{"id":191556,"date":"2025-12-22T13:28:09","date_gmt":"2025-12-22T12:28:09","guid":{"rendered":"https:\/\/helloproject.es\/vigilancia\/una-terapia-genica-para-la-anemia-de-fanconi-recibe-la-designacion-de-medicamento-huerfano-en-europa\/"},"modified":"2025-12-22T13:28:09","modified_gmt":"2025-12-22T12:28:09","slug":"una-terapia-genica-para-la-anemia-de-fanconi-recibe-la-designacion-de-medicamento-huerfano-en-europa","status":"publish","type":"post","link":"https:\/\/helloproject.es\/vigilancia\/una-terapia-genica-para-la-anemia-de-fanconi-recibe-la-designacion-de-medicamento-huerfano-en-europa\/","title":{"rendered":"Una terapia g\u00e9nica para la anemia de Fanconi recibe la designaci\u00f3n de medicamento hu\u00e9rfano en Europa"},"content":{"rendered":"

Personal investigador del Centro de Investigaciones Energ\u00e9ticas, Medioambientales y Tecnol\u00f3gicas (CIEMAT) ha desarrollado una nueva terapia g\u00e9nica antitumoral destinada a pacientes con anemia de Fanconi que ha obtenido la designaci\u00f3n de medicamento hu\u00e9rfano por parte de la Agencia Europea del Medicamento.<\/p>\n

El reconocimiento europeo respalda el avance de esta terapia dirigida a una patolog\u00eda poco frecuente y con importantes necesidades m\u00e9dicas no cubiertas<\/p>\n

El reconocimiento europeo respalda el avance de una opci\u00f3n terap\u00e9utica dirigida a una patolog\u00eda poco frecuente y con importantes necesidades m\u00e9dicas no cubiertas.<\/p>\n

La investigaci\u00f3n, realizada desde la Unidad de Innovaci\u00f3n Biom\u00e9dica del CIEMAT en colaboraci\u00f3n con el Centro de Investigaci\u00f3n Biom\u00e9dica en Red de Enfermedades Raras y el Instituto de Investigaci\u00f3n Sanitaria Fundaci\u00f3n Jim\u00e9nez D\u00edaz, se basa en el uso de linfocitos T modificados gen\u00e9ticamente, conocidos como c\u00e9lulas CAR-T.<\/p>\n

<\/a><\/p>\n

Eliminar de forma selectiva las c\u00e9lulas tumorales<\/h2>\n

Estas c\u00e9lulas permiten reconocer y eliminar de forma selectiva las c\u00e9lulas tumorales, lo que reduce la agresividad asociada a los tratamientos oncol\u00f3gicos convencionales.<\/p>\n

El desarrollo incorpora un vector lentiviral mejorado, apoyado en trabajos cl\u00ednicos previos del King\u2019s College de Londres, que incrementa la eficiencia y la seguridad del procedimiento. Esta aproximaci\u00f3n resulta especialmente relevante para los pacientes con anemia de Fanconi, que presentan una elevada sensibilidad a la quimioterapia y la radioterapia tradicionales.<\/p>\n

La anemia de Fanconi es una enfermedad gen\u00e9tica rara causada por alteraciones en los mecanismos de reparaci\u00f3n del ADN<\/p>\n

La anemia de Fanconi es una enfermedad gen\u00e9tica rara causada por alteraciones en los mecanismos de reparaci\u00f3n del ADN. Se manifiesta con fallo de la m\u00e9dula \u00f3sea y conlleva una alta predisposici\u00f3n al desarrollo de\u00a0distintos tipos de c\u00e1ncer. En particular, las personas afectadas presentan un riesgo hasta 700 veces superior al de la poblaci\u00f3n general de desarrollar carcinomas escamosos de cabeza y cuello.<\/p>\n

En el \u00e1mbito europeo, la designaci\u00f3n de medicamento hu\u00e9rfano se concede a tratamientos destinados a enfermedades raras o con escasa rentabilidad comercial, con el objetivo de incentivar la investigaci\u00f3n y garantizar el acceso equitativo a nuevas terapias. En este contexto, el nuevo reconocimiento supone una herramienta clave para avanzar en el desarrollo cl\u00ednico de esta terapia g\u00e9nica y facilitar su futura llegada a los pacientes.<\/p>\n

<\/a><\/p>\n

Centros de investigaci\u00f3n y hospitales<\/h2>\n

Los investigadores del CIEMAT subrayan que este avance representa un paso significativo hacia una alternativa terap\u00e9utica m\u00e1s segura y eficaz para una poblaci\u00f3n especialmente vulnerable. Los resultados precl\u00ednicos obtenidos hasta el momento sientan las bases para continuar con los estudios necesarios que permitan evaluar su aplicaci\u00f3n cl\u00ednica con las m\u00e1ximas garant\u00edas de seguridad.<\/p>\n

El trabajo ha contado con la colaboraci\u00f3n de numerosos hospitales y centros de investigaci\u00f3n espa\u00f1oles, as\u00ed como con la participaci\u00f3n activa de pacientes, un factor determinante para el progreso de la investigaci\u00f3n en enfermedades raras. Adem\u00e1s de su impacto directo en la anemia de Fanconi, el hallazgo abre nuevas perspectivas para el desarrollo de tratamientos oncol\u00f3gicos m\u00e1s selectivos y menos agresivos.<\/p>","protected":false},"excerpt":{"rendered":"

Personal investigador del Centro de Investigaciones Energ\u00e9ticas, Medioambientales y Tecnol\u00f3gicas (CIEMAT) ha desarrollado una nueva terapia g\u00e9nica antitumoral destinada a pacientes con anemia de Fanconi que ha obtenido la designaci\u00f3n de medicamento hu\u00e9rfano por parte de la Agencia Europea del Medicamento. El reconocimiento europeo respalda el avance de esta terapia dirigida a una patolog\u00eda poco […]<\/p>\n","protected":false},"author":5,"featured_media":0,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"footnotes":""},"categories":[5],"tags":[],"class_list":["post-191556","post","type-post","status-publish","format-standard","hentry","category-noticias"],"yoast_head":"\nUna terapia g\u00e9nica para la anemia de Fanconi recibe la designaci\u00f3n de medicamento hu\u00e9rfano en Europa - bip4ex<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/helloproject.es\/vigilancia\/una-terapia-genica-para-la-anemia-de-fanconi-recibe-la-designacion-de-medicamento-huerfano-en-europa\/\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Una terapia g\u00e9nica para la anemia de Fanconi recibe la designaci\u00f3n de medicamento hu\u00e9rfano en Europa\" \/>\n<meta property=\"og:description\" content=\"Personal investigador del Centro de Investigaciones Energ\u00e9ticas, Medioambientales y Tecnol\u00f3gicas (CIEMAT) ha desarrollado una nueva terapia g\u00e9nica antitumoral destinada a pacientes con anemia de Fanconi que ha obtenido la designaci\u00f3n de medicamento hu\u00e9rfano por parte de la Agencia Europea del Medicamento. 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