Scientists are hopeful that an effective treatment for cerebral palsy using cord blood stem cells could be on the horizon now that the first results of a Phase 2 study have been published. Promising results will encourage more clinical trials which will make the therapy available to more patients.

Scientists' optimism

Dr. Min Young Kim and colleagues at the CHA Bundang Medical Center in South Korea recently published encouraging preliminary results of a Phase 2 study showing efficacy of cord blood stem cells to treat cerebral palsy (CP)1. The randomized, placebo-controlled, doubleblind study was the first of its kind and although the results are promising, more questions remain to be answered.
CP can affect brain and nervous system functions such as movement, learning, hearing, seeing, and thinking. This group of disorders affects as many as 10,000 infants per year in the U.S. and results in lifelong motor and cognitive functional deficits. Symptoms are usually seen before a child is 2 years
old, and sometimes begin as early as 3 months. They can include developmental delays in sitting, rolling, crawling or walking as well as difficulty in sucking or feeding, tight and/or weak muscles, an abnormal gait, tremors or seizures, speech, hearing or vision problems and learning disabilities. There is no cure for CP, only treatment to help the individual become as independent as possible.

Results of the study

Dr. Kim’s team treated 96 children between the ages of 10 months and 10 years. Patients were divided into three groups:

• those treated with HLA-matched cord blood from unrelated donors plus the growth factor erythropoietin (EPO)
• those treated with placebo plus EPO, and
• those treated with placebo alone

EPO was included in the study because of its previously demonstrated neural repair properties. Unfortunately, the investigators did not include a group of patients receiving cord blood alone, which will have to be addressed in a subsequent study.

Motor function and cognitive development changes were measured using a group of well-established tests performed at the initiation of the study and again at one, three and six-month intervals.
• The data consistently revealed superior outcomes in the group that received cord blood compared to the other two groups.

• The beneficial effects appeared in both motor and cognitive function.
• The degree of HLA matching correlated with outcomes: patients receiving closer matched units did better. These results imply that patients receiving autologous cord blood may have even better outcomes. HLA-matched allogeneic cord blood units were used in this study due to the unavailability of previously banked autologous cord blood.

Cerebral palsy has few warning signs

The study results provide optimism for parents of thousands of children suffering with CP and for unborn children at risk of acquiring CP. Since there are few, if any, early warning signs predicting who will be affected by CP, these results should encourage families to consider storing umbilical cord blood when the option is available to them.

Preliminary animal studies

The clue that cord blood infusions may provide a benefit for patients with brain damage due to lack of oxygen come from extensive studies in animal models of hypoxic ischemia from our group and others.
Cryo-Cell International and our research affiliates at Saneron CCEL Therapeutics, Inc. have investigated using cord blood in rodents to test effectiveness for traumatic brain injury, stroke and hemorrhage. The beneficial outcome appears to arise from anti-inflammatory effects as well as by increasing new neural cell and blood vessel formation. Early clinical trials in children are currently underway in the US. A team led by Joanne Kurtzberg, MD, at Duke University conducted a pilot study to determine the safety and feasibility of infusion of autologous cord blood in 184 pediatric patients who had their cord blood privately banked at birth and who subsequently acquired neurological injuries. That investigation demonstrated the safety and feasibility of the approach and has led to an on-going Phase 2 clinical trial to demonstrate efficacy. Initial study reports are expected this year.

Hope for the future

Together the studies demonstrate that cord blood infusions may represent a safe and effective therapeutic alternative for patients with CP. Clearly more work is required to understand the best stem cell source, mechanism of action, cell dose and time of infusion. Most importantly, the promising results will encourage more clinical trials which will make the therapy available to more patients. Cryo-Cell International and Saneron CCEL Therapeutics, Inc. are committed to seeing the therapeutic potential of banked umbilical cord blood in laboratory studies and clinical applications for CP as well as other brain injuries such as stroke, Alzheimer’s and Lou Gehrig’s Diseases.