Brain injury stem cell therapy advances

By Dr Michelle Mason

20 July 2016

Current academic views on cord blood banking are still conservative as the lifetime likelihood of developing a blood-related disease requiring transplantation remains low. However, we would like to highlight the enormous growth in perinatal stem cell research, particularly the non-haematological uses of both cord blood and cord tissue stem cells.

The field of regenerative medicine is especially promising. Exciting work is being done both pre-clinically and in clinical trials by world-renowned researchers and clinicians at reputable institutions. There have been noteworthy developments in the area of neurological disorders and brain injury (cerebral palsy, stroke, neuro-degenerative and demyelinating disease) at various clinical centres.

Dr Joanne Kurtzberg from Duke University has been pioneering in the brain injury field. Her work started over a decade ago when many patients who had received stem cell transplants for genetic metabolic disorders had marked improvement in their neurological status. ¹ This inspired her to give children with cerebral palsy their own cord blood. After several positive testimonials, they embarked on a large phase 2 clinical trial at Duke and Georgia Regents University.  This has been extended to include other categories of brain conditions, including autism, paediatric stroke, hydrocephalus and neonatal oxygen deprivation. ²

The results of the trial have taken longer than anticipated because of the difficulty in recruiting the number of patients required who fit the study parameters and have their cord blood stored. However, preliminary data show that cord blood infusions are safe and effective in improving the neurological outcomes of cerebral palsy patients and have been submitted for publication. They apply to the FDA for the standard of care to collect cord blood and treat any suspected birth-related brain injuries.

Duke University is taking on paying patients for autologous cord blood infusions for cerebral palsy. In November 2015, Duke started a second cerebral palsy trial using cord blood from siblings, and a trial in adult acute ischaemic stroke to be treated with non-HLA matched (ABO and Rh matched) cord blood. ³´⁴

Australia, through Monash University and The Ritchie Centre, has completed very promising animal model trials for cerebral palsy, demonstrating the benefits of cord blood infusions and neurological improvement. In March 2016, Australia started their first clinical trial treating cerebral palsy. This is a phase 1 trial of matched sibling cord blood at the Royal Children’s Hospital in Melbourne with funding from the Cerebral Palsy Alliance and Cell Care, Australia’s largest family cord blood bank.⁵

A double-blind, randomised, placebo-controlled trial using matched allogeneic cord blood in 96 children has been completed in the Republic of Korea led by Dr MinYoung Kim. Results showed that umbilical cord blood concomitantly administered with rhEPO ameliorated motor and cognitive dysfunction in children with CP undergoing active rehabilitation, accompanied by structural and metabolic changes in the brain.⁶

Dr Michael Chez at Sutter Health in Sacramento, USA, has been conducting a small randomised, placebo-controlled, blinded autism trial which, in preliminary data, showed the UCB infusion was safe and showed some trends with variable responses.  There was a statistically significant improvement in patients on the Vineland Social Scale. This study should be published later this year. Further studies are warranted to include a larger study group, longer observation and optimal dosing of cellular therapy.⁷

We look forward to stem cell therapies derived from perinatal stem cells becoming the standard of care and an efficacious treatment option for a wide variety of clinical conditions, particularly for brain injury, where the only other therapies available are adjunctive therapies such as physiotherapy, occupational and speech therapy.

To read further visit

Sun J.M., Kurtzberg J. Cord Blood for Brain injury. Cytotherapy. 2015; 17: 775-785

Duke University: A Randomized Study of Autologous Umbilical Cord Blood Reinfusion in Children with Cerebral Palsy

Duke University: assessment of Safety of Umbilical Cord Blood infusions in Children with Cerebral palsy

Duke University: Cord Blood infusion for Ischaemic Stroke:

Min et al .Umbilical Cord Blood Therapy Potentiated with Erythropoietin for Children with Cerebral Palsy: A Double-blind, Randomized, Placebo-Controlled Trial. Stem Cells 2013. 581-591; doi:  10.1002/stem.1304

Sutter Health: Autologous Cord Blood Stem Cells for Autism