Ironically, the first person to be diagnosed with what is now Alzheimer’s disease was missing its major risk factor. When she first began showing symptoms of dementia in 1901, Auguste Deter was not particularly old. Despite Deter’s case, aging is the largest risk factor for developing Alzheimer’s, by a large margin. But “geroscience has not been translated into drugs for Alzheimer’s disease,” Howard Fillit, Alzheimer’s Drug Discovery Foundation co-founder and chief scientific officer, told BioWorld. “We’re just starting to see that cross-fertilization now.” This first article of a three-part BioWorld series on Alzheimer’s disease looks at how a group of researchers, as well as some startups, are trying to approach Alzheimer’s via an aging lens.
Asha Therapeutics LLC has nominated a development candidate, ASHA-624, as a potential disease-modifying therapy for amyotrophic lateral sclerosis (ALS) with additional indications in chemotherapy-induced peripheral neuropathy, glaucoma, and traumatic brain and spinal cord injuries. ASHA-624 is expected to enter the clinic by year-end.
Analysis of the immune signatures of blood samples from patients with early-stage multiple sclerosis (MS) who were treatment naive has identified three distinct subtypes that have different disease trajectories and which respond differently to therapy.
Europe may still await its first disease-modifying Alzheimer’s drug after the EMA postponed its decision on Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd) on March 22, but leading members of the World Dementia Council were in an optimistic mood when they convened in London four days later. “We are working to make the inevitable happen earlier,” said Lenny Shallcross, executive director of WDC. “The inevitable will be rollout of medicines, rollout of better diagnostics and the improvement of care. All of those things over the next 10 years are inevitably going to happen.”
Acurex Biosciences Corp. has synthesized phenothiazines acting as ferroptosis inhibitors reported to be useful for the treatment of mitochondrial disease.
Japan Tobacco Inc. has disclosed pyrazolopyrimidine compounds acting as NLRP3 inflammasome inhibitors reported to be useful for the treatment of traumatic brain injury, inflammatory bowel disease and more.
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a congenital metabolic disorder that leads to the accumulation of partially degraded heparan sulfate, which triggers neurodegeneration.
Shanghai Huilun Pharmaceutical Co. Ltd. has identified sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, multiple sclerosis, Charcot-Marie-Tooth disease, urinary incontinence, cough and arrhythmia.
Atamyo Therapeutics SAS has received clinical trial application (CTA) authorizations in Italy and France for ATA-200, its gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).