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1. The Brain Tumor Segmentation in Pediatrics (BraTS-PEDs) Challenge: Focus on Pediatrics (CBTN-CONNECT-DIPGR-ASNR-MICCAI BraTS-PEDs)

Author

Kazerooni, Anahita Fathi, Khalili, Nastaran, Liu, Xinyang, Gandhi, Deep, Jiang, Zhifan, Anwar, Syed Muhammed, Albrecht, Jake, Adewole, Maruf, Anazodo, Udunna, Anderson, Hannah, Baid, Ujjwal, Bergquist, Timothy, Borja, Austin J., Calabrese, Evan, Chung, Verena, Conte, Gian-Marco, Dako, Farouk, Eddy, James, Ezhov, Ivan, Familiar, Ariana, Farahani, Keyvan, Franson, Andrea, Gottipati, Anurag, Haldar, Shuvanjan, Iglesias, Juan Eugenio, Janas, Anastasia, Johansen, Elaine, Jones, Blaise V, Khalili, Neda, Kofler, Florian, LaBella, Dominic, Lai, Hollie Anne, Van Leemput, Koen, Li, Hongwei Bran, Maleki, Nazanin, McAllister, Aaron S, Meier, Zeke, Menze, Bjoern, Moawad, Ahmed W, Nandolia, Khanak K, Pavaine, Julija, Piraud, Marie, Poussaint, Tina, Prabhu, Sanjay P, Reitman, Zachary, Rudie, Jeffrey D, Sanchez-Montano, Mariana, Shaikh, Ibraheem Salman, Sheth, Nakul, Tu, Wenxin, Wang, Chunhao, Ware, Jeffrey B, Wiestler, Benedikt, Zapaishchykova, Anna, Bornhorst, Miriam, Deutsch, Michelle, Fouladi, Maryam, Lazow, Margot, Mikael, Leonie, Hummel, Trent, Kann, Benjamin, de Blank, Peter, Hoffman, Lindsey, Aboian, Mariam, Nabavizadeh, Ali, Packer, Roger, Bakas, Spyridon, Resnick, Adam, Rood, Brian, Vossough, Arastoo, and Linguraru, Marius George

Subjects
Computer Science - Computer Vision and Pattern Recognition, Electrical Engineering and Systems Science - Image and Video Processing
Abstract

Pediatric tumors of the central nervous system are the most common cause of cancer-related death in children. The five-year survival rate for high-grade gliomas in children is less than 20%. Due to their rarity, the diagnosis of these entities is often delayed, their treatment is mainly based on historic treatment concepts, and clinical trials require multi-institutional collaborations. Here we present the CBTN-CONNECT-DIPGR-ASNR-MICCAI BraTS-PEDs challenge, focused on pediatric brain tumors with data acquired across multiple international consortia dedicated to pediatric neuro-oncology and clinical trials. The CBTN-CONNECT-DIPGR-ASNR-MICCAI BraTS-PEDs challenge brings together clinicians and AI/imaging scientists to lead to faster development of automated segmentation techniques that could benefit clinical trials, and ultimately the care of children with brain tumors., Comment: arXiv admin note: substantial text overlap with arXiv:2305.17033

Published
2024

2. Rational combination platform trial design for children and young adults with diffuse midline glioma: A report from PNOC.

Author

Plasschaert, Sabine, Molinaro, Annette, Koschmann, Carl, Nazarian, Javad, Mueller, Sabine, Kline, Cassie, Franson, Andrea, van der Lugt, Jasper, Prados, Michael, and Waszak, Sebastian

Subjects
ONC201, clinical trials, diffuse midline glioma, paxalisib, pediatric neuro-oncology, Humans, Glioma, Brain Neoplasms, Child, Young Adult, Adolescent, Antineoplastic Combined Chemotherapy Protocols, Pyrimidines, Adult, Female, Research Design, Prognosis, Male, Quality of Life
Abstract

Background Diffuse midline glioma (DMG) is a devastating pediatric brain tumor unresponsive to hundreds of clinical trials. Approximately 80% of DMGs harbor H3K27M oncohistones, which reprogram the epigenome to increase the metabolic profile of the tumor cells. Methods We have previously shown preclinical efficacy of targeting both oxidative phosphorylation and glycolysis through treatment with ONC201, which activates the mitochondrial protease ClpP, and paxalisib, which inhibits PI3K/mTOR, respectively. Results ONC201 and paxalisib combination treatment aimed at inducing metabolic distress led to the design of the first DMG-specific platform trial PNOC022 (NCT05009992). Conclusions Here, we expand on the PNOC022 rationale and discuss various considerations, including liquid biome, microbiome, and genomic biomarkers, quality-of-life endpoints, and novel imaging modalities, such that we offer direction on future clinical trials in DMG.

Published
2024

3. ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma

Author

Moertel, Christopher L., Hirbe, Angela C., Shuhaiber, Hans H., Bielamowicz, Kevin, Sidhu, Alpa, Viskochil, David, Weber, Michael D., Lokku, Armend, Smith, L. Mary, Foreman, Nicholas K., Hajjar, Fouad M., McNall-Knapp, Rene Y., Weintraub, Lauren, Antony, Reuben, Franson, Andrea T., Meade, Julia, Schiff, David, Walbert, Tobias, Ambady, Prakash, Bota, Daniela A., Campen, Cynthia J., Kaur, Gurcharanjeet, Klesse, Laura J., Maraka, Stefania, Moots, Paul L., Nevel, Kathryn, Bornhorst, Miriam, Aguilar-Bonilla, Ana, Chagnon, Sarah, Dalvi, Nagma, Gupta, Punita, Khatib, Ziad, Metrock, Laura K., Nghiemphu, P. Leia, Roberts, Ryan D., Robison, Nathan J., Sadighi, Zsila, Stapleton, Stacie, Babovic-Vuksanovic, Dusica, Gershon, Timothy R., Raslan, Ahmed, Sidhu, Alpa, Aguilar-Bonilla, Ana, Franson, Andrea T., Walter, Andrew, Hirbe, Angela C., Van Tine, Brian, Koschmann, Carl, Moertel, Christopher L., Campen, Cynthia, Bota, Daniela A., Schiff, David, Viskochil, David, Babovic-Vuksanovic, Dusica, Hajjar, Fouad M., Kaur, Gurcharanjeet, Shuhaiber, Hans H., Capal, Jamie K., Slopis, John, Gill, Jonathan, Meade, Julia, Nevel, Kathryn, Metrock, Laura K., Bielamowicz, Kevin, Klesse, Laura J., Weintraub, Lauren, Nghiemphu, Leia, Kilburn, Lindsay, Mrugala, Maciej M., Schmidt, Mary Lou, Bornhorst, Miriam, Dalvi, Nagma, Robison, Nathan J., Foreman, Nick K., Moots, Paul L., Ambady, Prakash, Gupta, Punita, Dhamija, Radhika, McNall-Knapp, Rene Y., Antony, Rueben, Roberts, Ryan D., Merrell, Ryan, Chagnon, Sarah, Stapleton, Stacie, Maraka, Stefania, Gershon, Timothy R., Walbert, Tobias, Khatib, Ziad, and Sadighi, Zsila

Published
2024
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4. Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways.

