Article
Oncology
Thomas Blom, Roosmarijn Lurvink, Leonie Aleven, Maarten Mensink, Tom Wolfs, Miranda Dierselhuis, Natasha van Eijkelenburg, Kathelijne Kraal, Max van Noesel, Martine van Grotel, Godelieve Tytgat
Summary: The study described treatment-related toxicities during immunotherapy with dinutuximab, IL-2, GM-CSF, and isotretinoin in high-risk neuroblastoma patients, showing that a significant proportion of patients discontinued immunotherapy due to disease progression, isotretinoin-associated liver toxicity, and catheter-related infection. Common grade >= 3 toxicities included pain, catheter-related infections, and fever.
FRONTIERS IN ONCOLOGY
(2021)
Article
Biochemistry & Molecular Biology
Andras Heczey, Xin Xu, Amy N. Courtney, Gengwen Tian, Gabriel A. Barragan, Linjie Guo, Claudia Martinez Amador, Nisha Ghatwai, Purva Rathi, Michael S. Wood, Yanchuan Li, Chunchao Zhang, Thorsten Demberg, Erica J. Di Pierro, Andrew C. Sher, Huimin Zhang, Birju Mehta, Sachin G. Thakkar, Bambi Grilley, Tao Wang, Brian D. Weiss, Antonino Montalbano, Meena Subramaniam, Chenling Xu, Chirag Sachar, Daniel K. Wells, Gianpietro Dotti, Leonid S. Metelitsa
Summary: This study presented updated interim results on the evaluation of GD2-specific CAR-expressing NKT cells co-expressing IL15 in children with NB. The results showed that GD2-CAR.15 NKT cells had anti-tumor activity, which could be enhanced by targeting BTG1.
Article
Biochemistry & Molecular Biology
Enrico Rango, Fabio Pastorino, Chiara Brignole, Arianna Mancini, Federica Poggialini, Salvatore Di Maria, Claudio Zamperini, Giulia Iovenitti, Anna Lucia Fallacara, Samantha Sabetta, Letizia Clementi, Massimo Valoti, Silvia Schenone, Adriano Angelucci, Mirco Ponzoni, Elena Dreassi, Maurizio Botta
Summary: Si306, an anticancer agent, has shown favorable activity against neuroblastoma models. However, its sub-optimal aqueous solubility poses a limitation for its development. In this study, Si306 was encapsulated into liposomes and modified with an antibody specific for neuroblastoma cells. The modified liposomes demonstrated increased cellular targeting and cytotoxic activity. In animal experiments, Si306 encapsulated in targeted liposomes showed significantly higher tumor uptake and improved survival rates.
Review
Oncology
Jaume Mora, Shakeel Modak, Joyce Kinsey, Carolyn E. Ragsdale, Hillard M. Lazarus
Summary: Colony-stimulating factors, such as GM-CSF, have been studied extensively for their potential to enhance the effectiveness of anti-GD2 monoclonal antibodies in high-risk neuroblastoma. However, access to these therapies is not uniform, and there is limited clinical data supporting the use of G-CSF as an alternative to GM-CSF. More research is needed to ensure equitable access to these treatments globally.
INTERNATIONAL JOURNAL OF CANCER
(2023)
Letter
Medicine, General & Internal
Concetta Quintarelli, Francesca del Bufalo, Franco Locatelli
Summary: Del Bufalo et al. describe a treatment using GD2-CART01 T cells that target GD2 and express a suicide gene for patients with relapsed or refractory pediatric neuroblastoma. Although this treatment suppresses tumor growth, T cell reexpansion and tumor GD2 expression were not observed in relapsed patients.
