{"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\/de\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/","title":{"rendered":"Erster klinischer Arzneimittelversuch gegen Progerie hat die Halbzeitmarke \u00fcberschritten!"},"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=\u201dtop_left\u201d background_repeat=\u201dwiederholen\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=\u201dwiederholen\u201d]<\/p>\n<p>PRF schreibt weiterhin Geschichte, denn fast alle an der Studie teilnehmenden Kinder sind f\u00fcr ihren einj\u00e4hrigen Besuch ins Children&#039;s Hospital Boston gekommen und haben damit die H\u00e4lfte des Weges bis zum Abschluss der Studie zur\u00fcckgelegt. <a href=\"https:\/\/www.progeriaresearch.org\/de\/clinical-trials\/\">klicken Sie hier<\/a> f\u00fcr Einzelheiten, wie Sie helfen k\u00f6nnen.<\/p>\n<p>Spannende Zeiten! Die klinische Arzneimittelstudie gegen Progerie begann am 7. Mai 2007 mit der Ankunft von zwei Kindern in Boston, MA, zu ihrem ersten von sieben Besuchen \u00fcber einen Zeitraum von zwei Jahren. Bei diesem ersten Besuch wurden sie ausf\u00fchrlich getestet und erhielten ihre erste Dosis des Medikaments. Seitdem fliegen durchschnittlich zwei Familien pro Woche nach Boston, und im Oktober 2007 waren alle Teilnehmer f\u00fcr die Studie angemeldet. Die Studie soll im Oktober 2009 enden und die Ergebnisse sollen 2010 ver\u00f6ffentlicht werden.<\/p>\n<p>Bis zum 1. Oktober 2008 hatten alle Kinder, bis auf eines, den einw\u00f6chigen, einj\u00e4hrigen Besuch abgeschlossen.<\/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 tr\u00e4gt stolz ihre 1-Jahres-Trial-Medaille, die sie am Ende ihrer letzten Reise nach Boston erhielt<\/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, aus Argentinien<\/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>\u201eMir ist keine andere seltene genetische Erkrankung bekannt, bei der es von der Genentdeckung bis zur klinischen Erprobung weniger als vier Jahre gedauert hat \u2013 ein ph\u00e4nomenaler Beweis f\u00fcr die harte Arbeit der Progeria Research Foundation.\u201c\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>Achtundzwanzig (28) Kinder aus sechzehn L\u00e4ndern<\/strong> nehmen teil, im Alter von 3 bis 15 Jahren. Die Kinder kommen alle vier Monate wieder ins Children&#039;s Hospital Boston, um sich untersuchen zu lassen und neue Medikamente zu erhalten. Bei jedem Besuch bleiben sie 4-8 Tage in Boston. W\u00e4hrend sie zu Hause sind, werden die Kinder von ihren \u00c4rzten genau beobachtet und monatlich an das Bostoner Forschungsteam \u00fcbermittelt.<\/div>\n<div>\u00a0<\/div>\n<div>F\u00fcr die Dauer der Studie reisen 1\u20132 Kinder pro Woche nach Boston, um daran teilzunehmen.<\/div>\n<div>\u00a0<\/div>\n<div>Kinder stammen aus folgenden L\u00e4ndern:<\/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>Argentinien<\/li>\n<li>Belgien<\/li>\n<li>Kanada<\/li>\n<li>D\u00e4nemark<\/li>\n<li>England<\/li>\n<li>Indien<\/li>\n<li>Israel<\/li>\n<li>Italien<\/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>\u201eDie beiden Megans\u201c, beide 6 Jahre alt, in Boston f\u00fcr die klinische Studie<\/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, 8 \u00bd, aus Belgien mit Hayley, 9 \u00bd, aus England im Juni im Children&#039;s Hospital Boston bei ihrem ersten Besuch.<\/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>Japan<\/li>\n<li>Mexiko<\/li>\n<li>Pakistan<\/li>\n<li>Polen<\/li>\n<li>Portugal<\/li>\n<li>Rum\u00e4nien<\/li>\n<li>USA<\/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>Der<\/strong> <strong>Klinische Arzneimittelstudie zur Progerie<\/strong><strong>:\u00a0<\/strong><strong>Wer, Wo, Wann, Wie und Wie viel\u2026<\/strong><\/p>\n<p><strong>Die klinische Studie wird von Mark Kieran MD, PhD geleitet. <\/strong>Direktor der Abteilung f\u00fcr p\u00e4diatrische Neuroonkologie am Dana-Farber Cancer Institute und Children&#039;s Hospital Boston; Assistenzprofessor an den Abteilungen f\u00fcr P\u00e4diatrie und H\u00e4matologie\/Onkologie an der Harvard Medical School. Dr. Kieran ist ein p\u00e4diatrischer Onkologe mit umfassender Erfahrung mit dem untersuchten Medikament (Farnesyltransferase oder FTI) bei Kindern.<\/p>\n<div>Die klinische Studie ist eine Gemeinschaftsanstrengung. Die Kinder werden von \u00c4rzten in <strong>Kinderkrankenhaus Boston, Dana-Farber Cancer Institute und Brigham and Women&#039;s Hospital,<\/strong> allen Einrichtungen der Harvard University. Dar\u00fcber hinaus sind \u00c4rzte und Wissenschaftler aus<strong> Die Warren Alpert Medical School an der Brown University, UCLA und NIH<\/strong> tragen dazu bei, dass dieser Versuch ein Erfolg wird. Viele Personen arbeiten zusammen, um diese Forschung durchzuf\u00fchren.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong>Wie sind wir an diesen Punkt gelangt?