Author

Sweha, Stefan, Mota, Mateus, Pun, Matthew, Deogharkar, Akash, Chung, Chan, Tarapore, Rohinton, Ramage, Samuel, Chi, Andrew, Wen, Patrick, Arrillaga-Romany, Isabel, Batchelor, Tracy, Butowski, Nicholas, Sumrall, Ashley, Shonka, Nicole, Harrison, Rebecca, de Groot, John, Mehta, Minesh, Hall, Matthew, Daghistani, Doured, Cloughesy, Timothy, Ellingson, Benjamin, Beccaria, Kevin, Varlet, Pascale, Kim, Michelle, Umemura, Yoshie, Garton, Hugh, Franson, Andrea, Schwartz, Jonathan, Jain, Rajan, Kachman, Maureen, Baum, Heidi, Burant, Charles, Mottl, Sophie, Cartaxo, Rodrigo, John, Vishal, Messinger, Dana, Qin, Tingting, Peterson, Erik, Sajjakulnukit, Peter, Ravi, Karthik, Waugh, Alyssa, Walling, Dustin, Ding, Yujie, Xia, Ziyun, Schwendeman, Anna, Hawes, Debra, Yang, Fusheng, Judkins, Alexander, Wahl, Daniel, Lyssiotis, Costas, de la Nava, Daniel, Alonso, Marta, Eze, Augustine, Spitzer, Jasper, Schmidt, Susanne, Duchatel, Ryan, Dun, Matthew, Cain, Jason, Jiang, Li, Stopka, Sylwia, Baquer, Gerard, Regan, Michael, Filbin, Mariella, Agar, Nathalie, Zhao, Lili, Kumar-Sinha, Chandan, Mody, Rajen, Chinnaiyan, Arul, Kurokawa, Ryo, Pratt, Drew, Yadav, Viveka, Grill, Jacques, Kline, Cassie, Venneti, Sriram, Kawakibi, Abed, Ji, Sunjong, Resnick, Adam, Nazarian, Javad, Allen, Joshua, Odia, Yazmin, Gardner, Sharon, Koschmann, Carl, Mueller, Sabine, and Waszak, Sebastian

Subjects
Humans, Glioma, Brain Neoplasms, Histones, Treatment Outcome, Epigenesis, Genetic, Mutation
Abstract

UNLABELLED: Patients with H3K27M-mutant diffuse midline glioma (DMG) have no proven effective therapies. ONC201 has recently demonstrated efficacy in these patients, but the mechanism behind this finding remains unknown. We assessed clinical outcomes, tumor sequencing, and tissue/cerebrospinal fluid (CSF) correlate samples from patients treated in two completed multisite clinical studies. Patients treated with ONC201 following initial radiation but prior to recurrence demonstrated a median overall survival of 21.7 months, whereas those treated after recurrence had a median overall survival of 9.3 months. Radiographic response was associated with increased expression of key tricarboxylic acid cycle-related genes in baseline tumor sequencing. ONC201 treatment increased 2-hydroxyglutarate levels in cultured H3K27M-DMG cells and patient CSF samples. This corresponded with increases in repressive H3K27me3 in vitro and in human tumors accompanied by epigenetic downregulation of cell cycle regulation and neuroglial differentiation genes. Overall, ONC201 demonstrates efficacy in H3K27M-DMG by disrupting integrated metabolic and epigenetic pathways and reversing pathognomonic H3K27me3 reduction. SIGNIFICANCE: The clinical, radiographic, and molecular analyses included in this study demonstrate the efficacy of ONC201 in H3K27M-mutant DMG and support ONC201 as the first monotherapy to improve outcomes in H3K27M-mutant DMG beyond radiation. Mechanistically, ONC201 disrupts integrated metabolic and epigenetic pathways and reverses pathognomonic H3K27me3 reduction. This article is featured in Selected Articles from This Issue, p. 2293.

Published
2023

5. Phase 1 study of high-dose DFMO, celecoxib, cyclophosphamide and topotecan for patients with relapsed neuroblastoma: a New Approaches to Neuroblastoma Therapy trial

Author

Hogarty, Michael D., Ziegler, David S., Franson, Andrea, Chi, Yueh-Yun, Tsao-Wei, Denice, Liu, Kangning, Vemu, Rohan, Gerner, Eugene W., Bruckheimer, Elizabeth, Shamirian, Anasheh, Hasenauer, Beth, Balis, Frank M., Groshen, Susan, Norris, Murray D., Haber, Michelle, Park, Julie R., Matthay, Katherine K., and Marachelian, Araz

Published
2024
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6. The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Author

Kilburn, Lindsay B., Khuong-Quang, Dong-Anh, Hansford, Jordan R., Landi, Daniel, van der Lugt, Jasper, Leary, Sarah E. S., Driever, Pablo Hernáiz, Bailey, Simon, Perreault, Sébastien, McCowage, Geoffrey, Waanders, Angela J., Ziegler, David S., Witt, Olaf, Baxter, Patricia A., Kang, Hyoung Jin, Hassall, Timothy E., Han, Jung Woo, Hargrave, Darren, Franson, Andrea T., Yalon Oren, Michal, Toledano, Helen, Larouche, Valérie, Kline, Cassie, Abdelbaki, Mohamed S., Jabado, Nada, Gottardo, Nicholas G., Gerber, Nicolas U., Whipple, Nicholas S., Segal, Devorah, Chi, Susan N., Oren, Liat, Tan, Enrica E. K., Mueller, Sabine, Cornelio, Izzy, McLeod, Lisa, Zhao, Xin, Walter, Ashley, Da Costa, Daniel, Manley, Peter, Blackman, Samuel C., Packer, Roger J., and Nysom, Karsten

Published
2024
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7. The children's brain tumor network (CBTN) - Accelerating research in pediatric central nervous system tumors through collaboration and open science

Author

Lilly, Jena V, Rokita, Jo Lynne, Mason, Jennifer L, Patton, Tatiana, Stefankiewiz, Stephanie, Higgins, David, Trooskin, Gerri, Larouci, Carina A, Arya, Kamnaa, Appert, Elizabeth, Heath, Allison P, Zhu, Yuankun, Brown, Miguel A, Zhang, Bo, Farrow, Bailey K, Robins, Shannon, Morgan, Allison M, Nguyen, Thinh Q, Frenkel, Elizabeth, Lehmann, Kaitlin, Drake, Emily, Sullivan, Catherine, Plisiewicz, Alexa, Coleman, Noel, Patterson, Luke, Koptyra, Mateusz, Helili, Zeinab, Van Kuren, Nicholas, Young, Nathan, Kim, Meen Chul, Friedman, Christopher, Lubneuski, Alex, Blackden, Christopher, Williams, Marti, Baubet, Valerie, Tauhid, Lamiya, Galanaugh, Jamie, Boucher, Katie, Ijaz, Heba, Cole, Kristina A, Choudhari, Namrata, Santi, Mariarita, Moulder, Robert W, Waller, Jonathan, Rife, Whitney, Diskin, Sharon J, Mateos, Marion, Parsons, Donald W, Pollack, Ian F, Goldman, Stewart, Leary, Sarah, Caporalini, Chiara, Buccoliero, Anna Maria, Scagnet, Mirko, Haussler, David, Hanson, Derek, Firestein, Ron, Cain, Jason, Phillips, Joanna J, Gupta, Nalin, Mueller, Sabine, Grant, Gerald, Monje-Deisseroth, Michelle, Partap, Sonia, Greenfield, Jeffrey P, Hashizume, Rintaro, Smith, Amy, Zhu, Shida, Johnston, James M, Fangusaro, Jason R, Miller, Matthew, Wood, Matthew D, Gardner, Sharon, Carter, Claire L, Prolo, Laura M, Pisapia, Jared, Pehlivan, Katherine, Franson, Andrea, Niazi, Toba, Rubin, Josh, Abdelbaki, Mohamed, Ziegler, David S, Lindsay, Holly B, Stucklin, Ana Guerreiro, Gerber, Nicolas, Vaske, Olena M, Quinsey, Carolyn, Rood, Brian R, Nazarian, Javad, Raabe, Eric, Jackson, Eric M, Stapleton, Stacie, Lober, Robert M, Kram, David E, Koschmann, Carl, Storm, Phillip B, Lulla, Rishi R, Prados, Michael, Resnick, Adam C, and Waanders, Angela J

