatment for the rapidly progressive disease ALS, or amyotrophic lateral sclerosis, has been called potentially “game-changing.”
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.
Medical Research Roundup: Dr. Tim Miller Awarded Research Grant from NIH (Links to an external site)
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.
Dr. Timothy Miller and Team Win the TDP-43 Biomarker Grand Challenge (Links to an external site)
The team is developing a unique imaging biomarker to track TDP-43, a protein found in almost all cases of ALS.
Phase 1 Clinical Trial to Test BIIB067 in People with ALS Currently Recruiting Participants
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.
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.”
ALS Trial Shows Novel Therapy is Safe (Links to an external site)
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 (Links to an external site)
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.