{"id":2091,"date":"2008-05-16T13:50:49","date_gmt":"2008-05-16T13:50:49","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?p=2091"},"modified":"2020-12-10T12:55:01","modified_gmt":"2020-12-10T17:55:01","slug":"first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark","status":"publish","type":"post","link":"https:\/\/www.progeriaresearch.org\/es\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/","title":{"rendered":"\u00a1El primer ensayo cl\u00ednico con un f\u00e1rmaco contra la progeria supera la mitad del camino!"},"content":{"rendered":"<p>[et_pb_section fb_built=\u201d1\u2033 _builder_version=\u201d3.22\u2033][et_pb_row _builder_version=\u201d3.25\u2033 background_size=\u201dinitial\u201d background_position=\u201dsuperior_izquierda\u201d background_repeat=\u201drepetir\u201d][et_pb_column type=\u201d4_4\u2033 _builder_version=\u201d3.25\u2033 custom_padding=\u201d|||\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text _builder_version=\u201d4.6.5\u2033 background_size=\u201dinitial\u201d background_position=\u201dsuperior_izquierda\u201d background_repeat=\u201drepetir\u201d]<\/p>\n<p>PRF contin\u00faa haciendo historia, ya que casi todos los ni\u00f1os inscritos en el ensayo han venido al Children&#039;s Hospital Boston para su visita de un a\u00f1o, lo que marca su punto medio hacia la finalizaci\u00f3n. <a href=\"https:\/\/www.progeriaresearch.org\/es\/clinical-trials\/\">haga clic aqu\u00ed<\/a> Para obtener detalles sobre c\u00f3mo puedes ayudar.<\/p>\n<p>\u00a1Tiempos emocionantes! El ensayo cl\u00ednico de medicamentos contra la progeria comenz\u00f3 el 7 de mayo de 2007 con dos ni\u00f1os que llegaron a Boston, Massachusetts, para la primera de siete visitas a lo largo de un per\u00edodo de dos a\u00f1os. En esta primera visita, se les realizaron pruebas exhaustivas y se les administraron las primeras dosis del medicamento. Desde entonces, un promedio de dos familias han estado viajando a Boston cada semana y, en octubre de 2007, el ensayo complet\u00f3 la inscripci\u00f3n. Se espera que el ensayo finalice en octubre de 2009 y que los resultados se publiquen en 2010.<\/p>\n<p>A partir del 1 de octubre de 2008, todos los ni\u00f1os, excepto uno, han completado la visita de una semana de duraci\u00f3n que dura un a\u00f1o.<\/p>\n<div><\/div>\n<div><\/div>\n<div>\n<div><strong><\/strong><\/div>\n<\/div>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8221;1_4,1_4,1_4,1_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_column type=&#8221;1_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2020\/12\/Megan-medal-May08.jpg&#8221; title_text=&#8221;Megan-medal-May08&#8243; admin_label=&#8221;meagan medal&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][\/et_pb_image][\/et_pb_column][et_pb_column type=&#8221;1_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<p><em><strong>Megan luce con orgullo su medalla de prueba de 1 a\u00f1o, que recibi\u00f3 al final de su reciente viaje a Boston.<\/strong><\/em><\/p>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8221;1_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2020\/12\/Julieta-Aug07.jpg&#8221; title_text=&#8221;Julieta-Aug07&#8243; admin_label=&#8221;Julieta&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][\/et_pb_image][\/et_pb_column][et_pb_column type=&#8221;1_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<p><em><strong>Julieta, de Argentina<\/strong><\/em><\/p>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_testimonial author=&#8221;Francis Collins, MD, PhD, Director of the National Human Genome Research Institute that mapped the human genome, workshop speaker and co-discoverer of the Progeria gene.&#8221; admin_label=&#8221;FC quote&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<p>\u201cNo conozco ninguna otra enfermedad gen\u00e9tica rara que haya pasado del descubrimiento gen\u00e9tico al ensayo cl\u00ednico en menos de cuatro a\u00f1os: un testimonio fenomenal del arduo trabajo de la Fundaci\u00f3n para la Investigaci\u00f3n de la Progeria\u201d.