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Accelerating drug development for children with cancer

No. 56 | 14/11/2017

Which drugs should be given priority for being tested against which childhood cancer types? A new European Union Innovative Medicines Initiative 2 project has been launched to address this question. Highly distinguished research institutions, big pharmaceutical companies and small biotech companies have joined forces to develop better models for investigating childhood cancer. The new research consortium is called "ITCC-P4" and will be coordinated jointly by the German Cancer Research Center (DKFZ) and the pharmaceutical company Eli Lilly.

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Despite major progress in research made over the past decades, approximately 6,000 children and adolescents still die of cancer in Europe each year. Therefore, there is an urgent need for new effective drugs that are particularly effective against cancer entities that typically occur in this age group. Among the many innovative compounds that are being developed primarily to treat cancer in adults, it is a challenge for scientists to identify those that are also active against pediatric cancers. For this purpose, high quality and meaningful results from preclinical research are crucial.

This is the goal of the new ITCC-P4 project (Innovative Therapies for Children with Cancer - Pediatric Preclinical Proof-of-concept Platform) that will receive over €16 million in funds over the next five years – as a public-private partnership supported by the European consortium 'Innovative Medicines Initiative' (IMI). The funds will be provided half by the European Union (Horizon 2020) and half by the participating enterprises.

"From the perspective of a pediatric oncologist, the ITCC-P4 project is ideally suited to accelerate science-driven development of new drugs against high-risk cancer entities in children – in a huge Europe-wide joint effort," says Professor Dr. Stefan Pfister from the Hopp Children's Tumor Center (KiTZ) at the NCT Heidelberg and the German Cancer Research Center (DKFZ). Pfister and Dr. Louis Stancato of Eli Lilly share the project leadership of ITCC-P4.

The scientists in the consortium plan to establish 400 patient-derived preclinical models for ten pediatric cancer types that often cannot be treated successfully to date. To this end, tumor cells from the individual patients will be transferred to mice in order to fully explore their biology and use them for preclinical testing.

The overarching goal of the initiative is to accelerate drug development for children with cancer and to develop new biomarkers in order to use the drugs tailored to each individual case. The tumor models that will be established at the participating academic research centers will subsequently be shared to build a comprehensive sustainable platform to use these models for future testing of drugs for pediatric cancer patients. The researchers expect that patient-individual tumor models will enable them to obtain more meaningful results for preparing clinical trials than it is currently possible based on the commonly used tests in cancer cells in the Petri dish or in genetically homogeneous mouse strains.

Many of Europe's most distinguished research institutions are participating in the ITCC-P4 project:

  • Deutsches Krebsforschungszentrum/ Hopp Children's Tumor Center at NCT (Heidelberg), Institute for Cancer Research (London), European consortium for Innovative Therapies for Children with Cancer, Institute Gustave Roussy (Paris), Alleanza Contro il Cancro (Rome), Zurich University (Zurich), Medical University Vienna, Fundació Sant Joan de Déu Barcelona, Academic Medical Center (AMC) (Amsterdam), Children's Cancer Research Institute (Vienna), Institute Curie (Paris), Charité Berlin, Princess Máxima Center (Utrecht)
  • Well-established small and medium-sized enterprises (SMEs): EPO-Berlin-Buch GmbH, XenTech (Evry, France)
  • A member of the European Biopharmaceutical Enterprises (EBE): PharmaMar (Madrid)
  • And members of the European Federation of Pharmaceutical Industries and Associations (EFPIA): Lilly, Roche, Pfizer, Bayer, Charles River.


With more than 3,000 employees, the German Cancer Research Center (Deutsches Krebsforschungszentrum, DKFZ) is Germany’s largest biomedical research institute. DKFZ scientists identify cancer risk factors, investigate how cancer progresses and develop new cancer prevention strategies. They are also developing new methods to diagnose tumors more precisely and treat cancer patients more successfully. The DKFZ's Cancer Information Service (KID) provides patients, interested citizens and experts with individual answers to questions relating to cancer.

To transfer promising approaches from cancer research to the clinic and thus improve the prognosis of cancer patients, the DKFZ cooperates with excellent research institutions and university hospitals throughout Germany:

  • National Center for Tumor Diseases (NCT, 6 sites)
  • German Cancer Consortium (DKTK, 8 sites)
  • Hopp Children's Cancer Center (KiTZ) Heidelberg
  • Helmholtz Institute for Translational Oncology (HI-TRON Mainz) - A Helmholtz Institute of the DKFZ
  • DKFZ-Hector Cancer Institute at the University Medical Center Mannheim
  • National Cancer Prevention Center (jointly with German Cancer Aid)
The DKFZ is 90 percent financed by the Federal Ministry of Education and Research and 10 percent by the state of Baden-Württemberg. The DKFZ is a member of the Helmholtz Association of German Research Centers.


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