The Murdoch Children’s Research Institute (MCRI) clinical research, reported in Cytotherapy, found that fully matched umbilical cord blood cells from one kid could be safely transplanted into a cerebral palsy sibling. The Cerebral Palsy Alliance Research Foundation and Cell Care cord blood bank financed the study.
Only one significant adverse reaction was reported as a result of the trial, while six of the twelve subjects experienced moderate adverse reactions. In the hospital, all adverse reactions were effectively treated.
According to Dr. Kylie Crompton of Murdoch Children’s Research Institute, while there is no treatment for cerebral palsy, cord blood cells have the potential to enhance brain injury and gross motor function due to their ability to stimulate healing processes and regenerate particular tissues in the human body.
“Our study discovered that infusing matched sibling cord blood cells into children with cerebral palsy is a relatively safe procedure,” she said. “However, it should only be performed in tertiary hospitals with facilities to treat the infrequent adverse reactions.”
The phase I clinical experiment included 12 volunteers from throughout Australia, aged 1–16 years, who received a sibling’s cord blood cells and were followed up on for 12 months after the infusion.
The majority of the patients, who were followed for a year, had usual developmental growth for children with cerebral palsy. Three months following the infusion, three children showed improvements in gross motor function. One year later, any changes were less noticeable.
According to Dr. Crompton, the most significant gross motor improvements were noticed in younger children who had not yet reached 90% of their anticipated gross motor skill capacity.
“This shows that early intervention may be more effective,” she said. To find out if this is the case, we and other researchers are preparing more experiments to better understand these consequences.”
Cerebral palsy is caused by brain injury in utero, during delivery, or in the first years of life. The congenital condition affects around two out of every 1,000 live births worldwide, making it the most prevalent physical handicap in childhood. Every 20 hours, a kid in Australia is born with cerebral palsy.
Professor Dinah Reddihough of Murdoch Children’s Hospital said the findings were a significant step in establishing the safety and viability of using umbilical cord blood in the treatment of cerebral palsy. However, not all children with cerebral palsy will have fully matching cells from their siblings.
“Investigating a cell product that is available to the larger cerebral palsy community, rather than only those with matched siblings who have cord blood banked,” she says.
“Further research is also required to determine whether different types of cerebral palsy are more receptive to change after cord blood cell infusion, what cell dose is required, and whether numerous infusions will produce a better outcome.”
Charlotte, who was diagnosed with cerebral palsy at the age of two, couldn’t hold a water bottle or a pen, couldn’t speak vocally, and hadn’t taken her first steps. Laura West, the mother who took part in the trial, said it felt like “a light switch had been turned on.”
Laura, on the other hand, stated that Charlotte’s developmental progress had stalled after the injection.
Laura opted to collect and retain cells from her umbilical cord when pregnant with her youngest daughter, Emma, to be later transplanted to Charlotte. Laura enrolled in Cell Care’s cord blood collection and storage programme for siblings of a cerebral palsy child. The free initiative ensures that these stem cells are gathered in a properly audited and government-approved way, allowing them to be used in future clinical trials in Australia.
Laura stated that the changes in Charlotte, now seven years old, were noticeable after the cell infusion began.
“Charlotte was pretty static in her development and advancement until the injection, and then we noticed this dramatic shift,” she explained.
“It was impressive at first, then settled into a gradual improvement.” Charlotte was always fed via tube, but she rapidly learned to sip from a bottle. We never expected her to be able to properly grip a pencil, yet all of a sudden she was.
“She progressed from sitting, standing, and running while supported up with pillows.” It meant a lot to see her so self-sufficient and capable of doing these things on her own. Charlotte’s development reduced to baby steps in the early aftermath because the rate of change was so rapid.
“We’re delighted the researchers will keep working to determine if the intervention can be more beneficial in the first few years of life, giving hope to other families.”
Professor Nadia Badawi, Cerebral Palsy Alliance Chair of Cerebral Palsy Research at The University of Sydney, described the work as a watershed moment that provided a fresh knowledge of how umbilical cord blood cells could one day be used to better the lives of children with the disorder.
“We are grateful to the children and families who took part in this research and helped pave the way for many future trials,” she said.
“At Cerebral Palsy Alliance, we are dedicated to conducting research that will empower persons with cerebral palsy and their families.” We know from surveys that people with cerebral palsy and their families desire additional research into potential therapies, and this trial is a crucial step forward in establishing the safety of utilising cord blood cells in an Australian hospital setting.”