\u00a0<\/p>\n<p>[\/et_pb_testimonial][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<div><strong>Veintiocho (28) ni\u00f1os de diecis\u00e9is pa\u00edses<\/strong> Participan ni\u00f1os de entre 3 y 15 a\u00f1os de edad. Los ni\u00f1os vuelven al Children&#039;s Hospital Boston cada cuatro meses para hacerse pruebas y recibir un nuevo suministro de medicamentos, y permanecen en Boston entre 4 y 8 d\u00edas en cada visita. Mientras est\u00e1n en casa, sus m\u00e9dicos vigilan de cerca a los ni\u00f1os y env\u00edan informes de salud mensuales al equipo de investigaci\u00f3n de Boston.<\/div>\n<div>\u00a0<\/div>\n<div>Durante la duraci\u00f3n del ensayo, entre 1 y 2 ni\u00f1os por semana viajar\u00e1n a Boston para participar.<\/div>\n<div>\u00a0<\/div>\n<div>Los ni\u00f1os proceden de los siguientes pa\u00edses:<\/div>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8221;1_2,1_2&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; custom_margin=&#8221;|-1px||auto||&#8221;][et_pb_column type=&#8221;1_2&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_text admin_label=&#8221;countries 1&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<ul>\n<li>Argentina<\/li>\n<li>B\u00e9lgica<\/li>\n<li>Canad\u00e1<\/li>\n<li>Dinamarca<\/li>\n<li>Inglaterra<\/li>\n<li>India<\/li>\n<li>Israel<\/li>\n<li>Italia<\/li>\n<\/ul>\n<p>[\/et_pb_text][et_pb_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2008\/05\/2-megans-frnt-cover.jpg&#8221; title_text=&#8221;2-megans-frnt-cover&#8221; admin_label=&#8221;2 meghans&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; custom_margin=&#8221;|219px||||&#8221;][\/et_pb_image][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; custom_padding=&#8221;||0px|||&#8221;]<\/p>\n<p><em>\u201cLas dos Megans\u201d, ambas de 6 a\u00f1os, en Boston para el ensayo cl\u00ednico<\/em><\/p>\n<p>[\/et_pb_text][et_pb_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2008\/05\/MichielHayleyJun07.jpg&#8221; title_text=&#8221;MichielHayleyJun07&#8243; admin_label=&#8221;m&#038;H&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][\/et_pb_image][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<p><em>Michiel, de 8 a\u00f1os y medio, de B\u00e9lgica, con Hayley, de 9 a\u00f1os y medio, de Inglaterra, en junio en el Children&#039;s Hospital Boston durante su primera visita.<\/em><\/p>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8221;1_2&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_text admin_label=&#8221;countries 2&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;]<\/p>\n<ul>\n<li>Jap\u00f3n<\/li>\n<li>M\u00e9xico<\/li>\n<li>Pakist\u00e1n<\/li>\n<li>Polonia<\/li>\n<li>Portugal<\/li>\n<li>Rumania<\/li>\n<li>EE.UU<\/li>\n<li>Venezuela<\/li>\n<\/ul>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221;][et_pb_text _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; hover_enabled=&#8221;0&#8243; sticky_enabled=&#8221;0&#8243;]<\/p>\n<p><strong>El<\/strong> <strong>Ensayo cl\u00ednico de un f\u00e1rmaco contra la progeria<\/strong><strong>:\u00a0<\/strong><strong>Qui\u00e9n, d\u00f3nde, cu\u00e1ndo, c\u00f3mo y cu\u00e1nto\u2026<\/strong><\/p>\n<p><strong>El ensayo cl\u00ednico est\u00e1 dirigido por el Dr. Mark Kieran, PhD, <\/strong>Director de Neurooncolog\u00eda M\u00e9dica Pedi\u00e1trica del Instituto de C\u00e1ncer Dana-Farber y del Hospital Infantil de Boston; Profesor Adjunto de los Departamentos de Pediatr\u00eda y Hematolog\u00eda\/Oncolog\u00eda de la Facultad de Medicina de Harvard. El Dr. Kieran es un onc\u00f3logo pedi\u00e1trico con amplia experiencia con el f\u00e1rmaco en estudio (farnesiltransferasa o FTI) en ni\u00f1os.