A phase 2 randomized trial of autologous mononuclear cells infused acutely after severe pediatric traumatic brain injury demonstrated a favorable safety profile and effective reduction in long-term brain volume loss.
Traumatic brain injury (TBI) is the leading cause of death in children in the U.S., and neurological disability among survivors contributes to significant lifelong morbidity. Acute care after TBI focuses on controlling and attempting to mitigate the effects of secondary brain injury, although effective neuroprotective therapeutics are lacking. Preclinical studies of autologous bone marrow–derived mononuclear cells (ABMMCs) demonstrated improved control of an aberrant inflammatory response seen in the secondary phase of TBI. Two U.S. children's hospitals have now conducted a phase 2 dose-escalation trial exploring the safety and efficacy of ABMMC infusion within 48 hours after injury in children with acute severe TBI.
Of 47 randomized patient…