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Exosome therapy offers a promising new approach to sensorineural hearing loss

Exosome therapy offers a promising new approach to sensorineural hearing loss
Credit: Association of Basic Medical Sciences of FBIH

Sensorineural hearing loss (SNHL) is the most prevalent form of permanent hearing loss, affecting over 6% of the global population, according to the World Health Organization. It results from damage to sensory hair cells in the inner ear or to the auditory nerve pathways that transmit sound to the brain.

This type of loss can be caused by factors such as prolonged noise exposure, certain medications (like chemotherapy or aminoglycosides), genetic conditions, or autoimmune diseases. SNHL often affects communication, cognition, and quality of life, and remains irreversible in most cases due to the limited regenerative capacity of inner ear cells.

Hearing depends on the proper function of the organ of Corti, a structure in the cochlea that houses around 15,000 inner and . These cells convert sound vibrations into electrical signals, which are interpreted by the brain. In mammals, the ability of these cells to regenerate is lost early in embryonic development, making recovery from damage extremely difficult.

As a result, current treatments rely on assistive technologies such as hearing aids and , which can improve sound perception but do not restore natural hearing or reverse cellular damage. In some cases, cochlear implants may also impair residual hearing or limit sound clarity in noisy environments.

To address these limitations, researchers have explored regenerative therapies, particularly using . Embryonic stem cells and induced (iPSCs) show potential but raise concerns related to ethics, tumor formation, and immune rejection. The work is in the journal Biomolecules and Biomedicine.

Mesenchymal stem cells (MSCs), derived from adult tissues like bone marrow or umbilical cord, offer a more accessible and practical alternative.

These cells can be manipulated in the lab and possess regenerative and anti-inflammatory properties. In animal studies, transplanted MSCs have been shown to improve hearing function, reduce inflammation, and help preserve critical structures in the cochlea.

However, using live stem cells comes with its own challenges. Cell survival, delivery to the inner ear, and the risk of unwanted immune responses remain significant hurdles.

As a result, attention has shifted toward a cell-free alternative: using —tiny, naturally secreted vesicles that carry therapeutic molecules from stem cells to other cells in the body.

Harnessing the power of MSC-derived exosomes

Exosomes are nanoscale particles secreted by most types of cells, including MSCs. These vesicles carry proteins, lipids, and RNA, and play a vital role in intercellular communication. Research has shown that exosomes can transfer their contents to target cells, influencing gene expression and promoting tissue repair.

Importantly, MSC-derived exosomes retain many of the regenerative benefits of their parent stem cells—such as reducing inflammation and supporting —without the risks of tumor formation or immune rejection.

Their small size also allows exosomes to cross biological barriers like the blood–labyrinth barrier, making them particularly well-suited for delivering therapies to the inner ear. In this way, exosomes represent a promising new platform for treating hearing loss caused by damage to the cochlea or auditory neurons.

Promising results in preclinical and early clinical research

Recent studies in animal models of SNHL have demonstrated that exosomes derived from MSCs can improve hearing thresholds, protect hair cells, and reduce cochlear inflammation.

In mice and rats exposed to the ototoxic drug cisplatin, MSC-exosomes helped reverse damage, improve survival of auditory neurons, and modulate gene expression in the cochlea. In another model, exosomes enhanced the growth and survival of spiral ganglion neurons—key cells involved in transmitting sound signals to the brain.

These appear to result from the exosomes' ability to reduce oxidative stress, suppress cell death pathways, and deliver regenerative signals through molecules such as microRNAs and heat shock proteins. Some exosomes even appear to stimulate cellular repair and remodeling of inner ear tissues.

In a preliminary clinical case, a patient undergoing cochlear implantation also received a dose of MSC-derived exosomes delivered directly into the cochlea. Over a 24-month follow-up period, the patient showed improved speech comprehension and signs of enhanced tissue activity around the implant electrodes, suggesting that exosomes may have positively influenced the local cellular environment.

Challenges and future directions

While the results from preclinical and early clinical work are encouraging, the path to clinical adoption involves several hurdles. Standardizing exosome isolation, scaling up production, and ensuring consistent dosing remain significant technical challenges. Different purification methods—such as ultracentrifugation or size-exclusion chromatography—can impact exosome yield and quality.

Engineering exosomes to carry targeted therapeutic molecules may enhance their benefits, but also introduces complexity and regulatory hurdles.

Moreover, there are currently no completed clinical trials evaluating MSC-exosomes specifically for hearing loss. Rigorous safety testing, reproducibility studies, and controlled human trials will be necessary before exosome therapies can be widely adopted.

Researchers are also working to better understand the molecular mechanisms by which exosomes promote repair in the inner ear, which could lead to more targeted and effective therapies in the future.

A new frontier in hearing loss treatment

MSC-derived exosomes represent a novel, minimally invasive strategy for treating . By delivering regenerative and anti-inflammatory signals directly to damaged inner ear cells, they offer a potential solution that avoids the risks associated with live cell transplantation.

If ongoing research confirms their safety and efficacy, exosome-based treatments could complement or even enhance existing therapies such as cochlear implants, and offer new hope to people with currently untreatable hearing loss.

As the field progresses, collaboration between scientists, clinicians, and regulatory bodies will be essential to transform this promising approach into a viable treatment option. With continued investment in research and clinical trials, exosome therapy could mark a major advance in the future of auditory health.

More information: Maria Perde-Schrepler et al, Mesenchymal stem cell- derived exosomes as cell-free therapeutics for sensorineural hearing loss, Biomolecules and Biomedicine (2025).

Citation: Exosome therapy offers a promising new approach to sensorineural hearing loss (2025, June 24) retrieved 7 August 2025 from /news/2025-06-exosome-therapy-approach-sensorineural-loss.html
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