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.