Subjects
Neurosciences, Pediatric Cancer, Brain Disorders, Pediatric Research Initiative, Pediatric, Brain Cancer, Rare Diseases, Cancer, Good Health and Well Being, Adult, Humans, Child, Quality of Life, Brain Neoplasms, Collaborative international research infrastructure, Pediatric brain tumors, Multi-omic data, Longitudinal clinical data, Biospecimens, Molecular clinical trials, Clinical Sciences, Oncology & Carcinogenesis
Abstract

Pediatric brain tumors are the leading cause of cancer-related death in children in the United States and contribute a disproportionate number of potential years of life lost compared to adult cancers. Moreover, survivors frequently suffer long-term side effects, including secondary cancers. The Children's Brain Tumor Network (CBTN) is a multi-institutional international clinical research consortium created to advance therapeutic development through the collection and rapid distribution of biospecimens and data via open-science research platforms for real-time access and use by the global research community. The CBTN's 32 member institutions utilize a shared regulatory governance architecture at the Children's Hospital of Philadelphia to accelerate and maximize the use of biospecimens and data. As of August 2022, CBTN has enrolled over 4700 subjects, over 1500 parents, and collected over 65,000 biospecimen aliquots for research. Additionally, over 80 preclinical models have been developed from collected tumors. Multi-omic data for over 1000 tumors and germline material are currently available with data generation for > 5000 samples underway. To our knowledge, CBTN provides the largest open-access pediatric brain tumor multi-omic dataset annotated with longitudinal clinical and outcome data, imaging, associated biospecimens, child-parent genomic pedigrees, and in vivo and in vitro preclinical models. Empowered by NIH-supported platforms such as the Kids First Data Resource and the Childhood Cancer Data Initiative, the CBTN continues to expand the resources needed for scientists to accelerate translational impact for improved outcomes and quality of life for children with brain and spinal cord tumors.

Published
2023

8. Author Correction: The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Author

Kilburn, Lindsay B., Khuong-Quang, Dong-Anh, Hansford, Jordan R., Landi, Daniel, van der Lugt, Jasper, Leary, Sarah E. S., Driever, Pablo Hernáiz, Bailey, Simon, Perreault, Sébastien, McCowage, Geoffrey, Waanders, Angela J., Ziegler, David S., Witt, Olaf, Baxter, Patricia A., Kang, Hyoung Jin, Hassall, Timothy E., Han, Jung Woo, Hargrave, Darren, Franson, Andrea T., Yalon Oren, Michal, Toledano, Helen, Larouche, Valérie, Kline, Cassie, Abdelbaki, Mohamed S., Jabado, Nada, Gottardo, Nicholas G., Gerber, Nicolas U., Whipple, Nicholas S., Segal, Devorah, Chi, Susan N., Oren, Liat, Tan, Enrica E. K., Mueller, Sabine, Cornelio, Izzy, McLeod, Lisa, Zhao, Xin, Walter, Ashley, Da Costa, Daniel, Manley, Peter, Blackman, Samuel C., Packer, Roger J., and Nysom, Karsten

Published
2024
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9. The children's brain tumor network (CBTN) - Accelerating research in pediatric central nervous system tumors through collaboration and open science

Author

Lilly, Jena V., Rokita, Jo Lynne, Mason, Jennifer L., Patton, Tatiana, Stefankiewiz, Stephanie, Higgins, David, Trooskin, Gerri, Larouci, Carina A., Arya, Kamnaa, Appert, Elizabeth, Heath, Allison P., Zhu, Yuankun, Brown, Miguel A., Zhang, Bo, Farrow, Bailey K., Robins, Shannon, Morgan, Allison M., Nguyen, Thinh Q., Frenkel, Elizabeth, Lehmann, Kaitlin, Drake, Emily, Sullivan, Catherine, Plisiewicz, Alexa, Coleman, Noel, Patterson, Luke, Koptyra, Mateusz, Helili, Zeinab, Van Kuren, Nicholas, Young, Nathan, Kim, Meen Chul, Friedman, Christopher, Lubneuski, Alex, Blackden, Christopher, Williams, Marti, Baubet, Valerie, Tauhid, Lamiya, Galanaugh, Jamie, Boucher, Katie, Ijaz, Heba, Cole, Kristina A., Choudhari, Namrata, Santi, Mariarita, Moulder, Robert W., Waller, Jonathan, Rife, Whitney, Diskin, Sharon J., Mateos, Marion, Parsons, Donald W., Pollack, Ian F., Goldman, Stewart, Leary, Sarah, Caporalini, Chiara, Buccoliero, Anna Maria, Scagnet, Mirko, Haussler, David, Hanson, Derek, Firestein, Ron, Cain, Jason, Phillips, Joanna J., Gupta, Nalin, Mueller, Sabine, Grant, Gerald, Monje-Deisseroth, Michelle, Partap, Sonia, Greenfield, Jeffrey P., Hashizume, Rintaro, Smith, Amy, Zhu, Shida, Johnston, James M., Fangusaro, Jason R., Miller, Matthew, Wood, Matthew D., Gardner, Sharon, Carter, Claire L., Prolo, Laura M., Pisapia, Jared, Pehlivan, Katherine, Franson, Andrea, Niazi, Toba, Rubin, Josh, Abdelbaki, Mohamed, Ziegler, David S., Lindsay, Holly B., Stucklin, Ana Guerreiro, Gerber, Nicolas, Vaske, Olena M., Quinsey, Carolyn, Rood, Brian R., Nazarian, Javad, Raabe, Eric, Jackson, Eric M., Stapleton, Stacie, Lober, Robert M., Kram, David E., Koschmann, Carl, Storm, Phillip B., Lulla, Rishi R., Prados, Michael, Resnick, Adam C., and Waanders, Angela J.

Published
2023
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12. The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Author

Kilburn, Lindsay B; https://orcid.org/0000-0003-1478-3619, Khuong-Quang, Dong-Anh; https://orcid.org/0000-0001-6305-7790, Hansford, Jordan R; https://orcid.org/0000-0001-7733-383X, Landi, Daniel, van der Lugt, Jasper; https://orcid.org/0000-0002-8186-338X, Leary, Sarah E S; https://orcid.org/0000-0003-0225-6184, Driever, Pablo Hernáiz; https://orcid.org/0000-0003-3135-3872, Bailey, Simon; https://orcid.org/0000-0003-4763-4329, Perreault, Sébastien, McCowage, Geoffrey, Waanders, Angela J, Ziegler, David S; https://orcid.org/0000-0001-7451-7916, Witt, Olaf, Baxter, Patricia A, Kang, Hyoung Jin; https://orcid.org/0000-0003-1009-6002, Hassall, Timothy E, Han, Jung Woo; https://orcid.org/0000-0001-8936-1205, Hargrave, Darren; https://orcid.org/0000-0001-8219-9807, Franson, Andrea T; https://orcid.org/0000-0002-5361-7683, Yalon Oren, Michal, Toledano, Helen, Larouche, Valérie; https://orcid.org/0000-0003-4082-7267, Kline, Cassie; https://orcid.org/0000-0001-7765-7690, Abdelbaki, Mohamed S, Jabado, Nada; https://orcid.org/0000-0003-2485-3692, Gottardo, Nicholas G; https://orcid.org/0000-0002-1082-6776, Gerber, Nicolas U; https://orcid.org/0000-0002-1783-631X, Whipple, Nicholas S, Segal, Devorah; https://orcid.org/0000-0002-9740-1286, Chi, Susan N, et al, Kilburn, Lindsay B; https://orcid.org/0000-0003-1478-3619, Khuong-Quang, Dong-Anh; https://orcid.org/0000-0001-6305-7790, Hansford, Jordan R; https://orcid.org/0000-0001-7733-383X, Landi, Daniel, van der Lugt, Jasper; https://orcid.org/0000-0002-8186-338X, Leary, Sarah E S; https://orcid.org/0000-0003-0225-6184, Driever, Pablo Hernáiz; https://orcid.org/0000-0003-3135-3872, Bailey, Simon; https://orcid.org/0000-0003-4763-4329, Perreault, Sébastien, McCowage, Geoffrey, Waanders, Angela J, Ziegler, David S; https://orcid.org/0000-0001-7451-7916, Witt, Olaf, Baxter, Patricia A, Kang, Hyoung Jin; https://orcid.org/0000-0003-1009-6002, Hassall, Timothy E, Han, Jung Woo; https://orcid.org/0000-0001-8936-1205, Hargrave, Darren; https://orcid.org/0000-0001-8219-9807, Franson, Andrea T; https://orcid.org/0000-0002-5361-7683, Yalon Oren, Michal, Toledano, Helen, Larouche, Valérie; https://orcid.org/0000-0003-4082-7267, Kline, Cassie; https://orcid.org/0000-0001-7765-7690, Abdelbaki, Mohamed S, Jabado, Nada; https://orcid.org/0000-0003-2485-3692, Gottardo, Nicholas G; https://orcid.org/0000-0002-1082-6776, Gerber, Nicolas U; https://orcid.org/0000-0002-1783-631X, Whipple, Nicholas S, Segal, Devorah; https://orcid.org/0000-0002-9740-1286, Chi, Susan N, and et al

