“I am not a fortune teller, nor am I a gambler. I will make no bets,” Lorraine Kalia told the audience at the 2023 International Congress of Parkinson’s Disease and Movement Disorders. “But I am optimistic.” At the meeting, which is being held in Copenhagen this week, Kalia, who is a scientist at Toronto Western Hospital’s Krembil Brain Institute and at the University of Toronto’s Tanz Centre for Research in Neurodegenerative Diseases, was giving an overview of “Emerging targets in the clinic” in a plenary session on “Therapeutic strategies for the future.”
Akava Therapeutics Inc. has announced FDA clearance of its IND application for AKV-9 (formerly NU-9) for the treatment of amyotrophic lateral sclerosis (ALS). The company is planning a first-in-human phase I study in healthy subjects.
Researchers from Oregon Health and Science University presented data from a study that aimed to identify and validate potential new therapeutic targets for the treatment of amyotrophic lateral sclerosis (ALS). Systems immunology analysis utilizing mass cytometry (CyTOF) was used to assess α5 integrin expression in 78,293 single cells, including 21,250 CD45+/CD11b+ myeloid cells from CNS of superoxide dismutase-1 (SOD) G93A mouse model of ALS (SOD1G93A).
Insilicotrials Technologies SpA has entered into a collaborative partnership with Axoltis Pharma SA in the field of central nervous system (CNS) diseases, with the aim of optimizing the clinical development plan for Axoltis’ drug candidate, NX-210c.
When a group of British scientists studied which proteins might be in the wrong place of the cell in amyotrophic lateral sclerosis (ALS) patients, they found hundreds of them mislocalized. Other studies had shown that TDP-43 protein was mislocalized. But it was not known that the phenomenon was widespread, and affected mRNA as well as proteins. “Our study revealed that these mislocalized proteins were heavily involved in RNA binding functions and exhibited high binding affinities to RNAs,” Rickie Patani told BioWorld.
With the launch of intravenous Leqembi (lecanemab-irmb) for Alzheimer’s disease (AD) underway, Priya Singhal, Biogen Inc.’s head of development and interim head of research, said the firm, along with partner Eisai Co. Ltd., aims to “address the long-term duration question” with a subcutaneous version, recently touted at the Alzheimer's Association International Conference.
Several neurodegenerative disorders have TAR DNA-binding protein 43 (TDP-43) inclusions as a pathological hallmark; thus, the development of PET tracers able to detect TDP-43 aggregates is essential to advance the diagnosis and treatment monitoring in diseases such as frontotemporal dementia, amyotrophic lateral sclerosis and others.
Aeterna Zentaris Inc. has offered an update on its development pipeline programs. Macimorelin acetate (AEZS-130), a ghrelin agonist approved and commercialized as a test for adult growth hormone deficiency, is in preclinical development for amyotrophic lateral sclerosis (ALS). The company has now successfully developed an alternative formulation suitable for use in ALS.
Spinogenix Inc. is opening enrollment in a first-in-human phase I trial of SPG-302 for the treatment of amyotrophic lateral sclerosis (ALS), having received approval from Australia's Human Research Ethics Committee (HREC).
