Exosome Therapy: The Holy Grail of Regenerative Medicine
Dec 9 Written By Mida Pezeshkian, PhD
Regeneration is essential to life and more common than we think. Our bodies can regenerate at three levels: molecular, cellular, and tissue. Though we may think of the more astonishing forms of regeneration such as an octopus regrowing a severed limb, this critical process occurs in our hair, skin, bones, and other tissues on a routine or as needed basis. The process of regeneration requires a conducive microenvironment with the right regeneration signals and down-regulation of inhibitory signals. At times, the regeneration process may become faulty or slowed, hindering proper repair and restoration.
Regenerative medicine aims to regulate and restore regeneration by facilitating the appropriate signals and creating a conducive environment. Exosome therapy is a type of regenerative medicine that seeks to repair tissue damage and restore function to damaged organs by delivering exosomes— small vesicles containing proteins, lipids, and genetic material—into the patient's body. Exosomes are naturally produced by cells, and their delivery is thought to stimulate the body's own healing mechanisms. This therapy has been used to treat a variety of diseases and conditions, including heart disease, arthritis, neurological disorders, and cancer.
In recent decades, stem cell research and its clinical application from transplant sources and enrichment of the host cells have advanced treatment and cure of several cancers and other diseases. These approached carry a host of downsides and difficulties such as cost, accessibility, infection transmission due to blood borne pathogens in handling/delivery, host/donor HLA-matching, and barriers in harnessing the appropriate quantity and quality of the self donor populations. Due to its cell-free nature, in many cases, exosome therapy can deliver significant benefits through modulating cell signaling with manageable & preventable risk - potentially becoming the holy grail of regenerative medicine.
A network analysis of data on “stem cells” and “exosomes” from all open source references shows key areas of interest in both topics and further highlights exosomes in cancer and mesenchymal stem cell derived exosomes (Figure 1 - bottom left clusters). This is consistent with published research on use of mesenchymal stem cell derived exosomes rendering promising results.
These early results have attracted both industry and public attention (Figure 2), creating demand for purification and treatment delivery in a clinical setting. Stem cell derived exosomes are cell-free products; therefore, eliminating risks associated with live cell biologics. However, great care and education is needed to ensure proper quality control, storage, handling, and treatment delivery. Unfortunately, the recent demand and potential for profit has attracted untrained operators, resulting in low quality products and increased risk of mishandling and unsupervised treatment.
The current regulatory environment for exosome therapy is in its early stages, with a patchwork of regulations in place across the United States. Currently, the FDA has not approved any therapies using exosomes. In 2020, the FDA announced that all regenerative medicine products intended for clinical use must go through their regulatory process. The CDC followed with additional information warning patients and providers on this issue. Additionally, different states have different regulations, and the FDA has cautioned that therapies delivered in some states may not be available in other states. This announcement prohibits clinical use through research of all unapproved products.
While the early results of exosome research for therapeutic use have demonstrated some positive results, large scale studies are needed to fully understand the benefits and applications in this area of regenerative medicine.
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