What the Cancer Moonshot Might Mean for Kids

When Lara Weberling of Bakersfield, California, heard about the Cancer Moonshot effort, announced in January by President Barack Obama, the news resonated deeply. As a mother who lost her young son to cancer and as a motivated cancer advocate, she's strongly aware of the need to put cancer in the national spotlight. "To hear the Moonshot is on the national radar makes us feel validated," she says. "It makes us feel on the map."

Moonshot's goal in a nutshell: achieve a decade's worth of progress in cancer prevention, diagnosis and treatment within five years. The initiative is led by Vice President Joe Biden, whose oldest son, Beau Biden, died of brain cancer in May 2015 at age 46. For families coping with childhood cancer, one question is how big of a priority pediatric cancer will be.

"There are just a lot of challenges in dealing with childhood cancers -- a lot of things like surgery that's not as available in these cancers as they are in adults," says Greg Simon, executive director of the White House Cancer Moonshot Task Force. "So this is definitely something that's been on the front burner from the beginning."

Unique Challenges

Dr. Peter Adamson, a pediatric oncologist at the Children's Hospital of Philadelphia, chair of the Children's Oncology Group and a member of the National Cancer Moonshot Blue Ribbon Panel, has met with Biden. "He's very passionate and committed, with an increasing depth of knowledge about the challenges," Adamson says.

On September 7, the Blue Ribbon Panel presented its report encompassing 10 key recommendations. Two relate directly to children, including one recommendation to intensify research on the major drivers of childhood cancer.

"What we've known for a number of cancers is that they're driven by something called a fusion oncoprotein," Adamson says. "That means two genes have come together in a way that was never meant to be. That's a common theme in a number of childhood cancers and in some adult cancers."

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Immunotherapy for Kids

Neuroblastoma, a cancer that originates in the nervous system and most commonly occurs in infants and young children, took the life of 9-year-old Hans Weberling in 2012. Hans was enrolled in several clinical trials, his mother says, including compassionate-use and off-label drug trials.

But he largely missed out on a rising new treatment. By allowing the body's immune system to recognize and get rid of cancer cells, immunotherapy has been called a transformative approach to fighting the disease.

Hans missed the window for a landmark phase III randomized clinical trial of immunotherapy. "He was just diagnosed too soon," Lara Weberling says. Even so, his doctors made immunotherapy available to him, for which she is grateful. Unfortunately, his disease progressed during treatment.

"Most children get treated inside clinical trials because every childhood cancer is fairly unique," Simon explains. "So the Blue Ribbon Panel is also creating a clinical trial network to make it easier for children to get into trials that use so-called immunotherapy drugs."

[See: 7 Innovations in Cancer Therapy.]

Partial Success

Far more children survive cancer than they used to. "If you take our most common cancer in children, acute lymphoblastic leukemia (ALL), a child born in the 1960s with ALL had a less than 10 percent chance of being cured," Adamson says. "That same child born today has almost a 90 percent chance of being cured."

But you have to look deeper, Adamson says. Although 80 percent of children achieve five-year survival, which often translates to long-term survival, childhood cancer remains the leading cause of death from disease in children.

"More importantly, what children have to endure to achieve long-term survival is unacceptable," Adamson says. "During treatment, many treatments are highly toxic. About 4 out of 5 children at some point during treatment experience life-threatening side effects. We've gotten very good at taking care of children with life-threatening side effects, but no one believes that's an acceptable approach to medical treatment."

Boosting childhood cancer research is a near and dear topic for Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center. As a member (and co-founder) of the Coalition for Pediatric Medical Research, Williams' group flew to the District of Columbia to meet with Biden's staff. "The gist of the Moonshot in pediatrics in particular is to address the currently unmet need of cancers we're not curing," Williams says, such as certain subsets of brain tumors and types of leukemia.

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About That Appropriation

Childhood cancer efforts are already outperforming those for adults in at least one Moonshot recommendation. "The good news is that the children's cancer community and the medical community have been much more organized around children's issues and clinical trials and research coordination than the adult community," Simon says.

But children's cancer lags far behind on funding sources. For cancer biomedical research, Adamson says, about 60 percent of money comes from the biopharmaceutical industry and 25 percent comes from the National Institutes of Health and National Cancer Institute. Children's cancer, however, is much more dependent on government funding and philanthropic resources.

For Moonshot recommendations to succeed, Adamson says, "There's going to have to be a substantial increase in the congressional appropriation to the NCI to support this." A sustained investment is needed to have an impact, he says, not just a single year of funding.

"I'm very optimistic that someone of the stature of the vice president will be able, even in this sort of political system that we live in, to sharpen the focus of people around this area," Williams says. "But I do hope this also comes with some real increase in resources."

"Everybody will be happier when we get the actual money," Simon says. "But many of the scientific recommendations that are in the Blue Ribbon Panel report can be done with existing programs by expanding them or focusing them. So the Moonshot will definitely benefit from an increased appropriation from Congress. But there are many, many things that we're doing without waiting for that. And we're also very optimistic that we will get some funding from Congress."

Parents and advocacy groups believe funding is "critically important," Weberling says. "There is a little bit of concern that children's cancers are underfunded because they're a smaller percent of the population that's affected by cancer." Consider the years of potential involved when a 3-year-old has cancer, she says: "We want our 85-year-old grandfathers who have prostate cancer to get excellent care and treatment, too, but there's just a different number of years of life on the line."

Lisa Esposito is a Patient Advice reporter at U.S. News. You can follow her on Twitter, connect with her on LinkedIn or email her at lesposito@usnews.com.