{"id":2082,"date":"2009-05-16T13:36:57","date_gmt":"2009-05-16T13:36:57","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?p=2082"},"modified":"2020-12-10T16:44:57","modified_gmt":"2020-12-10T21:44:57","slug":"moving-ahead-at-lightening-speed-as-the-progeria-triple-drug-trial-begins","status":"publish","type":"post","link":"https:\/\/www.progeriaresearch.org\/de\/2009\/05\/16\/moving-ahead-at-lightening-speed-as-the-progeria-triple-drug-trial-begins\/","title":{"rendered":"Mit Lichtgeschwindigkeit geht es voran: Die Dreifach-Medikamentenstudie gegen Progerie beginnt!"},"content":{"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_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2009\/05\/HayleyMichielAmber-Aug09.jpg&#8221; title_text=&#8221;HayleyMichielAmber-Aug09&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; hover_enabled=&#8221;0&#8243; align=&#8221;center&#8221; sticky_enabled=&#8221;0&#8243; custom_margin=&#8221;||-4px|||&#8221;][\/et_pb_image][et_pb_text admin_label=&#8221;caption HM&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; text_font_size=&#8221;12px&#8221;]<\/p>\n<p><strong><em>Hayley aus England und Michiel aus Belgien strahlen \u00fcber das ganze Gesicht, als sie am Freitag, den 14. August 2009, mit ihren Troph\u00e4en f\u00fcr den Abschluss der allerersten klinischen Arzneimittelstudie zu Progerie posieren. In dieser Woche absolvierten sie zusammen mit Michiels Schwester Amber (rechts) auch ihren ersten Besuch im Rahmen der Dreifach-Medikamentenstudie.<\/em><\/strong><\/p>\n<p>[\/et_pb_text][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;]<\/p>\n<p>Die Progeria Research Foundation und das Children&#039;s Hospital Boston arbeiten erneut zusammen, um eine zweite klinische Studie f\u00fcr Kinder mit Progerie durchzuf\u00fchren. An dieser spannenden und viel gr\u00f6\u00dferen Studie nehmen 45 Kinder aus 24 L\u00e4ndern teil, die 17 verschiedene Sprachen sprechen!<strong>\u00a0<\/strong><\/p>\n<p><strong>Zusammenfassung<\/strong>: Forscher haben zwei weitere Medikamente identifiziert, die in Kombination mit dem aktuell getesteten FTI-Medikament eine noch wirksamere Behandlung f\u00fcr Kinder mit Progerie erm\u00f6glichen k\u00f6nnten als FTI allein.<\/p>\n<div><strong>Wissenschaftliche Grundlagen der Behandlung<\/strong><\/div>\n<div>Progerie wird durch ein abnormales Protein namens Progerin verursacht. Das Progerie-Forschungsteam am Children&#039;s Hospital Boston wird die derzeitige Behandlung mit FTI um zwei Medikamente namens Pravastatin und Zoledronat erg\u00e4nzen.<\/div>\n<div>\u00a0<\/div>\n<div><strong><em>Strategie:<\/em><\/strong> Alle drei Medikamente zielen auf verschiedene Punkte auf dem Weg ab, der zur Produktion des krankheitserregenden Progerins f\u00fchrt. In spannenden Laborstudien, die der Spanier Dr. Carlos Lopez-Otin auf dem wissenschaftlichen Workshop der Progeria Research Foundation 2007 vorstellte, verbesserten die beiden neuen Medikamente die Krankheit in Progeria-Zellen und verl\u00e4ngerten die Lebensdauer in Progeria-Mausmodellen.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong><em>Ziel:<\/em><\/strong> Wenn die drei in dieser Studie verabreichten Medikamente die Bindung dieser Farnesylgruppe wirksam blockieren k\u00f6nnen, kann Progerin \u201egel\u00e4hmt\u201c und die Progerie gelindert werden. Wir hoffen, dass die Medikamente als Partner wirken und sich erg\u00e4nzen, sodass das Progerinprotein durch die Kombination der drei Medikamente st\u00e4rker beeinflusst wird als durch die Verwendung eines einzelnen Medikaments allein.<\/div>\n<div>\u00a0<\/div>\n<div><strong>Wer nimmt an der Dreifachstudie teil?<\/strong><\/div>\n<div><strong>Der Machbarkeitsversuch:<\/strong>\u00a0Das Team hat bereits eine Ministudie an f\u00fcnf Kindern mit Progerie durchgef\u00fchrt. In der kurzen, einmonatigen \u201eMachbarkeitsstudie\u201c wurde untersucht, ob die Kombination der drei Medikamente gut vertr\u00e4glich w\u00e4re, bevor eine gr\u00f6\u00dfere internationale Studie gestartet wurde. Die Nebenwirkungen waren akzeptabel, und das Team ist nun mit der gr\u00f6\u00dferen Wirksamkeitsstudie fortgefahren.