<\/p>\n<div>El ensayo cl\u00ednico es un esfuerzo colaborativo. Los ni\u00f1os est\u00e1n siendo examinados por m\u00e9dicos en <strong>Hospital de Ni\u00f1os de Boston, Instituto de C\u00e1ncer Dana-Farber y Hospital Brigham and Women&#039;s,<\/strong> todas las instituciones de la Universidad de Harvard. Adem\u00e1s, m\u00e9dicos y cient\u00edficos de<strong> La Facultad de Medicina Warren Alpert de la Universidad Brown, la UCLA y el NIH<\/strong> Est\u00e1n contribuyendo a que este ensayo sea un \u00e9xito. Muchas personas est\u00e1n trabajando juntas para llevar a cabo esta investigaci\u00f3n.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong>\u00bfC\u00f3mo llegamos a este punto?<\/strong> En 2002, el equipo de investigaci\u00f3n colaborativa de la Fundaci\u00f3n para la Investigaci\u00f3n de la Progeria <a href=\"https:\/\/www.progeriaresearch.org\/es\/2003\/04\/16\/identification-of-gene-gives-hope-to-children-with-progeria\/\" target=\"_blank\" rel=\"noopener noreferrer\">descubri\u00f3 el gen de la progeria.<\/a>\u00a0\u00a0Este descubrimiento no s\u00f3lo condujo a una mejor comprensi\u00f3n de la progeria, sino que los cient\u00edficos ahora saben que estudiar la progeria puede ayudarnos a aprender m\u00e1s sobre las enfermedades card\u00edacas y el proceso normal de envejecimiento que nos afecta a todos.<\/div>\n<div>\u00a0<\/div>\n<div>Desde el descubrimiento del gen, el apoyo de investigadores, m\u00e9dicos y familias de ni\u00f1os con progeria nos ha llevado a otra encrucijada en la b\u00fasqueda de un tratamiento. Los investigadores han identificado un posible tratamiento farmacol\u00f3gico para ni\u00f1os con progeria, llamado inhibidores de la farnesiltransferasa (FTI), y han realizado estudios en el laboratorio que respaldan un ensayo en humanos con el f\u00e1rmaco.\u00a0<a href=\"https:\/\/www.progeriaresearch.org\/es\/2006\/02\/16\/three-studies-released-that-bring-us-closer-than-ever-to-understanding-progeria-and-to-disease-treatment\/\">haga clic aqu\u00ed<\/a>\u00a0para m\u00e1s detalles sobre la investigaci\u00f3n.<\/div>\n<div>\u00a0<\/div>\n<div>\n<div><strong>\u00bfC\u00f3mo funcionar\u00e1 este medicamento en la progeria?<\/strong><\/div>\n<div>La prote\u00edna que creemos que es responsable de la progeria se llama progerina. Para bloquear la funci\u00f3n celular normal y causar progeria, una mol\u00e9cula llamada \u201cgrupo farnesilo\u201d debe unirse a la prote\u00edna progerina. Los FTI act\u00faan bloqueando (inhibiendo) la uni\u00f3n del grupo farnesilo a la progerina. Por lo tanto, si el medicamento FTI puede bloquear esta uni\u00f3n del grupo farnesilo en ni\u00f1os con progeria, entonces la progerina puede quedar \u201cparalizada\u201d y la progeria puede mejorar.<\/div>\n<\/div>\n<div style=\"text-align: center;\"><img loading=\"lazy\" decoding=\"async\" class=\"wp-image-2096 size-full aligncenter\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/FTI-3-cell-image-crpd.jpg\" alt=\"\" width=\"375\" height=\"89\" srcset=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/FTI-3-cell-image-crpd.jpg 375w, https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/FTI-3-cell-image-crpd-300x71.jpg 300w\" sizes=\"(max-width: 375px) 100vw, 375px\" \/> <em><strong>Las c\u00e9lulas de progeria se normalizan cuando se les aplican FTI. Capell et al., PNAS, 2005. C\u00e9lula normal. C\u00e9lula de progeria. C\u00e9lula de progeria despu\u00e9s de ser tratada con FTI<\/strong><\/em><\/div>\n<div>\u00a0<\/div>\n<div>\n<div><strong>\u00bfCu\u00e1nto le costar\u00e1 el juicio a PRF? <\/strong>\u00a0Calculamos que el ensayo le costar\u00e1 a PRF $2 millones de d\u00f3lares, que cubrir\u00e1n los gastos de pruebas cl\u00ednicas, traductores, vuelos, comida, alojamiento y algunos gastos m\u00e9dicos. <img loading=\"lazy\" decoding=\"async\" class=\"alignright\" src=\"https:\/\/web.archive.org\/web\/20170218013638im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/donate_images\/Capsule-1_9.jpg\" alt=\"\" width=\"150\" height=\"299\" align=\"right\" hspace=\"3\" vspace=\"3\" \/>durante ese per\u00edodo de dos a\u00f1os y medio.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong><em>Su donaci\u00f3n ayudar\u00e1 a que este ensayo se realice.