The ability to harness the potential of adult stem and precursor cells would be a major advance in the treatment of human disease. Stem cells have been derived from embryo, foetus, umbilical cord, blood, adult tissue, or from the reprogramming of adult somatic cells. They are remarkable cells characterized by their ability to divide and to differentiate via a number of steps to embryonic and adult somatic cell lineages. Such cells thus hold enormous promise both for in vitro screening tools for drug efficacy and toxicity testing, and especially for regenerative therapies treating a wide range of disorders with high unmet medical need such as neurodegenerative diseases, diabetes, heart disease, and vision loss.
Current approaches to cell programming chemistry
A number of treatment options have been investigated in recent years, including cell therapy and tissue engineering. The majority of companies in the cell programming 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 of 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 huge 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 will involve the activation of adult stem or precursor 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.
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.