From Bench to Bedside: Extrazelluläre Vesikel aus mesenchymalen Stammzellen der Nabelschnur als neuartige Therapie für das Innenohr bei Cochlea-Implantationen

Background:

Cochlear implantation can be compromised by inflammation and fibrosis due to foreign body reactions, and patients often lose residual hearing post-implantation. A new investigational medicinal product derived from umbilical cord mesenchymal stromal cells, called vesicle-rich secretome fraction (VSF), shows promise for providing immunomodulatory and neuroprotective effects when applied intracochlearly during implantation. After demonstrating feasibility in a named patient case, a first-in-human phase I/II clinical trial (ESCRT) has been accepted by regulatory authorities recently.

Methods:

Preclinical studies were conducted in mice and guinea pigs to evaluate safety, efficacy, and biodistribution of VSF after intracochlear application and cochlear implantation. Data have been compiled for clinical trial application.

Results:

VSF was well-tolerated in disease-relevant animal models with no abnormalities observed. It improved hearing after implantation trauma in guinea pigs. Labeled extracellular vesicles were detected in cochlear structures and kidney, indicating physiological degradation after local injection mimicking the therapeutic approach in the clinical setting.

Conclusion:

Developing VSF from basic research through preclinical testing has led to the initiation of the ESCRT phase I/II clinical trial to evaluate safety and efficacy in humans. This process demonstrates feasibility of translating a new drug candidate from bench to bedside despite various challenges in biopharmaceutical development.

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