<\/em><\/strong><\/div>\n<div>\n<p><em>PRF necesita recaudar aproximadamente $2 millones de d\u00f3lares para financiar este ensayo y, a julio de 2009, \u00a1hemos recaudado $1,9 millones!<\/em><\/p>\n<\/div>\n<div>\n<div><strong><img loading=\"lazy\" decoding=\"async\" class=\"alignleft\" src=\"https:\/\/web.archive.org\/web\/20170218013638im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/donate_images\/Circle_of_Hope_4sm.jpg\" alt=\"\" width=\"125\" height=\"130\" align=\"left\" \/><\/strong><\/div>\n<p><strong>Nuestro <a href=\"https:\/\/www.progeriaresearch.org\/es\/circle-of-hope-3\/\" target=\"_blank\" rel=\"noopener noreferrer\">C\u00edrculo de esperanza<\/a> se ha expandido\u2026<\/strong><\/p>\n<\/div>\n<div>\u00a0<\/div>\n<div>En 2006, se cre\u00f3 la campa\u00f1a Circle of Hope para recaudar 100.000 d\u00f3lares al a\u00f1o durante 5 a\u00f1os para mantener a toda marcha nuestro banco de c\u00e9lulas, pruebas de diagn\u00f3stico, financiaci\u00f3n de subvenciones para investigaci\u00f3n y otros programas. Alcanzar esta meta de recaudaci\u00f3n de fondos nos permitir\u00eda seguir el ritmo del creciente inter\u00e9s en la investigaci\u00f3n sobre la progeria y continuar con nuestro ritmo de progreso. \u00bfQui\u00e9n podr\u00eda haber imaginado que menos de un a\u00f1o despu\u00e9s estar\u00edamos en medio de una campa\u00f1a para recaudar 100.000 d\u00f3lares para financiar un ensayo de un f\u00e1rmaco para tratar la progeria? Nuestra campa\u00f1a Circle of Hope ahora incluye esta gran iniciativa.<\/div>\n<div>\u00a0<\/div>\n<div>Por favor ay\u00fadenos a mantener el <strong>C\u00edrculo <\/strong>intacto, por lo que el <strong>esperanza<\/strong> del tratamiento se convierte en un <strong>realidad.\u00a0<\/strong><a href=\"https:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR\">Donar<\/a> hoy.<\/div>\n<div><em>\u00a0<\/em><\/div>\n<div><strong>Este es el momento de encontrar un tratamiento para la progeria<em>.<\/em><\/strong><\/div>\n<div>\u00a0<strong>Juntos, nosotros\u00a0<em>VOLUNTAD<\/em>\u00a0\u00a1Encuentra la cura!<\/strong><\/div>\n<\/div>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>","protected":false},"excerpt":{"rendered":"<p>[et_pb_section fb_built=&#8221;1&#8243; _builder_version=&#8221;3.22&#8243;][et_pb_row _builder_version=&#8221;3.25&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;3.25&#8243; custom_padding=&#8221;|||&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.6.5&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221;] PRF continues to make history, as nearly all the children enrolled in the trial have come to Children&#8217;s Hospital Boston for their 1-year visit, marking their half-way point to completion. Click here for details on how you can help. Exciting [&hellip;]<\/p>","protected":false},"author":1,"featured_media":11695,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"<p>[vc_custom_heading text=\"October 2008: First-Ever Progeria Clinical Drug Trial Surpasses Half-Way Mark!\" font_container=\"tag:h1|text_align:left\" use_theme_fonts=\"yes\"]PRF continues to make history, as nearly all the children enrolled in the trial have come to Children's Hospital Boston for their 1-year visit, marking their half-way point to completion. <a href=\"https:\/\/www.progeriaresearch.org\/clinical-trials\/\">Click here<\/a> for details on how you can help.<\/p><p>Exciting times! The Progeria clinical drug trial began on May 7th, 2007 with two children arriving in Boston, MA for their first of seven visits over a 2-year period. At this first visit, they were given extensive tests and their first doses of the drug. An average of two families have been flying to Boston each week since then, and in October 2007, the trial became fully enrolled. The trial is expected to end in October 2009, with results published in 2010.<\/p><div>As of October 1, 2008, all but one child has completed the week-long, 1-year visit.