13 September 2018

Great progress made in investigating special stem cells that could be used treat children with neuroblastoma

stem cells to treat neuroblastoma

Neuroblastoma is one of the most common types of cancer seen in childhood with around 100 new cases diagnosed every year.


The severity of the disease can vary greatly. In about 20% of neuroblastoma tumours there is a change in a gene called MYCN.  This is known to make the tumour more aggressive as it causes it to grow rapidly. Children with this type of neuroblastoma undergo a gruelling course of chemotherapy, surgery and radiotherapy, yet still the prognosis is very poor.  More effective therapies are desperately needed.

The project

In this study, Professor Arturo Sala is investigating whether a type of stem cell that has been engineered to carry a cancer-killer gene can be used to find and eliminate neuroblastoma cells in patients.  These cells are already being manufactured for a large clinical trial in adult lung cancer to assess whether they can find and clear tumour cells.

Prof Sala and his team is extending this work by assessing whether the modified stem cells can clear tumours in mice transplanted with human neuroblastoma cells taken from patients.

The advantage of using this stem cell-based therapy over some other approaches is that all neuroblastoma cells should be killed, independently of their genetic makeup (tumours are made up of cells with different active genes making them harder to kill). If the team validate the effectiveness of the modified stem cells in mouse models of neuroblastoma, a clinical trial could be established very quickly.

Progress update

Professor Arturo Sala and his team at Brunel University have made great progress in investigating special stem cells that could be used treat children with neuroblastoma.

His team received neuroblastoma cells from six patients, which they used to create an infinite variety of human neuroblastoma cells to better test the cancer-fighting ability of these cells in test tubes. This led to some exciting results. They found that a combination of the modified stem cells and the cancer drug, bortezomib, killed more neuroblastoma cells compared to the modified stem cells or cancer drug alone.

To test if these cells could find neuroblastoma cells in the body, they next injected a mouse model of neuroblastoma with these modified stem cells and used specialist imaging techniques to show that the stem cells successfully found the neuroblastoma tumour and infiltrated it. They are now testing to see if these injections reduced the tumour size and whether the stem cells would hunt out human neuroblastoma cells that have been transplanted into mice in a similar way. If they can show that the cells find and eliminate human neuroblastoma cells, they would be one step closer to establishing a clinical trial.

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