NEW ENGLAND JOURNAL OF MEDICINE
(2023)
Article
Medicine, General & Internal
Francesca Del Bufalo, Biagio De Angelis, Ignazio Caruana, Giada Del Baldo, Maria A. De Ioris, Annalisa Serra, Angela Mastronuzzi, Maria G. Cefalo, Daria Pagliara, Matteo Amicucci, Giuseppina Li Pira, Giovanna Leone, Valentina Bertaina, Matilde Sinibaldi, Stefano Di Cecca, Marika Guercio, Zeinab Abbaszadeh, Laura Iaffaldano, Monica Gunetti, Stefano Iacovelli, Rossana Bugianesi, Stefania Macchia, Mattia Algeri, Pietro Merli, Federica Galaverna, Rachid Abbas, Maria C. Garganese, Maria F. Villani, Giovanna S. Colafati, Federico Bonetti, Marco Rabusin, Katia Perruccio, Veronica Folsi, Concetta Quintarelli, Franco Locatelli
Summary: Immunotherapy with GD2-CAR T cells expressing the inducible caspase 9 suicide gene is a feasible and safe treatment option for high-risk neuroblastoma patients, with a sustained antitumor effect and a significant improvement in overall survival and event-free survival.
NEW ENGLAND JOURNAL OF MEDICINE
(2023)
Article
Multidisciplinary Sciences
Babak Moghimi, Sakunthala Muthugounder, Samy Jambon, Rachelle Tibbetts, Long Hung, Hamid Bassiri, Michael D. Hogarty, David M. Barrett, Hiroyuki Shimada, Shahab Asgharzadeh
Summary: The study highlights the importance of developing gated systems for predicting clinical toxicity of CAR T cells in solid tumors. By utilizing murine and human CAR-T cells specific for tumor antigens, the researchers were able to generate a GD2-B7H3 CAR-T with improved metabolic fitness and efficacy in controlling neuroblastoma growth in preclinical models. This innovative approach shows promising results in suppressing neuroblastoma with minimal adverse effects.
NATURE COMMUNICATIONS
(2021)
Article
Biochemistry & Molecular Biology
Johanna Theruvath, Marie Menard, Benjamin A. H. Smith, Miles H. Linde, Garry L. Coles, Guillermo Nicolas Dalton, Wei Wu, Louise Kiru, Alberto Delaidelli, Elena Sotillo, John L. Silberstein, Anna C. Geraghty, Allison Banuelos, Molly Thomas Radosevich, Shaurya Dhingra, Sabine Heitzeneder, Aidan Tousley, John Lattin, Peng Xu, Jing Huang, Nicole Nasholm, Andy He, Tracy C. Kuo, Emma R. B. Sangalang, Jaume Pons, Amira Barkal, Rachel E. Brewer, Kristopher D. Marjon, Jose G. Vilches-Moure, Payton L. Marshall, Ricardo Fernandes, Michelle Monje, Jennifer R. Cochran, Poul H. Sorensen, Heike E. Daldrup-Link, Irving L. Weissman, Julien Sage, Ravindra Majeti, Carolyn R. Bertozzi, William A. Weiss, Crystal L. Mackall, Robbie G. Majzner
Summary: The combination of anti-GD2 and CD47 blockade demonstrates robust anti-tumor activity in mouse models, particularly effective against neuroblastoma, osteosarcoma, and small-cell lung cancer.
Article
Oncology
Marjolein C. Stip, Mitchell Evers, Maaike Nederend, Chilam Chan, Karli R. Reiding, Mirjam J. Damen, Albert J. R. Heck, Sofia Koustoulidou, Ruud Ramakers, Gerard C. Krijger, Remmert de Roos, Edouard Souteyrand, Annelisa M. Cornel, Miranda P. Dierselhuis, Marco Jansen, Mark de Boer, Thomas Valerius, Geert van Tetering, Jeanette H. W. Leusen, Friederike Meyer-Wentrup
Summary: Researchers engineered an antibody called IgA3.0 ch14.18, which shows promise as a new therapy for neuroblastoma. The antibody has a longer half-life, increased protein stability, and potent tumor-killing abilities.
JOURNAL FOR IMMUNOTHERAPY OF CANCER
(2023)
Article
Pediatrics
William S. Panosyan, Daniel E. Panosyan, Jan Koster, Eduard H. Panosyan
Summary: Oncometabolism is a potential target for developing less myelotoxic cancer therapies, and lactate dehydrogenase A (LDHA) is crucial to the Warburg effect in neuroblastoma (NBL). Although advanced surgical, cytotoxic, and cell-differentiating therapies have improved the survival of children with NBL, the clinical outcomes for high-risk NBL still need improvement. This study confirms the prognostic value of LDHA expression in NBL and proposes the development of an immunoliposome loaded with an LDHA inhibitor, Oxamate, for targeted delivery using anti-GD2 monoclonal antibodies (mAb).
