Researchers at the University of Sydney have uncovered a mechanism that may explain why glioblastoma returns after treatment, and the world-first discovery offers new clues for future therapies. Glioblastoma is one of the deadliest brain cancers, accounting for about half of all brain tumors, with a median survival rate of just 15 months. Despite surgery and chemotherapy, more than 1,250 clinical trials over the past 20 years have struggled to improve survival rates.
Scientists at Osaka University and Tokyo University of Science have described compounds targeting Claudin-5 (CLDN5) acting as blood-brain barrier (BBB) permeability regulators reported to be useful for the treatment of sepsis, cerebral edema, infections, epilepsy, multiple sclerosis, psychiatric disorders, Alzheimer’s disease and Parkinson’s disease, among others.
Researchers at Meiji Seika Pharma Co. Ltd., National Center of Neurology & Psychiatry and Tokyo University of Pharmacy & Life Sciences have synthesized NAD(+) H\hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of neurodegeneration.
Pheno Therapeutics Ltd. has disclosed uracil nucleotide/cysteinyl leukotriene receptor (GPR17; P2Y-Like) antagonists reported to be useful for the treatment of multiple sclerosis, amyotrophic lateral sclerosis, Alzheimer’s disease and Parkinson’s disease.
Tonix Pharmaceuticals Holding Corp. has licensed exclusive worldwide rights to TNX-4900 (formerly PW-507) from Rutgers University. TNX-4900 is a highly selective, small-molecule sigma-1 receptor (S1R) antagonist, which has demonstrated analgesic activity in multiple models of neuropathic pain.
Superoxide dismutase 1 (SOD1) mutations were among the first genetic causes identified in familial amyotrophic lateral sclerosis (ALS) and confer a toxic gain-of-function that drives motor neuron degeneration via protein misfolding, oxidative stress, mitochondrial dysfunction and neuroinflammation.
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder marked by progressive degeneration of upper and lower motor neurons, resulting in paralysis and death typically within 3-5 years of symptom onset. Historically, treatment options have been extremely limited. However, the identification of genetic contributors to ALS pathogenesis has enabled the application of antisense oligonucleotides (ASOs) to selectively modify or reduce the expression of disease-associated genes at the RNA level.
Researchers from Wuhan Institute of Biomedical Sciences, Jianghan University and collaborators recently showed that activating the endogenous transcription factors Ngn2 and Isl1 via CRISPRa can directly reprogram astrocytes into functional motor neurons in the mouse spinal cord.
Epilepsygtx Ltd. has raised a $33 million series A to fund a phase I/IIa trial of EPY-201, a gene therapy for treating drug-resistant focal epilepsy. EPY-201 uses an adeno-associated viral vector to deliver KCNA1, the gene encoding Kv1.1, a potassium ion channel that modulates neuronal excitability.
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