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4 March, 2021 - 14:19 By Tony Quested

Hope of new treatments for rare childhood cancer

The first proof of the origin of a rare childhood cancer has been discovered by researchers at the Wellcome Sanger Institute in Cambridge, the Princess Máxima Center for Pediatric Oncology in the Netherlands and collaborators.

The study involved malignant rhabdoid tumour (MRT) and found that MRT arises from developmental cells in the neural crest whose maturation is blocked by a genetic defect. 

The neural crest is a temporary group of stem cells from which many other cell types, including bone, cartilage and certain types of neural cell, arise during development. The team also identified two drugs that could be used to overcome this block and resume normal development, bringing hope of new treatments for the disease.

MRT is a rare soft tissue cancer that predominantly affects infants. Although these tumours may arise in any part of the body, they usually form in the kidney and the brain. MRT is one of the childhood cancers with the poorest outcomes.

The rarity of MRT, with only 4-5 cases per year in the UK, combined with its aggressiveness, make clinical trials extremely difficult. 

Until now, the origin of MRT has not been known and no reliably effective treatment currently exists. This new study sought to discover the root of MRT in the hope of identifying new treatments for the disease.

Dr Sam Behjati, co-lead author of the study from the Wellcome Sanger Institute, said: “We began our enquiry into the origins of malignant rhabdoid tumours in late 2019, so we have gone from hypothesis to discovery of origin to possible treatments for the disease in just over a year. 

“This was possible due to all the leading-edge tools available to us, from organoid technology to single-cell mRNA sequencing to drug screen databases. I hope this study will serve as the blueprint for discovering the origin of other childhood cancers and, ultimately, lead to better outcomes for children affected by these awful diseases.”

Dr Jarno Drost, co-lead author of the study from the Princess Máxima Center for Pediatric Oncology, said: “To be able to identify where malignant rhabdoid tumour (MRT) comes from for the first time is an important step in being able to treat this disease, but to confirm that it is possible to overcome the genetic flaw that can cause these tumours is incredibly exciting. 

“The fact that two drugs already exist that we think can be used to treat the disease gives us hope that we can improve outcomes for children diagnosed with MRT.”

Professor Richard Grundy, Chair of the Children’s Cancer and Leukaemia Group, said: “It is fantastic to see this collaborative research bearing highly translatable outcomes in a childhood cancer with a currently poor prognosis. 

“It emphasises the significant benefit of a National Tumour banking system, that allows collection of rarer tumours and in turn, the best use of such precious tissue through agreement of the CCLG Biological Studies committee that oversees this resource. For this to result in such a meaningful outcome gives new hope to children with malignant rhabdoid tumour (MRT).”

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