Alchemedicine Inc. has divulged aldehyde dehydrogenase 2 (ALDH2) activators reported to be useful for the treatment of pain, cancer, ischemia-reperfusion injury, nonalcoholic fatty liver disease, peripheral arterial disease, Fanconi anemia, Parkinson’s and Alzheimer’s disease, among others.
The gene for Huntington’s disease “was cloned in 1993, and everyone thought there was going to be a treatment right around the corner,” Sarah Tabrizi told the audience at the 2023 Annual Meeting of the Society for Neuroscience. Then, “it took 25 years for the first trial targeting the Huntington gene.”
The U.S. FDA granted Roche Holding AG breakthrough device designation for its Elecsys Neurofilament Light Chain test for multiple sclerosis. The Elecsys NfL test can aid in the detection of disease activity in adults, 18-55 years old, with relapsing-remitting multiple sclerosis or secondary progressive multiple sclerosis. It also provides critical information for managing the disease.
Mindset Pharma Inc. has patented new indoline derivatives acting as 5-HT1A receptor agonists and thus reported to be useful for the treatment of neurological and psychiatric disorders.
Sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers have been reported in a Vertex Pharmaceuticals Inc. patent as potentially useful for the treatment of pain, arrhythmia, Charcot-Marie-Tooth disease, cough, urinary incontinence and multiple sclerosis.
Previous research found that transplanting human induced pluripotent stem cell-derived neural stem/progenitor cells (hiPSC-NS/PCs) promoted motor functional recovery in animal models of spinal cord injury (SCI). hiPSC-NS/PC transplantation has been recently evaluated for subacute SCI in a first clinical trial. However, animal studies revealed that the effectiveness of NS/PC transplantation varies depending on the state of the injured spinal microenvironment, with reduced therapeutic effects in severe models. Hepatocyte growth factor (HGF) is a potent growth factor that promotes tissue regeneration through the MET receptor and constitutes an interesting candidate to enhance the efficacy of NS/PC transplantation.
Researchers from the University of Michigan and affiliated organizations presented data from a study that identified a link between a new neurodevelopmental disorder and biallelic variants in the EPB41L3 gene, which encodes band 4.1-like protein 3 that plays important roles in neuronal development, myelination and cytoskeletal organization.
Pasithea Therapeutics Corp. has selected a lead therapeutic candidate for its PAS-003 program, a proprietary humanized monoclonal antibody that targets α5β1 integrin, a protein overexpressed in both humans and mice with amyotrophic lateral sclerosis (ALS).
The Alzheimer’s Drug Discovery Foundation (ADFF)’s Diagnostic Accelerator launched the first longitudinal, international study of vocal changes associated with Alzheimer’s disease (AD). Part of a $100 million effort to develop affordable biomarkers for AD, the study hopes to create the world’s largest database of speech and vocal data to facilitate diagnosis and monitoring of neurodegenerative disease.
Prism Biolab Co. Ltd. has identified new 7-membered ring-fused compounds acting as Notch signaling inhibitors and reported to be potentially useful for the treatment of neuronal injury and neurodegenerative diseases.
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