What patients and families need to know
The Children's National Research Institute
Our clinical oncology research involves the Pediatric Brain Tumor and Children's Brain Tumor Tissue consortiums, COllaborative Network for NEuro-oncology Trials, National Pediatric Cancer Foundation, Children's Oncology Group and Investigational Lead Early Phase Trials.
Pediatric Brain Tumor Consortium (PBTC)
The Pediatric Brain Tumor Consortium (PBTC) was established by the NCI in 1999 to improve the treatment of primary brain tumors in children. The consortium brings together the most prominent pediatric brain tumor programs in the country to perform early phase developmental therapeutic clinical trials:
- Eugene Hwang, M.D., serves as the Children’s National principal investigator for the PBTC as well as co-chair of the Immunotherapy Working Group.
- Lindsay Kilburn, M.D., is chair of the Quality Assurance Committee.
- Roger Packer, M.D., is both on the Steering Committee and is the site co-PI of the PBTC (the latter along with Brian Rood, M.D.), as well as lead of the Low-grade Glioma Working Group.
In the PBTC, Dr. Hwang is the PI and Dr. Kilburn is the co-PI of a trial utilizing checkpoint inhibition in high-grade pediatric cancers. Dr. Hwang is the PI of a project examining immunophenotypes in pediatric CNS tumors and the co-PI of a trial utilizing the Optune device for children with recurrent high-grade glioma and ependymoma.
COllaborative Network for NEuro-oncology Trials (CONNECT)
The COllaborative Network for NEuro-oncology Trials (CONNECT) is a new clinical trials consortium that strives to be both biology-driven as well as fill the gap between preclinical work and Phase I trials. Established by a large foundation initiative in 2017, Children’s National has been invited as an inaugural member. Eugene Hwang, M.D., is the PI for Children’s and Roger Packer, M.D., is on the Steering Committee for this consortium.
National Pediatric Cancer Foundation (NPCF)
Children’s National has recently joined the NPCF in order to lead and participate in trials focused on pediatric brain tumors. This has included the ReMATCH trial, utilizing a dendritic cell vaccine and adoptive cellular therapy in order to target recurrent medulloblastomas.
Children’s Brain Tumor Tissue Consortium (CBTTC)
Children’s National is a member of the Children’s Brain Tumor Tissue Consortium (CBTTC), a multi-institutional consortium that banks brain tumor specimens, including blood, buccal swabs, cerebrospinal fluid and urine, to be accessed by the pediatric brain tumor research community. Each specimen is annotated with clinical data in a continuously updated database. In addition, large data sets resulting from genomic, epigenomic and proteomic studies of those specimens are housed within the consortium in a computing environment that allows native analysis in the cloud. The CBTTC data repository, called CAVATICA, is the largest such repository in the world.
Children's Oncology Group (COG)
The clinical oncology research team at Children’s National specializes in treating patients with specific types of cancer, including leukemia, solid tumors and brain tumors. Their research provides innovative treatments and the highest quality of care for each child. Established in 2000, Children’s Oncology Group (COG)’s vision is to eliminate the personal, family and societal burden of cancer in children and adolescents. Children’s National has a long history of leadership and scientific contributions to the COG:
- Gregory Reaman, M.D., (Chief of Oncology, emeritus) served as the first chair of the NIH-funded COG until December 2010.
- Jeffrey Dome, M.D., Ph.D., currently serves as the COG Principal Investigator for Children’s National and served as Chair of the COG Renal Tumor Committee for over a decade. He now serves on the COG Scientific Council and Executive Committee and the study for standard-risk acute lymphoblastic leukemia (ALL), the largest therapeutic study within the COG.
- Catherine M. Bollard, M.B.Ch.B, M.D., is Chair of the COG Non-Hodgkin Lymphoma Committee and is Co-Chair of the ANHL1522 study of Rituximab (RTX) and Cytotoxic T-Lymphocytes in Pediatric Solid Organ Recipients (SOT) with EBV-Positive CD20-Positive Post-Transplant Lymphoproliferative Disease (PTLD).
- Holly Meany, M.D., is the Study Chair for the COG ANBL1232 study for non-high-risk neuroblastoma.
- Shana Jacobs, M.D., is on the steering committee of the COG Cancer Control Committee.
- Lindsay Kilburn, M.D., serves on the Developmental Therapeutics Committee.
- AeRang Kim, M.D., Ph.D., serves as the COG Pediatric Early Phase-Clinical Trial Network (PEP-CTN) Principal Investigator for Children’s National. Children’s National is one of a select group of institutions within North America to belong to the COG PEP-CTN developmental therapeutics consortium. Dr. Kim also serves as the study chair for APEC1621G, Phase 2 subprotocol of vemurafenib in patients with solid tumors and is on the Pediatric MATCH and Target and Agent Prioritization (TAP) committee. Active phase 1 and 2 cancer clinical trials are listed here. Dr. Kim also leads the malignant peripheral nerve sheath tumor (MPNST) steering committee (Roger Packer, M.D. is the PI) of the Neurofibromatosis Clinical Trials Consortium. Several clinical trials are open through this consortium including NF108 the Binimetinib trial, NF105 the cabozantinib trial, and recently completed NF106 PD0325901 trial.
- Anne Angiolillo, M.D.
- Catherine M. Bollard, M.B.Ch.B, M.D.
- Jennifer Dean, M.D.
- Jeffrey Dome, M.D., Ph.D.
- D. Ashley Hill, M.D.
- Pamela Hinds, RN, Ph.D.
- Eugene Hwang, M.D.
- Shana Jacobs, M.D.
- Kathy Kelly, RN, Ph.D.
- Lindsay Kilburn, M.D.
- AeRang Kim, M.D., Ph.D.
- Christopher Lawlor, M.D.
- Holly Meany, M.D.
- Roger Packer, M.D.
- Brian Rood, M.D.
- Reuven Schore, M.D.
- Amanda Thompson, Ph.D.
- Carly Varela, M.D.
Investigational Lead Early Phase Trials
AeRang Kim, M.D, Ph.D., serves as the Principal Investigator of a Phase I study of MR-guided high intensity focused ultrasound (MR-HIFU) for the ablation of recurrent pediatric solid tumors including desmoid tumors. She is also leading a NIH-funded Phase 1 Study of Lyso-thermosensitive Liposomal Doxorubicin (LTLD, ThermoDox) combined with MR-guided high intensity focused ultrasound for relapsed solid tumors in children. These are the first clinical studies using this novel device alone and in combination with temperature sensitive chemotherapy in children.
Children's National is one of few children's hospitals to participate in the Sarcoma Alliance through Collaboration (SARC) consortium dedicated to achieving breakthroughs in sarcoma research. Dr. Kim serves as the Overall PI of Department of Defense funded multi-institutional trial of SARC031, phase II study of selumetinib and vistusertib in patients with unresectable malignant peripheral nerve sheath tumors. Eugene Hwang, M.D., is the co-PI of an international study examining the utility of bevacizumab and vinblastine as front-line therapy for children with low-grade gliomas. He is also the PI of a Children’s National-led study testing the use of a novel gene therapy to restore p53 function in children with recurrent brain tumors, coupled with irradiation and chemotherapy.