<\/div>\n<div><strong>Der Wirksamkeitstest:<\/strong> Im Januar 2010 war die Dreifachstudie mit 45 Kindern vollst\u00e4ndig abgeschlossen. Dazu geh\u00f6ren Kinder, die an der FTI-Studie teilgenommen haben, die 5 Kinder der Machbarkeitsstudie und andere Kinder, die entweder zu jung waren, um an der ersten Studie teilzunehmen, oder Kinder, die wir in den letzten zwei Jahren entdeckt haben. Kinder, die an der FTI-Studie teilgenommen hatten, hatten die M\u00f6glichkeit, sich bei ihrer Ankunft f\u00fcr die Dreifachstudie anzumelden. Dadurch konnten die Kinder FTI weiter einnehmen, ohne Dosen zu vergessen.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong>Die Beziehung zwischen Behandlung und Progerie<\/strong><\/div>\n<div>Wie sind wir von der Genentdeckung zur medikament\u00f6sen Therapie f\u00fcr Kinder mit Progerie gekommen? Der Schl\u00fcssel war die Entdeckung des Gens f\u00fcr Progerie. Dieses Gen hei\u00dft <em>LMNA<\/em>, und es kodiert normalerweise ein Protein namens Prelamin A (dieses Protein wird weiter verarbeitet und wird zu Lamin A). Kinder mit Progerie haben eine Mutation in <em>LMNA<\/em> Dies f\u00fchrt zur Produktion einer abnormalen Form von Prelamin A, genannt \u201eProgerin\u201c. Viele Jahre grundlegender Forschung zu Prelamin A und Lamin A haben uns gezeigt, dass die in dieser Studie verabreichten Medikamente die Krankheit bei Progerie beeinflussen k\u00f6nnen. In den letzten sechs Jahren konzentrierte sich die Forschung auf das systematische Testen dieser Medikamente an Progeriezellen und Progeriem\u00e4usen.<\/div>\n<div>\u00a0<\/div>\n<div><strong>Das klinische Studienteam<\/strong><\/div>\n<div>Seit Mai 2007 behandelt ein 28-k\u00f6pfiges Team Kinder mit Progerie aus aller Welt. Die Mitglieder des Teams sind nicht nur Experten f\u00fcr Progerie, sondern auch f\u00fcr die drei Medikamente, die in dieser Studie verabreicht werden.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong>Testmedikamente im \u00dcberblick<\/strong><\/div>\n<div><strong>Pravastatin<\/strong> (vermarktet als Pravachol oder Selektine) geh\u00f6rt zur Arzneimittelklasse der Statine. Es wird normalerweise zur Senkung des Cholesterinspiegels und zur Vorbeugung von Herz-Kreislauf-Erkrankungen eingesetzt.<\/div>\n<div><strong>Zoledrons\u00e4ure <\/strong>ist ein <strong>Bisphosphonat, <\/strong>wird \u00fcblicherweise als Knochenmedikament zur Linderung von Osteoporose und zur Vorbeugung von Skelettbr\u00fcchen bei Menschen mit bestimmten Krebsarten eingesetzt.<\/div>\n<div><strong>Lonafarnib<\/strong> ist ein <strong>FTI<\/strong> (Farnesyltransferase-Hemmer), ein Medikament, das im Labor eine Anomalie in Progeriezellen r\u00fcckg\u00e4ngig machen kann und bei Progerie-M\u00e4usen eine Besserung der Krankheit bewirkt hat.<\/div>\n<div><em>Alle drei Medikamente blockieren die Produktion des Farnesylmolek\u00fcls, das Progerin zur Entstehung der Krankheit Progerie ben\u00f6tigt.<\/em><\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong>Timing<\/strong><\/div>\n<div>Die Patienten reisen \u00fcber einen Zeitraum von 2 Jahren alle 6 Monate f\u00fcr 4-7 Tage dauernde Tests und Untersuchungen nach Boston. Bei der FTI-Studie allein fanden die Besuche in Boston alle vier Monate statt.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong>Kosten<\/strong><br \/>Nat\u00fcrlich ist die Durchf\u00fchrung einer klinischen Studie dieser Gr\u00f6\u00dfenordnung kostspielig und es gibt nur wenige F\u00f6rderquellen f\u00fcr die Erforschung seltener Krankheiten. Doch im Oktober 2009 vergab das National Heart, Lung, and Blood Institute des NIH an das dreifache Studienteam ein prestigetr\u00e4chtiges \u201eGrand Opportunities\u201c-Stipendium, das einen Gro\u00dfteil der Kosten abdecken wird. Wir freuen uns sehr \u00fcber diese au\u00dferordentliche Unterst\u00fctzung durch das NIH.