Abstract

BRAF genomic alterations are the most common oncogenic drivers in pediatric low-grade glioma (pLGG). Arm 1 (n = 77) of the ongoing phase 2 FIREFLY-1 (PNOC026) trial investigated the efficacy of the oral, selective, central nervous system-penetrant, type II RAF inhibitor tovorafenib (420 mg m$^{-}$$^{2}$ once weekly; 600 mg maximum) in patients with BRAF-altered, relapsed/refractory pLGG. Arm 2 (n = 60) is an extension cohort, which provided treatment access for patients with RAF-altered pLGG after arm 1 closure. Based on independent review, according to Response Assessment in Neuro-Oncology High-Grade Glioma (RANO-HGG) criteria, the overall response rate (ORR) of 67% met the arm 1 prespecified primary endpoint; median duration of response (DOR) was 16.6 months; and median time to response (TTR) was 3.0 months (secondary endpoints). Other select arm 1 secondary endpoints included ORR, DOR and TTR as assessed by Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO) criteria and safety (assessed in all treated patients and the primary endpoint for arm 2, n = 137). The ORR according to RAPNO criteria (including minor responses) was 51%; median DOR was 13.8 months; and median TTR was 5.3 months. The most common treatment-related adverse events (TRAEs) were hair color changes (76%), elevated creatine phosphokinase (56%) and anemia (49%). Grade ≥3 TRAEs occurred in 42% of patients. Nine (7%) patients had TRAEs leading to discontinuation of tovorafenib. These data indicate that tovorafenib could be an effective therapy for BRAF-altered, relapsed/refractory pLGG. ClinicalTrials.gov registration: NCT04775485 .

Published
2024

18. Single-molecule systems for detection and monitoring of plasma circulating nucleosomes and oncoproteins in Diffuse Midline Glioma

Author

Erez, Nir, primary, Furth, Noa, additional, Fedyuk, Vadim, additional, Wadden, Jack, additional, Aittaleb, Rayan, additional, Schwark, Kallen, additional, Niculcea, Micheal, additional, Miclea, Madeline, additional, Mody, Rajen, additional, Franson, Andrea, additional, Eze, Augistine, additional, Nourmohammadi, Niku, additional, Nazarian, Javad, additional, Venneti, Sriram, additional, Koschmann, Carl, additional, and Shema, Efrat, additional

Published
2023
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19. The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Author

Kilburn, Lindsay B., primary, Khuong-Quang, Dong-Anh, additional, Hansford, Jordan R., additional, Landi, Daniel, additional, van der Lugt, Jasper, additional, Leary, Sarah E. S., additional, Driever, Pablo Hernáiz, additional, Bailey, Simon, additional, Perreault, Sébastien, additional, McCowage, Geoffrey, additional, Waanders, Angela J., additional, Ziegler, David S., additional, Witt, Olaf, additional, Baxter, Patricia A., additional, Kang, Hyoung Jin, additional, Hassall, Timothy E., additional, Han, Jung Woo, additional, Hargrave, Darren, additional, Franson, Andrea T., additional, Yalon Oren, Michal, additional, Toledano, Helen, additional, Larouche, Valérie, additional, Kline, Cassie, additional, Abdelbaki, Mohamed S., additional, Jabado, Nada, additional, Gottardo, Nicholas G., additional, Gerber, Nicolas U., additional, Whipple, Nicholas S., additional, Segal, Devorah, additional, Chi, Susan N., additional, Oren, Liat, additional, Tan, Enrica E. K., additional, Mueller, Sabine, additional, Cornelio, Izzy, additional, McLeod, Lisa, additional, Zhao, Xin, additional, Walter, Ashley, additional, Da Costa, Daniel, additional, Manley, Peter, additional, Blackman, Samuel C., additional, Packer, Roger J., additional, and Nysom, Karsten, additional

Published
2023
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20. Table S4 from Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, primary, Ji, Sunjong, primary, Waszak, Sebastian M., primary, Sweha, Stefan R., primary, Mota, Mateus, primary, Pun, Matthew, primary, Deogharkar, Akash, primary, Chung, Chan, primary, Tarapore, Rohinton S., primary, Ramage, Samuel, primary, Chi, Andrew, primary, Wen, Patrick Y., primary, Arrillaga-Romany, Isabel, primary, Batchelor, Tracy T., primary, Butowski, Nicholas A., primary, Sumrall, Ashley, primary, Shonka, Nicole, primary, Harrison, Rebecca A., primary, de Groot, John, primary, Mehta, Minesh, primary, Hall, Matthew D., primary, Daghistani, Doured, primary, Cloughesy, Timothy F., primary, Ellingson, Benjamin M., primary, Beccaria, Kevin, primary, Varlet, Pascale, primary, Kim, Michelle M., primary, Umemura, Yoshie, primary, Garton, Hugh, primary, Franson, Andrea, primary, Schwartz, Jonathan, primary, Jain, Rajan, primary, Kachman, Maureen, primary, Baum, Heidi, primary, Burant, Charles F., primary, Mottl, Sophie L., primary, Cartaxo, Rodrigo T., primary, John, Vishal, primary, Messinger, Dana, primary, Qin, Tingting, primary, Peterson, Erik, primary, Sajjakulnukit, Peter, primary, Ravi, Karthik, primary, Waugh, Alyssa, primary, Walling, Dustin, primary, Ding, Yujie, primary, Xia, Ziyun, primary, Schwendeman, Anna, primary, Hawes, Debra, primary, Yang, Fusheng, primary, Judkins, Alexander R., primary, Wahl, Daniel, primary, Lyssiotis, Costas A., primary, de la Nava, Daniel, primary, Alonso, Marta M., primary, Eze, Augustine, primary, Spitzer, Jasper, primary, Schmidt, Susanne V., primary, Duchatel, Ryan J., primary, Dun, Matthew D., primary, Cain, Jason E., primary, Jiang, Li, primary, Stopka, Sylwia A., primary, Baquer, Gerard, primary, Regan, Michael S., primary, Filbin, Mariella G., primary, Agar, Nathalie Y.R., primary, Zhao, Lili, primary, Kumar-Sinha, Chandan, primary, Mody, Rajen, primary, Chinnaiyan, Arul, primary, Kurokawa, Ryo, primary, Pratt, Drew, primary, Yadav, Viveka N., primary, Grill, Jacques, primary, Kline, Cassie, primary, Mueller, Sabine, primary, Resnick, Adam, primary, Nazarian, Javad, primary, Allen, Joshua E., primary, Odia, Yazmin, primary, Gardner, Sharon L., primary, and Koschmann, Carl, primary

