Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials

Authors: Río, Paula, Zubicaray, Josune, Navarro, Susana, Gálvez, Eva, Sánchez-Domínguez, Rebeca, Nicoletti, Eileen, Sebastián, Elena, Rothe, Michael, Pujol, Roser, Bogliolo, Massimo, John-Neek, Philipp, Bastone, Antonella L, Schambach, Axel, Wang, Wei, Schmidt, Manfred, Larcher, Lise, Segovia, José C, Yáñez, Rosa M, Pérez-Maroto, Florencio, de la Cruz, Ana, Casado, José A, Giménez, Yari, Álvarez, Lara, Alberquilla, Omaira, Díez, Begoña, Fernández-García, María, García-García, Laura, Gómez, Ana, Galán, Almudena, Ramírez, Manuel, Salgado, Rocío, Galy, Anne, Lefrere, Francois, Cavazzana, Marina, Leblanc, Thierry, Garcia de Andoin, Nagore, López-Almaraz, Ricardo, Catalá, Albert, Barquinero, Jordi, Rodríguez-Perales, Sandra, Rao, Gayatri, Surrallés, Jordi, Soulier, Jean, Díaz-de-Heredia, Cristina, Schwartz, Jonathan D, Sevilla, Julián, Bueren, Juan A, Bastone, Antonella Lucía, García de Andoin, Nagore

Source: In The Lancet 21 December 2024-3 January 2025 404(10471):2584-2592

Predicting disease progression and the need for tumor-directed treatment in tectal plate gliomas

Authors: Bauman, Megan M. J., Giesken, Max B., Naylor, Ryan M., Keating, Gesina F., Schwartz, Jonathan D., Daniels, David J.

Source: Journal of neurosurgery.Pediatrics. 32(3):332-342

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCRN633441855&indx=1&recIds=ETOCRN633441855

Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD

Authors: Schwartz, Jonathan D., Barcellini, Wilma, Grace, Rachel F., Bianchi, Paola, Zanella, Alberto, López Lorenzo, José Luis, Sevilla, Julián, Shah, Ami J., Glader, Bertil, Nicoletti, Eileen, Navarro Ordoñez, Susana, Segovia, José Carlos

Source: American journal of hematology. 97(3):E120-E125

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100139959055.0x000001&indx=1&recIds=ETOCvdc_100139959055.0x000001

Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I

Authors: Mesa-Núñez, Cristina, Damián, Carlos, Fernández-García, María, Díez, Begoña, Rao, Gayatri, Schwartz, Jonathan D., Law, Ken M., Sevilla, Julián, Río, Paula, Yáñez, Rosa, Bueren, Juan A., Almarza, Elena

Source: In Molecular Therapy - Methods & Clinical Development 8 September 2022 26:459-470

Drug delivery across the blood-brain barrier for the treatment of pediatric brain tumors – An update

Authors: Power, Erica A., Rechberger, Julian S., Gupta, Sumit, Schwartz, Jonathan D., Daniels, David J., Khatua, Soumen

Source: In Advanced Drug Delivery Reviews June 2022 185

Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment

Authors: Navarro, Susana, Quintana-Bustamante, Oscar, Sanchez-Dominguez, Rebeca, Lopez-Manzaneda, Sergio, Ojeda-Perez, Isabel, Garcia-Torralba, Aida, Alberquilla, Omaira, Law, Kenneth, Beard, Brian C., Bastone, Antonella, Rothe, Michael, Villanueva, Mariela, Ramirez, Juan C., Fañanas-Baquero, Sara, Nieto-Romero, Virginia, Molinos-Vicente, Andrea, Gutierrez, Sonia, Nicoletti, Eileen, García-Bravo, María, Bueren, Juan A., Schwartz, Jonathan D., Segovia, Jose-Carlos

Source: In Molecular Therapy - Methods & Clinical Development 10 September 2021 22:350-359

Precision Medicine in Pediatric Bithalamic Glioma: Significance of the EGFR exon 20 Insertion Mutation

Authors: Goyal, Anshit, Nesvick, Cody L., Raghunathan, Aditya, Schwartz, Jonathan D., Daniels, David J.

