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Abstract
Stem cell therapeutics offer novel treatments for disease and tissue injury that cannot be achieved by other means. As these therapeutic are developed, validating correct developmental progression, optimizing patient compatibility and applicability for use in clinical use are vital for success. Here we show the progression of two developing therapies, one for neural deficits and one for bone formation, in their various stages of development. Both of these therapeutics offer hope for very different disorders, and progression to the clinic presents unique hurdles due to the complex nature of their role in healing the patients body. We present some of the aspects of these potential therapies as they progress to becoming a viable treatment.Pluripotent cell derived neural cell therapy offers the potential aide in the treatment of multiple neurodegenerative disorders, however complexities involved in treating these diseases require thorough examination and testing before cellular therapies can be used. ESC derived neural progenitor differentiation into neurons showed structural changes similar to what is seen in the developing nervous system in vivo. Additionally, using neural cells for therapy requires that the cells integrate into the patients body to treat the disease, and generating cells that are compatible with the patients immune system will prevent the need for lifelong immunosupressive treatments. Here we show that blood cells transduced with POU5F1/OCT4 were capable of generating cells of all germ layers and differentiated neurons. In a vastly different therapeutic for bone regeneration, genetically modified mesenchymal stem encapsulated in a poly (ethylene glycol) allows the use of a biological barrier to prevent the cells from migrating away from the site of injury and protect the encapsulated cells from the patients immune system. Here we develop a method for cryopreserving this therapeutic in a ready to use format that can facilitate the distribution to centers for use in animal models and in the future, to the clinic. Stem cell therapies offer the opportunity to provide unique treatments for many needed diseases. Optimizing efficacy and safety will allow for the best potential outcome as the stem cells cross the bridge between a potential therapeutic and effective treatment.