Se trata de la primera financiaci\u00f3n del CIRM que se espera que conduzca expl\u00edcitamente a la aprobaci\u00f3n de la FDA para la realizaci\u00f3n de ensayos cl\u00ednicos. Las subvenciones se otorgaron a equipos multidisciplinarios formados por investigadores b\u00e1sicos, m\u00e9dicos y representantes de la industria, en colaboraci\u00f3n con dos socios internacionales: el Consejo de Investigaci\u00f3n M\u00e9dica (MRC) del Reino Unido y el Consorcio de C\u00e9lulas Madre Cancerosas de Canad\u00e1.<\/p>\n
![\"Sam](\"https:\/\/www.salk.edu\/wp-content\/uploads\/2015\/03\/pfaff_389.jpg\")
<\/p>\n
Denis Poroy\/AP, \u00a9 HHMI<\/p>\n<\/div>\n
\nLa ELA es una enfermedad neurodegenerativa progresiva que afecta a las c\u00e9lulas nerviosas del cerebro y la m\u00e9dula espinal. Cuando las neuronas motoras mueren, el cerebro pierde la capacidad de iniciar y controlar el movimiento muscular. La degeneraci\u00f3n progresiva de las neuronas motoras en la ELA acaba provocando la par\u00e1lisis y la muerte.\n<\/p>\n
El equipo de investigaci\u00f3n, del que forman parte los investigadores principales adjuntos, los doctores Larry Goldstein y Don Cleveland, ambos de la UCSD, se centrar\u00e1 en los astrocitos, unas c\u00e9lulas de soporte con forma de estrella que proporcionan nutrientes a las neuronas motoras cercanas. Trabajando con seis l\u00edneas diferentes de c\u00e9lulas madre embrionarias humanas (hESC), Pfaff y el equipo de investigadores cultivar\u00e1n c\u00e9lulas precursoras de astrocitos de grado cl\u00ednico e identificar\u00e1n la l\u00ednea m\u00e1s adecuada para su implantaci\u00f3n en modelos de laboratorio.<\/p>\n
\nSu hip\u00f3tesis es que los precursores de astrocitos humanos (hAP) trasplantados madurar\u00e1n hasta convertirse en astrocitos in vivo y proporcionar\u00e1n apoyo a las neuronas motoras espinales afectadas por la enfermedad. Los astrocitos tambi\u00e9n son capaces de eliminar el exceso de glutamato neurot\u00f3xico y, por lo tanto, podr\u00edan ralentizar o detener la progresi\u00f3n de la ELA al prevenir la degeneraci\u00f3n de las neuronas motoras.<\/p>\n
\nUna vez que se haya evaluado la eficacia y la seguridad de los precursores de astrocitos para minimizar la posibilidad de tumorig\u00e9nesis, el siguiente paso ser\u00e1 avanzar hacia los ensayos cl\u00ednicos en humanos tras la aprobaci\u00f3n de la FDA, afirma Pfaff.<\/p>\n
\n\u201cEsta subvenci\u00f3n para el equipo encaja a la perfecci\u00f3n con San Diego, ya que aprovecha la solidez de la investigaci\u00f3n sobre enfermedades neuromusculares que lleva a cabo la comunidad cient\u00edfica local\u201d, afirma Pfaff. \u201cEl list\u00f3n se ha puesto muy alto en este proyecto, ya que nuestro objetivo es cultivar una poblaci\u00f3n segura de astrocitos que pueda implantarse en los pacientes. Nuestro \u00e9xito se medir\u00e1 por si esto contribuye a prolongar la vida de los pacientes que padecen ELA\u201d.\u201d\n<\/p>\n
El presidente del CIRM, Alan Trounson, se\u00f1al\u00f3 que el ritmo de los proyectos del Equipo de Enfermedades contrasta con la d\u00e9cada o m\u00e1s que suele requerirse para llegar a los ensayos cl\u00ednicos. \u201cLos cient\u00edficos llevan a\u00f1os hablando de la necesidad de encontrar formas de acelerar el ritmo de los descubrimientos\u201d, afirm\u00f3. \u201cAl animar a los solicitantes a formar equipos compuestos por los mejores investigadores de todo el mundo, creemos que el CIRM establecer\u00e1 un nuevo est\u00e1ndar sobre c\u00f3mo debe financiarse la investigaci\u00f3n traslacional\u201d.\u201d\n<\/p>\n
\nAcerca de Sam Pfaff:<\/strong>
\nEl laboratorio de Pfaff se dedica al estudio del desarrollo embrionario de la m\u00e9dula espinal; en concreto, se centra en investigar c\u00f3mo se forman las neuronas motoras y c\u00f3mo establecen sus conexiones predeterminadas entre la m\u00e9dula espinal y los m\u00fasculos del cuerpo. Estas conexiones son necesarias para todos los movimientos corporales. La alteraci\u00f3n de la funci\u00f3n de las neuronas motoras suele ser la causa principal de la par\u00e1lisis derivada de lesiones o enfermedades de la m\u00e9dula espinal.\n<\/p>\n\nSobre Larry Goldstein:<\/strong>
\nEl Dr. Larry Goldstein, profesor del Departamento de Medicina Celular y Molecular de la Facultad de Medicina de la Universidad de California en San Diego e investigador del Instituto M\u00e9dico Howard Hughes, es director del Programa de C\u00e9lulas Madre de la Universidad de California en San Diego. Goldstein es un l\u00edder nacional en investigaci\u00f3n y pol\u00edticas sobre c\u00e9lulas madre, y se desempe\u00f1\u00f3 como copresidente del comit\u00e9 asesor cient\u00edfico de la campa\u00f1a para aprobar la Proposici\u00f3n 71 de California, la medida respaldada por los votantes para proporcionar 1.000 millones de d\u00f3lares en fondos para la investigaci\u00f3n con c\u00e9lulas madre en California. Goldstein tambi\u00e9n ha comparecido en numerosas ocasiones ante la legislatura de California y el Congreso y el Senado de los Estados Unidos para testificar en apoyo de la investigaci\u00f3n con c\u00e9lulas madre.\n<\/p>\nAcerca de Don Cleveland:<\/strong>