PEDIATRIC RESEARCH
(2023)
Review
Biochemistry & Molecular Biology
Godfrey Chi-Fung Chan, Carol Matias Chan
Summary: Neuroblastoma is a childhood cancer with a specific antigen known as GD2. Monoclonal antibodies targeting GD2 have been used successfully in high-risk cases, but there are variations in the products and administration regimens. The long-term efficacy and challenges related to immune evasion and resistance still need further investigation.
Article
Oncology
Amalia Varo, Alicia Castaneda, Saray Chamorro, Juan Pablo Munoz, Maite Gorostegui, Monica S. Celma, Sandra Lopez, Margarida Simao, Sara Perez-Jaume, Jaume Mora
Summary: A novel method of administering the GD2-binding mAb naxitamab, termed Step-Up infusion (STU), was developed to reduce the risk of severe adverse events. The STU regimen showed a reduction in the frequency of infusions associated with Grade >= 3 adverse events.
FRONTIERS IN ONCOLOGY
(2023)
Article
Oncology
Fiorella Iglesias Cardenas, Audrey Mauguen, Irene Y. Cheung, Kim Kramer, Brian H. Kushner, Govind Ragupathi, Nai-Kong V. Cheung, Shakeel Modak
Summary: Beta glucans, along with 3F8, showed anti-neuroblastoma activity with no major toxicity, providing partial or complete response in some treated patients. Positive responses were associated with specific genetic factors and not correlated with beta glucans dose or serum levels.
Article
Oncology
Matias J. Stagno, Andreas Schmidt, Jonas Bochem, Cristian Urla, Rupert Handgretinger, Karin M. Cabanillas Stanchi, Rafael Saup, Manon Queudeville, Joerg Fuchs, Steven W. Warmann, Evi Schmid
Summary: This study analyzed the role of epitope detection in monocytes (EDIM) technique for liquid biopsy in neuroblastoma (NB) patients. The results showed that the EDIM test could serve as a non-invasive tool for liquid biopsy in pediatric NB patients, aiding in risk stratification, tumor biology research, treatment monitoring, and early detection of tumor relapses.
BRITISH JOURNAL OF CANCER
(2022)
Article
Oncology
Wayne L. Furman, Beth McCarville, Barry L. Shulkin, Andrew Davidoff, Matthew Krasin, Chia-Wei Hsu, Haitao Pan, Jianrong Wu, Rachel Brennan, Michael W. Bishop, Sara Helmig, Elizabeth Stewart, Fariba Navid, Brandon Triplett, Victor Santana, Teresa Santiago, Jacquelyn A. Hank, Stephen D. Gillies, Alice Yu, Paul M. Sondel, Wing H. Leung, Alberto Pappo, Sara M. Federico
Summary: Combining hu14.18K322A with chemotherapy improves early response and outcomes in high-risk neuroblastoma. This treatment is well-tolerated and reduces tumor volume in most patients, leading to improved end-of-induction response rates and encouraging 3-year event-free survival.
JOURNAL OF CLINICAL ONCOLOGY
(2022)
Editorial Material
Neuroimaging
Joseph Anthony Carnevale, Jacob Goldberg, Gary Kocharian, Maricruz Rivera, Alexandra Giantini Larsen, Andrew Garton, Alexander Ramos, Jasmine H. Francis, David H. Abramson, Y. Pierre Gobin
JOURNAL OF NEUROINTERVENTIONAL SURGERY
(2023)
Article
Clinical Neurology
Carolin Melcher, Ibrahim Hussain, Sertac Kirnaz, Jacob L. Goldberg, Fabian Sommer, Rodrigo Navarro-Ramirez, Branden Medary, Roger Hartl
Summary: This prospective study aimed to evaluate the educational benefit of using a high-fidelity simulator for surgical trainees performing minimally invasive unilateral laminotomy for bilateral decompression (ULBD) for lumbar stenosis. The results showed improved procedural metrics, decreased errors and skipped steps, and increased surgical proficiency among the trainees. The study suggests that surgical simulation using realistic simulators can enhance skills and confidence in trainees, especially in junior residents.
