The ability to harness the potential of adult stem and progenitor cells would be a major advance in the treatment of human disease. Stem/progenitor cells are remarkable; characterized by their ability to divide to form another stem/progenitor cell and to differentiate, via a number of steps, to adult somatic cell lineages. OxStem’s approach stimulating stem/progenitor cells thus holds promise for regenerative therapy and could be applicable to treatment of a wide range of disorders with high unmet medical need such as neurodegenerative diseases, diabetes, heart disease, and vision loss.
Current approaches to regenerative medicine
A number of treatment options have been investigated in recent years, including cell transplantation therapy and tissue engineering. The majority of companies in the regenerative medicine space are focused on developing autologous or allogeneic stem cell transplantation therapies, often performing in vitro modulation of the stem cells followed by direct injection into the patient. OxStem uses a very different in situ approach – endogenous cell activation therapy (ECAT) - as explained in the following section.
OxStem approach- Endogenous Cell Activation Therapy
The discovery of small molecules to control cell fate has attracted interest in recent years as it can offer significant advantages over alternative techniques in terms of cost effectiveness and the ability to manipulate cells reversibly and at will. One of the potentially most powerful applications of using small molecules to direct cell fate would be the ability to manipulate a patient’s own endogenous tissue cells in situ solely through administering a drug, thereby precluding the need for cell transplantation. This forms the basis of ECAT, which involves the activation of adult stem or progenitor cells in situ, by small molecules, to induce regeneration or repair of adult tissue.
There are many examples of this regenerative capacity in nature, for example the salamander, and other amphibians, that can regrow detached limbs. This is a phenomenon we can also observe in humans as many cell types are continuously replaced into adulthood, for instance blood/bone, gut epithelium and skin/hair. Many other tissues retain a more limited capacity to regenerate and, importantly, repair mechanisms can be stimulated in response to injury, e.g. in the brain and heart, but not to a sufficient level for long-term health.
OxStem aims to develop a collection of drug candidates to augment, or reactivate, these innate repair processes as a therapeutic paradigm applicable to a range of degenerative diseases.