<\/div><div>[caption id=\"attachment_2092\" align=\"alignleft\" width=\"175\"]<img class=\"size-full wp-image-2092\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Megan-medal-May08.jpg\" alt=\"\" width=\"175\" height=\"275\" \/> Megan proudly wears her 1-year Trial Medal, which she received at the end of her recent trip to Boston[\/caption]<p><strong>\u201cI know of no other rare genetic disease that has gone from gene discovery to clinical trial in under four years - a phenomenal testament to the hard work of The Progeria Research Foundation.\u201d\u00a0<\/strong><\/p><p>\u00a0<\/p>[caption id=\"attachment_2093\" align=\"alignright\" width=\"175\"]<img class=\"size-full wp-image-2093\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Julieta-Aug07.jpg\" alt=\"\" width=\"175\" height=\"194\" \/> <em><strong>Julieta, from Argentina.<\/strong><\/em>[\/caption]<p>\u00a0<\/p><p><em><strong>Francis Collins, MD, PhD, Director of the National Human Genome Research Institute that mapped the human genome, workshop speaker and co-discoverer of the Progeria gene.<\/strong> <\/em><\/p><\/div><div><strong>Twenty-eight (28) children from sixteen countries<\/strong> are participating, ages 3 to 15 years.\u00a0Children return to Children\u2019s Hospital Boston every four months, for testing and to receive new drug supply, and stay in Boston for 4-8 days each visit. \u00a0While at home, their doctors keep a close watch over the children and submit monthly health reports to the Boston research team.<\/div><div>\u00a0<\/div><div>For the duration of the trial, 1- 2 children per week will travel to Boston to participate.<\/div><div>\u00a0<\/div><div>\u00a0<\/div><div>\u00a0<\/div><div>\u00a0<\/div><div><div>Children Originate from the Following Countries:<\/div><div>\u00a0<\/div><ul><li>Argentina<\/li><li>Belgium<\/li><li>Canada<\/li><li>Denmark<\/li><li>England<\/li><li>India<\/li><li>Israel<\/li><li>Italy<\/li><li>Japan<\/li><li>Mexico<\/li><li>Pakistan<\/li><li>Poland<\/li><li>Portugal<\/li><li>Romania<\/li><li>USA<\/li><li>Venezuela<\/li><\/ul><\/div>[caption id=\"attachment_2094\" align=\"alignleft\" width=\"113\"]<img class=\"size-full wp-image-2094\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/2-megans-frnt-cover.jpg\" alt=\"\" width=\"113\" height=\"225\" \/> \u201cThe two Megans\u201d, both 6 years old, in Boston for the clinical trial[\/caption]<p>\u00a0<\/p>[caption id=\"attachment_2095\" align=\"alignnone\" width=\"200\"]<img class=\"size-full wp-image-2095\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/MichielHayleyJun07.jpg\" alt=\"\" width=\"200\" height=\"177\" \/> Michiel, 8 \u00bd , from Belgium with Hayley, 9 \u00bd , from England in June at Children\u2019s Hospital Boston during their first visit.[\/caption]<p><strong>The<\/strong> <strong>Progeria Clinical Research Drug Trial<\/strong><strong>:\u00a0<\/strong><strong>Who, Where, When, How and How Much\u2026<\/strong><\/p><p><strong>The clinical trial is led by Mark Kieran MD, PhD, <\/strong>Director, Pediatric Medical Neuro-Oncology, Dana-Farber Cancer Institute and Children\u2019s Hospital Boston; Assistant Professor, Departments of Pediatrics and Hematology\/Oncology, Harvard Medical School.\u00a0Dr. Kieran is a pediatric oncologist with extensive experience with the drug under study (farnesyltransferase, or FTI) in children.<\/p><div>The clinical trial is a collaborative effort.\u00a0The children are being seen by physicians at <strong>Children\u2019s Hospital Boston, Dana-Farber Cancer Institute, and Brigham and Women\u2019s Hospital,<\/strong> all Harvard University institutions.\u00a0In addition, physicians and scientists from<strong> The Warren Alpert Medical School at Brown University, UCLA, and NIH<\/strong> are helping to make this trial a success. Many individuals are working together to perform this research.<\/div><div><strong>\u00a0<\/strong><\/div><div><strong>How did we get to this point?