Giselle Saulnier Sholler, MD, Division Chief for Pediatric Hematology/Oncology at Penn State University and Founder of the Beat Childhood Cancer Research Consortium, discusses the organization’s work in neuroblastoma.
Neuroblastoma is a rare cancer that largely afflicts infants or children. It develops from neuroblasts in the sympathetic nervous system.The tumor most often develops in the adrenal gland, but may develop in the neck, chest, or spinal cord. In most cases, the tumor has metastasized by the time it is diagnosed. A neuroblastoma can cause a variety of signs and symptoms, including a lump where the tumor is growing, bone pain, diarrhea, and various neurological symptoms.
The Beat Childhood Cancer Research Consortium is a group of over 50 universities and children’s hospitals that provides a worldwide network of pediatric cancer clinical trials. Their mission is to continue the use of precision medicine and immunotherapy to bring forward new therapies for children with cancer with the goal of finding a cure for all patients.
The consortium consists of physicians, researchers, and parent advocates, who Dr. Sholler emphasizes as the most important contributor to the group’s mission. The group currently treat patients in the United States and Canada but plans are in place to reach the European and South American populations. By expanding their reach, the organization hopes to enroll and finalize clinical trials even quicker.
The consortium has led a few landmark studies over the past 15 years, including the development of difluoromethylornithine (DFMO) for high-risk neuroblastoma. DFMO specifically targets cancer stem cells left behind following treatment to prevent relapse, a main concern in patients with this condition. In a phase 2, single-arm trial, DFMO illustrated the ability to decrease relapse rates by over 50%. That study was instrumental in getting the drug approved by the U.S. Food and Drug Administration (FDA).
Additionally, the Beat Childhood Cancer Research Consortium is working to understand the specific biology of cancer in individual patients through the use of genomic sequencing. This method is being utilized with the goal of finding targeted, less toxic therapies that can be integrated into personalized treatment plans. A study done on this showed the ability to find effective treatment options in 70% of patients who otherwise have no treatment options left. For more information on studies Dr. Sholler is involved with, click here.
Currently, the consortium is currently enrolling eight clinical trials and more in the pipeline for next year. The group hopes to continue to move forward in precision medicine through the understanding of individual genomics and looking at using the immune system for specific targeting to reduce the toxicity of current treatment options.
To learn more about neuroblastoma and other rare cancer, visit https://checkrare.com/diseases/cancers/