Using Stem Cells to Treat Ailments of the Nervous System Crista Chavez, Jacqueline Doody, Nina Roxo, Aleksandra Sabov & Zachary Taylor Faculty Mentor:

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Using Stem Cells to Treat Ailments of the Nervous System Crista Chavez, Jacqueline Doody, Nina Roxo, Aleksandra Sabov & Zachary Taylor Faculty Mentor: Dr. Andrew Grosovsky; Dean of CSM Objective and Rationale Objective: to become more informed of methods and research that utilize stem cell treatments to cure diseases or damages to the nervous system where previously no treatments existed We chose to research this topic because as a group, we are all very interested in neurological disease and nerve damage. Many people suffer greatly from these problems, including some people we know, so researching possible cures seemed very interesting to us. Stem cells used to develop new nerve cells in patients who have lost nerve cell function The nervous system consists organs composed of neurons, which send signals throughout the body, and also glial cells, which aid in the speeding of signals over long distances If these glial cells are damaged, signals take longer to reach their destination which can cause a number of detrimental effects Embryonic and muscle-derived stem cells have proven to be making progress in treating diseases such as Parkinson’s disease and injuries affecting the spinal cord Geron: first company to be cleared by FDA to implement stem cell therapy for nerve damage via spinal injuries using GRNOPC1 GRNOPC1: derived from human embryonic stem cells; oligodendrocyte progenitor cells Injected directly into the site of the injury Instead of prescribing a drug, this type of treatment would introduce replacement cells to restore tissues, which would then restore function University of Pittsburgh School of Medicine: hypothesized that muscle-derived stem cells taken from the site of neuron damage, when grown in a medium suitable for nerve cells, could differentiate into neurons and glial cells glial cells also include Schwann cells, which form the myelin sheath Experiment on mice where sciatic nerves were damaged and human muscle derived stem cells were placed at the site After 12 weeks, the nerve had completely regenerated in the treated mice, but not in the untreated mice Can stem cells can replace cells in the brain, treating neurological diseases? It is hypothesized that malfunctioning cells would be removed and replaced with functioning stem cells in order restore full function Stem cells used as replacements must be able to create authentic replacement neurons in order to provide proper function Animal Trials: Stem cells used by taking fetal tissue from mice and transplanting the tissue into an adult rat eye Showed that the tissues would develop into mature neurons The neurons were found to produce dopamine and reduce or reverse symptoms of Parkinson’s in rats and monkeys damaged areas Human Trials: tissue transplants from aborted fetuses into the striatum of patients Low percentage saw improvement Younger patients with milder symptoms responded well to the grafts Scans indicated that the neurons in such patients had matured and started producing dopamine Another study that involved the grafting of cells both into the striatum and the substantia nigra of three patients showed no adverse effects and some modest improvement in patient movement Based on current research, high hopes for patients with degenerative neurological disorders by treating with stem cells. Through the use of stem cells, patients may be able to replace damaged nerves Much more work to be done, but progress that has been made in studies for conditions such as Parkinson’s and nerve injuries Stems cells can be helpful in neurological disorders in other ways than making neurons; can provide support to the diseased cells, helping them survive or recover We would like to acknowledge Dean Andrew Grosovsky for sharing his knowledge of stem cells to aid in this research. We would also like to thank Kristin Osborne and Velina Batchvarov with the organization of these presentations. Finally, we would like to thank Dr Sigmon for sharing his deep knowledge of stems cells with us and providing feedback. Driving Question: How can stem cells treat neuro-degenerative diseases and nerve damage? Introduction Treating Injuries with Stem Cells: The Geron Case Study Figure 1:This diagram describes the derivation of human embryonic stem cells, as well as the two ways the stem cells can break off into. ( Treating Injuries with Stem Cells: Other Case Studies Asteria: the second FDA-approved company to use stem cells as treatment for neck injuries Thirteen-person study in which the same oligodendrocyte progenitor cells as in the Geron study used Gave participants a 10x stronger dosage This trial only began recruiting people this year. Treating Neurological Diseases with Stem Cells: Parkinson’s Disease Figure 2: This figure describes the process in which induced neural stem cells are created and then modified into neurons ( Figure 3. Images from a human Parkinson's patient before and after fetal tissue transplantation. Red indicates dopamine concentration before and after treatment ( Conclusions BeforeAfter Acknowledgements