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.
The transition from complex and costly ex vivo strategies to platforms that enable direct cellular intervention within the body, known as in vivo therapies, is marking a paradigm change in the field of gene and cell therapies by simplifying manufacturing, improving tissue targeting and expanding clinical access to treatments.
Northern Illinois University and Stream Neuroscience Inc. have patented benzothiazole derivatives acting as phosphodiesterase‑4 (PDE4) inhibitors and reported to be useful for the treatment of cancer, cognitive, inflammatory and mood disorders.
The pathogenesis of multiple sclerosis (MS) has been tied to ineffective immune control of Epstein-Barr virus-driven autoimmune responses. Patients with MS are deficient in protective adaptive natural killer cells (pNK cells) in contrast to healthy individuals. These pNK cells are positive for NKG2A, NKG2C and NKG2D and recognize and kill autoreactive B cells in a selective and efficient manner.
Muna Therapeutics ApS has patented new triggering receptor expressed on myeloid cells 2 (TREM2) agonists reported to be useful for the treatment of amyotrophic lateral sclerosis, frontotemporal dementia, osteoporosis, rheumatoid arthritis, systemic lupus erythematosus, type 2 diabetes and obesity, among others.
Chinese researchers have published preclinical data regarding the potential of never in mitosis A (NIMA)-related kinase 2 (NEK2) as a therapeutic target in multiple sclerosis (MS), an inflammatory demyelinating disease of the central nervous system (CNS) in which B cells play a key role.
Humanwell Pharmaceutical US Inc. has disclosed new potassium voltage-gated channel subfamily KQT member 2 (KCNQ2; Kv7.2) and Kv7.3 activators. They are described as potentially useful for the treatment of epilepsy, pain, depression, bipolar disorder, amyotrophic lateral sclerosis and neurodegeneration.
Activation of cannabinoid receptors in the peripheral nervous system may help treat inflammatory and gastrointestinal disorders as well as pain, and several full agonists have been reported but they present safety concerns. Partial agonists, such as those that activate receptors to 20%-50% of full activity, may be safer, yet so far such agonists have emerged serendipitously.
Dem Biopharma Inc. has divulged G-protein coupled receptor 84 (GPR84) agonists and phagocytosis inducers reported to be useful for the treatment of cancer, infections, neurological disorders and rheumatoid arthritis.