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2023
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21. Supplementary Figures 1-15 from Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, primary, Ji, Sunjong, primary, Waszak, Sebastian M., primary, Sweha, Stefan R., primary, Mota, Mateus, primary, Pun, Matthew, primary, Deogharkar, Akash, primary, Chung, Chan, primary, Tarapore, Rohinton S., primary, Ramage, Samuel, primary, Chi, Andrew, primary, Wen, Patrick Y., primary, Arrillaga-Romany, Isabel, primary, Batchelor, Tracy T., primary, Butowski, Nicholas A., primary, Sumrall, Ashley, primary, Shonka, Nicole, primary, Harrison, Rebecca A., primary, de Groot, John, primary, Mehta, Minesh, primary, Hall, Matthew D., primary, Daghistani, Doured, primary, Cloughesy, Timothy F., primary, Ellingson, Benjamin M., primary, Beccaria, Kevin, primary, Varlet, Pascale, primary, Kim, Michelle M., primary, Umemura, Yoshie, primary, Garton, Hugh, primary, Franson, Andrea, primary, Schwartz, Jonathan, primary, Jain, Rajan, primary, Kachman, Maureen, primary, Baum, Heidi, primary, Burant, Charles F., primary, Mottl, Sophie L., primary, Cartaxo, Rodrigo T., primary, John, Vishal, primary, Messinger, Dana, primary, Qin, Tingting, primary, Peterson, Erik, primary, Sajjakulnukit, Peter, primary, Ravi, Karthik, primary, Waugh, Alyssa, primary, Walling, Dustin, primary, Ding, Yujie, primary, Xia, Ziyun, primary, Schwendeman, Anna, primary, Hawes, Debra, primary, Yang, Fusheng, primary, Judkins, Alexander R., primary, Wahl, Daniel, primary, Lyssiotis, Costas A., primary, de la Nava, Daniel, primary, Alonso, Marta M., primary, Eze, Augustine, primary, Spitzer, Jasper, primary, Schmidt, Susanne V., primary, Duchatel, Ryan J., primary, Dun, Matthew D., primary, Cain, Jason E., primary, Jiang, Li, primary, Stopka, Sylwia A., primary, Baquer, Gerard, primary, Regan, Michael S., primary, Filbin, Mariella G., primary, Agar, Nathalie Y.R., primary, Zhao, Lili, primary, Kumar-Sinha, Chandan, primary, Mody, Rajen, primary, Chinnaiyan, Arul, primary, Kurokawa, Ryo, primary, Pratt, Drew, primary, Yadav, Viveka N., primary, Grill, Jacques, primary, Kline, Cassie, primary, Mueller, Sabine, primary, Resnick, Adam, primary, Nazarian, Javad, primary, Allen, Joshua E., primary, Odia, Yazmin, primary, Gardner, Sharon L., primary, and Koschmann, Carl, primary

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2023
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22. Supplementary Data S2 from Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, primary, Ji, Sunjong, primary, Waszak, Sebastian M., primary, Sweha, Stefan R., primary, Mota, Mateus, primary, Pun, Matthew, primary, Deogharkar, Akash, primary, Chung, Chan, primary, Tarapore, Rohinton S., primary, Ramage, Samuel, primary, Chi, Andrew, primary, Wen, Patrick Y., primary, Arrillaga-Romany, Isabel, primary, Batchelor, Tracy T., primary, Butowski, Nicholas A., primary, Sumrall, Ashley, primary, Shonka, Nicole, primary, Harrison, Rebecca A., primary, de Groot, John, primary, Mehta, Minesh, primary, Hall, Matthew D., primary, Daghistani, Doured, primary, Cloughesy, Timothy F., primary, Ellingson, Benjamin M., primary, Beccaria, Kevin, primary, Varlet, Pascale, primary, Kim, Michelle M., primary, Umemura, Yoshie, primary, Garton, Hugh, primary, Franson, Andrea, primary, Schwartz, Jonathan, primary, Jain, Rajan, primary, Kachman, Maureen, primary, Baum, Heidi, primary, Burant, Charles F., primary, Mottl, Sophie L., primary, Cartaxo, Rodrigo T., primary, John, Vishal, primary, Messinger, Dana, primary, Qin, Tingting, primary, Peterson, Erik, primary, Sajjakulnukit, Peter, primary, Ravi, Karthik, primary, Waugh, Alyssa, primary, Walling, Dustin, primary, Ding, Yujie, primary, Xia, Ziyun, primary, Schwendeman, Anna, primary, Hawes, Debra, primary, Yang, Fusheng, primary, Judkins, Alexander R., primary, Wahl, Daniel, primary, Lyssiotis, Costas A., primary, de la Nava, Daniel, primary, Alonso, Marta M., primary, Eze, Augustine, primary, Spitzer, Jasper, primary, Schmidt, Susanne V., primary, Duchatel, Ryan J., primary, Dun, Matthew D., primary, Cain, Jason E., primary, Jiang, Li, primary, Stopka, Sylwia A., primary, Baquer, Gerard, primary, Regan, Michael S., primary, Filbin, Mariella G., primary, Agar, Nathalie Y.R., primary, Zhao, Lili, primary, Kumar-Sinha, Chandan, primary, Mody, Rajen, primary, Chinnaiyan, Arul, primary, Kurokawa, Ryo, primary, Pratt, Drew, primary, Yadav, Viveka N., primary, Grill, Jacques, primary, Kline, Cassie, primary, Mueller, Sabine, primary, Resnick, Adam, primary, Nazarian, Javad, primary, Allen, Joshua E., primary, Odia, Yazmin, primary, Gardner, Sharon L., primary, and Koschmann, Carl, primary

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2023
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23. TMET-02. CLINICAL EFFICACY OF ONC201 IN H3K27M-MUTANT DIFFUSE MIDLINE GLIOMAS IS DRIVEN BY DISRUPTION OF INTEGRATED METABOLIC AND EPIGENETIC PATHWAYS

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, additional, Ji, Sunjong, additional, Waszak, Sebastian, additional, Sweha, Stefan, additional, Mota, Mateus, additional, Pun, Matthew, additional, Deogharkar, Akash, additional, Chung, Chan, additional, Tarapore, Rohinton, additional, Ramage, Samuel, additional, Wen, Patrick, additional, Arrillaga-Romany, Isabel, additional, Garton, Hugh, additional, Franson, Andrea, additional, Schwartz, Jonathan, additional, Jain, Rajan, additional, Mottl, Sophie, additional, Cartaxo, Rodrigo, additional, John, Vishal, additional, Messinger, Dana, additional, Qin, Tingting, additional, Peterson, Erik, additional, Sajjakulnukit, Peter, additional, Judkins, Alexander, additional, Wahl, Daniel, additional, Lyssiotis, Costas, additional, de la Nava, Daniel, additional, Alonso, Martha, additional, Duchatel, Ryan, additional, Dun, Matthew, additional, Cain, Jason, additional, Jiang, Li, additional, Filbin, Mariella, additional, Zhao, Lili, additional, Kumar-Sinha, Chandan, additional, Mody, Rajen, additional, Chinnaiyan, Arul, additional, Kurokawa, Ryo, additional, Pratt, Drew, additional, Yadav, Viveka, additional, Grill, Jacques, additional, Kline, Cassie, additional, Mueller, Sabine, additional, Resnick, Adam, additional, Nazarian, Javad, additional, Allen, Joshua, additional, Odia, Yazmin, additional, Gardner, Sharon, additional, and Koschmann, Carl, additional

