The following is a compilation of two articles about Cerebral Palsy in the newsletter of Parents Guide to Cord Blood. It has been edited for focus and length.
Cerebral palsy is a diagnosis that covers a variety of sequelae to brain injury that occurred in utero, at birth, or in infancy. The actual cause of the injury could be bleeding in the brain (stroke), excessive pressure from fluid in the brain (e.g. hydrocephalus), loss of oxygen, traumatic brain injury, etc. The clinical symptoms of cerebral palsy may include impaired movement associated with exaggerated reflexes, floppiness or rigidity of the limbs and trunk, abnormal posture, involuntary movements, unsteadiness of walking, or some combination of these. Children with cerebral palsy often have learning disabilities as well. Here in the United Sates about 1 in 300 children have cerebral palsy (CDC).
Dr. Joanne Kurtzberg's group at Duke University was using cord blood transplants to cure children suffering from metabolic disorders. Along the way, the researchers found that children receiving cord blood transplants also experienced significant improvements in their motor and cognitive skills. The surprising neurologic improvements seen in children receiving cord blood transplants led to the proposal to give cord blood stem cells to children with brain injuries.
In 2010 Duke launched a Phase 2 clinical trial for spastic cerebral palsy, a category which comprises about 80% of children with cerebral palsy (CDC). Having already demonstrated that cord blood therapy for cerebral palsy was safe, this trial was designed to measure the efficacy of the treatment against a control. The trial was designed to enroll 120 children, and by the end of 2013 had achieved 65% accrual, with 40 patients who had completed their one year follow-up visit. Between this and other clinical trials, plus children who do not meet any trial enrollment criteria but receive compassionate care off-trial, Dr. Kurtzberg estimates that about 125 children per year receive autologous cord blood therapy at Duke.
There is growing acceptance in the medical community that stem cells derived from cord blood have value as a therapy for pediatric neurologic injury. When the FDA approves this therapy, there should be thousands of children worldwide who already have a cerebral palsy diagnosis and have their own cord blood in storage. At Duke University Medical Center, the current therapy for cerebral palsy requires the child to have his or her own cord blood (Sun et al. 2010). In theory the use of donated cord blood could open the door to therapy for many more cerebral palsy patients who do not have their own cord blood in storage. In practice, therapy with donated cord blood introduces two steep hurdles. The first hurdle is that the patient must take immunosuppressive drugs for the duration of therapy, in order to prevent the patient's body from immediately rejecting the donor cord blood. This in turn makes it necessary to take extra precautions against infection while the patient is immune compromised. This patient management is significantly more complicated and expensive than the autologous therapy which only requires medical supervision on the day of infusion.
Another hurdle is the process of finding a matched cord blood unit. This hurdle has many components - genetic, political, and financial. Patients from ethnic minorities and mixed racial backgrounds will have a harder time finding matched donors. But what can be even harder than finding the matching unit, is convincing a public cord blood bank to release that unit for a therapy that is not a traditional hematology or oncology indication. The cost of a public bank sample is over $30,000. That would be a staggering burden for a family pursuing a clinical trial that is not covered by insurance.
Families will probably find that the easiest way to access a cord blood unit for a child who does not have his or her own cord blood in storage, is to use sibling cord blood. The odds of a perfect match naturally occurring with a full sibling are 25%, but a usable sample is closer to 50%.
In summary, the potential market of current patients who could benefit from cord blood therapy for cerebral palsy numbers in the thousands and is distributed worldwide. Someday our approach towards all cord blood banking may be modified by the realization that a certain fraction of infants will need to use their cord blood stem cells for therapy. Dr. Kurtzberg of Duke has suggested that the cord blood donated to public banks should be "embargoed" for the first year of storage, in case the donor needs it back.
Frances Verter, PhD, is the founder and director of the Parent's Guide to Cord Blood Foundation, a 501(c)(3) charity and educational resource. The main public outreach activities of the Foundation are its website ParentsGuideCordBlood.org, and its free educational brochures. Dr. Verter has been monitoring the cord blood banking industry and answering parents' questions about cord blood since 1998. In her first career she was an astrophysicist, but she became motivated to raise awareness of the stem cells in umbilical cord blood after her daughter Shai died of cancer. The foundation she started provides educational services to both parents and physicians, and maintains unique databases about the cord blood banking industry.
