Despite the many challenges caused by COVID-19 this past year, ALS research made strides toward finding effective treatments for ALS. As 2020 winds down, we wanted to share an update from our partners at Biogen on some of their ongoing research projects.
Back in 2000, when Veronique Belzil was working as a counselor in Canada, she found a new path after watching her husband’s uncle quickly succumb to ALS.
The ALS Association and I AM ALS on Friday submitted a petition to the Food and Drug Administration calling on the agency and Amylyx Pharmaceuticals to act swiftly and with urgency to make AMX0035 available as soon as possible. The petition was signed by more than 50,000 people from across the country who have been affected by ALS.
Clinical trial participants who took AMX0035, a promising new drug therapy developed by Amylyx, showed a statistically significant 6.5 month increase in survivability compared to patients who did not receive the drug in the initial trial, according to data published in the journal Muscle and Nerve in October 2020. These findings validate calls led by The ALS Association and I AM ALS for Amylyx and the FDA to make AMX0035 available as quickly as possible.
ALS doesn’t stop and neither do we. The reality is, people living with ALS can’t wait for treatments and a cure, and just as importantly, the tireless researchers working together around the world can’t wait to make the next breakthrough.
Meet Jinsy Andrews, M.D., MSc, FAAN, Director of Neuromuscular Clinical Trials at Columbia University, and member of The ALS Association Board of Trustees. Dr. Andrews is a clinical neurologist, neuromuscular specialist, and an ALS specialist.
More than 43,000 people signed The ALS Association’s petition, launched September 3 with I Am ALS, to call on the FDA and Amylyx Pharmaceuticals to work together to speed up the process of getting AMX0035 available for people living with ALS as quickly as possible.
The ALS Association and I AM ALS have launched a petition calling on the FDA and Amylyx Pharmaceuticals to bring AMX0035, a promising new drug developed by Amylyx, to market as soon as possible.
People with ALS and their caregivers face a substantial burden accessing and understanding insurance coverage and paying for medical treatments and services, causing high stress, added work burden and debt for the ALS community.
We recently talked with Dr. Emily Thompson from the Rothstein Lab at Johns Hopkins University to learn about her unique research project focused on how the loss of a cortical astroglia subpopulation exacerbates dendritic and synaptic defects of upper motor neurons in ALS.
We recently talked with Dr. Gerbino from the Maniatis Lab at Columbia University to learn about her unique research project focused on identifying how mutations in TBK1, one of the genes associated with ALS, differentially affect the cells of the spinal cord involved in the pathogenesis of ALS.
We recently spoke with Dr. Paul McKeever from the Rogaeva lab at the Tanz Centre for Research in Neurodegenerative Diseases at the University of Toronto. Paul’s current research project is focused on uncovering the molecular programming which make individual brain cells and populations of cells susceptible or resilient to the disease process so that new therapeutic avenues can be developed for patients with ALS and FTD.
We recently talked with Dr. Lauren Laboissonniere from the Ranum lab at the University of Florida to learn about her unique research project focused on the development of novel therapeutics for the treatment of C9orf72 ALS/FTD and related repeat-associated disorders.
Biogen, a partner of The ALS Association, recently published promising results from its phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS and is now actively enrolling participants for their Phase 3 Valor study. It also announced that there is an open-label extension available in the study.
We recently talked with Dr. Zhe Zhang from the Sun Lab at the Johns Hopkins School of Medicine to learn about her unique research project focused on screening for expansion in the C9ORF72 gene, the most common genetic cause of ALS.
Research supported by The ALS Association found that blood plasma analysis could be key to speeding up the process of diagnosing the disease and monitoring disease progression. The research was led by Dr. Michael Bereman from North Carolina State University and supported by a $100,000 grant from The ALS Association, including funding from the North Carolina Chapter.
We support the A.C.T. for ALS Act (H.R. 7071) and believe it should be strengthened by helping fund ALS research and by ensuring people with ALS in clinical trials can continue receiving treatments that may be helping them. We believe these steps will help improve its chances for passage and ensure it helps even more people with ALS.
The National Institutes of Health on Wednesday announced plans to spend an additional $25 million to create a new program that will speed up ALS research and support cutting-edge approaches to understanding the disease and developing treatments. The money is scheduled to be spent over five years targeting innovative research through a program called Accelerating Leading-edge Science in ALS – or ALS2.
The ALS Association and I AM ALS have awarded a $500,000 grant to BrainStorm Cell Therapeutics, a biotechnology company, to support its ALS biomarker research study.
The lack of defined biomarkers for ALS has been a significant challenge to clinicians and researchers who are keen to identify disease risk and onset much earlier and also, to verify the effects of treatments in clinical trials. The funding partnership among The Association, I AM ALS and BrainStorm will draw insights from data and samples collected from patients enrolled in BrainStorm’s ongoing phase 3 clinical trial of its NurOwn treatment to see if the therapy is hitting its targets in the nervous system and generating measurable changes in biomarkers that would signal that the drug works.
