Trial Aims for Safer Bone Marrow Transplants

The Children’s Cancer Foundation is supporting the first nationwide trial of new technology that aims to dramatically improve the chances for children with otherwise incurable leukaemias.   

For children with the most high-risk forms of leukaemia who do not respond to conventional treatments, stem cell transplantation is the only available cure.

However, transplant success relies on getting the closest possible genetic match for each patient, ideally a brother or sister.

“There’s probably about 10% of kids who we just can’t find a good match for and in those cases one alternative is to use a parent as a match,” says Associate Professor David Ziegler who is coordinating the trial from the Sydney Children’s Hospital.

“But the problem is that in normal circumstances you only get half of your genes from each parent. At best it’s only going to be a 50% match.”

A transplant from a parent donor carries increased risks of serious complications, some of which can be fatal.

However, recent research breakthroughs have developed a new technology that allows doctors to select out the cells from a parent that cause transplant complications.

“This technology is about manipulating the parent’s donated stem cells to get rid of those cells which are going to create problems and prevent those mismatched stem cells from attacking the recipient,” says Associate Professor Ziegler.

“It will give those patients a much safer and more effective treatment. There’s really no more high-risk medical procedure that we could do than this sort of transplant and the aim is to drastically reduce the risk of toxicity.”

All the Australian paediatric bone marrow transplant centres in Australia are collaborating with the three-year trial.

“The plan is for 24 patients to really test this as a viable treatment technique. Once that is done this would potentially become a standard therapy that could be available to any patient in that situation,” says Associate Professor Ziegler.

The Children’s Cancer Foundation is providing funding to coordinate the trial nationally. Dr Ziegler says that this financial support is vital.

“The government pays for things that are already proven and we know that work but with this cutting-edge treatment and technology we have to do these trials first. Each oncology centre is very experienced at opening and managing patients in trials, but what isn’t done so commonly is coordinating those trials nationally,” he says.

“So without this support from the Children’s Cancer Foundation we wouldn’t be able to run these sorts of trials, they just wouldn’t happen.

“It’s really providing that critical resource, that critical infrastructure that allows us to do this – and most importantly to do it nationally and coordinate it around Australia.”

Dr Ziegler says the study aims to improve the chances of survival for children who undergo stem cell transplant therapy and increase the number of children who can access this potentially life-saving treatment.

“This is really testing a technology that allows us to much more safely do transplants of bone marrow for children who otherwise wouldn’t have a very good donor.”

Stem Cell Trial image