GLOBAL SPINE JOURNAL
(2023)
Article
Oncology
Kyle J. Morgan, Andrew Dudas, Wayne L. Furman, M. Beth McCarville, Barry L. Shulkin, Zhaohua Lu, Himani Darji, Doralina L. Anghelescu
Summary: This study examined the association between opioid consumption and tumor response in children with neuroblastoma. The results showed that there was no significant correlation between opioid use and tumor volume or burden.
PEDIATRIC BLOOD & CANCER
(2023)
Editorial Material
Oncology
Hollie A. Lai, Susan E. Sharp, Aashim Bhatia, Kelly R. Dietz, Beth McCarville, Dhanashree Rajderkar, Sabah Servaes, Barry L. Shulkin, Sudh A. Singh, Andrew T. Trout, Pankaj Watal, Marguerite T. Parisi
Summary: This manuscript provides consensus-based imaging recommendations for diagnosing and monitoring pediatric neuroblastoma patients.
PEDIATRIC BLOOD & CANCER
(2023)
Article
Oncology
Mitchell A. Rees, Cara E. Morin, Gerald G. Behr, J. Christopher Davis, Hollie Lai, Ajaykumar C. Morani, Marguerite T. Parisi, Gaurav Saigal, Sudha Singh, Sireesha Yedururi, Alexander J. Towbin, Barry L. Shulkin
Summary: This paper provides consensus recommendations for imaging of pediatric patients with adrenal malignancies other than neuroblastoma.
PEDIATRIC BLOOD & CANCER
(2023)
Review
Radiology, Nuclear Medicine & Medical Imaging
Hedieh Khalatbari, Barry L. Shulkin, Marguerite T. Parisi
Summary: The most common indication for 18F-FDG PET/CT is tumor imaging, but in pediatric nuclear medicine, most coincidental findings of infectious, inflammatory, and autoimmune processes are not the main indication and it is important to interpret these findings correctly. This review focuses on recent literature on fever of unknown origin and inflammation of unknown origin in pediatrics, as well as the PET/CT imaging findings in two specific diseases with increased immune reactivity.
SEMINARS IN NUCLEAR MEDICINE
(2023)
Article
Neurosciences
James R. McIntosh, Evan F. Joiner, Jacob L. Goldberg, Lynda M. Murray, Bushra Yasin, Anil Mendiratta, Steven C. Karceski, Earl Thuet, Oleg Modik, Evgeny Shelkov, Joseph M. Lombardi, Zeeshan M. Sardar, Ronald A. Lehman, Christopher Mandigo, K. Daniel Riew, Noam Y. Harel, Michael S. Virk, Jason B. Carmel
Summary: During clinically indicated surgery, cervical epidural stimulation was found to activate arm and hand muscles most effectively when stimulating laterally compared to midline. This map of the cervical spinal cord reveals organization and may guide stimulation for stronger and selective activation of arm and hand muscles.
JOURNAL OF NEUROPHYSIOLOGY
(2023)
Article
Oncology
Samuel N. Rogers, Unni Udayasankar, Sumit Pruthi, Hollie A. Lai, Nadja Kadom, Julie Guerin, Michael Malinzak, Barry L. Shulkin, Tina Y. Poussaint, Zoltan Patay, Asim K. Bag
Summary: This paper provides consensus-based recommendations for optimized imaging of childhood spinal tumors, including diagnosis, follow-up during and after therapy, and response assessment.
PEDIATRIC BLOOD & CANCER
(2023)
Review
Clinical Neurology
Taylor Wong, Aneek Patel, Danielle Golub, Sertac Kirnaz, Jacob L. Goldberg, Fabian Sommer, Franziska A. Schmidt, Raj Nangunoori, Ibrahim Hussain, Roger Hartl
Summary: Patients with LDH have a higher prevalence of long-term LBP compared to the general population (46.2% vs. 11.9%). The relationship between radiological findings and pain outcomes in LDH remains unclear and requires further research.