<\/div>\n<div>\n<p>Der Zuschuss deckt jedoch nicht alle Kosten des Versuchs. PRF muss noch etwa $150.000 f\u00fcr einige Kosten aufbringen, die nicht durch den Zuschuss abgedeckt sind.<\/p>\n<p><a href=\"https:\/\/www.progeriaresearch.org\/de\/other-ways-to-support-prf\/\">klicken Sie hier<\/a> Spenden Sie f\u00fcr die Dreifach-Medikamentenstudie und helfen Sie dabei, das ZIEL der Behandlung und Heilung WIRKLICHKEIT zu verwirklichen!<strong><br \/><\/strong><\/p>\n<\/div>\n<div><strong><em>* <\/em>\u201eKombinierte Behandlung mit Statinen und Aminobisphosphonaten verl\u00e4ngert die Lebenserwartung in einem Mausmodell der vorzeitigen Alterung des Menschen\u201c,<\/strong> von Ignacio Varela, Sandrine Pereira, Alejandro P. Ugalde, Claire L. Navarro, Mar\u00eda F. Suarez, Pierre Cau, Juan Cadinanos, Fernando G. Osorio, Nicolas Foray, Juan Cobo, Felix de Carlos, Nicolas Levy, Jose MP Freije und Carlos Lopez-Ot\u00e1n. <em>Naturmedizin, <\/em>2008. 14(7): S. 767-72<em>.<\/em><\/div>\n<div><em>\u00a0<\/em><em><br \/><\/em><strong>T<\/strong><strong>Die neue Generation von Kindern, der PRF hilft \u2026<\/strong><\/div>\n<div>Im M\u00e4rz 2009 nahmen f\u00fcnf Kinder im Alter von 2 bis 3 Jahren an einer einmonatigen Machbarkeitsstudie teil, um festzustellen, ob die Nebenwirkungen der drei Medikamente zusammen ertr\u00e4glich sind. Die Ergebnisse waren positiv und ebneten den Weg f\u00fcr die vollst\u00e4ndige, zweij\u00e4hrige Dreifachstudie, an der bis zu 45 Kinder mit Progerie teilnehmen sollten.\u00a0<strong>Hut ab vor diesen tollen Familien!<\/strong><\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong>Hier sind einige ihrer Aussagen:<\/strong><\/div>\n<p><img loading=\"lazy\" decoding=\"async\" class=\"size-full wp-image-2084 alignleft\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/AdaliaJAN09.jpg\" alt=\"\" width=\"125\" height=\"188\" \/><em>\u201e<\/em><em>ALLE waren so wunderbar. F\u00fcr uns seid ihr ALLE von Gott gesandt und wir SCH\u00c4TZEN alles, was ihr f\u00fcr diese kleinen Engel tut. Unsere Familie ist so \u00fcberw\u00e4ltigt von der Aufregung und allen m\u00f6glichen Emotionen wegen Adalias Reise nach Boston an diesem Wochenende, dass ich gar nicht in Worte fassen kann, wie wir uns f\u00fchlen.<\/em><em>\u201c.<\/em><\/p>\n<p><em><\/em><\/p>\n<div><em><img loading=\"lazy\" decoding=\"async\" class=\"alignright size-full wp-image-2085\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/ZachJune09.jpg\" alt=\"\" width=\"125\" height=\"138\" \/>\u201eDieses neue Medikament f\u00fcr Zach gibt uns neue Hoffnung, dass sein Herz st\u00e4rker, sein L\u00e4cheln strahlender und sein Leben l\u00e4nger wird. Dieser neue Medikamententest ist eine Antwort auf unsere Gebete. Vielen Dank an alle, die bei PRF mitgearbeitet haben und dies m\u00f6glich gemacht haben \u2026 die \u00c4rzte, die Forscher und das Personal. Ihr seid unsere Helden!\u201c<\/em><\/div>\n<div>\u00a0<\/div>\n<div>\n<div><div id=\"attachment_2086\" style=\"width: 160px\" class=\"wp-caption alignright\"><img loading=\"lazy\" decoding=\"async\" aria-describedby=\"caption-attachment-2086\" class=\"size-full wp-image-2086\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Cam-and-DadMarch09.jpg\" alt=\"\" width=\"150\" height=\"144\" \/><p id=\"caption-attachment-2086\" class=\"wp-caption-text\">Cam und sein Vater lernen, wie man das FTI-Medikament mit einem S\u00fc\u00dfstoff mischt.