<\/strong> In 2002, The Progeria Research Foundation\u2019s collaborative research team <a href=\"https:\/\/web.archive.org\/web\/20170218013638\/https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/progeria_gene_discovered.html\">discovered the Progeria gene.<\/a>\u00a0\u00a0This discovery not only led to further understanding of Progeria, but scientists now know that studying Progeria can help us learn more about heart disease and the normal aging process that affects us all.<\/div><div>\u00a0<\/div><div>Since\u00a0the\u00a0gene discovery, the support of researchers, clinicians, families of children with Progeria have brought us to another crossroads in the search for a treatment. Researchers have identified\u00a0a potential\u00a0drug treatment\u00a0for children with\u00a0Progeria,\u00a0called farnesyltransferase inhibitors (FTIs), and have conducted studies in the lab that support a human trial with the drug.\u00a0<a href=\"https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/more_studies_provide_strong_support.html\">Click here<\/a>\u00a0for more details on the research.<\/div><div>\u00a0<\/div><div><div><strong>How will this drug work in Progeria?<\/strong><\/div><div>The protein that we believe is responsible for Progeria is called progerin.\u00a0In order to block normal cell function and cause Progeria, a molecule called a \u201cfarnesyl group\u201d must be attached to the progerin protein. FTIs act by blocking (inhibiting) the attachment of the farnesyl group onto progerin. So if the FTI drug can block this farnesyl group attachment in children with Progeria, then progerin may be \u201cparalyzed\u201d and Progeria improved.<\/div><\/div><div>[caption id=\"attachment_2096\" align=\"alignleft\" width=\"375\"]<img class=\"wp-image-2096 size-full\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/FTI-3-cell-image-crpd.jpg\" alt=\"\" width=\"375\" height=\"89\" \/> Progeria cells become normalized when FTIs are applied. Capell et al., PNAS, 2005. Normal cell. Progeria cell. Progeria cell after being treated with FTI[\/caption]<\/div><div><a href=\"https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/the_fti_drug.html\">Click here<\/a> for more information on FTIs.<\/div><div><div><strong>What will the trial cost PRF? <\/strong>\u00a0We estimate the trial will cost PRF $2 million dollars.\u00a0This will pay for clinical testing, translators, flights, food, lodging, and certain medical costs <img class=\"alignright\" src=\"https:\/\/web.archive.org\/web\/20170218013638im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/donate_images\/Capsule-1_9.jpg\" alt=\"\" width=\"150\" height=\"299\" align=\"right\" hspace=\"3\" vspace=\"3\" \/>during that 2 \u00bd year time period.<\/div><div>\u00a0<\/div><div><strong>\u00a0<\/strong><strong>Support the <a href=\"https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/clinical_trial.html\">Progeria Clinical Drug Trial<\/a><\/strong><\/div><div><strong>\u00a0<\/strong><\/div><div><strong><em>Your donation will help make this trial happen.<\/em><\/strong><\/div><div><strong>\u00a0<\/strong><\/div><div><p><em>PRF needs to raise approximately $2 million dollars to fund this trial, and as of July 2009, we have raised $1.9 million!<\/em><\/p><\/div><div><div><strong><img class=\"alignleft\" src=\"https:\/\/web.archive.org\/web\/20170218013638im_\/https:\/\/www.progeriaresearch.org\/assets\/images\/donate_images\/Circle_of_Hope_4sm.jpg\" alt=\"\" width=\"125\" height=\"130\" align=\"left\" \/><\/strong><\/div><p><strong>Our <a href=\"https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/circle_of_hope.html\">Circle of Hope<\/a> has expanded\u2026<\/strong><\/p><\/div><div>\u00a0<\/div><div>In 2006, the Circle of Hope campaign was created to raise $100,000 per year for 5 years to keep our Cell Bank, Diagnostics Testing, research grant funding and other programs running full speed ahead.\u00a0Meeting this fundraising goal would allow us to keep up with the increasing interest in Progeria research, and to continue our pace of progress. Who could have imagined that less than a year later, we would be in the midst of a campaign to raise $2 million to fund a drug trial to treat Progeria?!