The Pediatric Cancer Summit at The Children’s Hospital of Philadelphia gave childhood cancer advocacy and community fundraising groups and members of the media the opportunity to listen to five of CHOP’s scientific experts discuss their work.
Attendees had a unique opportunity to ask questions of the very people who split their time between treating patients in CHOP’s Cancer Center and working in the state-of-the-art labs housed in CHOP’s Colket Translational Research Building.
Dr. John Maris, director of the Center for Childhood Cancer Research at The Children’s Hospital of Philadelphia, in his welcoming remarks, told participants, “We are on the verge of a second revolution in childhood cancer.”
The first revolution, he explained, started about 35 years ago, when mortality rates in pediatric cancers began a steady decline. “But cure rates have been stuck at a plateau for the past decade. Now greater understanding of the molecular basis of different cancers and advances in psychosocial care are bringing us closer to a new era of advances,” he added. Some of these advances were presented at the event that followed.
We captured a virtual version of the Pediatric Cancer Summit at CHOP to share here. Read on for details and downloads of each presentation.
In the lab with Dr. Maris at the Pediatric Cancer Summit at CHOP.
Dr. Maris spoke about recent advances in the treatment of high-risk neuroblastoma, a tumor of the nerve tissue that develops in infants and children.
Dr. Maris and colleagues at the Center for Childhood Cancer Research occasionally saw cases of neuroblastoma where there was a family history of the disease. They suspected that some types of neuroblastoma were hereditary and began looking for the associated gene.
The Cancer Center found in 2008 that the majority of hereditary neuroblastomas are caused by germline mutations in the ALK gene. This mutation leads to changes in the blueprint of this gene that alter the genetic message and cause unregulated growth of the tumor cell that initiates neuroblastoma.
As a result of this finding, CHOP now offers diagnostic testing for children diagnosed with neuroblastoma. Knowing if a child has the ALK mutation potentially provides more treatment options for those who suffer a relapse. Ultimately, the hope is that newly diagnosed patients could be treated with a personalized approach from the start of treatment.
Dr. Maris talks about the recent advances in neuroblastoma.
Dr. Balis speaks to a group at the Pediatric Cancer Summit at CHOP.The Cancer Center is committed to clinical research, which Dr. Balis discussed at the Pediatric Cancer Summit. Clinical cancer research measures the impact of new research in patients either by directly treating patients or by using tissue samples from patients. At CHOP, clinical research priorities are in:
Dr. Balis highlighted two specific areas of clinical research:
Fertility preservation is an important issue for young cancer patients, whose developing reproductive systems can be damaged by some cancer treatments. In the CHOP Cancer Survivorship Clinic, clinicians use a technique of cryopreservation to preserve the reproductive tissue of prepubescent boys and girls before they begin cancer treatment. Researchers are investigating methods to use this preserved tissue to allow these patients to achieve fertility when they become adults.
Dosing is critical to the development of new treatments for childhood cancer. Overdosing or underdosing in a clinical trial could mean an anti-cancer drug that could be effective at the appropriate dose is overlooked. Adjusting for body surface area is the standard method for translating an adult drug dose to a child’s dose. But this approach results in widely variable, less-than-optimal drug exposures. Clinical pharmacology researchers at the Cancer Center are instead developing new ways to establish safe and effective doses by, for instance, individualizing dosages based on markers of kidney and liver function. Researchers are shifting their emphasis to determining the optimal dose based on the drug’s therapeutic effects rather than its toxicity.
Dr. Balis talks about clinical cancer research.
In the lab with Dr. Grupp at the Pediatric Cancer Summit at CHOP.Dr. Grupp’s presentation at the Pediatric Cancer Summit focused on his clinical trial, CART19, which is currently enrolling new patients. CART19 is a trial of a new immunotherapy technique to treat acute lymphoblastic leukemia (ALL), the most common childhood cancer.
