Study links abnormal RNA modifications to TDP-43 function

TDP-43, a protein that is known to have toxic effects in amyotrophic lateral sclerosis (ALS), is able to interact with RNA molecules carrying a chemical modification called m6A, and this chemical modification is increased in ALS nerve cells, a new study shows. The findings add to a growing body…

NeuroSense Therapeutics has joined researchers at Massachusetts General Hospital to study the impact of its investigational therapy PrimeC on key amyotrophic lateral sclerosis (ALS) processes. The collaboration will the headed by Ghazaleh Sadri-Vakili, director of the Neuroepigenetics laboratory at the MassGeneral Institute for Neurodegenerative Disease. Using a new…

The ALS Society of Canada (ALS Canada) and Brain Canada are collaboratively investing CA$1.4 million (about $1.1M US) in nine grants for studies ranging from the development of new treatments for amyotrophic lateral sclerosis (ALS) to finding ways to predict disease progression. The ALS Canada Discovery…

Cytokinetics announced that it is stopping COURAGE-ALS, a Phase 3 trial investigating oral reldesemtiv in treating people with amyotrophic lateral sclerosis (ALS), due to a lack of efficacy compared with a placebo. In a review of unblinded interim trial findings at a second planned data monitoring committee meeting,…

A Phase 3b study evaluating the long-term safety and effectiveness of two dosing regimens of Radicava ORS (edaravone) — an approved oral formulation of the medicine — in people with amyotrophic lateral sclerosis (ALS) is now fully enrolled. The study was designed to fulfill a post-marketing commitment that…

The other day my husband, Todd, told me he forgot to ask the caregiver to spray out his clicker tube for his HeadMouse. Because he’s paralyzed from ALS, he clicks his computer mouse by sipping and puffing on a straw, and we only need to replace it once…

The U.S. Food and Drug Administration (FDA) will hold an advisory committee meeting to discuss BrainStorm Cell Therapeutics‘ application to approve NurOwn for amyotrophic lateral sclerosis (ALS). Such a meeting, called an ADCOM, is normally for when a therapy is under regulatory review so experts can discuss its…

Difficulties in measuring the progression of amyotrophic lateral sclerosis (ALS) among patients with advanced disease can cause complications in clinical trials, according to a new analysis that highlights how such problems may have affected the failed NurOwn study. The work was led by scientists at BrainStorm…

In most cases of amyotrophic lateral sclerosis (ALS), dysfunction of a protein known as TDP-43 leads to abnormally low levels of another protein, stathmin-2, which is sufficient to drive nerve cell dysfunction, new data suggest. In an aim to restore stathmin-2 levels, researchers created a designer DNA treatment. Now,…

“So tell us, Dagmar, what’s your latest project?” The question came from my neurologist as we chatted during my recent ALS clinic visit. She asked not only for herself, but for the medical student who was also in the room. As I prattled on, part of my brain reflected…