Vanishing white matter disease (VWM) is a rare and progressive leukoencephalopathy caused by loss-of-function mutations, in a recessive pattern of inheritance, in any of the genes encoding eIF2B, a guanine nucleotide exchange factor for eIF2 and an effector of the integrated stress response (ISR). At last week’s American Academy of Neurology meeting, Calico Life Sciences LLC and Abbvie Inc. presented preclinical results for their brain-penetrant compound ABBV-CLS-7262 (fosigotifator sodium tromethamine) in VWM.

Wista Laboratories Ltd. has disclosed thiazole-containing compounds acting as microtubule-associated protein tau (PHF-tau; MAPT) aggregation inhibitors reported to be useful for the treatment of corticobasal syndrome, Alzheimer’s disease, Down syndrome and more.

Biointervene Inc. has described adenosine analogues acting as adenosine A3 receptor (ADORA3) agonists reported to be useful for the treatment of mild cognitive impairment, diabetic neuropathy, irritable bowel syndrome, neurodegeneration, ototoxicity, neuropathic pain, chemotherapy-induced peripheral neuropathy and spinocerebellar ataxia, among others.

Neurocrine Biosciences Inc. has synthesized vesicular monoamine transporter 2 (VMAT2) inhibitors reported to be useful for the treatment of hyperkinesia and psychosis.

University of California Oakland has described potassium channel subfamily K member 2 (TREK-1; KCNK2) and/or potassium channel subfamily K member 10 (TREK2; KCNK10) activators reported to be useful for the treatment of Caisson disease (decompression sickness), depression, headache, pulmonary hypertension, insomnia, chronic pain, lung and neuronal Injury.