Consortium-Based Oncology Research
At Nicklaus Children’s Hospital, our mission, vision and values guide us to continually improve the care we offer children. One vital way we do that is through ongoing clinical research.
“When talking about pediatric oncology, the number of cases simply isn’t as high as it is with adults,” says Guillermo De Angulo, MD, Director of Leukemia/Lymphoma, Director of Immunotherapy, Director of the Hemophilia Treatment Center and board-certified pediatric hematologist/oncologist at Nicklaus Children’s Hospital. “A single adult breast cancer trial can have thousands of participants, but participation in some pediatric oncology trials is in the low hundreds. The only way to get legitimate data is by joining other organizations in a cohort environment and enlisting children in clinical trials together.”
To that end, Nicklaus Children’s is part of two research-centric consortiums:
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Beat Childhood Cancer (BCC). BCC is a boutique consortium that gives oncologists an opportunity to collaborate weekly on particularly difficult cases, such as relapsed and refractory cancers. The group includes approximately thirty oncologists across the United States.
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Children’s Oncology Group (COG). As the world’s largest childhood and adolescent cancer research organization, COG and its members have played a significant role in improved outcomes for many childhood cancers since the merge of four research groups in 2000 with histories dating back as far as 1955.
Finding Solutions in Unchartered Territory
“Pediatric oncology is very evidence-based,” Dr. De Angulo says. “However, with the dearth of pediatric-specific research, we are often in unchartered waters.”
Bouncing ideas off pediatric experts beyond the walls of Nicklaus Children’s allows clinicians to learn from others who have faced similar situations and to hear of literature that speaks on a specific situation. This lets clinicians reach beyond their personal expertise and experience, leading to a more comprehensive approach when facing a given tumor.
Clinical trials grant opportunities to develop evidence-based best practices. In recent years, research from COG and BCC has had significant effect on patient care. One COG trial was so successful that it ended early, as the experimental arm outperformed the nonexperimental arm in overwhelming fashion. The outcome of this trial established a new best practice. Now, all patients with standard- or high-risk leukemia receive immunotherapy at the start of their treatment regimen.
“Until recently, the population of children with refractory leukemia and lymphoma had a terminal prognosis,” Dr. De Angulo says. “Targeted therapies have improved survival rates for these specific patients, and we expect greater gains moving forward.”
Eager to better serve their specific patient population, Nicklaus Children’s researchers are also making efforts to better understand disease discrepancies experienced by Latin American children. These findings will come with time, and Dr. De Angulo anticipates ongoing collaboration with cohort participants.
In the meantime, clinicians at Nicklaus Children’s are engaged in an array of innovative research efforts. One study turns off the gene responsible for growing brain tumors, allowing improved results in patients whose tumors are not fully resectable. A similar study reviews the patient’s genes to determine the volume and type of therapy necessary for individual leukemia patients. Future studies will seek similar advances for children with sickle cell disease and hemophilia.
The Patient’s Genetic Journey
Genetic testing, once a novelty with potential, is now standard protocol when treating pediatric leukemia and is becoming more common with solid and brain tumors. This advanced approach gives fuller insight that helps determine how an individual cancer will respond to various treatments.
“We used to rely on our eyes through histopathology,” Dr. De Angulo says. “Now, I want to know more about the biology of a tumor, as our care is based on the patient’s pediatric genetics and the genetics of the tumor itself.”
With genetic testing, this is possible. This insight then guides clinicians in developing a treatment plan. Thanks to the advent and widespread acceptance of targeted therapies via genetic testing, the patient journey now adheres to the following path:
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Following diagnosis, the solid tumor or brain tumor is removed and biopsied for molecular biology analysis.
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Based on genetic findings from the molecular biology team, clinicians can rule out certain treatments and identify those therapies with a higher probability of success.
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Therapy begins or is adjusted with approved targeted therapy options. When appropriate, the care team enrolls a patient in a clinical trial in an effort to develop a treatment that addresses the patient’s specific genetic makeup.
Though genetic testing improves cancer care, Dr. De Angulo admits significant strides remain. Many genes responsible for causing cancer have yet to be discovered, and the biology of all tumors is not fully understood.
Despite this, science is moving in a positive direction, offering newfound hope to families dealing with a pediatric cancer diagnosis.
“The war on cancer is starting to shift in our favor, with survival rates consistently trending upward,” Dr. De Angulo says. “With added therapies that are tailored to a patient’s genetic makeup, the C word people will begin hearing more and more when seeing an oncologist is curable.”
Are you caring for a pediatric patient in need of novel therapeutic approaches? Email a Nicklaus Children’s physician liaison to refer a patient to one of Nicklaus Children’s oncology clinical trials.