Aquinnah Pharmaceuticals has partnered with Roche to further develop its brain-penetrating, oral small molecules for the treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. These small molecules are designed to eliminate the persistent stress granules that contribute to the buildup of toxic protein clumps in…

A team of scientists in the U.K. and Japan has determined the structure of aggregated TDP-43, the protein whose abnormal clumps are characteristic of amyotrophic lateral sclerosis (ALS). Their work, reportedly the first to reveal the molecular structure of aggregated TDP-43, identified a “double-spiral fold” of the protein in patients’ brain…

AcuraStem’s experimental therapy AS-202 significantly reduced neurodegeneration and improved motor function and survival in a mouse model of aggressive amyotrophic lateral sclerosis (ALS) associated with toxic clumps of the TDP-43 protein, the company announced. Preclinical studies also confirmed the therapy’s biological activity and target engagement and suggested that…

An Italian scientist was awarded $20,000 for research that may help pave the way for a TDP-43 antibody-based treatment for amyotrophic lateral sclerosis (ALS). The annual Paulo Gontijo Award from the Brazilian Paulo Gontijo Institute was granted to Silvia Tozzi, PhD, for her research, titled “Monoclonal full-length antibody…

An investigational antisense oligonucleotide (ASO) molecule that works by increasing the production of Stathmin-2 protein effectively reversed a number of neurodegenerative processes in lab-grown motor neurons, the cells that are damaged in amyotrophic lateral sclerosis (ALS), a study found. The rescue occurred even in the absence of TDP-43, a…

Amydis announced plans to launch a first clinical trial next year into the ability of its small molecule tracer to detect toxic TDP-43 clumps — a hallmark of amyotrophic lateral sclerosis (ALS) — in the eye’s retina. Its announcement follows a preliminary meeting with U.S. Food and Drug Administration…

While it’s long been believed that the death of motor neurons in the spinal cord is the main cause of amyotrophic lateral sclerosis (ALS), new research suggests that degeneration of motor neurons in the brain takes place independently and may also contribute to the disease. The study’s work in…

BioArctic AB announced that it is advancing work into selective antibodies against TAR DNA-binding protein 43 (TDP-43) that when misfolded can form the toxic clumps, or aggregates, believed to be involved in the development of amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. One potential therapy approach is called…

Recent findings and ongoing efforts to more fully understand the underlying molecular mechanisms of amyotrophic lateral sclerosis (ALS), and to identify new and better ways of helping patients were discussed at the 11th Annual Les Turner Symposium on ALS. The daylong event, held virtually Nov. 1, was sponsored by the …

AC Immune has extended its research collaboration with scientists at the University of Pennsylvania (UPenn) working to uncover how TDP-43 protein clumps contribute to different disease manifestations in amyotrophic lateral sclerosis (ALS). In the partnership’s first two years, researchers with the Perelman School of Medicine at UPenn received…