At the 2025 European College of Neuropsychopharmacology (ECNP) Congress in Amsterdam this week, researchers presented new findings on the role of endocannabinoids in stress, addiction … and the integrity of the blood-brain-barrier.
Nonsteroidal anti-inflammatory drugs (NSAIDs) are commonly used to treat inflammatory pain, but they can have severe side effects, including potentially life-threatening gastrointestinal, renal and cardiac toxicity. Their analgesic effect is driven by inhibition of prostaglandin (PG) biosynthesis and subsequent inflammation, but this inhibitory effect on inflammation could delay pain resolution. An optimal approach to managing PG-mediated pain would selectively relieve pain while preserving the PGs’ essential inflammatory and protective functions.
Lysosomal homeostasis is crucial to the metabolism of certain proteins and lipids that would otherwise accumulate, thus leading to cellular stress and pathology. This is common in diseases such as Parkinson’s disease and Gaucher disease. Researchers set out to find a brain-penetrant small-molecule agonist of the lysosomal channel TRPML1, also known as mucolipin-1.
Researchers at the Institute for Bioengineering of Catalonia (IBEC) and collaborators have introduced a novel therapeutic strategy for Alzheimer’s disease (AD) that leverages the multivalency of supramolecular nanomedicines to reprogram blood-brain barrier (BBB) function, facilitating efficient amyloid-β (Aβ) clearance and restoring cognitive function in animal models.
Myrobalan Therapeutics Nanjing Co. Ltd. has divulged 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.
Sichuan Kelun Pharmaceutical Research Institute Co. Ltd. and Suzhou Kelun Pharmaceutical Co. Ltd. have identified carboxylic acid ester compounds reported to be useful for anesthesia.
Parkinson’s disease (PD) is a progressive disease characterized by loss of dopaminergic neurons in the substantia nigra, which lead to motor symptoms. Aromatic-L-amino-acid decarboxylase (AADC) converts levodopa into dopamine and glial cell line-derived neurotrophic factor (GDNF) promotes the survival, growth and regeneration of dopaminergic neurons. VGN-R09b is an adeno-associated viral vector (AAV)-based AADC and GDNF combination gene therapy that is delivered to the striatum for the treatment of PD.
Casma Therapeutics Inc. has developed bicyclic heteroaromatic compounds acting as mucolipin-1 (MCOLN1; TRPML1) activators reported to be useful for the treatment of Duchenne muscular dystrophy, neurodegeneration, atherosclerosis, cancer, infections, liver, lysosomal storage and inflammatory bowel diseases.
Oligomeric forms of α-synuclein are increasingly recognized as the primary neurotoxic species in Parkinson’s disease (PD) and other synucleinopathies, contributing to synaptic dysfunction, mitochondrial impairment and the prion-like propagation of pathology. Targeting these early aggregates represents a promising strategy for disease modification.