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’s 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 and 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 numbers of patients required who fit the study parameters and have their own cord blood stored. However preliminary data shows that cord blood infusions are safe and effective in improving in the neurological outcomes of cerebral palsy patients and have been submitted for publication. They are applying to the FDA for 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, as well as 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 have completed very promising animal model trials for cerebral palsy demonstrating the benefits of cord blood infusions and neurological improvement. In March 2016 Australia have started their first clinical trial for the treatment of 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, randomized placebo controlled trial using matched allogeneic cord blood in 96 children has been completed in the Republic of Korea lead 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 in particular for brain injury where there the only other therapies available are adjunctive therapies such as physiotherapy, occupational and speech therapy.
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