Research Topic

Pioneering for the Orphan Cancer Domain: Community-Facing Roadmaps for the Most Rare Pediatric and/or Adult Cancers with Poor Survival Outcomes

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About this Research Topic

This Research Topic brings the biomedical and clinical research communities in collaboration with the patient community in order to develop roadmaps for the rare pediatric & adult cancers with longstanding poor survival outcomes. The goal of this topic is to summarize both the (i) resources and (ii) ...

This Research Topic brings the biomedical and clinical research communities in collaboration with the patient community in order to develop roadmaps for the rare pediatric & adult cancers with longstanding poor survival outcomes. The goal of this topic is to summarize both the (i) resources and (ii) potential therapeutic targets/approaches for a given rare cancer. Roadmaps will also be required to establish highly specific “next steps” for the field, which may in many instances involve patient community – academic – industry partnerships.

The motivation of this Research Topic is the recognition that a better understanding of rare cancers (malignant or benign) could offer a tremendous opportunity for Oncology drug discovery. Currently these tumors are understudied because there is a lack of resources. For instance, study section referees often are conflicted by the unmet clinical need that contrasts with the observation that project proposals are underpowered when patient sample collections are small or disease models are limited (i.e., cell lines, patient-derived xenografts).

Examples of such rare and orphan cancers are NF-1 associated pilocytic astrocytoma, intracranial germ cell tumor, hepatoblastoma and epithelioid sarcoma.

Roadmap reviews should address the full path from patient identification to drug approval, including:
• Existence or potential for patient registries that extend across all age ranges
• Presence of centralized or distributed biobanks
• Potentially actionable biomarkers assessed from clinical samples (not cell lines)
• Potential therapeutic targets and corresponding experimental model systems
• Demographics and biological features of cell lines & animal models for the given disease
• Availability of team science efforts to accelerate the drug discovery process

Roadmaps that address cancer predisposition syndromes and cancer surveillance and specialized considerations for therapy (e.g., Bloom Syndrome) are also welcome.

By way of this unique Research Topic, it is hoped that patient-centered partnerships bring critical mass to rare pediatric and adult cancers, resulting in scientific discovery and clinical trials, in a way that has been seen for cystic fibrosis, Pompe disease, chordoma and other orphan diseases.

The image for this Research Topic is original artwork from Luke Avery and was inspired by research turning small round blue childhood muscle cancer cells (rhabdomyosarcoma) into non-cancerous muscle cells (red, elongated).


Keywords: Childhood, pediatric, cancer, orphan, pediatric priority review voucher


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