Stem Cell Treatment for Septo-optic dysplasia (SOD)

Stem cells are cells that are “pluripotent”, meaning they can differentiate into all other cells due to their self-renewing abilities. They can develop into ectodermal (ex. skin and some neurological structures), mesodermal (ex. bones, cartilage, and blood cells), or endodermal cells (ex. cells of internal body organs). Therefore, injecting stem cells – from a donor – should theoretically allow them to differentiate and “replace” the damaged neurons seen in different NTDs (4). Stem cell therapy has provided new hope in curing or at least improving the neurological symptoms associated with spina bifida, in order to provide patients a better quality of life. Following the testing of stem cell treatment on people with spina bifida, in addition to their self-renewing abilities, stem cells have proven to have additional benefits other than tissue replacement; including (5, 6):

• Replacing and repairing the damaged neurons: As mentioned, this is their original function; to replace the damaged tissue – i.e. spinal cord neurons in spina bifida – through differentiating into its cells.
• Increasing the production of neurotrophic factors that promote nervous cell proliferation and differentiation (ex. glia derived neurotrophic factor (GDNF) and brain derived neurotrophic factor (BDNF)): These factors can locally enhance cellular recruitment, proliferation and maturation within the damaged or affected neurons.
• Modulating the immune system and the ongoing inflammatory process: Stem cells produce different antioxidants; thereby reducing the neurodestructive and atrophic process characterizing different NTDs.
• Promoting vascular supply to the nervous system: by stimulating the generation of new blood vessels (neovascularization/angiogenesis) through stimulating different vascular stimulating growth factors (ex. VEGF). Better blood supply allows for better and faster neuron regeneration.
• Preventing cell death: Through releasing substances that can inhibit the process of apoptosis, or programmed cell death, of the damaged cells until they are adequately repaired by stem cells.

Over the last few decades, there have been numerous “animal” trials testing the efficacy and safety of injecting stem cells in-utero (during pregnancy) to allow for proper neurological development, and these trials have shown promising results (5, 7). However, their application in humans requires further trials to prove definite efficacy. We are still waiting for the results from the first FDA-approved human-based in-utero stem cell therapy trial to be able to use it on people with spina bifida (3). This is why, despite the extensive animal-based results on the benefits of in-utero stem cell injections, we can still only used conventional stem cell therapy after birth due to their extensive usage in different neurological disorders.