Because dysregulated energy metabolism is common across both familial and sporadic forms of amyotrophic lateral sclerosis (ALS), targeting energy production is believed to have therapeutic potential. University of Edinburgh and University of Oxford researchers have tested modulating the expression of the glycolysis enzyme phosphoglycerate kinase 1 (PGK1) in preclinical models of ALS by using terazosin, a PGK1 activator, with the aim of confirming it as a therapeutic target in the treatment of the disease.

Parkinson's disease (PD) is the second most common neurodegenerative disorder and is characterized by the progressive loss of dopaminergic neurons in the substantia nigra and the abnormal accumulation and aggregation of alpha-synuclein.

China Hinye Pharmaceutical has presented new phenol derivatives acting as GABA(A) receptor alpha1beta2gamma2S positive allosteric modulators (PAMs) reported to be useful for the treatment of anxiety disorders, convulsions, migraine, nausea and vomiting and neurodegeneration.

XtalPi has discovered heterocyclic amide derivatives acting as prostaglandin E2 receptor EP4 subtype (PTGER4; EP4) antagonists reported to be useful for the treatment of pain, rheumatoid arthritis, osteoarthritis and cancer.

Learning and memory are complex processes that involve many genes, cell types and brain circuits. Genetic screening performed by researchers have identified potassium voltage-gated channel subfamily C member 3 (KCNC3) to be involved in the hippocampal learning and memory processes in mice.

Two large-scale studies provide new data on genes, inherited variations, and de novo mutations associated with autism spectrum disorder (ASD). Some of them are also associated with other neurological conditions, like developmental delay (DD), or schizophrenia.

Insusense has divulged pyridine derivatives targeting sortilin (neurotensin NTR3; NT3; Gp95) receptors reported to be useful for the treatment of neurodegenerative disorders, cancer, pain, diabetes mellitus, diabetic retinopathy, cardiovascular disease, hereditary eye conditions and hearing loss.

Neuron-targeting of caveolin-1 (Cav-1) using AAV9 delivery and a synapsin-driven Cav-1 engineered construct, SynCav1, showed therapeutic potential in the hSOD1G93A model of familial amyotrophic lateral sclerosis (ALS).

A fusion protein removed beta-amyloid plaque without producing the neurotoxic inflammation associated with other treatments, such as aducanumab immunotherapy. It is based on the alphaA Beta-Gas6 fusion protein developed in a mouse model of Alzheimer's disease (AD) by a team of researchers at The Korea Advanced Institute of Science and Technology (KAIST) in South Korea.

Biofront Therapeutics has identified fMet-Leu-Phe receptor (fMLP; FPR1) antagonists reported to be useful for the treatment of glioblastoma, traumatic brain injury and stroke.