Researchers funded through The ALS Association’s Lawrence and Isabel Barnett Drug Development Program, uncovered a new way to prevent muscle paralysis in an ALS mouse model. Dr. Steven Burden at New York University Medical School and colleagues at Columbia University Medical School used a stimulatory antibody to increase the activity of MuSK, a protein critical for maintaining the connections between muscle and motor neurons.
This treatment preserved nerve-muscle synapses, improved diaphragm muscle function, and modestly extended lifespan of the mice. Their findings reveal a novel therapeutic strategy using an antibody to potentially target ALS and other neuromuscular diseases.
At the recent Leadership Conference for The ALS Association, Joshua Cohen and Justin Klee, CEO and president, respectively, and founders of Amylyx Pharmaceuticals, updated us on their ongoing CENTAUR phase II clinical trial. The Association is supporting their trial through ALS Ice Bucket Challenge donations, with a $2.96 million grant in partnership with ALS Finding a Cure.
“The ALS Association’s contribution has made a substantial difference in accelerating our clinical study, which, hopefully will result in new insights and a new therapeutic for ALS,” said Cohen. Klee added, “We are enormously grateful for their continuing support and for the support of everybody in the ALS community.”
For this Throwback Thursday, we’re taking it back to February 10, 2017. The article was titled, “Nuedexta Trial Demonstrates Promising Results Impacting Bulbar Function in ALS Patients.”
In the January 9, 2017, issue of Neurotherapeutics, Dr. Richard Smith, Director of the Center for Neurologic Study in La Jolla, Calif. published promising results of a phase II trial testing the effect of Nuedexta on bulbar function. Overall, he and his co-authors found that Nuedexta had a significant palliative effect on speech, swallowing, and salivation in people living with ALS. The ALS Association contributed to the funding of this trial.
Research funded by The ALS Association using Ice Bucket Challenge donations recently led to a significant discovery in understanding a disease pathway behind the most frequent cause of inherited ALS and frontotemporal dementia (FTD).
Top ALS researchers published three high-impact papers in Nature Communications, delving into the underlying mechanism of how the C9orf72 gene expansion is translated into toxic proteins. Information uncovered in these papers inform therapeutic development of drugs targeting the C9 expansion, which have the potential to make an impact on the largest population of people living with familial, or inherited, ALS.
This morning, President Trump signed the Bipartisan Budget Act of 2018 (H.R. 1892), which included the Steve Gleason Enduring Voices Act. The Act will permanently fix the current Centers for Medicare and Medicaid Services (CMS) policy limiting access to Speech Generating Devices (SGD) for people with degenerative diseases.
Read our article from earlier this week for more details.
In honor of Throwback Thursday, we’re taking it back to February 9, 2017, to an article titled, “Duke, UNC Coaches Unite to Raise Awareness of ALS.”
The men’s basketball coaching staffs of Duke University and the University of North Carolina (UNC) wore The ALS Association lapel pins on February 9, 2017, in honor of Jeff Capel, Jr., who had recently been diagnosed with ALS. Capel was the head coach at Old Dominion University (ODU) for seven seasons and also served as an assistant for the Charlotte Bobcats and the Philadelphia 76ers.
FORTITUDE-ALS is a clinical trial of an investigational oral drug for the treatment of amyotrophic lateral sclerosis (ALS). This clinical trial is now enrolling participants in both the United States and Canada.
FORTITUDE-ALS is designed to evaluate whether CK-2127107 has an effect on participants with amyotrophic lateral sclerosis (ALS) and what kind of side effects may occur in these participants taking CK-2127107.
CK-2127107 is an investigational medicine that is being evaluated as a potential new medicine which could slow the decline of skeletal muscle function in diseases and conditions associated with muscle weakness or fatigue such as ALS.