1. Billett, Amy MD

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Vice President Joseph Biden's Cancer Moonshot will help accelerate the wide range of clinical, academic, and business efforts to improve cure rates and care for cancer patients. With cancer the leading disease-related cause of death in American children, I came away from the summer Moonshot summit in Washington, D.C., more convinced than ever it is critical for pediatric oncology to be fully integrated into all phases of the Moonshot rather than addressed separately.

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Meanwhile, the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act, introduced in Congress with bipartisan support, would help young children gain access to lifesaving treatments, propel research into childhood cancers, and maximize survivors' quality of life.


As we observe September as Childhood Cancer Awareness Month, here are additional ways to integrate pediatric cancer into the Moonshot.


Drug Development

None of the last 57 drugs FDA approved to treat adult cancers had a pediatric application. Likewise, over the past 23 years, the FDA has approved only three drugs specifically developed to treat pediatric cancers. This must change. Despite well-intentioned legislation like the Pediatric Research Equity Act and the Best Pharmaceuticals for Children's Act, holes in the process remain.


When submitting a drug to the FDA for approval, drug companies, for instance, are required to include pediatric data only if the adult cancer in which the drug was tested and shown to be active also occurs in children. Yet the vast majority of adult cancers are not the cancers that children get. However, as we are now learning, seemingly distinct pediatric and adult cancers might share the same molecular pathways and/or genetic aberrations. If we simply switch the focus to molecular pathways, rather than type of cancer, suddenly the door opens to integrating pediatric studies early in the drug development process.


Precision Medicine & Immunotherapy

It is time to bring pediatric cancer fully on board precision medicine and immunotherapy initiatives. There is growing data in pediatrics that genetic sequencing of children's cancers points to enough potential avenues of treatment and other clinically relevant information to make such sequencing appropriate. In addition, the NCI is expected to open a pediatric MATCH (Molecular Analysis for Therapy Choice) trial later this year.


We must accelerate this research, offer appropriate sequencing to young patients and, as noted above, include pediatric cancers early in the drug development process. Immunotherapy is another promising frontier, with CAR T-cell therapy for children with leukemia and the recently approved monoclonal antibody, dinutuximab, for children with neuroblastoma. We must continue this path, exploring these avenues of immunotherapy for pediatric patients while also pushing forward with new clinical trials investigating the pediatric potential of checkpoint inhibitors, such as the immunotherapy that successfully attacked President Carter's cancer.



Prevention is a major theme of the Cancer Moonshot. Yet cancer prevention doesn't align well with pediatric cancers, whose onset, unlike a number of adult cancers, is largely unrelated to lifestyle and/or environmental exposures. For pediatric cancer, prevention means preventing the harmful side effects and late effects of treatment. Here precision medicine offers great promise. Because it is targeted therapy, rather than broadly toxic chemotherapy and radiation, it has the potential to greatly reduce late effects of treatment. While exploring its potential to increase cure rates with less toxicity, we also must explore precision medicine's potential to determine which patients are most at risk for side effects and late effects and tailor treatment accordingly. We also must continue to look for ways to cut back on toxic treatments of pediatric cancers we are most successful at curing.


Survivorship & Palliative Care

At a time when the overall cure rate for childhood cancers hovers around 80 percent, there are now about 800,000 survivors of pediatric cancer in the U.S. Research finds that two-thirds of survivors will face at least one late effect of treatment, and one-third will confront at least one serious late effect of the toxic treatment that cured them. The 30-year-old who had cancer at age 10, for instance, may suffer treatment-related cardiac disease usually seen in much older adults.


With an expected lifespan that extends decades beyond their cancer cure, the needs of survivors of pediatric cancer must be addressed. At the other end of the spectrum, we must also do a better job providing palliative and end-of-life care for the 20 percent of children who do not survive their cancer. As discussed at a recent Institute of Medicine workshop, access to high-quality palliative care for pediatric cancer is thoroughly inadequate. While advances in treatment prolong life, many children still eventually die of their cancer. With that comes a need for palliative care that includes symptom management and a focus on quality of life as well as supportive end-of-life care.


Patient- & Family-Centered Care

Pediatrics is the poster child in the delivery of patient- and family-centered care. With true patient- and family-centered care, however, patients and families would be "at the table" with the health care team designing effective care delivery, not just receiving that care.


If I don't solicit input from patients and families, for instance, I might schedule a return visit to clinic for blood work. Perhaps that child and family would prefer to have a visiting nurse come to the home and have the results sent to the clinic This would eliminate travel time (and costs), allow the child to spend more hours in school, and allow the parent to spend more time at work, with less overall disruption and economic impact on the family. How do we design care with you rather than for you? If we want to deliver the optimal best care, we must continuously ask patients and families for guidance on how to do that.



Partnerships and collaboration are emerging as major themes of the Cancer Moonshot. Because all pediatric cancers are rare, collaboration is a hallmark of pediatric cancer research. The Children's Oncology Group is a wonderful example of this, as are a growing number of consortia, but they are not sufficient. With many successful treatments involving a combination of medications, drug companies are going to have to work with each other. If IBM's Watson works with 10 cancer centers, do all 10 centers include pediatric cancer?


The bottom line is it is much more difficult to harness special interests than to incorporate them into the whole. For the Moonshot to truly propel efforts to improve outcomes for children with cancer, we can ill afford to relegate pediatric cancer to the sidelines.


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