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Update on clinical trials investigating cord blood as a treatment of cerebral palsy
Over the past few years, a number of clinical trials have been initiated to investigate whether cord blood (CB) could be used in the treatment of cerebral palsy (CP). The rationale for these trials comes from preclinical studies which demonstrated CB could lessen the impact of hypoxic brain injury and stroke in animal models.31 Additionally, in animal models and in human patients with inborn errors of metabolism treated with an unrelated donor CB transplant, CB has been shown to engraft and differentiate in the brain, facilitating neural cell repair.32 The largest of these clinical trials is a Phase 2 trial being conducted by Dr. Joanne Kurtzberg at Duke University in North Carolina.33 This trial was initiated in 2010 with the aim to treat 120 CP patients with their own cord blood. It was open to children with spastic CP, between the ages of 1 – 6 years, who had access to their own cord blood and who had a Gross Motor Function Measurement (GMFM) scores between Level II – IV. The GMFM score is a measure of the severity of CP. Patients accepted into this trial underwent an initial evaluation, then were administered either CB or a placebo. One year later, patients were re-evaluated. Patients initially treated with CB were subsequently infused with placebo, whereas those initially treated with placebo were subsequently infused with their own CB. After another year, patients were again re-evaluated. To date, patient accrual into this study has been completed; all patients have been infused with CB and are currently awaiting final evaluations.At the beginning of the trial, Dr. Kurtzberg hypothesized that, in the setting of brain injury, infusion of the child’s own CB would facilitate neural cell repair resulting in improved function in pediatric patients with cerebral palsy. The targeted improved function was defined as a >30% increase in the predicted GMFM score at 1 year. Preliminary results from this trial were announced recently at the International Cord Blood Symposium in June 2015.34 In response to treatment with CB, it appears that the >30% increase in the predicted GMFM score was met, suggesting that CB does provide a benefit to children with CP. Additionally, MRI studies of the brain were conducted to determine whether, or how, CB infusion affected brain development. Results demonstrated that children treated with CB had increases in white matter connectivity within the brain and that the increase in connectivity correlated with functional improvement. Though the recently announced results are preliminary, they are exciting and support further investigation for the use of cord blood to treat cerebral palsy and other neurological disorders.