Director of Clinical Research in the Department of Neuro-Oncology at the Children's Hospital of Philadelphia
Children's Hospital of Philadelphia
Pediatric Neuro-Oncology, Clinical research, Early phase clinical trials in neuro-oncology, Developmental therapeutics
Children’s Hospital of Philadelphia
Investigating Disease Mechanism In Adamantinomatous Craniopharyngioma Using New Cell Line Models
Adamantiomatious craniopharyngioma (ACP) is an intracranial tumor with no currently available targeted drug therapeutics. Using cell lines provided by the Children’s Brain Tumor Network, researchers will develop new models for use in identifying therapeutic opportunities for this disease.
Precision Imaging of Pediatric High-grade Gliomas with Quantitative Diffusion Weighted Imaging and Texture Analysis to Identify Imaging Biomarkers That Predict Tumor Genetics and Patient Outcomes
The recently characterized molecular marker, histone H3 K27M, could prove important to the treatment of pediatric gliomas. Researchers will interrogate data from the Pediatric Brain Tumor Atlas in order to better understand this new class of malignant gliomas.
Diversity, Equity, and Inclusion: Translation of Informed Consent Forms
In November 2020, the FDA released guidance to help promote clinical trial enrollment practices that could lead to clinical trials that better reflect diverse populations who may benefit from treatments, medical devices, or drugs that are being investigated. Despite previous efforts, there remain gaps in inclusion of under-represented populations in many clinical trials. One of the barriers to being inclusive among all groups are the languages that are offered for informed consent practices. Currently, the Children’s Hospital of Philadelphia and collaborating institutions typically offer multilingual staff and informed consents in limited languages. But, the options available at each site vary and can be costly and burdensome at the level of each individual institution/clinical research team. Our goal is to remove this potential barrier by providing consortium level informed consents and study documents in multiple languages to ensure enrollment and recruitment of under-represented minorities (URM) in languages understood by a diverse population of patients and families. By providing these necessary resources, non-English speaking patients and families will be afforded the same opportunity to understand and participate in clinical trial enrollments as English-speaking patients and families.
All Brain Tumor Types
Childhood craniopharyngiomas are rare tumors usually found near the pituitary gland (a pea-sized organ at the bottom of the brain that controls other glands) and the hypothalamus (a small cone-shaped organ connected to the pituitary gland by nerves).Craniopharyngiomas are usually part solid mass and
High-grade Gliomas (HGG) or astrocytomas in children nearly always result in a dismal prognosis. Although novel therapeutic approaches are currently in development, preclinical testing has been limited, due to a lack of pediatric-specific HGG preclinical models. These models are needed to help test
Diffuse Intrinsic Pontine Glioma
A presumptive diagnosis of DIPG based on classic imaging features, in the absence of a histologic diagnosis, has been routinely employed. Increasingly however, histologic confirmation is obtained for both entry into research studies and molecular characterization of the tumor. New approaches with
Indigenous peoples and inclusion in clinical and genomic research: Understanding the history and navigating contemporary engagement
The survival rates for pediatric cancer patients have improved significantly, but there are still significant disparities among underrepresented racial and ethnic groups. To address this issue, the Pacific Pediatric Neuro-Oncology Consortium (PNOC) and Children's Brain Tumor Network (CBTN) created a
Angela Waanders, Alex Brown, Nadine R. Caron, Alexa Plisiewicz, Sean T. McHugh, Thinh Q. Nguyen, Kaitlin Lehmann, Jeffrey Stevens, Phillip J. Storm, Adam Resnick, Tom Belle Davidson, Sabine Mueller, Cassie Kline
Retrospective dataset and survey analyses identify gaps in data collection for craniopharyngioma and priorities of patients and families affected by the disease
Craniopharyngioma is a rare type of brain tumor located in the suprasellar region of the brain, close to important structures like the pituitary gland. This study aimed to better understand patient outcomes associated with existing therapies and to inform new treatment strategies. Our team collected
Emily Marshall, Nikhil Joshi, Julia Crowley, Shana McCormack, Sylvia Cheng, Walter Faig, Phillip B. Storm, Adam Resnick, Sabine Mueller, Fatema Malbari, Cassie Kline
Clinical Cancer Research
Upfront Biology-Guided Therapy in Diffuse Intrinsic Pontine Glioma: Therapeutic, Molecular, and Biomarker Outcomes from PNOC003
PNOC003 is a multicenter precision medicine trial for children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG).
Cassie Kline, Payal Jain, Lindsay Kilburn, Erin R. Bonner, Nalin Gupta, John R. Crawford, Anu Banerjee, Roger J. Packer; Javier Villanueva-Meyer, Tracy Luks, Yalan Zhang, Madhuri Kambhampati, Jie Zhang, Sridevi Yadavilli, Bo Zhang, Krutika S. Gaonkar, Jo Lynne Rokita, Adam Kraya, John Kuhn, Winnie Liang; Sara Byron, Michael Berens, Annette Molinaro, Michael Prados, Adam Resnick, Sebastian M. Waszak, Javad Nazarian, Sabine Mueller
Scientific Reports - Nature
Harmonization of Postmortem Donations for Pediatric Brain Tumors and Molecular Characterization of Diffuse Midline Gliomas
The whole genome sequencing and RNA sequencing data related to pediatric diffuse midline gliomas that is generated by researchers on this project will be shared widely with support from CBTN.
Kambhampati M, Panditharatna E, Yadavilli S, Saoud K, Lee S, Eze A, Almira-Suarez MI, Hancock L, Bonner ER, Gittens J, Stampar M, Gaonkar K, Resnick AC, Kline C, Ho C-Y, Waanders AJ, Georgescu M-M, Rance NE, Kim Y, Johnson C, Rood BR, Kilburn LB, Hwang EI, Mueller S, Packer RJ, Bornhorst M, Nazarian J.