{"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\/pt\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/","title":{"rendered":"Primeiro ensaio cl\u00ednico com medicamento Progeria ultrapassa a marca da metade!"},"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=\u201dinicial\u201d background_position=\u201dsuperior_esquerdo\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=\u201dinicial\u201d background_position=\u201dsuperior_esquerdo\u201d background_repeat=\u201drepetir\u201d]<\/p>\n<p>O PRF continua fazendo hist\u00f3ria, j\u00e1 que quase todas as crian\u00e7as inscritas no estudo compareceram ao Hospital Infantil de Boston para sua visita de 1 ano, marcando a metade do caminho para a conclus\u00e3o. <a href=\"https:\/\/www.progeriaresearch.org\/pt\/clinical-trials\/\">Clique aqui<\/a> para obter detalhes sobre como voc\u00ea pode ajudar.<\/p>\n<p>Tempos emocionantes! O teste cl\u00ednico do medicamento Progeria come\u00e7ou em 7 de maio de 2007 com duas crian\u00e7as chegando em Boston, MA, para sua primeira de sete visitas ao longo de um per\u00edodo de 2 anos. Nesta primeira visita, elas passaram por testes extensivos e suas primeiras doses do medicamento. Uma m\u00e9dia de duas fam\u00edlias tem voado para Boston a cada semana desde ent\u00e3o, e em outubro de 2007, o teste foi totalmente inscrito. O teste deve terminar em outubro de 2009, com os resultados publicados em 2010.<\/p>\n<p>Em 1\u00ba de outubro de 2008, todas as crian\u00e7as, exceto uma, completaram a visita de uma semana e um ano.<\/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=\u201d1_4,1_4,1_4,1_4\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_column type=\u201d1_4\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2020\/12\/Megan-medal-May08.jpg\u201d title_text=\u201dMegan-medal-May08\u2033 admin_label=\u201dmeagan medal\u201d _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][\/et_pb_image][\/et_pb_column][et_pb_column type=\u201d1_4\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_text _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d]<\/p>\n<p><em><strong>Megan usa com orgulho sua Medalha de Julgamento de 1 ano, que recebeu no final de sua recente viagem a Boston<\/strong><\/em><\/p>\n<p>[et_pb_text][\/et_pb_column][et_pb_column type=\u201d1_4\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2020\/12\/Julieta-Aug07.jpg\u201d title_text=\u201dJulieta-Aug07\u2033 admin_label=\u201dJulieta\u201d _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][\/et_pb_image][\/et_pb_column][et_pb_column type=\u201d1_4\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_text _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d]<\/p>\n<p><em><strong>Julieta, da Argentina<\/strong><\/em><\/p>\n<p>[et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_column type=\u201d4_4\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_testimonial author=\u201dFrancis Collins, MD, PhD, Diretor do Instituto Nacional de Pesquisa do Genoma Humano, que mapeou o genoma humano, palestrante do workshop e codescobridor do gene da Progeria.\u201d admin_label=\u201dCita\u00e7\u00e3o de FC\u201d _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d]<\/p>\n<p>\u201cN\u00e3o conhe\u00e7o nenhuma outra doen\u00e7a gen\u00e9tica rara que tenha passado da descoberta gen\u00e9tica para o ensaio cl\u00ednico em menos de quatro anos \u2013 um testemunho fenomenal do trabalho \u00e1rduo da The Progeria Research Foundation.\u201d\u00a0<\/p>\n<p>[\/et_pb_testimonial][et_pb_text _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d]<\/p>\n<div><strong>Vinte e oito (28) crian\u00e7as de dezasseis pa\u00edses<\/strong> est\u00e3o participando, com idades entre 3 e 15 anos. As crian\u00e7as retornam ao Children&#039;s Hospital Boston a cada quatro meses, para testes e para receber novos suprimentos de medicamentos, e ficam em Boston por 4 a 8 dias a cada visita. Enquanto est\u00e3o em casa, seus m\u00e9dicos mant\u00eam uma vigil\u00e2ncia rigorosa sobre as crian\u00e7as e enviam relat\u00f3rios mensais de sa\u00fade para a equipe de pesquisa de Boston.<\/div>\n<div>\u00a0<\/div>\n<div>Durante o teste, 1 a 2 crian\u00e7as por semana viajar\u00e3o para Boston para participar.