<\/p><\/div><\/p>\n<p><em>\u201eIm Namen von Cam und unserer Familie danke ich Ihnen allen bei PRF f\u00fcr alles, was Sie getan haben! Ohne Sie w\u00e4ren wir in einer Welt voller Verwirrung und Trauer verloren gewesen. Stattdessen leben wir in einer Welt voller Hoffnung und Zielstrebigkeit. Nochmals vielen Dank! Mit viel Liebe und Respekt\u201c,<\/em><\/p>\n<\/div>\n<div>\u00a0<\/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_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2009\/05\/HayleyMichielAmber-Aug09.jpg&#8221; title_text=&#8221;HayleyMichielAmber-Aug09&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; hover_enabled=&#8221;0&#8243; align=&#8221;center&#8221; sticky_enabled=&#8221;0&#8243; custom_margin=&#8221;||-4px|||&#8221;][\/et_pb_image][et_pb_text admin_label=&#8221;caption HM&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; text_font_size=&#8221;12px&#8221;] Hayley from England and Michiel from Belgium are all smiles as they pose with their trophies for completing the first-ever Progeria clinical drug trial on Friday, August 14, 2009. They also, [&hellip;]<\/p>","protected":false},"author":1,"featured_media":0,"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=\"Moving Ahead at Lightening Speed as The Progeria Triple Drug Trial Begins!\" font_container=\"tag:h1|text_align:left\" use_theme_fonts=\"yes\"]<\/p><div>[caption id=\"attachment_2083\" align=\"alignleft\" width=\"200\"]<img class=\"size-full wp-image-2083\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/HayleyMichielAmber-Aug09.jpg\" alt=\"\" width=\"200\" height=\"196\" \/> Hayley from England and Michiel from Belgium are all smiles as they pose with their trophies for completing the first-ever Progeria clinical drug trial on Friday, August 14, 2009. They also, along with Michiel\u2019s sister Amber (right), completed their first visit for the triple drug trial that week.[\/caption]<p>The Progeria Research Foundation and Children\u2019s Hospital Boston are once again partnering to conduct a second clinical trial for children with Progeria. This exciting and much larger trial includes 45 children from 24 countries, speaking 17 different languages!<strong>\u00a0<\/strong><\/p><p><strong>Summary<\/strong>: Researchers have identified two additional drugs that, when used in combination with the current FTI drug being tested, may provide an even more effective treatment for children with Progeria than FTI\u2019s alone.<\/p><div><strong>Scientific Basis for Treatment<\/strong><\/div><div>Progeria is caused by an abnormal protein named progerin.\u00a0The Progeria research team at Children\u2019s Hospital Boston will add two drugs, called pravastatin and zoledronate, to the current treatment with FTI.<\/div><div>\u00a0<\/div><div><strong><em>Strategy:<\/em><\/strong> All three drugs will target different points along the pathway leading to production of the disease-causing progerin.\u00a0In exciting laboratory studies presented by Dr. Carlos Lopez-Otin of Spain at the 2007 Progeria Research Foundation Scientific Workshop, the two new drugs improved disease in Progeria cells and extended lifespan in mouse models of Progeria.<\/div><div><strong>\u00a0<\/strong><\/div><div><strong><em>Goal:<\/em><\/strong> If the three drugs administered in this trial can effectively block this farnesyl group attachment, then progerin may be \u201cparalyzed\u201d and Progeria may be improved.\u00a0We hope that the drugs will work as partners, to complement each other so that the progerin protein is affected more by combining the three drugs than using any one drug alone.<\/div><div>\u00a0<\/div><div><strong>Who is Enrolled in the Triple Drug Trial?<\/strong><\/div><div><strong>The Feasibility Trial:<\/strong>\u00a0The team has already conducted a mini-trial for 5 children with Progeria.\u00a0The short, one month \u201cfeasibility\u201d trial, asked whether the three-drug combination would be well-tolerated, prior to embarking on a larger international trial.\u00a0Side effects were acceptable, and the team has moved ahead to the larger efficacy trial.