Our researchers at University College London Great Ormond Street Institute of Child Heath have developed a new way to deliver drugs that can shut down cancer-promoting mutations in neuroblastoma. The findings show that the new delivery method, which uses tiny bubbles to deliver their cargo directly to the tumour cells, could form the basis for a new treatment for this common childhood cancer.
Our findings show that this approach could be a new potential therapy for neuroblastoma. The next steps would be to develop methods of scaling up production to clinical grade, and to show that the treatment is safe.
Neuroblastoma is the most common solid tumour found in children and accounts for about 15% of all cancer-related deaths in children.
Tumours develop from certain types of nerve cells and are most commonly found in the abdomen. Children who are diagnosed above the age of one often fail to respond to treatment or relapse at a later time, meaning that there is an urgent need for new treatment options.
MYCN is a gene that is associated with poor prognosis and is found to be mutated or overactive in about 20% of neuroblastoma cases. The gene is usually expressed during foetal development and is involved in cell growth and development. Neuroblastoma cells continue to express too much MYCN, leading to uncontrolled cell growth and division and preventing cancer cells from dying.
These researchers have now found a way to silence MYCN by delivering a certain type of genetic material, called siRNA, directly to the tumour cells. They developed nanoparticles – or tiny bubbles – that use the leaky blood vessels around the tumour and certain features that are only present on tumour cells to home in on the tumours.
The research was published in the journal Advanced Functional Materials.
We are funding researchers like Professor Hart to start new cancer cures. This innovative research shows just how important investment in early-stage discovery research is. 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.
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