WORLD NEUROSURGERY
(2023)
Article
Clinical Neurology
Jacob L. Goldberg, Joseph A. Carnevale, Jimmy Xia, Fabian Sommer, Pravesh Gadjradj, Branden Medary, Alexandra Giantini-Larsen, Rodrigo Navarro-Ramirez, A. John Tsiouris, Vikram Chakravarthy, Justin T. Schwarz, Lynn B. McGrath, Michael S. Virk, Kai-Ming Fu, K. Daniel Riew, Ibrahim Hussain, Roger Hartl
Summary: This study describes the site of pedicle attachment to the vertebral body in the subaxial cervical spine. It found that pedicles mostly attach to the top third of the vertebral body, but there is also significant variation. The rate of variation is highest at C3 and decreases linearly with caudal progression down the subaxial cervical spine to T1. This is the first report investigating this morphological phenomenon.
OPERATIVE NEUROSURGERY
(2023)
Article
Oncology
Amy K. Erbe, Mitch B. Diccianni, Rajen Mody, Arlene Naranjo, Fan F. Zhang, Jen Birstler, KyungMann Kim, Arika S. Feils, Jung-Tung Hung, Wendy B. London, Barry L. Shulkin, Varsha Mathew, Marguerite T. Parisi, Sabah Servaes, Shahab Asgharzadeh, John M. Maris, Julie Park, Alice L. Yu, Paul M. Sondel, Rochelle Bagatell
Summary: In the Children's Oncology Group ANBL1221 trial, the presence or absence of certain killer immunoglobulin-like receptors (KIRs) and their ligands was associated with clinical outcomes in patients with high-risk neuroblastoma treated with chemoimmunotherapy. The study confirms the importance of KIR/KIR-ligand genotype in the context of this treatment regimen.
JOURNAL FOR IMMUNOTHERAPY OF CANCER
(2023)
Article
Clinical Neurology
Scott L. Zuckerman, Jacob L. Goldberg, Meghan Cerpa, Meghana Vulapalli, Mychael W. Delgardo, Xena E. Flowers, Sandra Leskinen, Mena G. Kerolus, Ian A. Buchanan, Alex S. Ha, K. Daniel Riew
Summary: Grip strength significantly improved in approximately 42% of patients undergoing cervical spine surgery. Patients with radiculopathy were more likely to experience grip strength improvement compared to patients with myelopathy. Patients undergoing surgery at the C2/3-C5/6 and C6/7-T1/2 levels showed significant grip strength improvement at 3-month postoperative follow-up.
GLOBAL SPINE JOURNAL
(2023)
Article
Clinical Neurology
Alexander Ramos, Joseph A. Carnevale, Kashif Majeed, Gary Kocharian, Ibrahim Hussain, Jacob L. Goldberg, Justin Schwarz, David Kutler, Jared Knopman, Philip Stieg
Summary: This study describes a series of patients with carotid body tumors (CBTs) successfully treated via resection following preoperative embolization and compares the results with previous reported outcomes. The results show that preoperative embolization is a safe and effective technique for tumor devascularization, making it an important consideration in the management of CBT. Moreover, a multidisciplinary approach is crucial for optimal management of patients with CBT.
JOURNAL OF NEUROSURGERY
(2023)
Article
Clinical Neurology
Scott L. Zuckerman, Jacob L. Goldberg, Meghan Cerpa, Meghana Vulapalli, Mychael W. Delgardo, Xena E. Flowers, Sandra Leskinen, Mena G. Kerolus, Ian A. Buchanan, Alex S. Ha, K. Daniel Riew
Summary: This retrospective cohort study examined changes in hand function in patients undergoing cervical spine surgery. The results showed that grip strength significantly improved in 42% of patients. Patients with radiculopathy were more likely to experience improvement compared to those with myelopathy.
GLOBAL SPINE JOURNAL
(2023)
Article
Oncology
John Thomas Lucas, Daniel Victor Wakefield, Michael Doubrovin, Yimei Li, Teresa Santiago, Sara Michele Federico, Thomas E. Merchant, Andrew M. Davidoff, Matthew J. Krasin, Barry L. Shulkin, Victor M. Santana, Wayne Lee Furman
Summary: This retrospective study aimed to identify predictors of metastatic site failure in high-risk neuroblastoma patients. The study found that improvements in systemic therapy influenced the patterns and type of metastatic site failure.
CLINICAL AND TRANSLATIONAL RADIATION ONCOLOGY
(2022)