<\/div><div><strong>The Efficacy Trial:<\/strong> In Janaury 2010, the triple trial beamce fully enrolled with 45 children. This includes children participating in the FTI-only trial, the 5 in the feasibility trial, and other children that were either too young to participate in the first trial or children that we\u2019ve discovered over the past 2 years. Children enrolled in the FTI-only trial had the opportunity to enroll in the triple trial when they arrived for that first trial.\u00a0This allowed the children to continue taking FTI without any missed doses.<\/div><div><strong>\u00a0<\/strong><\/div><div><strong>The Treatment\/Progeria Relationship<\/strong><\/div><div>How did we get from gene discovery to drug therapy for children with Progeria?\u00a0Finding the gene for Progeria was the key. This gene is called <em>LMNA<\/em>, and it normally encodes a protein called prelamin A (this protein is further processed and becomes lamin A). Children with Progeria have a mutation in <em>LMNA<\/em> which leads to the production of an abnormal form of prelamin A called \u201cprogerin.\u201d \u00a0Many years\u2019 worth of basic research on prelamin A and lamin A gave us the ability to understand that the drugs administered in this trial may affect disease in Progeria. \u00a0Over the past six years, research has focused on systematically testing these drugs on Progeria cells and Progeria mice.<\/div><div>\u00a0<\/div><div><strong>The Clinical Trial Team<\/strong><\/div><div>Since May 2007, a 28-member team has treated children with Progeria from around the globe.\u00a0Members of the team have expertise not only in Progeria, but also in the three drugs administered in this trial.<\/div><div><strong>\u00a0<\/strong><\/div><div><strong>Trial Medications at a Glance<\/strong><\/div><div><strong>Pravastatin<\/strong> (marketed as Pravachol or Selektine) is a member of the drug class of statins.\u00a0It is usually used for lowering cholesterol and preventing cardiovascular disease.<\/div><div><strong>Zoledronic acid <\/strong>is a <strong>bisphosphonate, <\/strong>usually used as a bone drug for improving osteoporosis, and to prevent skeletal fractures in people suffering from some forms of cancer.<\/div><div><strong>Lonafarnib<\/strong> is an <strong>FTI<\/strong> (Farnesyltransferase inhibitor), a drug that can reverse an abnormality in Progeria cells in the laboratory, and has improved disease in Progeria mice.<\/div><div><em>All 3 drugs block the production of the farnesyl molecule that is needed for progerin to create disease in Progeria.<\/em><\/div><div><strong>\u00a0<\/strong><\/div><div><strong>Timing<\/strong><\/div><div>Patients are traveling to Boston for testing and examinations lasting 4-7 days, every 6 months for a period of 2 years. For the FTI-only trial, Boston visits occurred every four months.<\/div><div><strong>\u00a0<\/strong><\/div><div><strong>Cost<\/strong><br \/>Of course, a clinical trial of this magnitude is expensive to manage, and there are precious few sources of support for rare disease research.\u00a0 But in October 2009, the NIH's National Heart, Lung, and Blood Institute awarded the triple trial team a prestigious \"Grand Opportunities\" grant that will cover many of the costs.\u00a0 We are thrilled to have earned this extraordinary support from NIH.<\/div><div><p>However, the grant doesn't cover all of the trial expenses.\u00a0 PRF still needs to raise about $150,000 for some costs not covered by the grant.<\/p><p><a href=\"https:\/\/web.archive.org\/web\/20170216041635\/https:\/\/www.progeriaresearch.org\/assets\/plugins\/fckeditor\/editor\/ways_to_donate\/\">Click here<\/a> to donate to the triple drug trial and help make the GOAL of treatment and cure a REALITY!<strong><br \/><\/strong><\/p><\/div><div><strong><em>* <\/em>\u201cCombined treatment with statins and aminobisphosphonates extends longevity in a mouse model of human premature aging\u201d,<\/strong> by Ignacio Varela, Sandrine Pereira, Alejandro P. Ugalde, Claire L. Navarro, Mar\u00c4\u00b1a F. Suarez, Pierre Cau, Juan Cadinanos, Fernando G. Osorio, Nicolas Foray, Juan Cobo, Felix de Carlos, Nicolas Levy, Jose MP Freije and Carlos Lopez-Ot\u00c4\u00b1n. <em>Nature Medicine, <\/em>2008. 