Stem cell trial to begin for children suffering from cerebral palsy


Pioneer Founding member
By Kate Hagan Health Reporter Nov. 23, 2014

About 20 Australian children with cerebral palsy will be infused with their own umbilical cord blood in a trial due to start next year, as physicians warn families against travelling overseas for experimental stem cell treatments.

The long-awaited Australian trial will provide some of the world's first evidence about the safety and effectiveness of using stem cells from umbilical cord blood to repair brain injury that leads to cerebral palsy.

Researchers are waiting on ethics approval for the trial which will provide treatment to families who have chosen to store their child's cord blood at private banks.

In some cases, children with cerebral palsy will be able to receive a sibling's cord blood if this is available.

Cerebral Palsy Alliance head of research Iona Novak said the study, led by the Murdoch Childrens Research Institute, will recruit children from around Australia who have access to privately banked cord blood.

Children aged one to 10 will receive infusions at private blood banks in Melbourne, Sydney and Brisbane, and will be assessed before and after the treatment to check for improvements.

Researchers will be unable to access cord blood from a public bank, which collects blood to treat blood disorders such as leukaemia and cannot be used for untested new therapies.

Associate Professor Novak said the trial was an important first step towards establishing whether stem cells could help repair the brain injury that leads to cerebral palsy, a series of disabilities associated with movement and posture.

Cord blood is rich in stem cells, which have the ability to develop into other cells in the body. They have shown promise in animal and some human studies at repairing damaged tissues, including in the brain, but the evidence is at an early stage.

Trial details come as a new position paper by the Royal Australasian College of Physicians warns families against travelling overseas for experimental stem cell treatments.

Clinics in several countries including Panama, China, India and Russia offer stem cell treatments to children with cerebral palsy but the exact type of cells and their source was often unknown.

College fellow and Melbourne-based paediatric neurologist Michael Fahey said unproven treatments were being offered in some clinics "without adequate scientific rigour or safety controls".

He said he worked with families who were desperate to find a treatment for their children. Many considered stem cell tourism, he said, but decided against it after considering the risks.

Jen Williams said she investigated options for stem cell treatments overseas for her six-year-old son Tom Roach, who has cerebral palsy after being born at 29 weeks' gestation..

"I spent a lot of time second-guessing, am I doing my son a disservice because I haven't looked at this?" she said.

Ms Williams said the family had previously sought treatment outside Australia, including at a specialised clinic in Austria to wean Tom off a feeding tube and annual trips to an intensive physical therapy program in the United States.

But they decided against seeking stem cell treatment overseas after seeking advice from their neurologist, who highlighted safety concerns.

Ms Williams said Tom, who started school this year with a full-time aide, was making terrific progress through speech and physical therapy but stem cell treatments "could be the icing on the cake".

"I know stem cell research is on the cards here in Australia, and maybe it can help Tom one day," she said. Until then, she said, "I could never forgive myself if I made something worse."