A six-year-old girl named Zara has become the first child in Australia to receive an infusion of her own stored cord blood as treatment for cerebral palsy outside of a clinical trial. Experts hope that this milestone will pave the way towards broader availability, therefore removing the need for Australian children with cerebral palsy to travel elsewhere for treatment.[1]
Zara’s story
Zara was born prematurely, and was diagnosed with cerebral palsy after she began missing milestones. Before she was born, her parents had made the decision to store her cord blood with an Australian stem cell bank, due to her mother having type 1 diabetes. After the cerebral palsy diagnosis, her parents wondered if the stored cord blood might help her.[1]
Initially, they considered travelling overseas, as cord blood treatment for cerebral palsy is not generally available in Australia. In fact, excluding Zara, only 12 other children have received this treatment, and all were part of a clinical trial. Conversely, Australian charity Cerebral Palsy Alliance (CPA) estimates that hundreds of families have had to spend tens of thousands of dollars to seek this treatment abroad.[1][2]
Planning and securing the required approvals for the treatment took more than a year. It was a coordinated group effort, involving not only Zara’s parents and her paediatric neurologist, Professor Michael Fahey, but also the stem cell bank, CPA, Melbourne’s Monash Children’s Hospital and Hudson Institute of Medical Research and several local politicians.[1]
Zara received her cord blood in April of this year, and her progress will be monitored in the months and years ahead. The biggest gains are expected between three and six months after the treatment, but Zara’s parents are already seeing improvements in her balance and movement, as well as reduced muscle stiffness. “At soccer training, she can weave between the cones easier,” her mother said. “At physio the other day, for the first time in her life, she walked up and down four steps without having to hold on to the rail.”[1]
What does cord blood treatment for cerebral palsy entail?
After careful thawing and preparation, the cord blood treatment is given as an infusion, through a drip into the arm. The process takes 20-30 minutes and is very similar to a standard blood transfusion.[3][1] It is believed that the cord blood treatment works by helping the brain form new pathways; these are then strengthened and refined by a cycle of rehabilitation, done after the treatment.[3] A recently-published review paper confirms, through extensive data analysis, that cord blood treatment followed by rehabilitation improves motor skills significantly more than rehabilitation alone.[4][5]
The treatment can use either autologous (the child’s own) cord blood, as in Zara’s case, or allogeneic (a donor’s). In the latter case, a sibling is often the cord blood donor. At Cells4Life, we have released six cord blood samples for treatment of a sibling with cerebral palsy, most recently in 2024; sibling donors were also used in the single clinical trial performed in Australia.[2]
Why is this treatment not more widely available?
The science supports the effectiveness of cord blood treatment for cerebral palsy, particularly in younger children as well as those with less severe forms of the condition.[4][5] However, it is not yet approved in any country, including Australia and the UK, and as such is not readily available as a therapy option. This is because large-scale phase 3 clinical trials are needed for approval, and are expensive and complicated to organise.[5]
In the meantime, outside of clinical trials, cord blood treatment for cerebral palsy is available only through expanded access or compassionate use pathways. This can require either individual approval for the treatment, as in Zara’s case, or participation in an existing expanded access programme, such as the one at Duke University in the USA.
Both of those routes to treatment are likely to require a child having access to either their own banked cord blood, or a sibling’s. This is because while there are public cord blood banks, the cord blood stored there may be available for use only for approved treatments or clinical trials.
Banking your baby’s cord blood at birth could therefore be essential to ensure access to this treatment if they, or a sibling, develop cerebral palsy. To learn more about cord blood banking, as well as other potential uses of cord blood for other diseases and conditions, fill in the form below to request your free guide.
References
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