14(7): p. 767-72<em>.<\/em><\/div><div><em>\u00a0<\/em><em><br \/><\/em><strong>T<\/strong><strong>he new generation of children PRF is helping\u2026<\/strong><\/div><div>In March, 2009, five children, ages 2-3, participated in a one-month feasibility study to determine if the side effects of the three drugs taken together were tolerable.\u00a0The results were positive, paving the way for the full, two-year, Triple Drug Trial to enroll up to 45 children with Progeria.\u00a0<strong>Hats off to these amazing families!<\/strong><\/div><div><strong>\u00a0<\/strong><\/div><div><strong>Here is what some of them had to say:<\/strong><\/div><p><img class=\"size-full wp-image-2084 alignleft\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/AdaliaJAN09.jpg\" alt=\"\" width=\"125\" height=\"188\" \/><em>\u201c<\/em><em>EVERYONE has been so wonderful.\u00a0 To us you are ALL GOD SENT and we APPRECIATE all that you do for these little angels.\u00a0 Our family is so overwhelmed with excitement and all sorts of emotions with Adalia\u2019s trip to Boston this weekend, I can\u2019t even begin to type the words of how we are feeling<\/em><em>\u201d.<\/em><\/p><\/div><div><em><img class=\"alignright size-full wp-image-2085\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/ZachJune09.jpg\" alt=\"\" width=\"125\" height=\"138\" \/>\u201cThis new medication for Zach gives us a renewed hope that his heart will be stronger, his smile will be brighter and his life will be longer.\u00a0 This new drug trial is an answer to our prayers. \u00a0 Thank you to everyone involved with PRF who made this happen...the doctors, the researchers and the staff.\u00a0 You are our heroes!\u201d<\/em><\/div><div>\u00a0<\/div><div>\u00a0<\/div><div>\u00a0<\/div><div>\u00a0<\/div><div><div>[caption id=\"attachment_2086\" align=\"alignright\" width=\"150\"]<img class=\"size-full wp-image-2086\" src=\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Cam-and-DadMarch09.jpg\" alt=\"\" width=\"150\" height=\"144\" \/> Cam and his dad learn how to mix the FTI drug with a sweetener.[\/caption]<p><em>\u201cOn behalf of Cam and our family, thank you all at PRF so much for all you have done!\u00a0 We would have been lost in a world of confusion and grief without you.\u00a0 Instead, we live in a world of hope and purpose.\u00a0 Thank you again and again!\u00a0\u00a0With much love and respect,\u201d<\/em><\/p><\/div><div>\u00a0<\/div><\/div>","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[2],"tags":[],"class_list":["post-2082","post","type-post","status-publish","format-standard","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>Moving Ahead at Lightening Speed as The Progeria Triple Drug Trial Begins! - 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\/2009\/05\/16\/moving-ahead-at-lightening-speed-as-the-progeria-triple-drug-trial-begins\/\" \/>\n<meta property=\"og:locale\" content=\"de_DE\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Moving Ahead at Lightening Speed as The Progeria Triple Drug Trial Begins! - 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_image src=&#8221;https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2009\/05\/HayleyMichielAmber-Aug09.jpg&#8221; title_text=&#8221;HayleyMichielAmber-Aug09&#8243; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; hover_enabled=&#8221;0&#8243; align=&#8221;center&#8221; sticky_enabled=&#8221;0&#8243; custom_margin=&#8221;||-4px|||&#8221;][\/et_pb_image][et_pb_text admin_label=&#8221;caption HM&#8221; _builder_version=&#8221;4.6.5&#8243; _module_preset=&#8221;default&#8221; text_font_size=&#8221;12px&#8221;] Hayley from England and Michiel from Belgium are all smiles as they pose with their trophies for completing the first-ever Progeria clinical drug trial on Friday, August 14, 2009. 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