“Mom, why do I have cancer and other kids don’t?”
Patrick was only 3 when he was diagnosed with neuroblastoma. Three-year-olds should be playing superheroes, not getting chemotherapy through a central line. This was June 2006, and we were in shock that on this gorgeous summer day – when all of our friends and our friends’ their children were at the pool – our new oncologist was using words such as “neuroblastoma” and “surgery” and “stem cell transplants.” Most memorable were the odds of survival, which were diplomatically put: either zero or 100 percent.
WE KNOW NOW that neuroblastoma is one of the most common childhood cancers in children younger than 5. It’s a tricky cancer, comprised of the body’s own immature nerve cells that grow out of control. It can be highly curable in the rare cases when it is caught early – but if stage four neuroblastoma relapses, it does so with a vengeance.
Over the next nine months, we followed standard frontline therapy with surgery, high-dose chemotherapy and a stem cell transplant. Patrick wore his Spider-Man costume often – so much so that we even cut a slit in it so his nurses could access his chemotherapy catheter.
Despite receiving chemotherapy so toxic that he had to receive his own stem cells back to survive the toxicity, Patrick still had detectable cancer in his bone marrow. We decided to try immunotherapy treatments at Memorial Sloan Kettering Cancer Center, which would help Patrick’s immune system act like soldiers to fight off the disease. Despite those terrible, often painful treatments (and uprooting our family to live part-time in New York City for two years), life was precious and amazing. Summers were spent outside under a clear blue Georgia sky, and winters were busy with school and siblings and time at home.
Immunotherapy kept Patrick in remission, but he could remain on the therapy for only two years. We had our last trip to New York in the spring of 2009, and left with no evidence of disease and plenty of hope.
Then three months later, relapse – with no known cure.
He was 6, and Spider-Man had given way to Legos and Star Wars. Still, “Cancer’s my job, mom,” he said. “In the hospital, I get treatment and that’s my job, and then I come home and I’m done.”
When you’re a parent of a child with cancer, you do everything possible to chase down that cure. And we did. Patrick would undergo four metaiodobenzylguanidine (MIBG) treatments – targeted radiation therapy – and participate in every clinical trial for which he qualified. Patrick’s initial treatments had followed the standard recipe for how neuroblastoma should be treated. But after relapse, there is no guidebook, no standard treatment. And estimates put the risk of relapse as high as 60 percent in kids with high-risk disease.
THE RESEARCH just isn’t there, which may seem strange considering the billions that go into cancer research as a whole every year just in the United States. Although legislation such as the 2003 Pediatric Research Equity Act requires companies developing drugs for adults to also develop them for children, an unintended loophole has gutted the effectiveness of the PREA.
PREA requires research if a cancer in an adult arises in the same organ in children, but cancers in children are for the most part different from the cancers that occur in adults. Children don’t get lung cancer, prostate cancer or breast cancer—so companies aren’t required to conduct pediatric clinical trials on drugs developed for those diseases, even though those same drugs could treat a childhood cancer in a different organ.
And even though a cancer such as neuroblastoma is one of the most common pediatric cancers, children with cancer make up only a tiny part of the population of cancer patients in the United States – only 1 percent. It’s a harsh reality for children with cancer, but it’s simply not profitable or a priority for companies to pursue these latest therapies for kids, especially when a clinical trial process could last decades.
The RACE for Children Act (House Resolution 5858, Senate Bill 3239) plans to change that. Introduced by U.S. Sens. Michael Bennet, D-Colo., and Marco Rubio, R-Fla., along with U.S. Reps. Michael McCaul, R-Texas,, G.K. Butterfield, D-N.C., Chris Van Hollen, D-Md., and Sean Duffy, R-Wyo., the bipartisan, bicameral bill would update PREA to give kids with cancer access to clinical trials of the most promising cancer drugs with molecular targets.
We need to change our cancer nomenclature. We need to distinguish cancers not by the organ involved, but by its molecular properties.
IT’S AN IMPORTANT distinction. Targeted therapy takes aim at the specific molecules that speed tumor growth and progression—and these molecules often are shared by both pediatric and adult cancers. As an example, a cancer such as Patrick’s that arose on his adrenal gland shares molecular targets (such as the ALK gene) with adult non-small-cell lung cancer. Under the current PREA, researchers would not be required to run clinical trials with pediatric cancer patients because of the differing primary sites of the cancers. RACE would fix this loophole, focusing on the shared molecular target instead.
Health-care organizations across the United States are supporting this legislation, including the Coalition for Pediatric Medical Research, Children’s Hospital Colorado; the Dana-Farber Cancer Institute; Duke University Medical Center; Augusta University Health, Massachusetts General Hospital for Children; the MD Anderson Cancer Center; Nemours Children’s Health System; NYU Langone Medical Center; Rady Children’s Hospital-San Diego; St. Jude Children’s Research Hospital; and Texas Children’s Hospital.
Memorial Sloan Kettering Cancer Center and the Children’s Hospital of Philadelphia, both hospitals where Patrick received treatment, also are on that list. More than 100 pediatric cancer organizations – including Press On (www.pressonfund.org), the foundation we started with Patrick in 2006 – also are in support.
When Patrick asked me that tough question—why me?—I told him, “I honestly don’t know.” But I explained that was the kind of thing Press On was funding, research to find out why some kids have cancer and others don’t – and to help cure it.
On his ninth birthday, Patrick told us that he wasn’t scared. And on that birthday, Jan. 9, 2012 – surrounded by family, friends, nurses and doctors in the comforting surroundings of Children’s Healthcare of Atlanta, where his journey began – he found his cure.
Patrick has been gone now for more than four years – our curious, funny boy. Sometimes when people ask why we continue to press on, I share this story: When he was in first grade, Patrick was asked what he wanted to be when he grew up, and he said he wanted to heal people. Now that he’s gone, we believe we are his healing hands here on Earth.
WE AREN’T doctors, but we can heal people for Patrick by raising funds that allow clinicians and researchers to do what they do best.
With its partners, Press On has facilitated more than $7.9 million in grants to date to St. Jude Children’s Research Hospital; Children’s Healthcare of Atlanta; The Children’s Hospital of Philadelphia; Memorial Sloan-Kettering Cancer Center; Children’s Hospital Los Angeles; the Georgia Cancer Center at Augusta University; and Ronald McDonald House Charities of Augusta.
We recognize that were it not for the work that went into pediatric cancer research 20 years ago, we wouldn’t have had our time with Patrick – so brief, yet a longer time than would have been possible without that research. The RACE for Children Act can help multiply the impact of our dollars so we can pay forward the gift of time.
(The writers are the founders of Press On, whose mission is to identify feasible and groundbreaking alternative therapies for childhood cancers. Its partners include the Arms Wide Open Childhood Cancer Foundation; the Augusta University Foundation; The Band of Parents Inc.; Brooke’s Blossoming Hope for Childhood Cancer Foundation Inc.; Cannonball Kids Cancer; Janus Research Group Inc.; the I Back Jack Foundation Inc.; the Isabella Santos Foundation; the Rising Tide Foundation for Clinical Cancer Research; and Zev’s Fund.)