Sibling Cord Blood Therapy for Cerebral Palsy
It has been a decade since the first child received an infusion of his own cord blood stem cells to treat cerebral palsy, and finally this therapy is becoming more widely available to children who do not have their own stem cells in storage. Now it is possible for children with cerebral palsy to be treated with cord blood stem cells from a sibling.
Dr. Joanne Kurtzberg has told this story each time she gives a talk: In the course of giving cord blood transplants to children with metabolic disorders, her team at Duke University Medical Center noticed that their patients also had remarkable improvements in cognitive development. This inspired her to give children with cerebral palsy and other acquired neurologic disorders their own (autologous) cord blood stem cells on a compassionate basis.
As a result of that pioneering work, a number of testimonials emerged of children who had made dramatic functional improvements after receiving their own cord blood stem cells. But in order to measure the efficacy of the cord blood stem cells, they had to be tested against a control group in a phase 2 trial. The first two phase 2 trials of autologous cord blood for cerebral palsy launched in the United Sates in 2010: one at Georgia Regents University (NCT01072370) and one at Duke University (NCT01147653). Both trials were designed so that all the children received stem cell therapy, but the control group received the stem cells later.
For the past few years, the cerebral palsy patient community has been impatiently waiting for results from these trials. The frustrating reality is that many families who have a child with cerebral palsy did not bank that child’s cord blood. In fact, the phase 2 trials have taken longer than anticipated because of the difficulty accruing patients who fit the study parameters. Last year the phase 2 trial at Duke closed with about half of the originally planned enrollment and the results have been submitted for publication.
Now the tide is turning. In Nov 2015 a phase 1 trial opened at Duke (NCT02599207) that is treating cerebral palsy patients with sibling cord blood; already 10 children out of 15 planned enrollments have completed therapy.
This month Australia launched their first clinical trial ACTRN12616000403437 of cord blood stem cell therapy for cerebral palsy. This is a phase 1 trial of sibling cord blood, led by the Murdoch Children’s Research Institute. The trial will recruit 12 patients between the ages of 1 and 11 years nationally, and take place at The Royal Children’s Hospital in Melbourne. Lead investigator Dr. Dinah Reddihough commented,
"We are very excited about the announcement of this study which will primarily assess the safety of using sibling cord blood in children with CP. The study will also make preliminary investigations into changes in motor skills in these children."
National regulations in Australia prohibit cord blood that has been donated to public banks from being released for clinical trials; hence all the patients in this study will be relying on sibling cord blood that their parents stored in a family cord blood bank. These studies are using cord blood from a sibling who is a full HLA match for the child with cerebral palsy. The odds that a sibling will be a perfect match are 25% for each sibling.
Every 15 hours, an Australian child is born with cerebral palsy, making it the most common physical disability of children in Australia. This new trial is jointly funded by the Cerebral Palsy Alliance, an international charity on behalf of cerebral palsy patients and their families, and by Cell Care, Australia’s largest family cord blood bank. Dr. Iona Novak head of the Cerebral Palsy Alliance Research Institute, says the importance of the study can not be underestimated.
"Unfortunately we hear of many Australian children with CP and their families travelling overseas to receive unregulated stem cell treatments at great cost… This study, using cord blood which has been stored under Australian government-regulated conditions, is an important first step towards potentially improving treatment."
Cell Care recently commenced offering free collection and storage of cord blood for siblings of children with cerebral palsy. This is an important step towards improving the treatment options available for Australian children with cerebral palsy. This stem cell storage is fully licensed by the Therapeutic Goods Administration (TGA) of the Australian government so that it will be available for use in future clinical trials in Australia.
Ultimately, patients with cerebral palsy will have even more treatment options if they can receive cord blood transplants from matched unrelated donors or even partially matched unrelated donors. This option is available via clinical trials in South Korea, but the inventory of donated cord blood in Korean banks is not likely to match non-Korean children. A path towards cell therapy access for children in the west with cerebral palsy requires that researchers must convince regulatory authorities of the safety of treating brain injury with cord blood stem cells from unrelated donors.