The clinical trial to test RNS60, a new compound for the treatment of ALS, which was supported in part from a $1.0 million grant through the ALS ACT initiative funded by The ALS Association and ALS Finding A Cure®, is now actively enrolling. This randomized placebo-controlled phase II study is being run by the IRCCS Mario Negri Institute for Pharmacological Research in Milan and the ALS Center of the Maggiore University Hospital in Novara Italy. We are happy to report that the first patient was dosed in mid-July 2017.
It’s been three years since the viral fundraising phenomenon known as the ALS Ice Bucket Challenge, which allowed us to dedicate millions of dollars to our global TREAT ALS™ research program.
Because research takes time, we are now starting to see results of our investments. This is a very exciting time in ALS research!
Meet Dr. Tania Gendron, Assistant Professor of Neuroscience at the Mayo Clinic in Jacksonville, Fla. She fights ALS by working hard every day in the lab to find a cure for this devastating disease. Her work focuses on optimizing ALS biomarkers to track and better understand the most common genetic mutation in inherited ALS, called C9orf72.
Dr. Brian Wainger of Massachusetts General Hospital and Stephen Winthrop, Chairman of The ALS Association Board of Trustees, gave their unique clinical trial perspectives during the Northeast ALS Consortium (NEALS) webinar titled, “Retigabine Clinical Trial Update & Discussion with ALS Patient Advocate Stephen Winthrop.”
CENTAUR is a 24-week, randomized, double-blind, placebo-controlled Phase II clinical trial. The trial’s primary objectives are to evaluate the safety and tolerability of AMX0035 and assess the drug’s impact on disease progression as measured by the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) over the 24-week study period.
In the July 13, 2017 issue of The New England Journal of Medicine, leaders in the ALS field, Drs. Robert Brown of University of Massachusetts Medical School and Dr. Ammar Al-Chalabi of King’s College London, came together to write a comprehensive ALS review.
On July 17th, our partner Northeast ALS Consortium (NEALS) hosted a webinar, “NurOwn® Clinical Development Program,” which comprehensively explained past phase II results, the upcoming phase III study and Israeli Hospital Exemption Program.
Today, we sat down with Dr. Javier Jara, Research Assistant Professor at Northwestern University Feinberg School of Medicine, who just published groundbreaking work focused on brain inflammation caused by ALS.
Genentech, a member of the Roche group, has initiated a Phase I clinical study evaluating GDC-0134, an oral investigational medicine designed to block dual leucine zipper kinase (DLK), for the potential treatment of ALS.
The ALS Association is proud to announce three large research organizations -- Project MinE USA, Answer ALS and the New York Genome Center (NYGC) -- will be working collaboratively toward their mission for treatments and a cure for ALS.
The June issue of Scientific American on newsstands this month features, “Unlocking the Mystery of ALS,” which details the significant advances of ALS research over the years. The authors, Drs. Leonard Petrucelli at the Mayo Clinic in Jacksonville and Aaron Gitler at the Stanford University School of Medicine, thoughtfully explained the complicated science behind ALS, while weaving a story of its breakthroughs and the steps needed to get to the ultimate goal – an end to ALS.
The ALS Association is at the forefront of the global research effort to find treatments and a cure for ALS. We believe that innovation and collaboration will be the key to winning this important fight. Only by coming together with others around the world who are experts in their fields will we make significant progress. We know collaboration leads to progress.
One of the questions that people living with ALS often ask is – what can I do to help ALS research? People with ALS can do that, with a personal contribution to the National ALS Registry (Registry) – in the newly launched National ALS Biorepository (Biorepository).
But researchers are working to change that. The increased awareness and donations provided by events like the ALS Ice Bucket Challenge—and by people like you—are making a real difference in the pace of discoveries, bringing us ever closer to the end of ALS.
At the largest-ever Drug Company Working Group meeting held in Boston in April, The ALS Association featured the first details of exciting new “antisense” target that may be relevant to most people with ALS, not just those with an inherited ALS gene.
The biotechnology company, Aquinnah Pharmaceuticals is dedicated to identifying new therapeutic agents for ALS and Alzheimer’s disease, based on a new scientific approach of RNA binding proteins involved in neurodegenerative disease. Last week, Aquinnah announced a $10 million investment from two world leader pharmaceutical companies Pfizer, Inc. and AbbVie Inc. to support therapeutic development to treat ALS, Alzheimer’s disease and other neurodegenerative diseases.
This year at the 69th Annual American Academy of Neurology Meeting in Boston, thousands of neuroscientists came together to share their research and collaborate. Here we report the latest ALS research findings presented at the meeting by distinguished researchers, who were chosen to speak based on scientific merit.
Today, we are pleased to feature ALS researcher Dr. Sabrina Paganoni from Massachusetts General Hospital and Spaulding Rehab Hospital. She is this year’s recipient of the Clinician Scientist Development Award in ALS Research given in partnership with the American Academy of Neurology (AAN).
Dr. John Ravits, Professor of Clinical Neurosciences and Head of the ALS Translational Research Program at the University of California, San Diego (UCSD) is a physician-scientist at the forefront of ALS thought and research of sporadic and familial ALS. Yesterday, at the 69th Annual American Academy of Neurology (AAN) Meeting in Boston, he was presented the prestigious 2017 Sheila Essey Award by Dick Essey, founder of the award named in honor of his wife Sheila who battled with ALS for ten years and died from the disease in 2004.
