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Autologous umbilical cord blood is an experimental cell therapy developed by Duke University, specifically through the Marcus Center for Cellular Cures, for the treatment of autism spectrum disorder (ASD). The therapy involves a single intravenous infusion of a child's own umbilical cord blood that was collected and cryopreserved at birth. The biological rationale for this treatment is based on the hypothesis that cord blood-derived cells, including various stem and progenitor populations, can modulate neuroinflammatory processes and promote neural repair or protection in the brain. Clinical development progressed through a Phase I safety and feasibility study (published in 2017) to a Phase II randomized, placebo-controlled trial (published in 2020). While the Phase II trial confirmed the safety and tolerability of the infusion, it did not demonstrate a statistically significant improvement in the primary outcome of social communication across the overall study population.
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