<\/div>\n<div>\u00a0<\/div>\n<div>As crian\u00e7as s\u00e3o origin\u00e1rias dos seguintes pa\u00edses:<\/div>\n<p>[et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=\u201d1_2,1_2\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d custom_margin=\u201d|-1px||auto||\u201d][et_pb_column type=\u201d1_2\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_text admin_label=\u201dcountries 1\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d]<\/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>\u00cdndia<\/li>\n<li>Israel<\/li>\n<li>It\u00e1lia<\/li>\n<\/ul>\n<p>[et_pb_text][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2008\/05\/2-megans-frnt-cover.jpg\u201d title_text=\u201d2-megans-frnt-cover\u201d admin_label=\u201d2 meghans\u201d _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d custom_margin=\u201d|219px||||\u201d][\/et_pb_image][et_pb_text _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d custom_padding=\u201d||0px|||\u201d]<\/p>\n<p><em>\u201cAs duas Megans\u201d, ambas de 6 anos, em Boston para o ensaio cl\u00ednico<\/em><\/p>\n<p>[et_pb_text][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2008\/05\/MichielHayleyJun07.jpg\u201d title_text=\u201dMichielHayleyJun07\u2033 admin_label=\u201dm&amp;H\u201d _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][\/et_pb_image][et_pb_text _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d]<\/p>\n<p><em>Michiel, 8 \u00bd, da B\u00e9lgica, com Hayley, 9 \u00bd, da Inglaterra, em junho, no Hospital Infantil de Boston, durante sua primeira visita.<\/em><\/p>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=\u201d1_2\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_text admin_label=\u201dcountries 2\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d]<\/p>\n<ul>\n<li>Jap\u00e3o<\/li>\n<li>M\u00e9xico<\/li>\n<li>Paquist\u00e3o<\/li>\n<li>Pol\u00f4nia<\/li>\n<li>Portugal<\/li>\n<li>Rom\u00eania<\/li>\n<li>EUA<\/li>\n<li>Venezuela<\/li>\n<\/ul>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_column type=\u201d4_4\u2033 _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d][et_pb_text _builder_version=\u201d4.6.5\u2033 _module_preset=\u201ddefault\u201d hover_enabled=\u201d0\u2033 sticky_enabled=\u201d0\u2033]<\/p>\n<p><strong>O<\/strong> <strong>Progeria Pesquisa Cl\u00ednica de Medicamentos<\/strong><strong>:\u00a0<\/strong><strong>Quem, onde, quando, como e quanto\u2026<\/strong><\/p>\n<p><strong>O ensaio cl\u00ednico \u00e9 liderado por Mark Kieran MD, PhD, <\/strong>Diretor, Pediatric Medical Neuro-Oncology, Dana-Farber Cancer Institute e Children&#039;s Hospital Boston; Professor Assistente, Departamentos de Pediatria e Hematologia\/Oncologia, Harvard Medical School. O Dr. Kieran \u00e9 um oncologista pedi\u00e1trico com ampla experi\u00eancia com o medicamento em estudo (farnesiltransferase, ou FTI) em crian\u00e7as.<\/p>\n<div>O ensaio cl\u00ednico \u00e9 um esfor\u00e7o colaborativo. As crian\u00e7as est\u00e3o sendo atendidas por m\u00e9dicos em <strong>Hospital Infantil de Boston, Instituto de C\u00e2ncer Dana-Farber e Hospital Brigham and Women&#039;s,<\/strong> todas as institui\u00e7\u00f5es da Universidade de Harvard. Al\u00e9m disso, m\u00e9dicos e cientistas de<strong> A Escola M\u00e9dica Warren Alpert da Universidade Brown, UCLA e NIH<\/strong> est\u00e3o ajudando a tornar este teste um sucesso. Muitas pessoas est\u00e3o trabalhando juntas para realizar esta pesquisa.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong>Como chegamos a esse ponto?<\/strong> Em 2002, a equipe de pesquisa colaborativa da Progeria Research Foundation <a href=\"https:\/\/www.progeriaresearch.org\/pt\/2003\/04\/16\/identification-of-gene-gives-hope-to-children-with-progeria\/\" target=\"_blank\" rel=\"noopener noreferrer\">descobriu o gene Progeria.<\/a>\u00a0\u00a0Essa descoberta n\u00e3o s\u00f3 levou a uma maior compreens\u00e3o da Prog\u00e9ria, mas os cientistas agora sabem que estudar a Prog\u00e9ria pode nos ajudar a aprender mais sobre doen\u00e7as card\u00edacas e o processo normal de envelhecimento que afeta a todos n\u00f3s.<\/div>\n<div>\u00a0<\/div>\n<div>Desde a descoberta do gene, o apoio de pesquisadores, cl\u00ednicos e fam\u00edlias de crian\u00e7as com Progeria nos trouxe a outra encruzilhada na busca por um tratamento. Pesquisadores identificaram um potencial tratamento medicamentoso para crian\u00e7as com Progeria, chamado inibidores de farnesiltransferase (FTIs), e conduziram estudos em laborat\u00f3rio que apoiam um teste humano com o medicamento.