Gene therapy to make cells invincible against chemotherapy

04 August 2014

One of the main problems with chemotherapy for children with brain cancer is that the drugs damage the bone marrow.

Researchers at the Kids Research Institute have been working for 10 years to develop a radical new treatment which uses gene therapy to stop this from happening. If successful, the technique will enable children to receive much higher drug doses than they can currently tolerate – and hopefully have their cancer eradicated.

To do this, the researchers take some stem cells out of the bone marrow and insert a new gene into their DNA which causes them to produce a protein to repair the damage caused by the chemotherapy drugs. Once transferred back to the body, the stem cells are resistant to the side effects of chemotherapy.

The altered genes are transferred back to the right cells of the body through ‘vector technology.’ Based on viruses, the vectors are manufactured at the Kids Research Institute using a specialised cleanroom. This space has its own air supply and to enter it researchers must wear sterile blue suits with hoods, masks and big overshoes.

Though still in its early days, the trial is teaching researchers some valuable lessons about how to implement a gene therapy strategy to try and improve treatments for patients with brain tumours, says Dr Belinda Kramer of the Children’s Cancer Research Unit.

“We know these cancers can be sensitive to the drugs if we give more of the chemotherapy, but at the moment we just can’t do it. For children who’s tumour has relapsed and who can’t ordinarily be given higher doses, this strategy could both reduce chemotherapy side effects and more effectively treat their tumour,” she says.

If the trial is successful, the drug resistance strategy will have applications for many diseases other than cancer. The trial has been funded by the very generous support of The Kid’s Cancer Project.

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