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Translating Genomics into Action in Pediatric Cancer

The Translational Genomics Research Institute (TGen) hosted the Pediatric Cancer Translational Genomics conference in Scottsdale, AZ on February 6-8. The 180 attendees were described as a “dream team” in pediatric oncology. They represented 29 U.S. states and 11 overseas nations, and their discussions focused on how to translate pediatric genomic data for personalized or precision oncology.

One challenge facing pediatric oncology, as explained by Peter Adamson, MD, chair of the Children’s Oncology Group (COG), is that its funding is currently much more dependent on the federal government than other types of medical oncology. Clinical research in pediatric oncology receives about 99% of its funds from the federal government, while medical oncology receives about 60% of its funds from industry. The business models play out that way because pediatric cancer does not have an economic driver. Pediatric cancer relies on the National Institutes of Health (NIH), which is likely to face a flat or decreased budget in the near future. Increasingly, public-private partnerships and perhaps more collaborative research funding from foundations will be needed to address the funding challenges of pediatric oncology.

The main concerns expressed by speakers and attendees about moving forward with translating genomic data from pediatric oncology were the need for more biopsies and the need for bioinformatics.

Because tumors change over time, they need to be sampled over time. When pediatric oncologists can sample tumors over time through multiple biopsies, they will be able to take advantage of differences at the time of recurrence so the treatment can adapt to the tumor that recurred. Tumors are a moving target, and findings from a biopsy can be game-changing. However, because biopsies pose more than a minimal risk to a child, regulations require that they must have the potential to benefit that child. The research community felt that they need to prove the potential benefits of biopsies to guide treatment decisions for recurrent tumors.

Bioinformatics was another area of concern, including the need to share, organize, store, and annotate information. Four major pediatric sequencing groups were represented: the KIDS (Knowledge Integration & Dynamic-interexchange System) Cloud project, which is a collaboration between TGen, Dell Computers, and the Van Andel Research Institute; the Pediatric Cancer Genome Project, which involves St Jude Children’s Research Hospital and The Genome Institute at Washington University; TARGET (Therapeutically Applicable Research to Generate Effective Treatments) from the National Cancer Institute; and MAGIC (Medulloblastoma Advanced Genetics International Consortium) from Sick Kids Hospital in Toronto, Canada.

“When it comes to access to clinical and pathological data, more is more,” stated Spyro Mousses, PhD, Director of the Center for BioIntelligence at TGen. Data sharing and collaborations were emphasized as a way to move research forward and connect current science with choices for patient treatments. Pediatric oncology includes rare and ultra-rare diseases, and only by pooling resources and data over several years can enough information be developed about these diseases to provide guidance on clinical decisions.

By Kathy Boltz PhD

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