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2023
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24. Data from Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, primary, Ji, Sunjong, primary, Waszak, Sebastian M., primary, Sweha, Stefan R., primary, Mota, Mateus, primary, Pun, Matthew, primary, Deogharkar, Akash, primary, Chung, Chan, primary, Tarapore, Rohinton S., primary, Ramage, Samuel, primary, Chi, Andrew, primary, Wen, Patrick Y., primary, Arrillaga-Romany, Isabel, primary, Batchelor, Tracy T., primary, Butowski, Nicholas A., primary, Sumrall, Ashley, primary, Shonka, Nicole, primary, Harrison, Rebecca A., primary, de Groot, John, primary, Mehta, Minesh, primary, Hall, Matthew D., primary, Daghistani, Doured, primary, Cloughesy, Timothy F., primary, Ellingson, Benjamin M., primary, Beccaria, Kevin, primary, Varlet, Pascale, primary, Kim, Michelle M., primary, Umemura, Yoshie, primary, Garton, Hugh, primary, Franson, Andrea, primary, Schwartz, Jonathan, primary, Jain, Rajan, primary, Kachman, Maureen, primary, Baum, Heidi, primary, Burant, Charles F., primary, Mottl, Sophie L., primary, Cartaxo, Rodrigo T., primary, John, Vishal, primary, Messinger, Dana, primary, Qin, Tingting, primary, Peterson, Erik, primary, Sajjakulnukit, Peter, primary, Ravi, Karthik, primary, Waugh, Alyssa, primary, Walling, Dustin, primary, Ding, Yujie, primary, Xia, Ziyun, primary, Schwendeman, Anna, primary, Hawes, Debra, primary, Yang, Fusheng, primary, Judkins, Alexander R., primary, Wahl, Daniel, primary, Lyssiotis, Costas A., primary, de la Nava, Daniel, primary, Alonso, Marta M., primary, Eze, Augustine, primary, Spitzer, Jasper, primary, Schmidt, Susanne V., primary, Duchatel, Ryan J., primary, Dun, Matthew D., primary, Cain, Jason E., primary, Jiang, Li, primary, Stopka, Sylwia A., primary, Baquer, Gerard, primary, Regan, Michael S., primary, Filbin, Mariella G., primary, Agar, Nathalie Y.R., primary, Zhao, Lili, primary, Kumar-Sinha, Chandan, primary, Mody, Rajen, primary, Chinnaiyan, Arul, primary, Kurokawa, Ryo, primary, Pratt, Drew, primary, Yadav, Viveka N., primary, Grill, Jacques, primary, Kline, Cassie, primary, Mueller, Sabine, primary, Resnick, Adam, primary, Nazarian, Javad, primary, Allen, Joshua E., primary, Odia, Yazmin, primary, Gardner, Sharon L., primary, and Koschmann, Carl, primary

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2023
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25. CTNI-24. CLINICAL ACTIVITY AND SAFETY OF THE RAF INHIBITOR TOVORAFENIB IN PATIENTS WITH OPTIC PATHWAY GLIOMAS IN THE REGISTRATIONAL PEDIATRIC LOW-GRADE GLIOMA ARM OF THE PHASE 2 FIREFLY-1 (PNOC026) STUDY

Author

Nysom, Karsten, primary, Kilburn, Lindsay, additional, Leary, Sarah, additional, Landi, Daniel, additional, de Vos-Kerkhof, E, additional, Perreault, Sébastien, additional, Witt, Olaf, additional, Ziegler, David, additional, Driever, Pablo Hernáiz, additional, Franson, Andrea, additional, Baxter, Patricia, additional, Whipple, Nicholas S, additional, Kline, Cassie, additional, Segal, Devorah, additional, Jabado, Nada, additional, Bailey, Simon, additional, McCowage, Geoffrey, additional, Hansford, Jordan, additional, Khuong-Quang, Dong-Anh, additional, Gottardo, Nicholas, additional, Hassall, Timothy, additional, Han, Jung Woo, additional, Oren, Michal Yalon, additional, Chi, Susan, additional, Lee, Vai, additional, McLeod, Lisa, additional, Qiu, Jiaheng, additional, Walter, Ashley, additional, Manley, Peter, additional, and Hargrave, Darren, additional

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2023
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26. CTNI-84. PNOC022: A COMBINATION THERAPY TRIAL USING AN ADAPTIVE PLATFORM DESIGN FOR PATIENTS WITH DIFFUSE MIDLINE GLIOMAS (DMGS) AT INITIAL DIAGNOSIS, POST-RADIATION THERAPY AND AT TIME OF PROGRESSION

Author

Kline, Cassie, primary, Franson, Andrea, additional, Banerjee, Anu, additional, Reddy, Alyssa T, additional, Raber, Shannon, additional, Hoffman, Carly, additional, Minturn, Jane, additional, van der Lugt, Jasper, additional, de Vos-Kerkhof, E, additional, van Vuurden, Dannis, additional, Gerber, Nicolas, additional, Stuecklin, Ana Guerreiro, additional, Kilburn, Lindsay, additional, Elster, Jennifer, additional, Whipple, Nicholas, additional, Cohen, Kenneth, additional, Oren, Michal Yalon, additional, Gardner, Sharon, additional, Cluster, Andrew, additional, Chi, Susan, additional, Landi, Daniel, additional, McCowage, Geoffrey, additional, Resnick, Adam, additional, Phillips, Joanna, additional, Bhatia, Aashim, additional, Vossough, Arastoo, additional, George, Elizabeth, additional, Nabavizadeh, Ali, additional, Braunstein, Steve, additional, Gupta, Nalin, additional, Prados, Michael, additional, Waszak, Sebastian, additional, Boyle-Lang, Janel, additional, Molinaro, Annette, additional, Dun, Matthew, additional, Koschmann, Carl, additional, Nazarian, Javad, additional, and Mueller, Sabine, additional

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2023
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27. Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, additional, Ji, Sunjong, additional, Waszak, Sebastian M., additional, Sweha, Stefan R., additional, Mota, Mateus, additional, Pun, Matthew, additional, Deogharkar, Akash, additional, Chung, Chan, additional, Tarapore, Rohinton S., additional, Ramage, Samuel, additional, Chi, Andrew, additional, Wen, Patrick Y., additional, Arrillaga-Romany, Isabel, additional, Batchelor, Tracy T., additional, Butowski, Nicholas A., additional, Sumrall, Ashley, additional, Shonka, Nicole, additional, Harrison, Rebecca A., additional, de Groot, John, additional, Mehta, Minesh, additional, Hall, Matthew D., additional, Daghistani, Doured, additional, Cloughesy, Timothy F., additional, Ellingson, Benjamin M., additional, Beccaria, Kevin, additional, Varlet, Pascale, additional, Kim, Michelle M., additional, Umemura, Yoshie, additional, Garton, Hugh, additional, Franson, Andrea, additional, Schwartz, Jonathan, additional, Jain, Rajan, additional, Kachman, Maureen, additional, Baum, Heidi, additional, Burant, Charles F., additional, Mottl, Sophie L., additional, Cartaxo, Rodrigo T., additional, John, Vishal, additional, Messinger, Dana, additional, Qin, Tingting, additional, Peterson, Erik, additional, Sajjakulnukit, Peter, additional, Ravi, Karthik, additional, Waugh, Alyssa, additional, Walling, Dustin, additional, Ding, Yujie, additional, Xia, Ziyun, additional, Schwendeman, Anna, additional, Hawes, Debra, additional, Yang, Fusheng, additional, Judkins, Alexander R., additional, Wahl, Daniel, additional, Lyssiotis, Costas A., additional, de la Nava, Daniel, additional, Alonso, Marta M., additional, Eze, Augustine, additional, Spitzer, Jasper, additional, Schmidt, Susanne V., additional, Duchatel, Ryan J., additional, Dun, Matthew D., additional, Cain, Jason E., additional, Jiang, Li, additional, Stopka, Sylwia A., additional, Baquer, Gerard, additional, Regan, Michael S., additional, Filbin, Mariella G., additional, Agar, Nathalie Y.R., additional, Zhao, Lili, additional, Kumar-Sinha, Chandan, additional, Mody, Rajen, additional, Chinnaiyan, Arul, additional, Kurokawa, Ryo, additional, Pratt, Drew, additional, Yadav, Viveka N., additional, Grill, Jacques, additional, Kline, Cassie, additional, Mueller, Sabine, additional, Resnick, Adam, additional, Nazarian, Javad, additional, Allen, Joshua E., additional, Odia, Yazmin, additional, Gardner, Sharon L., additional, and Koschmann, Carl, additional

