Oportunidades e limitações para a fabricação de células CAR-T de grau clínico
DOI:
https://doi.org/10.54353/ritp.v4i2.001Palavras-chave:
innovación en salud, cáncer, células CAR-T, terapias celulares, manufacturaResumo
A terapia com células CAR-T é um dos tratamentos mais promissores para o câncer; no entanto, ainda está em fase final de estudo em ambiente clínico. Envolve a extração de células T do doador, modificando-as geneticamente para expressar um receptor de antígeno quimérico e infundindo as células modificadas nos pacientes. O objetivo deste artigo é avaliar os produtos e processos de produção de células CAR-T oferecidos para fins clínicos por empresas internacionais para determinar o estado atual destas tecnologias e sua potencial aplicação no Peru. Conclui-se que até o momento é uma terapia inacessível no Peru devido a necessidades que incluem: otimização dos processos de fabricação, melhorias no acesso, eficácia e segurança das terapias CAR-T e maior número de licenças. tanto o câncer quanto outras doenças.
Downloads
Referências
Abou-El-Enein, M., Elsallab, M., Feldman, S. A., Fesnak, A. D., Heslop, H. E., Marks, P., Till, B. G., Bauer, G., & Savoldo, B. (2021). Scalable Manufacturing of CAR T cells for Cancer Immunotherapy. Blood cancer discovery, 2(5), 408–422.
Brentjens, R. J., Latouche, J. B., Santos, E., Marti, F., Gong, M. C., Lyddane, C., King, P. D., Larson, S., Weiss, M., Rivière, I., & Sadelain, M. (2003). Eradication of systemic B-cell tumors by genetically targeted human T lymphocytes co-stimulated by CD80 and interleukin-15. Nature medicine, 9(3), 279–286.
Bryan, W., et al. (s.f.). Summary Basis for Regulatory Action Template Breyanzi. Available: https://fda.report/media/146242/Summary+Basis+for+Regulatory+Action+-+BREYANZI.pdf
Cai, Y., Prochazkova, M., Jiang, C., Song, H. W., Jin, J., Moses, L., Gkitsas, N., Somerville, R. P., Highfill, S. L., Panch, S., Stroncek, D. F., & Jin, P. (2021). Establishment and validation of in-house cryopreserved CAR/TCR-T cell flow cytometry quality control. Journal of translational medicine, 19(1), 523. https://doi.org/10.1186/s12967-021-03193-7
Cancer.org. (2019). Terapia de células CAR-T y sus efectos secundarios. https://www.cancer.org/es/tratamiento/tratamientos-y-efectos-secundarios/tipos-de-tratamiento/inmunoterapia/terapia-de-celulas-t.html
Cohen, A. D., Garfall, A. L., Stadtmauer, E. A., Melenhorst, J. J., Lacey, S. F., Lancaster, E., Vogl, D. T., Weiss, B. M., Dengel, K., Nelson, A., Plesa, G., Chen, F., Davis, M. M., Hwang, W. T., Young, R. M., Brogdon, J. L., Isaacs, R., Pruteanu-Malinici, I., Siegel, D. L., Levine, B. L., … Milone, M. C. (2019). B cell maturation antigen-specific CAR T cells are clinically active in multiple myeloma. The Journal of clinical investigation, 129(6), 2210–2221.
Depil, S., Duchateau, P., Grupp, S.A. et al. ‘Off-the-shelf’ allogeneic CAR T cells: development and challenges. Nat Rev Drug Discov 19, 185–199 (2020).
Dreyzin, A., Panch, S. R., Shalabi, H., Yates, B., Highfill, S. L., Jin, P., Stroncek, D., & Shah, N. N. (2022). Cryopreserved anti-CD22 and bispecific anti-CD19/22 CAR T cells are as effective as freshly infused cells. Molecular therapy. Methods & clinical development, 28, 51–61. https://doi.org/10.1016/j.omtm.2022.12.004
EsSalud. (2009). Normas Para La Investigación Clínica En Terapia Celular y Medicina Regenerativa de ESSALUD. [En línea]. Disponible en: https://ww1.essalud.gob.pe/compendio/pdf/0000002878_pdf.pdf
Ghassemi, S., & Milone, M. C. (2019). Manufacturing Chimeric Antigen Receptor (CAR) T Cells for Adoptive Immunotherapy. Journal of visualized experiments : JoVE, (154), 10.3791/59949.
