Many diseases are due to a deficit in healthy, fully functioning cells caused by injury, trauma, infection, ageing or congenital deficiencies. Cell-based regenerative medicine describes the restoration of lost function at the site of the disease by the application of medicines based on cells including stem cells.
Stem cells are seen as a key tool in the future of medicine, for their abilities to regenerate damaged tissue, replace missing tissue and repair the effects of disease or injury. However, advancing knowledge in stem cell therapy is demonstrating that positive medical outcomes can be achieved without using the stem cells themselves as the therapeutic agent.
Instead, some see the essential therapeutic components are the cell-secreted extracellular vesicles (EVs), including exosomes.
Although exosomes were discovered more than 30 years ago, it was not until recently that the scientific community began to give credit to exosomes for a range of promising traits relevant to regenerative medicine. Today, exosomes are rapidly gaining momentum as a strategy for accessing the therapeutic effects of stem cells without the risks and difficulties of administering the cells to patients.
Studies have shown that exosomes can be as potent as adult stem cells in promoting regeneration and functional recovery in experimental animal models of human diseases including stroke, traumatic brain injury, pulmonary hypertension and wound healing.
These findings point to exosomes as the potential next-generation regenerative medicine product to address many medical problems and problems associated with ageing.
As its main focus, Exopharm is seeking to commercialise exosomes as therapeutic agents by investing in the clinical development of exosomes – by conducting clinical trials in a number of medical areas (including skin, eye and joints) as well as developing its manufacturing technology to support the clinical trials and future sales and supportive scientific and research activities.
Exosomes as therapeutic agents (in comparison to cells) have the potential to streamline the entire therapeutics value chain, including manufacturing, storage, delivery and clinical use.
Exosomes – a natural part of our cell biology
Exosomes are nano-sized vesicles ranging from 30–400 nm in size and facilitate a range of important cellular functions.
They are membranous bilayer lipid vesicles secreted by a variety of cell types (including stem cells and platelets) into the blood circulation and other bodily fluids.
Exosomes are effective communication vehicles that transfer bioactive proteins and genetic material (DNA and RNA) between cells to alter the function of the targeted cells, including complex regenerative and healing programs within the body.
Cells produce exosomes naturally. Exosomes are commonly found in blood, placenta, urine and milk among other biofluids.
Exosomes contribute to functions including tissue repair, neural communication and immune modulation.
Another attribute that makes exosomes powerful are the large quantity found within living systems – it is estimated that there are 1000 times more exosomes than stem cells within the human body.
Stem cell exosomes are versatile in that they can alter the function of the targeted cells that can be near (paracrine like synapses) or far (endocrine) systemically via the blood.
Stem cell exosomes have the potential to be used in a wide range of applications, including as therapeutics, diagnostic tools, and research products.
There is also the potential to incorporate exosomes into cosmeceuticals (cosmetic products that have or claim to have medicinal properties) or nutraceuticals (functional feeds).
Drug development is by its nature a lengthy, expensive process with a high failure rate. There are many risks that relate to development of exosomes as a therapeutic product as well as risks that relate to Exopharm as a business entity.