By Jill Yersak, Ph.D.
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!
All month, we are featuring stories of individuals who are having an impact in the fight against ALS.
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.
The ALS Association has supported Dr. Gendron’s work as part of the TREAT ALSTM global research program with the hope to accelerate ALS biomarker progress. We are happy to report that she and her colleagues have made significant advances and recently published a paper in Annals of Neurology titled, “Phosphorylated neurofilament heavy chain: A biomarker of survival for C9orf72-associated amyotrophic lateral sclerosis.” Read more to learn how Tania contributes to ALS research by chipping away at the barriers between getting a drug from the lab and into clinical trials in her fight against ALS.
Continue reading Every Scientist: Meet Dr. Tania Gendron
Last week, 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.” For many years, The ALS Association has proudly partnered with NEALS to run ALS centered webinars to disseminate the most up-to-date information to the ALS community. Today’s post discusses the actively enrolling Retigabine clinical trial, which The ALS Association funds, as well as an honest perspective on what it is like to participate in ALS clinical trials.
Continue reading A Patient Advocate and Doctor’s Perspective on Clinical Trials: Update on the Retigabine Phase II Trial
Amylyx Pharmaceuticals, championed by The ALS Association, is currently enrolling up to 132 individuals nationwide for a Phase II CENTAUR (Combination of Phenylbutyrate and Tauroursodeoxycholic Acid) clinical trial to assess the efficacy and safety of AMX0035 for the treatment of ALS. The Neurology Clinical Research Institute (NCRI) at Massachusetts General Hospital and the Northeast ALS Consortium (NEALS) will oversee the trial, and 25 NEALS member medical centers across the United States will participate. The ALS Association, along with ALS Finding a Cure® supported this important trial with a $2.96 million grant.
Continue reading Clinical Trial: Amylyx Pharmaceuticals is Now Enrolling in Phase II CENTAUR Clinical Trial
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. The article is far-reaching in that it covers topics from ALS epidemiology, genetics, pathology, identified disease pathways and potential ALS therapies. Familial (inherited) ALS and sporadic (not inherited) ALS are covered.
Continue reading New Comprehensive ALS Review Published
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. To give a full picture of the company’s past progress and future endeavors, the webinar was led by leadership at Brainstorm Cell Therapeutics, including President and CEO Chaim Lebovits and Chief Operating and Medical Officer Dr. Ralph Kern. Clinicians involved in the upcoming phase III trial also spoke, including Drs. Robert Brown, Anthony Windebank and Merit Cudkowicz. The Association has partnered with NEALS for over eight years and we are happy to collaborate to disseminate the most accurate, up-to-date information regarding ALS clinical trials.
Continue reading Update from Brainstorm Cell Therapeutics on Upcoming Phase III NurOwn® Clinical Trial
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. This work was published in the July issue of the Journal of Neuroinflammation. The ALS Association has proudly supported Dr. Jara since 2010 through both our Milton Safenowitz Postdoctoral Fellowship Program and a recent Investigator-Initiated Grant. We are happy to report on his achievements! Read more to learn about this exciting study and get to know Dr. Jara inside and outside the lab.