ALL usually strikes B cells in the immune system, making them work improperly and grow wildly. The T cells of the immune system are meant to fight off cells that don’t belong, but they are blind to the B cells that have become cancerous.
Dr. Grupp and team have custom-designed T cells that not only recognize and destroy the cancer cells, but also multiply quickly once inside the patient and remain in the patient’s body after the B cells have been destroyed. This therapy has been tested in three adult patients, two of whom entered complete remission. The CART19 trial will test the approach in children.
Dr. Grupp talks about the launch of CART19.
Dr. Curran discusses personalized cancer treatment at the Pediatric Cancer Summit.The largest pediatric biorepository in the world, with space for several million biological samples, is housed in the basement of the Colket building at CHOP. That’s where Dr. Curran hosted his Pediatric Cancer Summit presentation about a pediatric brain tumor called medulloblastoma and advances in personalized medicine. CHOP contributed resources and data from the biorepository to the Children’s Brain Tumor Tissue Foundation, which provided tumor samples to a Johns Hopkins-led medulloblastoma study published in Science in 2010.
In that study, researchers analyzed all the genes known to code for proteins of medulloblastoma tumors. Results suggested that compared to adult tumors, pediatric tumors might respond better to drugs that target the genes and pathways altered by mutations that initiate cancer. Findings like this one help drive advances in personalized medicine, where understanding the unique causes of each individual’s disease can lead to a more effective treatment approach.
The key to personalized cancer medicine is that you need to identify the correct patients, and the correct tumors, that can be treated with a given drug. Studying patient biospecimens helps to answer that question. CHOP is committed to collecting as many biospecimens as possible—primarily blood samples—to advance what’s known about the genetic causes of disease.
Scientists extract the DNA from blood samples and look for genetic predispositions to diseases, including diseases like medulloblastoma. They then extract DNA from tumors and compare that DNA to the DNA in the blood to determine what went wrong that caused the disease. Understanding that can lead to the development of therapies that distinguish between cancer cells and normal cells, and are personalized to the patient.
Dr. Curran talks about personalized cancer treatment.
Dr. Anne Kazak speaks at the Pediatric Cancer Summit.Dr. Kazak's Pediatric Cancer Summit presentation focused on research at CHOP related to:
Specifically, Dr. Kazak discussed research on medical traumatic stress. The Cancer Center is interested in better understanding how the experience of cancer impacts children and their families. CHOP is a leader in showing how cancer and treatment experiences can be traumatic events to a child and a family.
CHOP has developed a treatment model and interventions to reduce traumatic stress responses for all members of the family. CHOP has a center in the National Child Traumatic Stress Network related to medical trauma and a website for healthcare professionals to learn more about trauma informed care, www.healthcaretoolbox.org.
Assessing psychosocial risk at the time of the diagnosis of childhood cancer is a major emphasis of research within the Behavioral Oncology program. Dr. Kazak and her colleagues have developed the Psychosocial Assessment Tool (PAT), a brief parent report screener of psychosocial risk, that has been translated into several languages and is used in the US and internationally. By screening families for psychosocial vulnerabilities and strengths at diagnosis, we can better identify psychosocial treatments to improve their wellbeing over the course of treatment, and beyond.
Dr. Kazak also discussed research about the psychosocial development of childhood cancer survivors as they grow up and enter young adulthood. Childhood cancer survivors are at risk for ongoing health problems as a result of their treatment, and scientists like Dr. Kazak are learning how to keep survivors engaged in healthcare and in a healthy lifestyle.
Our four faculty members and fellows are actively involved in many other research projects. Some of these include: understanding how families decide whether to enter clinical trials in oncology; learning how families cope and adjust to the neurocognitive difficulties experienced by brain tumor survivors; assessing factors related to health disparities; understanding how sleep may be impacted in children with cancer; and examining psychological factors associated with genetic surveillance.
Dr. Kazak talks about behavioral oncology research.
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