Many patients with amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD) harbor the (G4C2)n pathogenic repeat expansion in the C9orf72 gene, which leads to aggregating dipeptide proteins, mainly poly-glycine-alanine (poly-GA).

Abata Therapeutics Inc. has obtained FDA clearance for its IND application enabling initiation of a first-in-human study of ABA-101 in patients with progressive multiple sclerosis (MS). A phase I study will open later this year.

Brains from people with heroin use disorder have an increased number of hypocretin (Hcrt, orexin) neurons, which in turn are significantly smaller than in healthy controls. Meanwhile, human narcolepsy is known to present an average 90% loss of Hcrt neurons and no risk for drug abuse or overdose, even under methamphetamine or methylphenidate prescription, because of a greatly reduced reward activation of the ventral tegmental area (VTA).

Multiple sclerosis (MS) is a chronic autoimmune demyelinating disorder in need of new treatment options. Inhibition of the phosphoinositide 3-kinase δ (PI3Kδ) pathway has shown efficacy in animals with experimental encephalomyelitis (EAE), a model of MS.

Anew Medical Inc. has announced plans to advance its Klotho gene therapy program for neurodegenerative disorders. Initial data suggest that maintaining elevated levels of Klotho in the body significantly contributes to longer, healthier life spans, while individuals with depleted or lower than normal levels of Klotho are more susceptible to neurodegenerative disorders.

Researchers from the University of Texas Medical Branch have developed a novel tau immunotherapy delivered via intranasal route, able to enter the brain, and recognize and successfully clear tau aggregates in mouse models of tauopathy. Aberrant tau aggregates cause neurodegenerative symptoms in Alzheimer’s disease (AD), progressive supranuclear palsy (PSP) and dementia with Lewy bodies (DLB). Although these conditions present phenotypic differences, the fact of sharing tau deposits as a major hallmark tags them as tauopathies.