\u00a0<a href=\"https:\/\/www.progeriaresearch.org\/pt\/2006\/02\/16\/three-studies-released-that-bring-us-closer-than-ever-to-understanding-progeria-and-to-disease-treatment\/\">Clique aqui<\/a>\u00a0para mais detalhes sobre a pesquisa.<\/div>\n<div>\u00a0<\/div>\n<div>\n<div><strong>Como esse medicamento funcionar\u00e1 na Prog\u00e9ria?<\/strong><\/div>\n<div>A prote\u00edna que acreditamos ser respons\u00e1vel pela Progeria \u00e9 chamada progerina. Para bloquear a fun\u00e7\u00e3o celular normal e causar Progeria, uma mol\u00e9cula chamada \u201cgrupo farnesil\u201d deve ser anexada \u00e0 prote\u00edna progerina. Os FTIs agem bloqueando (inibindo) a liga\u00e7\u00e3o do grupo farnesil \u00e0 progerina. Ent\u00e3o, se o medicamento FTI pode bloquear essa liga\u00e7\u00e3o do grupo farnesil em crian\u00e7as com Progeria, ent\u00e3o a progerina pode ser \u201cparalisada\u201d e a Progeria melhorada.<\/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>As c\u00e9lulas de Progeria se tornam normalizadas quando FTIs s\u00e3o aplicados. Capell et al., PNAS, 2005. C\u00e9lula normal. C\u00e9lula de Progeria. C\u00e9lula de Progeria ap\u00f3s ser tratada com FTI<\/strong><\/em><\/div>\n<div>\u00a0<\/div>\n<div>\n<div><strong>Quanto custar\u00e1 o teste para a PRF? <\/strong>\u00a0Estimamos que o teste custar\u00e1 \u00e0 PRF $2 milh\u00f5es de d\u00f3lares. Isso pagar\u00e1 por testes cl\u00ednicos, tradutores, voos, alimenta\u00e7\u00e3o, hospedagem e certos custos 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 esse per\u00edodo de 2 anos e meio.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong><em>Sua doa\u00e7\u00e3o ajudar\u00e1 a tornar esse teste realidade.<\/em><\/strong><\/div>\n<div>\n<p><em>A PRF precisa arrecadar aproximadamente $2 milh\u00f5es de d\u00f3lares para financiar este estudo e, at\u00e9 julho de 2009, arrecadamos $1,9 milh\u00f5es!<\/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>Nosso <a href=\"https:\/\/www.progeriaresearch.org\/pt\/circle-of-hope-3\/\" target=\"_blank\" rel=\"noopener noreferrer\">C\u00edrculo da Esperan\u00e7a<\/a> expandiu\u2026<\/strong><\/p>\n<\/div>\n<div>\u00a0<\/div>\n<div>Em 2006, a campanha Circle of Hope foi criada para arrecadar $100.000 por ano durante 5 anos para manter nosso Banco de C\u00e9lulas, Testes Diagn\u00f3sticos, financiamento de bolsas de pesquisa e outros programas funcionando a todo vapor. Atingir essa meta de arrecada\u00e7\u00e3o de fundos nos permitiria acompanhar o interesse crescente na pesquisa da Progeria e continuar nosso ritmo de progresso. Quem poderia imaginar que menos de um ano depois, estar\u00edamos no meio de uma campanha para arrecadar $2 milh\u00f5es para financiar um teste de medicamento para tratar a Progeria?! Nossa campanha Circle of Hope agora inclui esse empreendimento.<\/div>\n<div>\u00a0<\/div>\n<div>Por favor, ajude-nos a manter o <strong>C\u00edrculo <\/strong>intacto, ent\u00e3o o <strong>ter esperan\u00e7a<\/strong> de tratamento torna-se um <strong>realidade.\u00a0<\/strong><a href=\"https:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR\">Doar<\/a> hoje.<\/div>\n<div><em>\u00a0<\/em><\/div>\n<div><strong>Este \u00e9 o momento de encontrar um tratamento para a Progeria<em>.<\/em><\/strong><\/div>\n<div>\u00a0<strong>Juntos, n\u00f3s\u00a0<em>VAI<\/em>\u00a0encontre a 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=\u201d1\u2033 _builder_version=\u201d3.22\u2033][et_pb_row _builder_version=\u201d3.25\u2033 background_size=\u201dinitial\u201d background_position=\u201dtop_left\u201d background_repeat=\u201drepeat\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=\u201dtop_left\u201d background_repeat=\u201drepeat\u201d] O PRF continua a fazer hist\u00f3ria, pois quase todas as crian\u00e7as inscritas no estudo j\u00e1 compareceram \u00e0 consulta de acompanhamento de 1 ano no Children&#039;s Hospital Boston, marcando a metade do caminho para a conclus\u00e3o do estudo. Clique aqui para saber como voc\u00ea pode ajudar. Excitante [\u2026]<\/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\/pt\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\" \/>\n<meta property=\"og:locale\" content=\"pt_PT\" \/>\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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