Porosome Therapeutics has reported preclinical results showing that restoring porosome function in human brain organoids reversed key features of Alzheimer’s disease, including structural abnormalities and impaired neuronal signalling. The company has published its findings in bioRxiv and plans to seek peer-reviewed publication.

The work centres on the porosome, a cellular structure known to regulate secretion across multiple cell types. Researchers used a therapeutic platform that combines porosome reconstitution with a small-molecule flavonoid. According to the team, targeting both disrupted cellular metabolism and faulty communication between neurons helped to restore growth, morphology and electrical activity in the Alzheimer’s model.

Guillermo Marmol, chief executive officer and co-founder of Porosome Therapeutics, said: “These preclinical findings reflect the progress we have made in translating decades of foundational research in porosome biology into a novel therapeutic strategy for one of the most devastating neurological diseases to date.” He added that the company aims to build on the results as it explores the porosome as a target for the millions of people living with the condition.

The study used human brain organoids derived from induced pluripotent stem cells, in line with systems recommended by NIH and FDA guidance for modelling disease-relevant neuronal biology. Organoids were exposed to the beta amyloid peptide Aβ1-42, a widely used method for inducing Alzheimer’s-like pathology. Researchers assessed neuronal activity using the UHD-CMOS-MEA platform, which was jointly developed by Sony Semiconductor Solutions, Screen Holdings and VitroVo to capture high-resolution electrical signals from living cells.

Bhanu Jena, chairman and co-founder of Porosome Therapeutics, added: “While defects in neuronal signalling are a hallmark of Alzheimer’s disease, our study is the first to show that restoring porosome function—alongside correcting neuronal metabolic deficiencies—can reverse impairments in neuronal growth, morphology, and synaptic activity.

“It is exciting to see that using iPSC-derived human brain organoids, as recommended by the NIH and FDA, we demonstrate both morphological and functional reversal of Alzheimer’s following porosome reconstitution therapy. This milestone not only advances Alzheimer’s treatment but also opens new possibilities for other neurosecretory disorders such as Parkinson’s.”

The company plans to showcase the work at the Society for Neuroscience conference, where the results will be displayed at Sony’s booth in the San Diego Convention Center. The article, titled Porosome reconstitution reverses Alzheimers in human brain organoids, is now online.