Source: In World Neurosurgery May 2021 149:271-273

Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal

Authors: Moscatelli, Ilana, Almarza, Elena, Schambach, Axel, Ricks, David, Schulz, Ansgar, Herzog, Christopher D., Henriksen, Kim, Askmyr, Maria, Schwartz, Jonathan D., Richter, Johan

Source: In Molecular Therapy - Methods & Clinical Development 12 March 2021 20:389-397

Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients

Authors: Ramírez, María José, Pujol, Roser, Trujillo‐Quintero, Juan Pablo, Minguillón, Jordi, Bogliolo, Massimo, Río, Paula, Navarro, Susana, Casado, José A., Badell, Isabel, Carrasco, Estela, Balmaña, Judith, Català, Albert, Sevilla, Julián, Beléndez, Cristina, Argilés, Bienvenida, López, Mónica, Díaz de Heredia, Cristina, Rao, Gayatri, Nicoletti, Eileen, Schwartz, Jonathan D., Bueren, Juan A., Surrallés, Jordi

Source: American journal of hematology. 96(8):989-999

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100123595252.0x000001&indx=1&recIds=ETOCvdc_100123595252.0x000001

Mosaicism in Fanconi anemia: concise review and evaluation of published cases with focus on clinical course of blood count normalization

Authors: Nicoletti, Eileen, Rao, Gayatri, Bueren, Juan A., Río, Paula, Navarro, Susana, Surrallés, Jordi, Choi, Grace, Schwartz, Jonathan D.

Source: Annals of hematology. 99(5):913-924

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100167866599.0x000001&indx=1&recIds=ETOCvdc_100167866599.0x000001

Diffuse Midline Gliomas without the H3 K27M Mutation: Surgical Biopsy is Needed to Better our Diagnostic Understanding

Authors: Lu, Victor M., Raghunathan, Aditya, Schwartz, Jonathan D., Daniels, David J.

Source: In World Neurosurgery May 2020 137:472-473

Survival Outcomes and Patterns of Care for Patients Diagnosed with Optic Pathway Glioma: Analysis of the Surveillance Epidemiology and End Results (SEER) Database.

Authors: Brown, Nolan J., Kuo, Cathleen C., Pennington, Zach, Bauman, Megan M., Gendreau, Julian, Givens, Drew B., Schwartz, Jonathan D., Daniels, David J

Source: Journal of Neurological Surgery. Part B. Skull Base; 2024 Supplement, Vol. 85, pS1-S398, 398p

Subject Terms: SURVIVAL rate, DATABASES, GLIOMAS, PROPORTIONAL hazards models, PATIENT care

Busulfan and subsequent malignancy: An evidence-based risk assessment.

Authors: Long-Boyle, Janel R., Kohn, Donald B., Shah, Ami J., Spencer, Sueli Marques, Sevilla, Julian, Booth, Claire, López Lorenzo, José Luis, Nicoletti, Eileen, Shah, Arpita, Reatz, Meredith, Matos, Joana, Schwartz, Jonathan D.

Source: Pediatric Blood & Cancer; Jan2024, Vol. 71 Issue 1, p1-11, 11p

A randomised non-comparative phase II trial of cixutumumab (IMC-A12) or ramucirumab (IMC-1121B) plus mitoxantrone and prednisone in men with metastatic docetaxel-pretreated castration-resistant prostate cancer

Authors: Hussain, Maha, Rathkopf, Dana, Liu, Glenn, Armstrong, Andrew, Kelly, Wm. Kevin, Ferrari, Anna, Hainsworth, John, Joshi, Adarsh, Hozak, Rebecca R., Yang, Ling, Schwartz, Jonathan D., Higano, Celestia S.

