Repeat injections of mesenchymal stem cells (MSCs) into the spinal canal were safe and well tolerated in patients with amyotrophic lateral sclerosis (ALS), and showed potential to significantly slow the rate of disease progression, a small Phase 2 clinical trial found. “Larger studies are warranted to confirm our observations…
News
Brainstorm Cell Therapeutics‘ investigational cell-based therapy, NurOwn, might be able to slow disease progression and be of “meaningful” benefit to people with less severe amyotrophic lateral sclerosis (ALS), according to new analyses of Phase 3 trial data. The company announced last year that its Phase 3 trial…
A combined gene therapy that delivered two nerve growth factors — NRG1-I and NRG1-III — to muscle and nerve cells improved motor function and delayed disease onset in a mouse model of amyotrophic lateral sclerosis (ALS), a study demonstrated. However, the combo therapy did not show a synergistic effect,…
The U.S. Food and Drug Administration (FDA) has cleared AB Science to resume patient enrollment in its confirmatory Phase 3 trial investigating masitinib as an add-on treatment in people with amyotrophic lateral sclerosis (ALS). AB Science put a temporary hold on all its masitinib clinical trials due…
The loss of chemical modifications in the regulatory sequence of the TARDBP gene, called DNA methylation, seems to contribute to increased levels and clumping of the TDP-43 protein in the motor cortex of people with amyotrophic lateral sclerosis (ALS), a study found. As methylation in this gene — which…
A Phase 2 clinical trial evaluating AL-S Pharma AG’s experimental therapy AP-101 in people with amyotrophic lateral sclerosis (ALS) has enrolled its first patient. The study (NCT05039099) already is recruiting up to 63 adults with sporadic and SOD1-related familial ALS at one of its Canadian sites.
An abnormal version of tau, a protein associated with Alzheimer’s disease, may contribute to the progression of amyotrophic lateral sclerosis (ALS) by damaging mitochondria in neurons, according to a new study. The findings suggest that decreasing tau levels might be a new therapeutic strategy to lessen mitochondrial dysfunction 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…
Prilenia Therapeutics has raised $43 million to support potential regulatory submissions and marketing of its lead candidate, pridopidine, for the treatment of amyotrophic lateral sclerosis (ALS). The funding, obtained through a Series B financing round, also will be used to support the potential marketing of the candidate therapy for…
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 …
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