<\/strong> Im Jahr 2002 hat das Forschungsteam der Progeria Research Foundation <a href=\"https:\/\/www.progeriaresearch.org\/de\/2003\/04\/16\/identification-of-gene-gives-hope-to-children-with-progeria\/\" target=\"_blank\" rel=\"noopener noreferrer\">entdeckte das Progerie-Gen.<\/a>\u00a0\u00a0Diese Entdeckung f\u00fchrte nicht nur zu einem besseren Verst\u00e4ndnis von Progerie, sondern Wissenschaftler wissen jetzt auch, dass uns die Erforschung von Progerie dabei helfen kann, mehr \u00fcber Herzkrankheiten und den normalen Alterungsprozess zu erfahren, der uns alle betrifft.<\/div>\n<div>\u00a0<\/div>\n<div>Seit der Entdeckung des Gens hat uns die Unterst\u00fctzung von Forschern, Klinikern und Familien von Kindern mit Progerie an einen weiteren Scheideweg bei der Suche nach einer Behandlung gef\u00fchrt. Forscher haben eine m\u00f6gliche medikament\u00f6se Behandlung f\u00fcr Kinder mit Progerie identifiziert, die sogenannten Farnesyltransferase-Inhibitoren (FTIs), und im Labor Studien durchgef\u00fchrt, die einen Versuch mit dem Medikament am Menschen unterst\u00fctzen.\u00a0<a href=\"https:\/\/www.progeriaresearch.org\/de\/2006\/02\/16\/three-studies-released-that-bring-us-closer-than-ever-to-understanding-progeria-and-to-disease-treatment\/\">klicken Sie hier<\/a>\u00a0f\u00fcr weitere Einzelheiten zur Forschung.<\/div>\n<div>\u00a0<\/div>\n<div>\n<div><strong>Wie wirkt dieses Medikament bei Progerie?<\/strong><\/div>\n<div>Das Protein, das unserer Meinung nach f\u00fcr Progerie verantwortlich ist, hei\u00dft Progerin. Um die normale Zellfunktion zu blockieren und Progerie zu verursachen, muss ein Molek\u00fcl namens \u201eFarnesylgruppe\u201c an das Progerinprotein angeh\u00e4ngt werden. FTIs wirken, indem sie die Anheftung der Farnesylgruppe an Progerin blockieren (hemmen). Wenn also das FTI-Medikament diese Anheftung der Farnesylgruppe bei Kindern mit Progerie blockieren kann, kann Progerin \u201egel\u00e4hmt\u201c und die Progerie gelindert werden.<\/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>Progeriezellen normalisieren sich, wenn FTIs angewendet werden. Capell et al., PNAS, 2005. Normale Zelle. Progeriezelle. Progeriezelle nach Behandlung mit FTI<\/strong><\/em><\/div>\n<div>\u00a0<\/div>\n<div>\n<div><strong>Was kostet die Testversion bei PRF? <\/strong>\u00a0Wir sch\u00e4tzen, dass die Studie PRF $2 Millionen Dollar kosten wird. Damit werden klinische Tests, \u00dcbersetzer, Fl\u00fcge, Verpflegung, Unterkunft und bestimmte medizinische Kosten abgedeckt. <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\" \/>w\u00e4hrend dieses Zeitraums von 2 \u00bd Jahren.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong><em>Ihre Spende wird dazu beitragen, dass dieser Versuch stattfinden kann.<\/em><\/strong><\/div>\n<div>\n<p><em>Zur Finanzierung dieser Studie muss die PRF etwa $2 Millionen Dollar aufbringen, und bis Juli 2009 haben wir bereits $1,9 Millionen aufgebracht!<\/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>Unser <a href=\"https:\/\/www.progeriaresearch.org\/de\/circle-of-hope-3\/\" target=\"_blank\" rel=\"noopener noreferrer\">Kreis der Hoffnung<\/a> hat sich erweitert\u2026<\/strong><\/p>\n<\/div>\n<div>\u00a0<\/div>\n<div>Im Jahr 2006 wurde die Kampagne \u201eCircle of Hope\u201c ins Leben gerufen, um 5 Jahre lang j\u00e4hrlich 140.000.000 TP2 zu sammeln, damit unsere Zellbank, unsere Diagnosetests, unsere Forschungsstipendien und andere Programme auf Hochtouren laufen. Das Erreichen dieses Spendenziels w\u00fcrde es uns erm\u00f6glichen, mit dem zunehmenden Interesse an der Progerieforschung Schritt zu halten und unser Fortschrittstempo beizubehalten. Wer h\u00e4tte gedacht, dass wir weniger als ein Jahr sp\u00e4ter mitten in einer Kampagne stecken w\u00fcrden, um 140.000.000 TP2 f\u00fcr die Finanzierung eines Medikamententests zur Behandlung von Progerie zu sammeln?! Unsere Kampagne \u201eCircle of Hope\u201c umfasst nun auch dieses Vorhaben.<\/div>\n<div>\u00a0<\/div>\n<div>Bitte helfen Sie uns, die <strong>Kreis <\/strong>intakt, so dass die <strong>Hoffnung<\/strong> der Behandlung wird zu einem <strong>Wirklichkeit.\u00a0<\/strong><a href=\"https:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR\">Spenden<\/a> Heute.<\/div>\n<div><em>\u00a0<\/em><\/div>\n<div><strong>Jetzt ist es an der Zeit, eine Behandlung f\u00fcr Progerie zu finden<em>.<\/em><\/strong><\/div>\n<div>\u00a0<strong>Gemeinsam\u00a0<em>WILLE<\/em>\u00a0Finde die Heilung!<\/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\/de\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\" \/>\n<meta property=\"og:locale\" content=\"de_DE\" \/>\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\/de\/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 Minuten\" \/>\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\":\"de\",\"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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