Though pediatric cancers account for just one percent of new cancer cases in the U.S., cancer is the leading cause of disease-related death in American children.
No parent should ever have to watch their child battle this disease, so it’s critical that we advance medical research in the field of pediatric oncology to improve cure rates and reduce side effects.
Advancing Pediatric Cancer Research
In recent years, we’ve made great strides when it comes to adult cancers. From immunotherapy for the treatment of melanoma to combination therapies for patients with late-stage disease, we’ve made progress in improving outcomes and prolonging life for adult patients who previously didn’t have many treatment options.
During the first 30 years of treatment for childhood cancer, cure rates increased dramatically. It’s unfortunate to say we haven’t seen this progress continue for pediatric cancers. Cancer therapy in children really has been driven by research, much more so than for adult cancers. With approximately 15,000 patients a year diagnosed with pediatric cancers in more than 30 types of cancer, there are fewer children with each type of cancer than adult types, which makes research all the more important in order to continue the advances in cure.
Though we work diligently to get children to participate in studies, there haven’t been new innovations coming out in pediatrics like there are in adult therapies. In the last decade, the FDA has approved more 62 new medicines for adult cancers. By comparison, over a 55-year period from 1948 to 2003, the agency approved just 15 new drugs for pediatric cancer. In pediatric oncology, there’s been only one new drug approved for use in children in the last decade— Unituxin, or dinutuximab, an immunotherapy treatment for children with high-risk neuroblastoma.
We’ve used the same drugs for the past 30 to 40 years. Though cure rates have increased slowly, and are excellent compared to outcomes in adult cancers, it’s been difficult for us to continue moving forward in recent years. We use a lot of therapies that emerge from the world of adult cancers, but they weren’t really invented for children.
As with many things, funding is the biggest concern for efforts in advancing new cures. Although some pharmaceutical companies are trying to show a good faith effort, there are still few studies of experimental drugs in pediatric oncology and the vast majority of funding goes to clinical trials in adult cancers. Even in the federal government the majority of funding is given to adult cancer research, with less than four percent of funding from the National Cancer Institute devoted to pediatric cancer.
In the last decade, the majority of funding for pediatric cancer outside of the government has come from private donations, families and corporations who have tried to fill the gap. At the Haley Center for Children’s Cancer & Blood Disorders at Arnold Palmer Hospital for Children, we’ve received major gifts from generous Central Florida philanthropists to help advance our cause, including a $5 million gift last year from local philanthropists Roy and Dee Haley that expanded our pediatric oncology services. We now have a bone marrow transplant and cellular therapy program and an early phase treatment center so families have the opportunity to get their children the treatment they need locally.
The Cutting Edge of Therapy
These donations help us advance pediatric oncology, but we need to do even more.
Unituxin, the new neuroblastoma drug, shows that we can make headway in finding treatments for even the most difficult-to-treat pediatric cancers. Though it took nearly 15 years to get it approved, it was a success in our field. Based on this advancement, we’ve focused on trying to use new medications to improve cure rates in children and lower side effects after they’re cured.
The second part of that equation is particularly important: many children are cured but wind up with side effects after they’re treatment. Newer treatments that are designed for specific cancers bring with them the hope of increased cure but also fewer side effects. We want to pave the way for our patients to live good, healthy, and long lives.
At Arnold Palmer Hospital, we’ve been participating in the first national trial in children that uses a patient’s own tumor to determine what treatment is most beneficial based on the molecular analysis of the tumor. This is a “personalized medicine approach,” which we believe is the direction many cancer treatments are headed in the near future. We’ve also added several specialized treatments that aren’t available at every center. Because of this, patients who are at high risk for a relapse now have some options for experimental therapies.
While we’re fortunate to have skilled oncology specialists at Arnold Palmer Hospital, one major challenge we need to overcome is Florida’s lack of a large early phase treatment center. We have 17 small-to-medium sized centers in the state, but until you can get to a certain size it’s hard to provide rare, cutting edge treatments. To meet this need, we have utilized the help of philanthropy to start an early phase treatment center. Without philanthropic support, many children would go without the treatment they need. Florida Medicaid will only cover treatment within the state of Florida and this has a significant impact on the patient population we serve, since half of our pediatric cancer patients rely on Medicaid to fund their cure.
Still, we’ve come a long way. The overall cure rate for pediatric cancer today is over 80%, but our goal is to reach 100% cure rate, and the only way to achieve this is with more research. Today we have gone about as far as we can go with the medicines we have, so we need new treatment options now. The therapies in the adult world are helpful, but we need to know which of these therapies will be helpful to children and how immunotherapy can be applied as widely to these patients. Only new innovations in treatment will be the game-changer for pediatric cancer and for thousands of families whose lives are forever changed by these diseases.
By Don E. Eslin, MD
Don Eslin, MD, is a practicing pediatric hematologist and oncologist at the Arnold Palmer Center for Children’s Cancer and Blood Disorders, and a member of the Pediatric Hematology/Oncology Department at UF Health Cancer Center – Orlando Health. He is board certified by the American Board of Pediatrics in general pediatrics and pediatric hematology/oncology.
Dr. Eslin earned his medical degree from Marshall University School of Medicine in Huntington, West Virginia, and completed a residency in pediatrics through Arnold Palmer Hospital for Children, a part of Arnold Palmer Medical Center in Orlando. He fulfilled a fellowship in pediatric hematology/oncology at Children’s Hospital of Philadelphia, and earned a National Research Fellowship Award from the Kennedy Krieger Institute in Baltimore.
Dr. Eslin also serves as clinical associate professor of pediatrics at the Florida State University in Tallahassee and serves as an assistant professor of pediatrics at the University of Central Florida in Orlando, Florida.
He holds membership in the American Academy of Pediatrics, the American Society of Clinical Oncology, the American Society of Pediatric Hematology/Oncology, the Florida Medical Association and the Orange County Medical Association.