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Children's Brain Tumor Charity

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“Supporting research as the charity does brings an opportunity to develop new therapies to improve survival rates of children with brain tumours, which can be translated into better quality of life and reduced toxicities related to therapy in surviving patients."

Gemma Llargues

Fast facts

  • Official title: XX

  • Lead researcher: XX

  • Where: XX

  • When: XX

  • Cost: XX

  • Research type: XX

 

What is it?

A research project focused on identifying and exploiting medulloblastoma tumour growth dependencies as a means to find novel therapeutic approaches. The study involves comprehensive screening of drug libraries, together with biological profiling, to identify drug-sensitivities in this specific group of aggressive tumours.

“One third of medulloblastoma patients are still incurable, and those that survive can suffer dramatic side effects due to current therapies, such as neurological disorders. This is still a big area that needs to be studied and a lot of improvement needs to be made.”

Dr Laure Bihannic, St Jude Children's Research Hospital

Why is it important?

Despite comprehensive multi-modal treatments, survival rates for medulloblastoma patients have not improved significantly in recent times. MYC-amplified Group3 medulloblastomas are among the hardest to cure, with very low survival rates. To increase cure rates, it is of crucial importance to develop new targeted therapy strategies, based on an understanding the biology of the disease.

Who will it help?

The results from our studies will support the development and administration of better clinical treatments for children with aggressive forms of medulloblastoma.

“This research will lead to new personalised or patient-centred therapies with increased survival and lower therapy-associated late effects.”

Professor Steve Clifford, Northern Institute for Cancer Research, Newcastle

Milestones

Achieved

A series of genetic medulloblastoma dependencies have been identified, which can be targeted with small molecule inhibitors and used as therapeutic strategies against medulloblastoma. The drugs and molecular targets identified have been pharmacologically and genetically validated to prioritise therapies for further development.

Publication in Lancet

BBC News Health Breakthrough

Upcoming

The therapies identified are now being advanced into pre-clinical trials, to prioritise those inhibitors to be used in the clinic. Our studies will provide the essential pre-clinical evidence to support the development of regimens for use in early clinical trials, with the aim of improving outcomes for children with highly-aggressive medulloblastomas.