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2023
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31. Supplementary Table S3A and S3B from Electronic DNA Analysis of CSF Cell-free Tumor DNA to Quantify Multi-gene Molecular Response in Pediatric High-grade Glioma

Author

Bruzek, Amy K., primary, Ravi, Karthik, primary, Muruganand, Ashwath, primary, Wadden, Jack, primary, Babila, Clarissa May, primary, Cantor, Evan, primary, Tunkle, Leo, primary, Wierzbicki, Kyle, primary, Stallard, Stefanie, primary, Dickson, Robert P., primary, Wolfe, Ian, primary, Mody, Rajen, primary, Schwartz, Jonathan, primary, Franson, Andrea, primary, Robertson, Patricia L., primary, Muraszko, Karin M., primary, Maher, Cormac O., primary, Garton, Hugh J.L., primary, Qin, Tingtin, primary, and Koschmann, Carl, primary

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2023
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32. Supplementary Data from Electronic DNA Analysis of CSF Cell-free Tumor DNA to Quantify Multi-gene Molecular Response in Pediatric High-grade Glioma

Author

Bruzek, Amy K., primary, Ravi, Karthik, primary, Muruganand, Ashwath, primary, Wadden, Jack, primary, Babila, Clarissa May, primary, Cantor, Evan, primary, Tunkle, Leo, primary, Wierzbicki, Kyle, primary, Stallard, Stefanie, primary, Dickson, Robert P., primary, Wolfe, Ian, primary, Mody, Rajen, primary, Schwartz, Jonathan, primary, Franson, Andrea, primary, Robertson, Patricia L., primary, Muraszko, Karin M., primary, Maher, Cormac O., primary, Garton, Hugh J.L., primary, Qin, Tingtin, primary, and Koschmann, Carl, primary

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2023
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36. GABAergic neuron-to-glioma synapses in diffuse midline gliomas

Author

Barron, Tara, primary, Yalçın, Belgin, additional, Mochizuki, Aaron, additional, Cantor, Evan, additional, Shamardani, Kiarash, additional, Tlais, Dana, additional, Franson, Andrea, additional, Lyons, Samantha, additional, Mehta, Vilina, additional, Jahan, Samin Maleki, additional, Taylor, Kathryn R., additional, Keough, Michael B., additional, Xu, Haojun, additional, Su, Minhui, additional, Quezada, Michael A., additional, Woo, Pamelyn J, additional, Fisher, Paul G., additional, Campen, Cynthia J., additional, Partap, Sonia, additional, Koschmann, Carl, additional, and Monje, Michelle, additional

Published
2022
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38. CTNI-09. DIFFUSE MIDLINE GLIOMA-ADAPTIVE COMBINATORY TRIAL (DMG-ACT): A COMBINATION PLATFORM TRIAL IN PEDIATRIC AND YOUNG ADULT PATIENTS WITH DIFFUSE MIDLINE GLIOMA

Author

Franson, Andrea, primary, Kline, Cassie, additional, Molinaro, Annette, additional, Zhang, Yalan, additional, Hitchner, Kelly, additional, Gupta, Nalin, additional, Koschmann, Carl, additional, Prados, Michael, additional, Nazarian, Javad, additional, and Mueller, Sabine, additional

Published
2022
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39. CTNI-68. FIREFLY-1 (PNOC026): PHASE 2 STUDY OF PAN-RAF INHIBITOR TOVORAFENIB IN PEDIATRIC AND YOUNG ADULT PATIENTS WITH RAF-ALTERED RECURRENT OR PROGRESSIVE LOW-GRADE GLIOMA OR ADVANCED SOLID TUMORS

Author

Kilburn, Lindsay, primary, Landi, Daniel, additional, Leary, Sarah, additional, Ziegler, David, additional, Baxter, Patricia, additional, Franson, Andrea, additional, McCowage, Geoffrey, additional, Waanders, Angela, additional, Van der Lugt, Jasper, additional, Oren, Michal Yalon, additional, Gerber, Nicolas, additional, Gottardo, Nicholas, additional, Khuong-Quang, Dong-Anh, additional, Nysom, Karsten, additional, Bailey, Simon, additional, Driever, Pablo Hernáiz, additional, Perreault, Sebastien, additional, Witt, Olaf, additional, Hahn, Seungmin, additional, Hargrave, Darren, additional, Hassall, Timothy, additional, Jabado, Nada, additional, Kang, Hyoung Jin, additional, Larouche, Valerie, additional, Toledano, Helen, additional, Kline, Cassie, additional, Abdelbaki, Mohamed, additional, Chi, Susan, additional, Gardner, Sharon, additional, Whipple, Nicholas, additional, Mueller, Sabine, additional, Blackman, Samuel, additional, Zhao, Xin, additional, Da Costa, Daniel, additional, Cox, Michael, additional, Packer, Roger, additional, and Hansford, Jordan, additional

Published
2022
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41. CTNI-61. CLINICAL EFFICACY AND PREDICTIVE BIOMARKERS OF ONC201 IN H3K27M-MUTANT DIFFUSE MIDLINE GLIOMA

Author

Kawakibi, Abed Rahman, primary, Tarapore, Rohinton, additional, Gardner, Sharon, additional, Chi, Andrew, additional, Kurz, Sylvia, additional, Wen, Patrick Y, additional, Arrillaga-Romany, Isabel, additional, Batchelor, Tracy, additional, Butowski, Nicholas, additional, Sumrall, Ashley, additional, Shonka, Nicole, additional, Harrison, Rebecca, additional, DeGroot, John, additional, Mehta, Minesh, additional, Odia, Yazmin, additional, Hall, Matthew, additional, Daghistani, Doured, additional, Cloughesy, Timothy, additional, Ellingson, Benjamin, additional, Kim, Michelle, additional, Umemura, Yoshie, additional, Garton, Hugh, additional, Franson, Andrea, additional, Schwartz, Jonathan, additional, Li, Sunjong, additional, Cartaxo, Rodrigo, additional, Ravi, Karthik, additional, Cantor, Evan, additional, Cummings, Jessica, additional, Paul, Alyssa, additional, Walling, Dustin, additional, Dun, Matthew, additional, Cain, Jason, additional, Li, Jiang, additional, Filbin, Mariella, additional, Zhao, Lili, additional, Kumar-Sinha, Chandan, additional, Mody, Rajen, additional, Chinnaiyan, Arul, additional, Kurokawa, Ryo, additional, Pratt, Drew, additional, Venneti, Sriram, additional, Grill, Jacques, additional, Kline, Cassie, additional, Mueller, Sabine, additional, Resnick, Adam C, additional, Nazarian, Javad, additional, Waszak, Sebastian, additional, Allen, Joshua E, additional, and Koschmann, Carl, additional

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2022
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42. The Children’s Brain Tumor Network (CBTN) - Accelerating Research in Pediatric Central Nervous System Tumors through Collaboration and Open Science