Genotipia. (2022). Historia de la Terapia génica. https://genotipia.com/historia-de-la-terapia-genica/
Germann, A., Oh, Y.-J., Schmidt, T., Schön, U., Zimmermann, H., & von Briesen, H. (2013). Temperature fluctuations during deep temperature cryopreservation reduce PBMC recovery, viability and T-cell function. Cryobiology, 67(2), 193–200. https://doi.org/10.1016/j.cryobiol.2013.06.012
GLOBOCAN. (2020). Estimación de la prevalencia a los 5 años del diagnóstico de cáncer a nivel mundial para el año 2020. https://seom.org/images/LAS_CIFRAS_DEL_CANCER_EN_ESPANA_2022.pdf
Hanley, P. J. (2019). Fresh versus frozen: Effects of cryopreservation on CAR-T cells. Molecular Therapy, 27(7), 1213–1214.
Harris, E., & Elmer, J. J. (2018). Optimization of electroporation and other non‐viral gene delivery strategies for T cells. Biotechnology Progress, 37(1), e3066.
IASO Therapeutics. (s.f.). Science & Products - Manufacturing. IASO Therapeutics. Disponible: https://www.iasobio.com/operation.php
IASO Therapeutics. (s.f.). Science & Products - Pipeline. IASO Therapeutics. Disponible: https://www.iasobio.com/proline.php
Ino, K., Ageitos, A. G., Singh, R. K., & Talmadge, J. E. (2001). Activation-induced T cell apoptosis by monocytes from stem cell products. International immunopharmacology, 1(7), 1307–1319.
Instituto Nacional del Cáncer. (2021). ¿Qué es el cáncer? https://www.cancer.gov/espanol/cancer/naturaleza/que-es#:~:text=El%20c%C3%A1ncer%20que%20se%20disemin%C3%B3,cancerosas%20que%20el%20c%C3%A1ncer%20primario
Instituto Nacional del Cáncer. (2019). Cómo se diagnostica el cáncer. https://www.cancer.gov/espanol/cancer/diagnostico-estadificacion/diagnostico
Instituto Nacional del Cáncer. (2023). Efectos secundarios del tratamiento del cáncer. https://www.cancer.gov/espanol/cancer/tratamiento/efectos-secundarios
International Agency for Research on Cancer. (2020). Global Cancer Observatory: Cancer Today. Iarc.fr. https://gco.iarc.fr/today/home
Janssen. (2023). CARVYKTI - Manufacturing Process. Janssenscience.com. Disponible: https://www.janssenscience.com/products/carvykti/medical-content/carvykti-manufacturing-process
June C. H. (2007). Principles of adoptive T cell cancer therapy. The Journal of clinical investigation, 117(5), 1204–1212.
Köhl, U., Arsenieva, S., Holzinger, A., & Abken, H. (2018). CAR T Cells in Trials: Recent Achievements and Challenges that Remain in the Production of Modified T Cells for Clinical Applications. Human gene therapy, 29(5), 559–568.
Levine, B. Performance-enhancing drugs: design and production of redirected chimeric antigen receptor (CAR) T cells. Cancer Gene Ther 22, 79–84 (2015).
Lu, X. (2017). Summary Basis for Regulatory Action. [Online]. Disponible: https://www.fda.gov/files/vaccines%2C%20blood%20%26%20biologics/published/August-30--2017-Summary-Basis-for-Regulatory-Action---KYMRIAH.pdf
Lu, J., Jiang, G. The journey of CAR-T therapy in hematological malignancies. Mol Cancer 21, 194 (2022).
Málaga, Germán, & Zúñiga-Rivera, Ana. (2012). ¿Contribuyen los ensayos clínicos al
desarrollo de la investigación en el Perú?: ¿cómo lograrlo?. Revista Peruana de Medicina Experimental y Salud Publica, 29(4), 529-534. Recuperado en 27 de septiembre de 2023, de http://www.scielo.org.pe/scielo.php?script=sci_arttext&pid=S1726-46342012000400017&lng=es&tlng=es.
Master A. (2019). Kymriah vs. Yescarta [UPDATED] | Nucleus Biologics. Nucleus Biologics. https://nucleusbiologics.com/resources/kymriah-vs-yescarta/
McFarland, D. C., Zhang, C., Thomas, H. C., & T L, R. (2006). Confounding effects of platelets on flow cytometric analysis and cell-sorting experiments using blood-derived cells. Cytometry. Part A : the journal of the International Society for Analytical Cytology, 69(2), 86–94.
