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.
QurAlis, a biotech company focused on developing targeted therapeutics for ALS and a recipient of a $250,000 funding grant from The ALS Association in 2019, recently announced that it had raised $42 million in a Series A venture capital financing round to advance its research into ALS treatments. The private investment round brings the total amount raised by QurAlis to $50.5 million.
The ALS Association is excited to announce $2.5 million in grants to help develop promising new treatments for people living with ALS.
The grants are supported by the Association’s Lawrence and Isabel Barnett Drug Development Program, which supports drug discovery research in both academia and industry to develop new drug therapies and test them in a preclinical setting while moving those therapies closer to clinical use.
The ALS Association is excited to announce $1.4 million in grants to help develop technology that will enhance the quality of life for people living with ALS. The funding supports research in fields ranging from assistive communications and noninvasive ventilation to wearable sensors and a brain-computer interface.
Target ALS and The ALS Association recently announced a new partnership committed to the discovery of biomarkers for ALS. Structured as a precompetitive initiative between scientists in academia and the pharmaceutical/biotech industry, Target ALS and The ALS Association will fund two projects to better understand the chemical and physical structure of the TDP-43 protein, which is present in most cases of ALS.
We are getting enthusiastic feedback and questions about our partnership with Project ALS to fund a clinical research project for jacifusen, an experimental therapy being developed at Columbia University’s Eleanor and Lou Gehrig ALS Center for FUS-associated ALS. We thought it would be helpful to describe the strategy underlying the project.
Joel Shamaskin spent 30 years caring for others as a primary care physician before his ALS diagnosis rushed him into retirement. Over the course of his career he had cared for some patients who had the disease, so by the time he received confirmation of his diagnosis, he and his wife Ann, who was a primary care physician as well, felt like they already knew. But ALS hasn’t taken away his desire to care for others.
The ALS Association and Project ALS announced a joint two-year, $900,000 commitment to pilot a clinical research program for an investigational gene therapy for mutant FUS-associated ALS, at Columbia University’s Eleanor and Lou Gehrig ALS Center. This is the first step in developing a comprehensive strategy to treat multiple rare ALS genes.
The tallies are in for The ALS Association’s research program for the 2020 fiscal year, showing a total commitment of $21.5 million dollars in 70 promising research initiatives around the world.
This week, Time reported that flu patterns have been unusual this season, which means it is a good time to talk about ALS and the flu. People with ALS often experience a wide variety of challenges related to weakened swallowing and respiratory muscles, including difficulty managing saliva, coughing, and clearing secretions. Consequently, it’s imperative to take steps to avoid any respiratory infections, especially the flu.
This week The ALS Association launched a major initiative, investing $3 million ($1 million per year over three years) in the first ever ALS Platform Trial. The trial will take place at the Sean M. Healey & AMG Center for ALS at Mass General in collaboration with the Barrow Neurological Institute and the Northeast ALS Consortium (NEALS).
The ALS Association, our partner ALS organizations, and the wider ALS community are all hopeful that several therapies currently in Phase 3 clinical trials will prove to be successful in slowing, halting, or reversing ALS. One of the therapies now in Phase 3 trials, NurOwn, has been the subject of considerable discussion on social media. Some of what is being stated on social media is unfortunately not accurate, and risks misleading people with ALS and their caregivers.
A global, centralized, cloud-based repository of ALS genetic data is being assembled with funding from The ALS Association. This is the first such repository of its kind. Much of the research aimed at finding a cure for ALS is in the area of genetics. Such work requires genomic sequencing, a process whereby individuals' genes are mapped in a way that can be compared to the genes of others who do not have ALS in an effort to identify variations that may be factors in the development or progression of the disease.