\nEl Dr. Don Cleveland, profesor de Medicina, Neurociencias y Medicina Celular y Molecular, y director del Laboratorio de Biolog\u00eda Celular del Instituto Ludwig para la Investigaci\u00f3n del C\u00e1ncer, es director del Departamento de Medicina Celular y Molecular de la Facultad de Medicina de la Universidad de California en San Diego. Es uno de los principales expertos del pa\u00eds en ELA o enfermedad de Lou Gehrig. Sus descubrimientos pioneros sobre los mecanismos del movimiento cromos\u00f3mico y el control del ciclo celular durante la divisi\u00f3n celular normal, as\u00ed como sobre los principios del desarrollo de las c\u00e9lulas neuronales y su relaci\u00f3n con los defectos que contribuyen a las enfermedades neurodegenerativas hereditarias, le valieron ser nombrado miembro de la Academia Estadounidense de las Artes y las Ciencias, as\u00ed como de la Academia Nacional de Ciencias, en 2006. <\/p>\nAcerca del Instituto Salk de Estudios Biol\u00f3gicos:<\/strong>
\nEl Instituto Salk de Estudios Biol\u00f3gicos es una de las instituciones de investigaci\u00f3n b\u00e1sica m\u00e1s destacadas del mundo, donde un cuerpo docente de prestigio internacional investiga cuestiones fundamentales de las ciencias de la vida en un entorno \u00fanico, colaborativo y creativo. Centrados tanto en el descubrimiento como en la formaci\u00f3n de las futuras generaciones de investigadores, los cient\u00edficos del Salk realizan contribuciones revolucionarias a nuestra comprensi\u00f3n del c\u00e1ncer, el envejecimiento, el Alzheimer, la diabetes y las enfermedades cardiovasculares mediante el estudio de la neurociencia, la gen\u00e9tica, la biolog\u00eda celular y vegetal, y otras disciplinas relacionadas.\n<\/p>\n\nLos logros del cuerpo docente han sido reconocidos con numerosos galardones, entre los que se incluyen premios Nobel y la pertenencia a la Academia Nacional de Ciencias. Fundado en 1960 por el Dr. Jonas Salk, pionero en la vacuna contra la poliomielitis, el Instituto es una organizaci\u00f3n independiente sin fines de lucro y un hito arquitect\u00f3nico.<\/p>","protected":false},"featured_media":0,"template":"","faculty":[106],"disease-research":[],"class_list":["post-2005","disclosure","type-disclosure","status-publish","hentry","faculty-samuel-pfaff"],"acf":[],"yoast_head":"\n
Salk Institute scientist receives $15.6 million CIRM Disease Team Award to develop novel stem-cell derived therapy for Lou Gehrig's Disease - Salk Institute for Biological Studies<\/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:\/\/www.salk.edu\/es\/news-release\/salk-institute-scientist-receives-15-6-million-cirm-disease-team-award-to-develop-novel-stem-cell-derived-therapy-for-lou-gehrigs-disease\/\" \/>\n<meta property=\"og:locale\" content=\"es_MX\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Salk Institute scientist receives $15.6 million CIRM Disease Team Award to develop novel stem-cell derived therapy for Lou Gehrig's Disease - Salk Institute for Biological Studies\" \/>\n<meta property=\"og:description\" content=\"LA JOLLA\u2014The Salk Institute has been awarded a $10.8 million grant by the California Institute for Regenerative Medicine (CIRM) for translational research focusing on developing a novel stem-cell based therapy for Amyotrophic Lateral Sclerosis (ALS) \u2013 or Lou Gehrig’s Disease.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.salk.edu\/es\/news-release\/salk-institute-scientist-receives-15-6-million-cirm-disease-team-award-to-develop-novel-stem-cell-derived-therapy-for-lou-gehrigs-disease\/\" \/>\n<meta property=\"og:site_name\" content=\"Salk Institute for Biological Studies\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.salk.edu\/wp-content\/uploads\/2023\/03\/salk-institute-preview-image.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"628\" \/>\n\t<meta property=\"og:image:height\" content=\"329\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data1\" content=\"4 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.salk.edu\\\/news-release\\\/salk-institute-scientist-receives-15-6-million-cirm-disease-team-award-to-develop-novel-stem-cell-derived-therapy-for-lou-gehrigs-disease\\\/\",\"url\":\"https:\\\/\\\/www.salk.edu\\\/news-release\\\/salk-institute-scientist-receives-15-6-million-cirm-disease-team-award-to-develop-novel-stem-cell-derived-therapy-for-lou-gehrigs-disease\\\/\",\"name\":\"Salk Institute scientist receives $15.6 million CIRM Disease Team Award to develop novel stem-cell derived therapy for Lou Gehrig's Disease - 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