Author

Lilly, Jena V., primary, Rokita, Jo Lynne, additional, Mason, Jennifer L., additional, Patton, Tatiana, additional, Stefankiewiz, Stephanie, additional, Higgins, David, additional, Trooskin, Gerri, additional, Larouci, Carina A., additional, Arya, Kamnaa, additional, Appert, Elizabeth, additional, Heath, Allison P., additional, Zhu, Yuankun, additional, Brown, Miguel A., additional, Zhang, Bo, additional, Farrow, Bailey K., additional, Robins, Shannon, additional, Morgan, Allison M., additional, Nguyen, Thinh Q., additional, Frenkel, Elizabeth, additional, Lehmann, Kaitlin, additional, Drake, Emily, additional, Sullivan, Catherine, additional, Plisiewicz, Alexa, additional, Coleman, Noel, additional, Patterson, Luke, additional, Koptyra, Mateusz, additional, Helili, Zeinab, additional, Van Kuren, Nicholas, additional, Young, Nathan, additional, Kim, Meen Chul, additional, Friedman, Christopher, additional, Lubneuski, Alex, additional, Blackden, Christopher, additional, Williams, Marti, additional, Baubet, Valerie, additional, Tauhid, Lamiya, additional, Galanaugh, Jamie, additional, Boucher, Katie, additional, Ijaz, Heba, additional, Cole, Kristina A., additional, Choudhari, Namrata, additional, Santi, Mariarita, additional, Moulder, Robert W., additional, Waller, Jonathan, additional, Rife, Whitney, additional, Diskin, Sharon J., additional, Mateos, Marion, additional, Parsons, Donald W., additional, Pollack, Ian F., additional, Goldman, Stewart, additional, Leary, Sarah, additional, Caporalini, Chiara, additional, Buccoliero, Anna Maria, additional, Scagnet, Mirko, additional, Haussler, David, additional, Hanson, Derek, additional, Firestein, Ron, additional, Cain, Jason, additional, Phillips, Joanna J., additional, Gupta, Nalin, additional, Mueller, Sabine, additional, Grant, Gerald, additional, Monje-Deisseroth, Michelle, additional, Partap, Sonia, additional, Greenfield, Jeffrey P., additional, Hashizume, Rintaro, additional, Smith, Amy, additional, Zhu, Shida, additional, Johnston, James M., additional, Fangusaro, Jason R, additional, Miller, Matthew, additional, Wood, Matthew D., additional, Gardner, Sharon, additional, Carter, Claire L., additional, Prolo, Laura M., additional, Pisapia, Jared, additional, Pehlivan, Katherine, additional, Franson, Andrea, additional, Niazi, Toba, additional, Rubin, Josh, additional, Abdelbaki, Mohamed, additional, Ziegler, David S., additional, Lindsay, Holly B., additional, Stucklin, Ana Guerreiro, additional, Gerber, Nicolas, additional, Vaske, Olena M., additional, Quinsey, Carolyn, additional, Rood, Brian R., additional, Nazarian, Javad, additional, Raabe, Eric, additional, Jackson, Eric M., additional, Stapleton, Stacie, additional, Lober, Robert M., additional, Kram, David E., additional, Koschmann, Carl, additional, Storm, Phillip B., additional, Lulla, Rishi R., additional, Prados, Michael, additional, Resnick, Adam C., additional, and Waanders, Angela J., additional

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2022
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43. Development of immunotherapy for high-grade gliomas: Overcoming the immunosuppressive tumor microenvironment

Author

Franson, Andrea, primary, McClellan, Brandon L., additional, Varela, Maria Luisa, additional, Comba, Andrea, additional, Syed, Mohammad Faisal, additional, Banerjee, Kaushik, additional, Zhu, Ziwen, additional, Gonzalez, Nazareno, additional, Candolfi, Marianela, additional, Lowenstein, Pedro, additional, and Castro, Maria Graciela, additional

Published
2022
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44. FIREFLY-1 (PNOC 026): A phase 2 study to evaluate the safety and efficacy of tovorafenib (DAY101) in pediatric patients with RAF-altered recurrent or progressive low-grade glioma or advanced solid tumors.

Author

Landi, Daniel B, primary, Ziegler, David S., additional, Franson, Andrea Flynn, additional, Baxter, Patricia Ann, additional, Leary, Sarah, additional, Larouche, Valérie, additional, Waanders, Angela Jae, additional, Van der Lugt, Jasper, additional, McCowage, Geoffrey Brian, additional, Doz, Francois, additional, Jabado, Nada, additional, Schiavello, Elisabetta, additional, Zapotocky, Michal, additional, Cornelio, Izzy, additional, Blackman, Samuel C, additional, Da Costa, Daniel, additional, Cox, Michael Craig, additional, Witt, Olaf, additional, Kilburn, Lindsay Baker, additional, and Hansford, Jordan R., additional

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2022
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46. DIPG-49. International preclinical drug discovery and biomarker program informing an adoptive combinatorial trial for DMG

Author

Nazarian, Javad, primary, Dun, Matthew, additional, Kilburn, Lindsay, additional, Waszak, Sebastian, additional, Vitanza, Nicholas, additional, Franson, Andrea, additional, Prados, Mike, additional, Raabe, Eric, additional, Firestein, Ron, additional, Beck, Alexander, additional, Saratsis, Amanda, additional, Rotblat, Barak, additional, van Vuurder, Dannis, additional, Foster, Jessica, additional, Hulleman, Esther, additional, Kline, Cassie, additional, Gupta, Nalin, additional, Cain, Jason, additional, Koschmann, Carl, additional, and Muller, Sabine, additional

Published
2022
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48. HGG-11. Clinical characteristics and clinical evolution of a large cohort of pediatric patients with primary central nervous system (CNS) tumors and tropomyosin receptor kinase (TRK) fusion.

Author

Lamoureux, Audrey-Anne, primary, Fisher, Michael, additional, Lemelle, Lauriane, additional, Pfaff, Elke, additional, Kramm, Christof, additional, De Wilde, Bram, additional, Kazanowska, Bernarda, additional, Hutter, Caroline, additional, Pfister, Stefan M, additional, Sturm, Dominik, additional, Jones, David, additional, Orbach, Daniel, additional, Pierron, Gaëlle, additional, Raskin, Scott, additional, Drilon, Alexander, additional, Diamond, Eli, additional, Harada, Guilherme, additional, Zapotocky, Michal, additional, Ellezam, Benjamin, additional, Weil, Alexander G, additional, Venne, Dominic, additional, Barritault, Marc, additional, Leblond, Pierre, additional, Coltin, Hallie, additional, Hammad, Rawan, additional, Tabori, Uri, additional, Hawkins, Cynthia, additional, Hansford, Jordan R, additional, Meyran, Deborah, additional, Erker, Craig, additional, McFadden, Kathryn, additional, Sato, Mariko, additional, Gottardo, Nicholas G, additional, Dholaria, Hetal, additional, Nørøxe, Dorte Schou, additional, Goto, Hiroaki, additional, Ziegler, David S, additional, Lin, Frank Y, additional, Parsons, Donald Williams, additional, Lindsay, Holly, additional, Wong, Tai-Tong, additional, Liu, Yen-Lin, additional, Wu, Kuo-Sheng, additional, Franson, Andrea Flynn, additional, Hwang, Eugene, additional, Aguilar-Bonilla, Ana, additional, Cheng, Sylvia, additional, Cacciotti, Chantel, additional, Massimino, Maura, additional, Schiavello, Elisabetta, additional, Wood, Paul, additional, Hoffman, Lindsey M, additional, Cappellano, Andréa, additional, Lassaletta, Alvaro, additional, Van Damme, An, additional, Llort, Anna, additional, Gerber, Nicolas U, additional, Ceruso, Mariella Spalato, additional, Bendel, Anne E, additional, Skrypek, Maggie, additional, Hamideh, Dima, additional, Mushtaq, Naureen, additional, Walter, Andrew, additional, Jabado, Nada, additional, Alsahlawi, Aysha, additional, Farmer, Jean-Pierre, additional, Abadi, Christina Coleman, additional, Mueller, Sabine, additional, Mazewski, Claire, additional, Aguilera, Dolly, additional, Robison, Nathan, additional, O’Halloran, Katrina, additional, Abbou, Samuel, additional, Berlanga, Pablo, additional, Geoerger, Birgit, additional, Øra, Ingrid, additional, Moertel, Christopher L, additional, Razis, Evangelia D, additional, Vernadou, Anastasia, additional, Doz, François, additional, Laetsch, Theodore W, additional, and Perreault, Sébastien, additional

Published
2022
Full Text
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