McGrath E, Machalik P. The Regulatory Framework for CAR-T Cells in Europe: Current Status and Foreseeable Changes AND Centre Qualification by Competent Authorities and Manufacturers. 2022 Feb 7. In: Kröger N, Gribben J, Chabannon C, et al., editors. The EBMT/EHA CAR-T Cell Handbook [Internet]. Cham (CH): Springer; 2022. Chapter 37.
Memorial Sloan Kettering Cancer Center. (s.f.). Car T cells: Timeline of progress. https://www.mskcc.org/timeline/car-t-timeline-progress
Novartis Pharmaceuticals Corporation. (2018). Package insert - KymriahTM (tisagenlecleucel). https://www.fda.gov/media/107296/download
Novas, A. (2020). Costo de la terapia con Células Madre y por qué es tan costosa - Curso Celulas Madre. https://cursocelulasmadre.com/costo-de-la-terapia-con-celulas-madre/
Qin, C., Tian DS., Zhou, LQ. et al. Anti-BCMA CAR T-cell therapy CT103A in relapsed or refractory AQP4-IgG seropositive neuromyelitis optica spectrum disorders: phase 1 trial interim results. Sig Transduct Target Ther 8, 5 (2023).
Reddy, O. L., Stroncek, D. F., & Panch, S. R. (2020). Improving CAR T cell therapy by optimizing critical quality attributes. Seminars in hematology, 57(2), 33–38.
Rioufol, C., & Wichmann, C. (2022). Receiving, Handling, Storage, Thawing, Distribution, and Administration of CAR-T Cells Shipped from the Manufacturing Facility. In N. Kröger (Eds.) et. al., The EBMT/EHA CAR-T Cell Handbook. (pp. 37–43). Springer.
Rochman, Y., Spolski, R., & Leonard, W. (2009). New insights into the regulation of T cells by γc family cytokines. Nat Rev Immunol 9, 480–490.
Simmons, G. L., & Castaneda Puglianini, O. (2022). T-Cell-Based Cellular Immunotherapy of Multiple Myeloma: Current Developments. Cancers, 14(17), 4249.
U.S. Food and Drug Administration, Center for Drug Evaluation and Research. (2017). STN: BL 125643/0 approval letter. https://www.fda.gov/media/108458/download
U.S. Food and Drug Administration, Center for Drug Evaluation and Research. (2019). https://www.fda.gov/patients/drug-development-process/step-3-clinical-research
U.S. Food and Drug Administration, Center for Drug Evaluation and Research. (2021). STN: BL 125736/0 approval letter. https://www.fda.gov/media/147062/download
U.S. Food and Drug Administration, Center for Drug Evaluation and Research. (2022). STN: BL 125714/90 approval letter. https://www.fda.gov/media/159473/download
U.S. National Library of Medicine. (2021). Tratamientos para el Cáncer: Medlineplus Enciclopedia Médica. MedlinePlus. https://medlineplus.gov/spanish/ency/patientinstructions/000901.htm
Vormittag, P., Gunn, R., Ghorashian, S., & Veraitch, F. S. (2018). A guide to manufacturing CAR T cell therapies. Current opinion in biotechnology, 53, 164–181.
Wang, X., & Rivière, I. (2016). Clinical manufacturing of CAR T cells: foundation of a promising therapy. Molecular therapy oncolytics, 3, 16015.
World Health Organization. (2023). ICTRP search portal. World Health Organization. https://www.who.int/clinical-trials-registry-platform/the-ictrp-search-portal#:~:text=The%20ICTRP%20Search%20Portal%20aims,for%20content%20and%20quality%20control
Yang, M., Tkach, D., Boyne, A., Kazancioglu, S., Duclert, A., Poirot, L., Duchateau, P., & Juillerat, A. (2022). Optimized two-step electroporation process to achieve efficient nonviral-mediated gene insertion into primary T cells. FEBS open bio, 12(1), 38–50.
Ye, Z. (2022) Summary Basis for Regulatory Action Carvykti. [Online]. Disponible: https://www.fda.gov/media/156999/download
Publicado
Como Citar
Edição
Seção
Licença
Copyright (c) 2024 Fiorella Ojeda , José Félix , Fanny L. Casado
Este trabalho está licenciado sob uma licença Creative Commons Attribution 4.0 International License.
Esta obra está bajo licencia internacional Creative Commons Reconocimiento 4.0 Internacional (CC BY 4.0).
La "Revista de Innovación y Transferencia Productiva" del Instituto Tecnológico de la Producción, Lima, Perú se distribuye bajo una Licencia Creative Commons Reconocimiento 4.0 Internacional (CC BY 4.0).
