Research funded by The ALS Association found that defects in the mechanism that transports proteins and RNA between the nucleus and cytoplasm of cells within the human body may lead to ALS and that regulation of those systems could present a novel therapeutic strategy for ALS.
Details of the study are provided in a new paper entitled, “Modulation of actin polymerization affects nucleocytoplasmic transport in multiple forms of amyotrophic lateral sclerosis,” which was released August 23, 2019, in the journal Nature Communications.
Continue reading A New Study Supported by The ALS Association May Provide a Novel Therapeutic Strategy for ALS
The ALS Association is inviting researchers to submit proposals for its Managing ALS Research Program and The 2019 Lawrence and Isabel Barnett Drug Development Program. Applicants are encouraged to apply no later than October 7, 2019.
Continue reading The ALS Association Announces Two New Research Funding Opportunities
The ALS Association is deeply committed to accelerating the development of novel ALS therapies and ensuring they are accessible to those who need them. The Association is announcing today its latest strategic action with the release of Principles for Urgent, Patient-Centered ALS Clinical Trials: a series of recommendations to clinical trial sponsors and regulatory bodies worldwide.
The Principles set out a clear framework for the design and conduct of ALS clinical trials that are efficient, impactful, and respectful. We view them as essential to both maximize the participation of people with ALS and their caregivers and to ensure every available tool is used to streamline drug development and regulatory-review processes. Regardless of the detailed specifics that may arise from FDA guidelines in the next few weeks, we hope these principles can help focus trial sponsors and regulators on the most important aspects of trial design and lead to ever-improving clinical trials as our fight against ALS advances.
Continue reading Principles for Urgent, Patient-Centered ALS Clinical Trials Latest in Ongoing Effort to Improve Trial Design
By Neil Thakur, Ph.D
Executive Vice President, Mission Strategy
The ALS Association
Diagnosing ALS is a lengthy and frustrating process for clinicians and families alike. This is due in large part to the absence of a specific biological indicator, which would signal the presence of the disease in people experiencing the onset of ALS symptoms. Unlike cancer and diabetes, which can be diagnosed and monitored through laboratory tests, ALS has no unique biological markers to confirm an ALS diagnosis.
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. You may already be familiar with biomarkers in other diseases, like blood sugar for diabetes or cholesterol for heart disease. Biomarkers can help us diagnose ALS more quickly and monitor disease progression precisely. With biomarkers, we can run faster and smaller clinical trials, because they can help us select participants more precisely and measure clinical changes more accurately.
One example of promising biomarker research being done today is happening at Dr. Tim Miller’s lab at the Washington University School of Medicine in St. Louis. With funding generated in part through the ALS Ice Bucket Challenge, Dr. Miller and his team are looking to develop a unique imaging biomarker to track TDP-43, a protein associated with almost all cases of ALS. I’d like to introduce you to Dr. Miller, who can tell you, in his own words, about the push to find biomarkers that can help people with ALS find effective treatments sooner.
Continue reading Challenge Me to Find the Path to a Faster Diagnosis of ALS
To help provide more information about our research program, our spending decisions, our approach to certain experimental therapies, and other matters, we have put together the below Frequently Asked Questions.
Continue reading Frequently Asked Questions (FAQ) about Our Research Program, Experimental Therapies, and More
AB Science has completed a Phase 2/3 human clinical trial of masitinib in ALS with promising results. The company published the results of its trial, conducted in Spain, in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. A confirmatory Phase 3 study will be launched later in 2019 with all locations yet to be announced.
Continue reading AB Science Publishes the Positive Phase 2/3 Clinical Trial of Masitinib in ALS
The ALS Association, ALS Finding a Cure (ALSFAC), and MDA announced they have jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. The principal investigator is Stanley Appel, MD, co-director of Houston Methodist Neurological Institute, chair of the Stanley H. Appel Department of Neurology and the Peggy and Gary Edwards Distinguished Chair in ALS at Houston Methodist Hospital, and professor of Neurology at Weill Cornell Medical College. Dr. Appel is an internationally renowned researcher and neurologist and is one of the country’s foremost experts on amyotrophic lateral sclerosis (ALS), a progressive neuromuscular disease that destroys muscle-controlling nerve cells called motor neurons, eventually causing muscles to become nonfunctional.
Continue reading The ALS Association, ALS Finding A Cure, and MDA Co-Fund Clinical Trial Grant to Study Restoring Immune System Function to Treat ALS