Children’s Healthcare of Atlanta Physician Leads Children’s Oncology Group Trial to Compare Chemotherapy with Targeted Therapy for Brain Tumors


ATLANTA (July 14, 2020) – While classic chemotherapy is the standard of care for most kids diagnosed with a pediatric brain tumor, it often has severe side effects as it destroys both cancerous and normal cells. To preserve healthy cells and improve quality of life during treatment, Jason Fangusaro, MD, Director of Developmental Therapeutics for the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta, is leading a multi-site trial through the Children’s Oncology Group (COG) to potentially replace chemotherapy with a targeted treatment as the new standard of care in patients with low-grade glioma, the most common form of pediatric brain cancer.

“This trial is one of the first large prospective trials in over a decade to study a front-line therapy, the first treatment given, in children with newly diagnosed Neurofibromatosis-associated low-grade glioma,” said Dr. Fangusaro, the COG principal investigator.

A prior Pediatric Brain Tumor Consortium (PBTC) study also led by Dr. Fangusaro was one of the first to use the drug known as Selumetinib in children with recurrent low-grade glioma. It was shown to be an effective treatment strategy in children whose previous treatment was not working or progressed after stopping therapy. Now, in the follow-up trial for newly diagnosed patients who have never been treated with chemotherapy, this COG study will compare the Selumetinib therapy with standard classic chemotherapy.

During “A Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma,” Dr. Fangusaro hopes to learn which treatment is more successful at shrinking the tumor. The team will also conduct evaluations from the patients’ perspective to measure Selumetinib’s impact on quality of life, neuropsychological function, and functional outcomes like vision and motor ability. If the drug outperforms chemotherapy, it may replace the go-to therapy as the standard of care for children with low-grade glioma, revolutionizing treatment of the tumor.

“Selumetinib is a pill that can be taken at home, and we are working to develop a more child-friendly formula,” said Dr. Fangusaro, who also serves as Associate Professor for the Emory University School of Medicine Department of Pediatrics. “In the previous trial, patients had less severe side effects, and they only had to come to the clinic once a month. While on the classic intravenous chemotherapy, patients are seen about once a week for almost two years.”

There are about 4,000 cases of pediatric brain tumors each year in the U.S. and about half are low-grade gliomas, the most common central nervous system tumor in children. While this tumor does not grow rapidly, like an aggressive malignant tumor, it can cause significant functional deficits for children including vision abnormalities, motor dysfunction and hormone abnormalities, altering their quality of life.

Dr. Fangusaro and his team, in collaboration with the other participating COG sites, plan to enroll 220 patients ages 2-21 over the course of 5 to 6 years for the study (ACNS1831). The trial is sponsored by the Children’s Oncology Group, a National Cancer Institute-supported clinical trials group and the world’s largest organization for childhood and adolescent cancer research. The group includes over 200 children’s hospitals, universities, and cancer centers in North America, Australia and New Zealand.

“As we identify more targets in cancer, and develop drugs that can attack those targets, we are getting closer to minimizing the side effects from aggressive chemotherapy and improving outcomes for all cancers,” said Dr. Fangusaro. “Effective targeted therapies are novel. Very few show the promising results we’ve been lucky enough to see.”

The results from the prior study on Selumetinib conducted by the Pediatric Brain Tumor Consortium (PBTC) and led by Dr. Fangusaro were selected for inclusion in Clinical Care Advances 2020: ASCO’s Annual Report on Progress Against Cancer, an independent annual review of the year’s major achievements and emerging trends in clinical cancer research and care, which is available in the Journal of Clinical Oncology at ascopubs.org/journal/jco. Two additional trials were generated from this work. Dr. Fangusaro is principal investigator for the one discussed in this press release and vice chair on the other. He is also a member of the Children's Oncology Group Brain Tumor Steering Committee and the Vice Chair of the Pediatric Brain Tumor Consortium. To learn more about this trial, visit: https://clinicaltrials.gov/ct2/show/NCT03871257?term=ACNS1831&draw=2&rank=1.

About Children’s Healthcare of Atlanta

As the only freestanding pediatric healthcare system in Georgia, Children’s Healthcare of Atlanta is the trusted leader in caring for kids. The not-for-profit organization’s mission is to make kids better today and healthier tomorrow through more than 60 pediatric specialties and programs, top healthcare professionals, and leading research and technology. Children’s is one of the largest pediatric clinical care providers in the country, managing more than one million patient visits annually at three hospitals, Marcus Autism Center, the Center for Advanced Pediatrics and 20 neighborhood locations. Consistently ranked among the top children’s hospitals by U.S. News & World Report, Children’s Healthcare of Atlanta has impacted the lives of kids in Georgia, across the United States and around the world for more than 100 years thanks to generous support from the community. Visit www.choa.org for more information.

About Emory University School of Medicine

Emory University School of Medicine is a leading institution with the highest standards in education, biomedical research and patient care, with a commitment to recruiting and developing a diverse group of students and innovative leaders. Emory School of Medicine has more than 2,800 full- and part-time faculty, 556 medical students, 530 allied health students, 1,311 residents and fellows in 106 accredited programs, and 93 MD/PhD students in one of 48 NIH-sponsored Medical Scientist Training Programs. Medical school faculty received $456.3 million in external research funding in fiscal year 2018. The school is best known for its research and treatment in infectious disease, neurosciences, heart disease, cancer, transplantation, orthopaedics, pediatrics, renal disease, ophthalmology and geriatrics.

About The Children’s Oncology Group (COG)

COG (childrensoncologygroup.org), a member of the NCI National Clinical Trials Network (NCTN), is the world’s largest organization devoted exclusively to childhood and adolescent cancer research. COG unites over 10,000 experts in childhood cancer at more than 200 leading children’s hospitals, universities, and cancer centers across North America, Australia, and New Zealand in the fight against childhood cancer. Today, more than 90% of the 14,000 children and adolescents diagnosed with cancer each year in the United States are cared for at COG member institutions. Research performed by COG institutions over the past 50 years has transformed childhood cancer from a virtually incurable disease to one with a combined 5-year survival rate of 80%. COG’s mission is to improve the cure rate and outcomes for all children with cancer.

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