Source: In European Journal of Cancer September 2015 51(13):1714-1724

Ramucirumab versus placebo as second-line treatment in patients with advanced hepatocellular carcinoma following first-line therapy with sorafenib (REACH): a randomised, double-blind, multicentre, phase 3 trial

Authors: Zhu, Andrew X, Park, Joon Oh, Ryoo, Baek-Yeol, Yen, Chia-Jui, Poon, Ronnie, Pastorelli, Davide, Blanc, Jean-Frederic, Chung, Hyun Cheol, Baron, Ari D, Pfiffer, Tulio Eduardo Flesch, Okusaka, Takuji, Kubackova, Katerina, Trojan, Jorg, Sastre, Javier, Chau, Ian, Chang, Shao-Chun, Abada, Paolo B, Yang, Ling, Schwartz, Jonathan D, Kudo, Masatoshi

Source: In The Lancet Oncology July 2015 16(7):859-870

Letter to the Editor: Hematopoietic Stem and Progenitor Cell Mobilization and Collection for Patients Diagnosed with Osteopetrosis and Hurler Syndrome

Authors: Sevilla, Julián, Iriondo, June, Sebastian, Elena, Gonzalez-Vicent, Marta, Schwartz, Jonathan D., Zubicaray, Josune

Source: Human gene therapy. 33(3/4):213-214

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100140726909.0x000001&indx=1&recIds=ETOCvdc_100140726909.0x000001

Ramucirumab plus paclitaxel versus placebo plus paclitaxel in patients with previously treated advanced gastric or gastro-oesophageal junction adenocarcinoma (RAINBOW): a double-blind, randomised phase 3 trial

Authors: Wilke, Hansjochen, Muro, Kei, Van Cutsem, Eric, Oh, Sang-Cheul, Bodoky, György, Shimada, Yasuhiro, Hironaka, Shuichi, Sugimoto, Naotoshi, Lipatov, Oleg, Kim, Tae-You, Cunningham, David, Rougier, Philippe, Komatsu, Yoshito, Ajani, Jaffer, Emig, Michael, Carlesi, Roberto, Ferry, David, Chandrawansa, Kumari, Schwartz, Jonathan D, Ohtsu, Atsushi

Source: In Lancet Oncology October 2014 15(11):1224-1235

A phase II study of ramucirumab (IMC-1121B) in the treatment of persistent or recurrent epithelial ovarian, fallopian tube or primary peritoneal carcinoma

Authors: Penson, Richard T., Moore, Kathleen M., Fleming, Gini F., Braly, Patricia, Schimp, Veronica, Nguyen, Hoa, Matulonis, Ursula A., Banerjee, Susana, Haluska, Paul, Gore, Martin, Bodurka, Diane C., Hozak, Rebecca R., Joshi, Adarsh, Xu, Yihuan, Schwartz, Jonathan D., McGuire, William P.

Source: In Gynecologic Oncology September 2014 134(3):478-485

A phase 2 randomised study of ramucirumab (IMC-1121B) with or without dacarbazine in patients with metastatic melanoma

Authors: Carvajal, Richard D., Wong, Michael K., Thompson, John A., Gordon, Michael S., Lewis, Karl D., Pavlick, Anna C., Wolchok, Jedd D., Rojas, Patrick B., Schwartz, Jonathan D., Bedikian, Agop Y.

Source: In European Journal of Cancer August 2014 50(12):2099-2107

Ramucirumab monotherapy for previously treated advanced gastric or gastro-oesophageal junction adenocarcinoma (REGARD): an international, randomised, multicentre, placebo-controlled, phase 3 trial

Authors: Fuchs, Charles S, Tomasek, Jiri, Yong, Cho Jae, Dumitru, Filip, Passalacqua, Rodolfo, Goswami, Chanchal, Safran, Howard, dos Santos, Lucas Vieira, Aprile, Giuseppe, Ferry, David R, Melichar, Bohuslav, Tehfe, Mustapha, Topuzov, Eldar, Zalcberg, John Raymond, Chau, Ian, Campbell, William, Sivanandan, Choondal, Pikiel, Joanna, Koshiji, Minori, Hsu, Yanzhi, Liepa, Astra M, Gao, Ling, Schwartz, Jonathan D, Tabernero, Josep

Source: In The Lancet 4-10 January 2014 383(9911):31-39