Translational Research in Pediatric Cancer

Dear Colleagues,

Pediatric Oncology of the 20th century is seen as a story of success. Indeed, the improvement in survival (in high-income countries) showed an impressive jump from barely 20% in the 1960s to 70% in the year 2000. This success is based on many variables, including increased knowledge of the disease, cooperative work, and the unique biology of developmental cancer making these tumors particularly sensitive to DNA damage agents such as chemotherapy and radiation therapy. The progress, however, although impressive, has been stalled for the last 20 years, especially for some tumors such as central nervous system cancers or sarcomas. For instance, DIPG continues to be a devastating and incurable children’s brain tumor despite more than 250 clinical trials over 30 years of classical (chemo and radiation) 20th century oncology.

Following the DIPG example, 21st-century precision therapy or immunotherapy approaches have also failed. We need to admit that pediatric oncology has not provided hope to families affected by DIPG, or many other cancers such as metastatic/relapsed sarcomas, etc. Hence, a radically different approach is desperately needed.

Recently, many cooperative groups have contributed to a radically innovative biological redefinition of DIPG and many other tumor entities from the discovery of the biological roots such as histone H3 mutation as the primary (unique in oncology) event in DIPG, to the cell of origin in the form of an oligodendroglial precursor cell of the developing brainstem. Even more recently, the interactions of the tumor cell with normal neurons have been described for DIPG and functional dependencies identified. Animal models to reproduce the identity of the tumors have been developed, and initial analyses at single cell level have been reported. Nevertheless, this has not been sufficient to transform survival prospects for patients.

Following the paradigm proposed by V. Narayanamurti and T. Odumosu (Harvard University Press, 2016) of research as virtuous cycles in which some periods are dominated by knowledge creation (discovery), and others are dominated by the creation of new tools or processes (invention), I suggest that now is the time to move our increased knowledge on pediatric cancers as unique entities into new tools to better diagnose, prognosticate, and treat: Translational Research. This Special Issue will highlight the many investigations focused on pediatric cancers that promote new tools and processes: from deep mechanistic insights relevant to better understand the uniqueness of developmental tumors; strategies to identify diagnostic/prognostic biomarkers; innovative clinical trial designs that can defy the conventional track of drug approval; to unique therapeutic strategies.

Prof. Dr. Jaume Mora
Guest Editor

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• developmental cancer
• translational research
• mechanistic insights
• novel tools for diagnostics
• novel therapeutics