Cord Blood Stem Cells to Treat Pediatric Stroke

Cord Blood Registry (CBR) has announced the launch of an FDA-regulated clinical trial to investigate the use of autologous stem cell therapy in children who have been diagnosed as victims of prenatal or perinatal pediatric stroke. This trial will be conducted at the Florida Hospital for Children and is a Phase 1 trial. It is the first trial to examine the use of newborn autologous stem cell therapy in the treatment of pediatric stroke. Hopefully, this trial will lead to Phase 2 studies that will further assess safety and measure efficacy in the use of stem cells to improve common symptoms of this condition.

According to data compiled by the American Stroke Association, strokes occur in about one in every 3,500 new born infants, and is one of the leading causes of death in children between ages 1 and 19. Approximately 60 percent of those children who survive as stroke will have permanent neurological deficits, most commonly cerebral palsy and hemiparesis or hemiplegia, which refers to total or partial paralysis on one side of the body. Other long-term disabilities caused by a stroke occurring around the time of birth can include epileptic seizures, spasticity and bladder control issues. Stroke risk in children is greatest in the first year of life, and peaks during the perinatal period, approximately the few weeks before and immediately after birth.

“There is a critical need for the development of new treatments as the incidence and prevalence of pediatric stroke has increased over time,” said Dr. James Baumgartner, pediatric neurosurgeon and principal investigator of the trial. By supporting this and other trials and investing in the types of regenerative medicine research that hold significant potential to meet the future needs of families, CBR is helping advance newborn stem cell research and therapies.”

Recruitment is underway to enroll 10 children between the ages of 6 weeks and 6 years who experienced a stroke in utero or immediately after birth, and who have a CBR-processed cord blood unit that was collected at birth. Subjects will first receive a baseline neurologic evaluation which includes brain imaging, evaluation of epilepsy, nerve impulses, and bladder control issues, which will help assess the overall severity of the stroke’s impact prior to treatment. After this evaluation is complete, eligible patients will receive a single autologous stem cell infusion, with follow-up testing to occur after six and 12 months. The hypothesis is that the autologous stem cells will help repair the damage incurred during the stroke.

Stroke risk factors, symptoms, prevention efforts, and treatment are often different in children than those in adults. More research is needed to better understand the unique aspects of diagnosing and treating strokes in children. Families affected by pediatric stroke face numerous challenges when treating the most common symptoms, such as epileptic seizures, spasticity and bladder control issues.

First line treatment for epilepsy includes drugs known as anticonvulsants. These drugs are difficult to accurately prescribe and administer to children and seizures may still breakthrough even when anticonvulsants are administered. Common side effects such as extreme drowsiness can affect the quality of life for children and their parents. First-line treatment for spasticity is physical therapy which not only has variable efficacy but may be difficult to access and may not be covered by insurance for extended timeframes. First-line treatment for bladder related issues is catheterization, which can be cumbersome and difficult to manage.

“Our Cerebral Palsy Family Network (CPFN) has been following stem cell research and the impact it can have on children born with cerebral palsy whether it is by in-utero stroke or other brain damage. Because of our extensive reach within the CP community, our families have been asked and have participated in NIH studies including the funding of research projects. These clinical trials are critical to making a better life for children with neurological deficits,” said Janice Godwin, Executive Director of CPFN.

Once the initial 10 children are recruited, infused and evaluated at Florida Hospital for Children, the preliminary results could be available in less than three years.