Hudson Cell Therapies facilitates Australian first UCB stem cell treatment for cerebral palsy
By Rob Clancy, staff writer
For the first time in Australia, a child has received an infusion of their own umbilical cord blood stem cells to treat cerebral palsy (CP), and Hudson Cell Therapies played a crucial role in making it happen.
The therapy, not yet approved as a standard treatment for CP anywhere in the world, was administered to Zara, a 6-year-old with CP, at Melbourne’s Monash Children’s Hospital in April.
The Therapeutic Goods Administration (TGA) granted Zara compassionate access to the therapy under the Special Access Scheme, with support from Cerebral Palsy Alliance, Cell Care, and Hudson Institute of Medical Research.
Global research has found that cord blood treatment can help repair a brain injury and support improvements in gross motor skills for some children with CP. Zara’s medical team is hopeful that cord blood treatment, in combination with rehabilitation to train motor skills, will lead to an improvement in her communication, movement and interactions skills.
Zara’s umbilical cord blood, collected at birth and stored with Cell Care, was transferred to Hudson Cell Therapies in the Monash Medical Precinct, where the team in the lab processed the cells and prepared her infusion.
Cord blood stem cells dampen inflammation
Background: Mary-Anne Thomas (Victorian Minister for Health), Paul Mercurio AM.
CP is the most common physical disability in children. In Australia, a child is born with CP every 20 hours. The condition is caused by damage to the developing brain and cord blood treatment works by dampening inflammation and encouraging the brain to heal itself.
Director and CEO of Hudson Institute of Medical Research, Professor Elizabeth Hartland AM, paid tribute to the collaboration and coordination that exists between Monash Health, Monash University and Hudson Institute.
“Through the creation of Hudson Cell Therapies, we’re able to facilitate trials and treatments all in the one location, bringing benefits to scientists, clinicians and ultimately patients, from the latest in cell therapy technology.”
First to receive own stem cells for cerebral palsy
To date, a total of 13 children have been treated with cord blood for CP in Australia. Twelve children were part of a Phase 1 clinical trial where they received umbilical cord blood from a sibling. Zara is the first child to be treated for CP in Australia with their own cord blood and outside a clinical trial.
Cerebral Palsy Alliance estimates hundreds of Australian families have travelled overseas to access cord blood therapy treatment for cerebral palsy privately whilst access is not available locally.
It took a year to plan and obtain approvals for Zara’s infusion, coordinating the treatment between Monash Children’s Hospital, Cerebral Palsy Alliance, Hudson Institute of Medical Research and Cell Care.
On the day of her treatment, the cord blood was hand-delivered from the lab to specialist nurses at Monash Children’s Hospital Cancer Centre for infusion.
After preparation, the cord blood was delivered via a drip in Zara’s arm, much like a regular blood transfusion. During the 30-minute infusion, Zara was engaged in play therapy and accompanied by her parents, Fiona and Michelle, and younger sister, Tilly.
The infusion was completed safely, and Zara’s progress will be monitored in the months and years ahead, as part of her standard clinical care. It can take several months to see any improvements after treatment.
Stars aligned for breakthrough treatment
Zara’s treating Paediatric Neurologist, Professor Michael Fahey, said the stars aligned for Zara: “She had cord blood stored at birth and had a good dose available; she was the right level of cerebral palsy – she was still walking; her parents were very keen to have this treatment; and she was young enough that we could infuse the cells and know that she would get the maximum benefit.”
“This treatment is something new in Australia; no one has received autologous cells – their own cells – in this setting before. The procedure was meticulously planned to the minute, and we didn’t encounter any surprises. Staff across neurology and haematology-oncology at Monash Children’s Hospital collaborated closely to provide this treatment, and I am really proud of their work.”
“Based on the studies, we are expecting that there will be a level of improvement in the way that Zara walks, the way that she communicates, and the way she interacts with others,” Prof Fahey said.
Researchers hope the case will serve as a stepping-stone towards establishing more research, as well as access options for children with CP to receive cord blood in Australia.
Funding for a large-scale trial is yet to be secured, but Victorian Minister for Health, Mary-Anne Thomas was impressed with what she saw when she and Zara’s family returned to Monash Children’s Hospital: “Victoria’s public health system is world leading and this remarkable, pioneering treatment that Monash Health has provided to little Zara, is proof of that.”
“We know how lucky we were to be able to make this treatment happen for Zara. We hope that this is the start of stem cell treatment becoming something that is routinely available for other children with cerebral palsy in Australia.”
“While it’s still early days, we already think we are seeing improvements in Zara’s movement and balance. As her parents, we know that we can’t cure her cerebral palsy, but like most parents, we wanted to do anything we could to make her life even just a tiny bit easier.”
Michelle Forrest, Zara’s mum
“Umbilical cord blood for cerebral palsy is an evidence-backed treatment option, proven to be safe and improve movement skills for children.”
“We want to see more children, like Zara, have the opportunity to be treated with cord blood in Australia and not need to rely on overseas treatment options.”
Dr Megan Finch-Edmondson, Senior Research Fellow and Program Lead, Regeneration, Cerebral Palsy Alliance
CEREBRAL PALSY FACTS
- Around 34,000 Australians have cerebral palsy.
- Every 20 hours, an Australian baby is born with a brain injury that underlies cerebral palsy.
- It is a permanent, life-long condition with no cure.
- Causes include oxygen starvation to the brain (asphyxia), infections, prenatal stroke or head injuries during birth or within the first few years
- Up to 50 per cent of children with cerebral palsy were born preterm.
- Babies born at 32 weeks or earlier are at a particularly high risk.
Collaborators | Monash Children’s Hospital, Cerebral Palsy Alliance and Cell Care.
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