Childhood-onset neurodegeneration with cerebellar atrophy (CONDCA) is an autosomal recessive disorder that causes progressive motor and cognitive impairment in children. The disease arises as a result of inactivating mutations in cytosolic carboxypeptidase 1 (CCP1), leading to excessive polyglutamylation of tubulin in the brain. Researchers at Shimane University have shown in a mouse model that delivering a truncated form of CCP1 into the brain can substantially mitigate Purkinje cell degeneration and improve motor function.

Seal Rock Therapeutics Inc. has joined The Michael J. Fox Foundation for Parkinson’s Research (MJFF) LRRK2 Investigative Therapeutics Exchange (LITE) program. The program supports the development of new therapies targeting LRRK2 for the treatment of Parkinson’s disease patients and fosters international collaboration across more than 30 academic and clinical centers and more than a dozen companies.

In Parkinson’s disease, α-synuclein accumulates in neurons and may thereby contribute to their degeneration. Reducing expression of α-synuclein may be an effective therapy, but delivering short interfering RNA (siRNA) to the brain noninvasively is notoriously ineffective, in part because siRNA does not pass the blood-brain barrier efficiently.

A little-known tissue composed of a cluster of immune cells could offer novel insights into the development of neurological disorders. Meninges' immune system changes with age and neurodegeneration. Are they protecting the brain or fueling disease? Mapping and analyzing the so-called ectopic lymphoid structures (ELSs) in the meninges at different ages in preclinical models of neurodegenerative diseases such as Alzheimer's may help clarify whether they are good, bad, or ugly, as in the iconic film by Sergio Leone.

The reach of the med-tech patent wars now includes devices for electroencephalography thanks to a complaint recently filed by Sunnyvale, Calif.-based Ceribell Inc. with the U.S. International Trade Commission. The company alleged infringement of six of its patents by two units under the Natus umbrella of companies, but this conflict is also under review in Delaware district court, promising a drawn-out conflict that may not resolve for years.

Quince Therapeutics Inc.’s mid-July completion of enrollment in the pivotal phase III study in ataxia-telangiectasia (A-T) marked an important milestone for the South San Francisco-based firm, which is not the only contender in the space. The study called NEAT – a rough acronym for “Neurological Effects of eDSP on Subjects with A-T” – will evaluate Quince’s lead asset, eDSP, in the neurodegenerative illness.

G protein-biased agonists enhance opioid-induced analgesia by selectively avoiding β-arrestin-2 (βarr2) signaling, which has been associated with reduced efficacy and adverse effects. Similarly, directing neurotensin receptor 1 (NTSR1) signaling toward β-arrestin pathways may promote analgesia via alternative mechanisms while minimizing side effects linked to G protein activation.

CLN3 disease, also known as juvenile neuronal ceroid lipofuscinosis, is a rare neurodegenerative disorder affecting neuronopathic lysosomal storage that severely impacts the central nervous system while also inducing notable peripheral neuromuscular symptoms. Researchers from Washington University School of Medicine have demonstrated the potential of gene therapy for CLN3 disease.