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Hopes for new treatment for neuroblastoma cancer in children

Researchers hope that the technology could also be used to treat a variety of other diseases

An effective new treatment for the most common form of childhood cancer could be available within five years after experiments in mice substantially slowed down tumour growth.

The technique works by shutting down the activity of a genetic mutation which is responsible for about a fifth of neuroblastoma cases.

This form of cancer occurs most commonly in the adrenal glands above the kidneys, or in the nerve tissue that runs alongside the spinal cord in the neck, chest, tummy or pelvis.

The treatment is injected directly into the tumour cells in miniscule “nanobubbles” that are 2,500 times smaller than a grain of salt.

Experiments in mice showed the bubbles slowed down the tumour growth and significantly prolonged the time the mice survived the cancer.

“These findings show that this approach is a new potential therapy for neuroblastoma,” said Stephen Hart, Professor at University College London Great Ormond Street Institute of Child Heath.

“Current therapies such as surgery, radio and chemotherapy are effective at removing the primary tumour but, unfortunately, in many cases the tumour will return at other sites in the body, which is much harder to treat. We hope that this therapy might augment conventional therapies and provide a way of targeting the therapy to these new tumour sites,” he said.

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In a “best case” scenario, the treatment would be found to be safe and effective in humans and enter clinical practice within five years, he said.

However, there was no guarantee that it would work in people and the process could take much longer if it did, he cautioned.

Nonetheless, the nanobubble approach holds promise – both for neuroblastoma as well as a range of other conditions.

“We have been working on this approach for many years and it is starting to show promise for cancers, such as neuroblastoma but also for other genetic diseases like lung disease of cystic fibrosis.”

“We are also working with other colleagues to generate a new class of therapies in skin, liver and kidney so there is a lot of promise from this approach,” Professor Hart said.

Helen Rippon, chief executive at Worldwide Cancer Research, which funded the research, said: “Using new methods, such as nanoparticles, to deliver treatment straight to the heart of cancer is an incredibly exciting area of research. These new results now offer hope to patients and their families by paving the way for effective new treatment options.”

Neuroblastoma is the most common solid tumour found in children and accounts for about 15 per cent of all cancer-related deaths in children.

The study is published in the journal Advanced Functional Materials.

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