**6. Conclusions**

A meta-analysis on the efficacy of all reported stem cell trials for children with CP was recently performed, demonstrating a statistically significant intervention effect when patients were followed short-term to 6 months following treatment [145]. Furthermore, the effect was greatest in the trials using UCB, and overall, the treatment effect highly favors the use of UCB

**Table 1.** Current clinical trials being conducted, or recently completed, using umbilical cord blood in regenerative

medicine therapies for the management of cerebral palsy and ischemic brain injury in the newborn.

**Study title Main objective Institution Treatment Current status Trial** 

Duke University, United States

General Hospital of Chinese Armed Police Forces, Beijing China

Infusions autologous volume reduced cord blood cells

Mesenchymal stem cells derived from umbilical cord blood

A pilot study to test feasibility of collection, preparation and infusion of a baby's own umbilical cord blood in the first 14 days after birth if the baby is born with signs of brain

118 Umbilical Cord Blood Banking for Clinical Application and Regenerative Medicine

To compare the efficacy of cell therapy and rehabilitation treatments for cerebral palsy patients

injury

Information obtained from ClinicalTrials.gov.

Cord blood for neonatal hypoxic-ischemic encephalopathy

Efficacy of stem cell transplantation compared to rehabilitation treatment of patients with cerebral paralysis

(CP)

**identifier**

NCT00593242

Active, not recruiting

Recruiting

The first autologous transplant of UCB for pediatric ischemic stroke has recently been reported [146]. The work reports a child with right spastic hemiplegia who received 250 million UCB mononuclear cells at 5 years of age. At 3 months after treatment, there was an improvement in motor control, and further improvements were observed at 18 months, however, no change was

**Figure 2.** Forest plot showing the gross motor function changes from UCB transplantation for treatment of established

with or without rehabilitation to treat children with CP (**Figure 2**).

cerebral palsy (adapted with permission from Novak et al. [145]).

Cerebral palsy is caused by injury to the developing brain, with the timing and severity of the insult underlying the heterogeneity of CP. Clinical trials are already underway to treat established CP with UCB mononuclear cells with some positive results, however. It is widely appreciated that treating brain injury as early as possible will demonstrate the most profound benefits. Given the good safety profile of UCB therapies, with the low incidence of transplant rejection, due to the increased number of immature progenitors cells and naïve immune cells, it is clear that UCB is a safe source of cells for transplantation. Preclinical data are accumulating exciting evidence for the mechanisms of neuroprotection by stem cells, and meta-analysis of clinical trials shows that UCB cells mediate significant improvement for children with CP. The immediate imperative is to optimize the benefits of UCB therapy by conducting well-planned strategic animal studies followed by human clinical trials that can further inform the use of targeted neuroprotective cell therapies for the prevention or repair of perinatal brain injury in order to provide long-term improvements for children after compromised pregnancy or birth.
