A faster lane towards effective therapies of childhood brain cancer

Scientists of the „Hopp Children’s Cancer Center at the NCT Heidelberg” (KiTZ), the German Cancer Consortium (DKTK) and the German Cancer Research Center (DKFZ), in collaboration with colleagues from Seattle, U.S.A., have built a collection of tumor models that can be used to test anticancer drugs for treating childhood brain cancer. The biobank and database can be accessed by scientists worldwide and is an important resource for cancer research. Children suffering from brain cancer may also benefit from it. The researchers have now published their results in NATURE MEDICINE.
The „Hopp Children’s Cancer Center at the NCT Heidelberg” (KiTZ) is a joint institution of the Heidelberg University Hospital and the German Cancer Research Center (DKFZ).

Microscopic examinations of tumor tissue (histology) make it possible to differentiate several subtypes of brain cancer. However, analyses of tumor genomes and investigations at molecular level have shown that tumors of similar histology can vary considerably in their biological behavior and their response to anticancer drugs.

Marcel Kool, who is a staff scientist at KiTZ and a group leader at DKFZ’s Pediatric Neurooncology Division, explains: “This means that a new targeted anticancer drug that is very effective in one patient may fail completely in another patient even though the tumors look very similar under the microscope. Since every ineffective therapy is a risk for the young patients and may also be extremely stressful for their minds and bodies, we need model systems that we can use to test the effectiveness of new therapy approaches in tumors with specific characteristics at molecular level before applying them in children.” Mouse transplant systems have been very successfully used as models in cancer medicine for adults because they offer a natural environment for brain tumors. In the artificial environment of a culture medium, the tumor cells most often fail to grow, or if they do, they show a completely different behavior than they do in the patient.

“In collaboration with our colleagues from the Seattle Children’s Hospital, the University of Washington and the Fred Hutchinson Cancer Research Center in Seattle, U.S.A., we have generated and characterized 30 tumor models representing 14 subtypes of pediatric brain tumors for the new biobank,” said Sebastian Brabetz, a KiTZ scientist and first author of the publication. He added: “It has become evident that our tumor models reflect the brain tumors in patients very well. Thus, we can expect highly meaningful results from tests of novel substances in these tumor models.” The scientists plan to further expand the biobank over the next few years to represent as many brain tumors as possible including rare subtypes.

A biobank with worldwide access
The working groups from Heidelberg and Seattle have made the tumor models and the corresponding data (r2platform.com/pdxexplorer) available in the biobank (http://www.btrl.org/) for the whole scientific community in order to advance cancer research worldwide. For translational oncology, the biobank is an important element bridging the gap between basic research and clinical application. In addition, it is an important resource for further research in cancer medicine from which ultimately all patients, children as well as adults, can benefit greatly. Gathering models of pediatric brain tumors in a biobank which is accessible for all scientists around the globe is a first and important step within a project called ITCC-P4 (Innovative Therapies for Children with Cancer – Pediatric Preclinical Proof-of-concept Platform), which was launched in 2017. Stefan Pfister, Director of the Preclinical Program at KiTZ and also a senior physician at Heidelberg University Hospital, explains the relevance of this biobank in the worldwide battle against cancer in children: “Among the many innovative compounds that have usually been developed to treat cancer in adults, it is very challenging to identify those that are also active against pediatric cancers. Within the ITCC-P4 project, which is undertaken as a joint venture with pharmaceutical companies, the European Union therefore supports the development of systems that make it easier and faster to identify effective cancer medications for children.”

Original publication:
Brabetz S. et al. 2018 “A biobank of patient-derived pediatric brain tumor models” https://rdcu.be/9Phy  . DOI: https://doi.org/10.1038/s41591-018-0207-3


The German Cancer Research Center (Deutsches Krebsforschungszentrum, DKFZ) with its more than 3,000 employees is the largest biomedical research institute in Germany. At DKFZ, more than 1,000 scientists investigate how cancer develops, identify cancer risk factors and endeavor to find new strategies to prevent people from getting cancer. They develop novel approaches to make tumor diagnosis more precise and treatment of cancer patients more successful. The staff of the Cancer Information Service (KID) offers information about the widespread disease of cancer for patients, their families, and the general public. Jointly with Heidelberg University Hospital, DKFZ has established the National Center for Tumor Diseases (NCT) Heidelberg, where promising approaches from cancer research are translated into the clinic. In the German Consortium for Translational Cancer Research (DKTK), one of six German Centers for Health Research, DKFZ maintains translational centers at seven university partnering sites. Combining excellent university hospitals with high-profile research at a Helmholtz Center is an important contribution to improving the chances of cancer patients. DKFZ is a member of the Helmholtz Association of National Research Centers, with ninety percent of its funding coming from the German Federal Ministry of Education and Research and the remaining ten percent from the State of Baden-Württemberg.



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