Look Into The Future Of Childhood Cancer Research

Despite the progress of the last half century there remain a number of challenges in childhood cancer. The focus of research in certain patient subsets with very high cure rates will be on quality of life endpoints. For example, retinoblastoma is curable in nearly 100% of cases, so preservation of sight and reduction of second malignancies (not survival) are now considered to be the primary goals and endpoints, and trials avoiding enucleation and eliminating external beam therapy are now the norm.

One would hope that future therapies for childhood cancer will be developed which would be more rational, less empirical and less toxic, relying more on strategies for growth control (e.g., anti-angiogenesis) and regulation of gene expression and cell proliferation, and/or induction of apoptotic pathways or blocking of anti-apoptotic signals, than on cytotoxic or ablative treatments. Assuming that deregulated and/or mutated cellular proto-oncogenes or loss of tumour suppressor genes are the proximate cause(s) of most forms of childhood cancer, then the genes and/or their protein products will very likely be the targets for the next generation of paediatric anti-cancer agents, many of which will likely be orphan drugs for orphan diseases.

With advances in translational research, the pie (universe of childhood cancer patients) will be divided into smaller but more homogeneous slices than ever before. International collaboration will probably be required in a substantial segment of cancer types in order to obtain sufficient patient numbers to conduct randomised trials. Enlightened partnerships between industry and academia, with the assistance of the FDA and NCI, will be needed for efficient development of new agents.

Finally, the skill sets necessary to conduct paediatric cancer research are expanding. Traditionally the field involved paediatric haematolo-gist/oncologists, surgeons, radiation oncologists, pathologists, nurses, clinical research associates, pharmacologists, epidemiologists and biostatisti-cians. Today, diagnostic imagers, bench scientists, geneticists, pharmacists, clinical psychologists, health economists and others also play significant roles in the research. In the future, other fields of expertise will surely need to be added to the team. The cooperation of a multi-disciplinary team and prompt referral of patients to paediatric cancer centres participating in clinical trials will be critical to achieving future goals of refining and improving therapy.

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