Patients with amyotrophic lateral sclerosis (ALS) have a median survival of 2 to 5 years. There are 3 FDA-approved drugs for ALS (riluzole, edaravone and Relyvrio [phenylbuturate/taurursodiol]), but they only lead to modest benefit. There are several pathways involved in the disease, but all of them lead to neuroinflammation.
Cytokinetics Inc. has disclosed fast skeletal muscle myosin inhibitors reported to be useful for the treatment of Becker muscular dystrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, spinal cord injury, limb girdle muscular dystrophy, stroke, tendinitis and tremor, among others.
Jawaharlal Nehru Centre for Advanced Scientific Research has divulged naphthalene monoimide compounds acting as modulators of liquid-liquid phase separation (LLPS) of tau protein reported to be useful for the treatment of traumatic encephalopathy, progressive supranuclear palsy, tauopathies, frontotemporal dementia and Alzheimer’s disease.
Recent studies have identified HAVCR2, which encodes immune checkpoint molecule TIM-3 (T-cell immunoglobulin mucin receptor 3), as a risk gene for late-onset Alzheimer’s disease (AD).
Ono Pharmaceutical Co. Ltd. has entered into a collaboration agreement with Sibylla Biotech SpA to generate novel drug candidates for neurological disorders. Under this collaboration, Sibylla will utilize its proprietary Pharmacological Protein Inactivation by Folding Intermediates Targeting (PPI-FIT) protein degradation technology platform.
Kv1.3 is a voltage-gated potassium channel that plays a crucial role in neuroinflammation and neurodegeneration in Parkinson’s disease and other disorders. Preclinical studies have shown that Kv1.3 inhibition confers neuroprotection against neurodegenerative disorders.
The University of California, Berkeley has announced the launch of the UC Berkeley Molecular Therapeutics Initiative (MTI) designed to accelerate drug discovery. The aim is to bridge fundamental research in rare neurological and metabolic diseases with drug discovery to identify and accelerate novel therapeutic modalities into the clinic.
Mapping brain circuits and studying the neural signals that are activated during post-traumatic stress could provide an answer to the generalized fear associated with this disorder. Scientists at the University of California, San Diego have identified a change in the expression of neurotransmitters as an adaptive response that could trigger this effect.
Xgene Pharmaceutical Pty Ltd., a subsidiary of Xgene Pharmaceutical Co. Ltd., has received approval in Australia to initiate a phase I trial of the selective TRPM8 blocker XG-2002 (RQ-00434739).
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