Genes associated with lysosomal dysfunction increase the risk of Parkinson’s disease (PD), according to a study led by scientists at Northwestern University. The discovery also explains why some people who carry a pathogenic variant of the GBA1 gene develop PD or dementia with Lewy bodies (DLB) and others do not. The key lies in the Commander complex, involved in the transport of proteins to this organelle. This discovery raises the need for combinatorial therapies that act on more than one pathway for this type of neurodegenerative disorder.

Researchers from Aarhus University and Synuca Therapeutics recently presented a novel drug-like small-molecule inhibitor of sarco/endoplasmic reticulum calcium ATPase (SERCA), SYN-4569, with superior pharmacological properties and evaluated its efficacy in vitro and in vivo. SYN-4569 is an orally available compound with good potency, pharmacokinetics and brain-penetrant properties.

A large-scale study has revealed the impact of germline variants on proteins in 10 cancer types. Scientists from the National Cancer Institute’s Clinical Proteomic Tumor Analysis Consortium (CPTAC) conducted a precision proteogenomic analysis in a pan-cancer study with data from 1,064 patients, identifying tumor heterogeneity and tumorigenesis associated with heritable genetic alterations. The results provide a broad view of cancer risk that could be useful for patient stratification and the design of prevention strategies.

At the 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (ADPD), researchers from Rutgers University investigated the therapeutic potential of CJRB-302, a live biotherapeutic product derived from healthy human gut microbiota, to improve PD motor symptoms and pathology.

At the 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (ADPD), researchers from Beijing Joekai Biotechnology LLC presented the discovery process and preclinical results for 50561.

At the 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (ADPD), Promis Neurosciences Inc. presented its new approach to optimizing the α-synuclein (α-Syn) vaccine composition in order to maximize targeting of toxic α-Syn species.