The Pediatric Brain Tumor Consortium, now in its 20th year, is composed of 11 academic centers and children's hospitals located across the United States which are competitively selected based on their scientific excellence and clinical expertise in pediatric brain tumors.
Participating sites: CAChildren’s Hospital Los Angeles, CALucile Packard Children's Hospital Stanford, COChildren's Hospital Colorado, DCChildren’s National Medical Center (Washington, DC), GAChildren's Healthcare of Atlanta, ILAnn & Robert H. Lurie Children’s Hospital of Chicago, MDNIH-Pediatric Oncology Branch, NYMemorial Sloan Kettering Cancer Center, OHCincinnati Children's Hospital Medical Center, PAChildren’s Hospital of Pittsburgh, TNSt. Jude Children’s Research Hospital, TXTexas Children's Cancer Center.
PBTC's mission is to contribute rapidly and effectively to the understanding and cure of pediatric brain tumors through the conduct of multi-center, multidisciplinary, innovative studies with designs and analyses based on uniformly high quality statistical science.
Since its inception in 1999, the PBTC's primary objective has been to rapidly conduct novel phase I and II clinical evaluations of new therapeutic agents, treatment delivery technologies and radiation treatment strategies in children from infancy to 21 years of age with primary central nervous system (CNS) tumors. In addition the Consortium aims to characterize reliable markers and predictors (direct or surrogate) of brain tumors' responses to new therapies. The Consortium conducts research on brain tumor specimens in the laboratory to further understand the biology of pediatric brain tumors.
The PBTC has a direct working relationship with the Children's Oncology Group (COG) to ensure that results from phase I and II trials can be confirmed through additional phase II and confirmatory phase III clinical trials in the COG. Results from PBTC studies are presented at professional meetings and reported in the published literature.
While the primary mission of the PBTC is to identify through laboratory and clinical science superior treatment strategies for children with brain cancers, the PBTC investigators recognize their profound responsibility to meet the special needs of the children and families as they face this enormous challenge. Members are committed to working within their institutions and communities to improve support services and follow up care for these patients and their families.
To accomplish its objectives, the PBTC has an Operations, Biostatistics and Data Management Core (OBDMC) based at St Jude Children’s Research Hospital in Memphis, TN, which provides the necessary expertise and capability to support the activities of the consortium. The OBDMC staff, led by Dr. Arzu Onar-Thomas, provide expertise in study design, protocol and database development, data monitoring and management as well as data analysis and reporting. The core also facilitates interactions among the various committees, is responsible for arranging and sponsoring semi-annual meetings, manages the PBTC website and provides various other support functions. All fiscal and regulatory affairs of the PBTC are also coordinated through the OBDMC.
The PBTC also aims to develop and coordinate innovative neuro-imaging techniques through its Neuro-Imaging Center (NIC) led by Dr. Tina Young Poussaint at Harvard Medical School. The NIC’s research focuses on evaluating new treatment response criteria and neuro-imaging methods to understand regional brain effects. These imaging techniques can also advance understanding of significant neuro-toxicity in a developing child's central nervous system. The Neuro-Imaging Center also provides central review of responses observed on PBTC studies and serves as a resource to PBTC member sites for radiology consultations related to the consortium trials.
Pharmacokinetic (PK) studies are overseen by the PBTC PK Core which is led by Dr. Clinton Stewart at St Jude Children’s Research Hospital in Memphis, TN. They are an integral component of new agent clinical trials within the PBTC and help develop a better understanding of the disposition of the new agents and gain insight into differences between how adults and children eliminate drugs leading to better insights into the variability in drug disposition. PK studies also aid in understanding the relationship between the amount of drug in a child’s body and the effect that a drug might have (toxicity as well as efficacy). This relationship (called pharmacodynamics) can help investigators use the new agent better in future studies.
The PBTC Genomics Core is led by Dr. Will Parsons from Baylor College of Medicine with an overall objective to support genomic studies related to PBTC clinical trials and actively integrate state-of-the-art genomics methodologies into these protocols. Research efforts led by the core range from focused analyses of specific molecular markers with proven relevance to the tumor types and/or novel agents being studied in individual PBTC trials to more comprehensive molecular profiling such as whole-exome sequencing to inform post-hoc analyses, link treatment response to somatic mutations in the given tumor(s) and when possible, identify genetic changes occurring during emergence of resistance.
The PBTC Pathology Central Review and Biorepository (CRB) is located at Children’s Hospital Los Angeles and is led by Drs. Alex Judkins and Jennifer Cotter. The CRB‘s function is to collect, store and distribute specimens for central pathology review and for planned correlative studies which support the laboratory objectives of PBTC studies. The CRB also serves as a central repository for specimens collected for future research from patients who consent to long term storage.