US scientists share research of children’s cancers to accelerate progress

In collaboration with the Press Association

Scientists at Howard Hughes Medical Institute have developed unique mouse models of 12 children’s cancers that could provide the basis for testing drugs and other life-saving research.

The US institute offered up years of this research in hope that sharing it for free with the scientific community will accelerate research into children’s cancer and potentially new drug development.

The results of the project, published in the journal Nature, cover the genetic make-up of 12 types of childhood tumours, as well as how these cancers react to certain drugs in the lab.

"We want this data at the fingertips of clinicians, translational researchers, and basic scientists," says Dr Mark Dyer, the lead investigator in the study, based at St. Jude Children's Research Hospital in the US. 

"It's a great resource for people interested in moving new therapies forward," he said.

“This remarkable study provides an incredibly valuable new resource for scientists studying childhood cancer.” Professor Richard Gilbertson, Cancer Research UK

The most common way to make an experimental model of cancer in mice is to inject tumour cells under the mouse’s skin. 

Many of the tumours grown in mice kept the complex genetic makeup found in the original human sample, suggesting they are a good representation of the disease.

But Dyer’s team wanted to grow the tumours in the tissues they started in, as the development of a tumour is significantly influenced by its environment.

They did this successfully in 97 models and then compared molecular and cellular details of the resulting tumours to those of the original patient.

This study is important because not all tumour cells in a tumour are genetically identical. "It's a mixture of different cells," said Dyer.

Tumour cells left behind after treatment are often responsible for cancer coming back. But treatment can change their genetic makeup.

Dyer said that researchers can now "capture that complexity".

Professor Richard Gilbertson, a Cancer Research UK-funded children’s cancer expert, said: “This remarkable study provides an incredibly valuable new resource for scientists studying childhood cancer.”

He said that the number of experimental models produced by the scientists and the depth of genetic information that they provided was particularly impressive.

“They will help us better understand the genetics and treatment of childhood cancer,” he added.

The team then used the experimental models to test 156 cancer drugs and examined their effect on the tumour and the tumour cells left behind.

They found one drug that was particularly effective against a rare type of childhood cancer.

“Using these models the team has already identified a potential new treatment of the soft tissue cancer, rhabdomyosarcoma,” said Gilbertson.

The researchers now hope that other teams across the world will use these models to find other drugs effective against this range of children’s cancers.

“The fact the researchers have made the resource freely available to the scientific community will further increase the positive impact of the study for children with cancer,” said Gilbertson

The types of childhood cancer the team created models for were:

  • Neuroblastoma  
  • Osteosarcoma
  • Rhabdomyosarcoma
  • Retinoblastoma
  • Wilms tumours
  • Ewing sarcomas
  • High-grade sarcoma
  • Malignant rhabdoid tumour
  • Synovial sarcoma
  • Renal medullary carcinoma
  • Desmoplastic small round cell tumor
  • Epithelioid sarcoma

References

Dyer, M. et al. (2017) Orthotopic patient-derived xenografts of paediatric solid tumours. Nature doi:10.1038/nature23647