\u00a0Our Circle of Hope campaign now includes this g undertaking.<\/div><div>\u00a0<\/div><div>Please help us keep the <strong>Circle <\/strong>intact, so the <strong>hope<\/strong> of treatment becomes a <strong>reality.\u00a0<\/strong><a href=\"https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/ways_to_donate\/\">Donate<\/a> today.<\/div><div><em>\u00a0<\/em><\/div><div><strong>This is the time to find a treatment for Progeria<em>.<\/em><\/strong><\/div><div>\u00a0<strong>Together, we\u00a0<em>WILL<\/em>\u00a0find the cure!<\/strong><\/div><\/div>","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[2],"tags":[],"class_list":["post-2091","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>First-Ever Progeria Clinical Drug Trial Surpasses Half-Way Mark! - The Progeria Research Foundation<\/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.progeriaresearch.org\/es\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"First-Ever Progeria Clinical Drug Trial Surpasses Half-Way Mark! - The Progeria Research Foundation\" \/>\n<meta property=\"og:description\" content=\"[et_pb_section fb_built=&#8221;1&#8243; _builder_version=&#8221;3.22&#8243;][et_pb_row _builder_version=&#8221;3.25&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221;][et_pb_column type=&#8221;4_4&#8243; _builder_version=&#8221;3.25&#8243; custom_padding=&#8221;|||&#8221; custom_padding__hover=&#8221;|||&#8221;][et_pb_text _builder_version=&#8221;4.6.5&#8243; background_size=&#8221;initial&#8221; background_position=&#8221;top_left&#8221; background_repeat=&#8221;repeat&#8221;] PRF continues to make history, as nearly all the children enrolled in the trial have come to Children&#8217;s Hospital Boston for their 1-year visit, marking their half-way point to completion. 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Exciting [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.progeriaresearch.org\/es\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\" \/>\n<meta property=\"og:site_name\" content=\"The Progeria Research Foundation\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/ProgeriaResearch\/\" \/>\n<meta property=\"article:published_time\" content=\"2008-05-16T13:50:49+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2020-12-10T17:55:01+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2020\/12\/Megan-medal-May08.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"175\" \/>\n\t<meta property=\"og:image:height\" content=\"275\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"gravoc\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:creator\" content=\"@Progeria\" \/>\n<meta name=\"twitter:site\" content=\"@Progeria\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"gravoc\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"7 minutos\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\"},\"author\":{\"name\":\"gravoc\",\"@id\":\"https:\/\/www.progeriaresearch.org\/ta\/#\/schema\/person\/a3c935a81e92242c6a77ae0018542928\"},\"headline\":\"First-Ever Progeria Clinical Drug Trial Surpasses Half-Way Mark!\",\"datePublished\":\"2008-05-16T13:50:49+00:00\",\"dateModified\":\"2020-12-10T17:55:01+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\"},\"wordCount\":1327,\"commentCount\":0,\"publisher\":{\"@id\":\"https:\/\/www.progeriaresearch.org\/ta\/#organization\"},\"image\":{\"@id\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2020\/12\/Megan-medal-May08.jpg\",\"articleSection\":[\"News\"],\"inLanguage\":\"es\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/#respond\"]}]},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\",\"url\":\"https:\/\/www.progeriaresearch.org\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\",\"name\":\"First-Ever Progeria Clinical Drug Trial Surpasses Half-Way Mark! 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