{"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\/id\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/","title":{"rendered":"Uji Klinis Obat Progeria Pertama Kalinya Melampaui Setengah Jalan!"},"content":{"rendered":"<p>[et_pb_section fb_built=\u201d1\u2033 _builder_version=\u201d3.22\u2033][et_pb_row _builder_version=\u201d3.25\u2033 ukuran_latar belakang=\u201dawal\u201d posisi_latar belakang=\u201dkiri_atas\u201d pengulangan_latar belakang=\u201dulangi\u201d][et_pb_column jenis=\u201d4_4\u2033 _builder_version=\u201d3.25\u2033 bantalan_khusus=\u201d|||\u201d bantalan_khusus__hover=\u201d|||\u201d][et_pb_text _builder_version=\u201d4.6.5\u2033 ukuran_latar belakang=\u201dawal\u201d posisi_latar belakang=\u201dkiri_atas\u201d pengulangan_latar belakang=\u201dulangi\u201d]<\/p>\n<p>PRF terus membuat sejarah, karena hampir semua anak yang terdaftar dalam uji coba telah datang ke Rumah Sakit Anak Boston untuk kunjungan 1 tahun mereka, menandai titik tengah menuju penyelesaian. <a href=\"https:\/\/www.progeriaresearch.org\/id\/clinical-trials\/\">klik disini<\/a> untuk mengetahui rincian tentang bagaimana Anda dapat membantu.<\/p>\n<p>Saat-saat yang mengasyikkan! Uji klinis obat Progeria dimulai pada tanggal 7 Mei 2007 dengan kedatangan dua anak di Boston, MA untuk kunjungan pertama dari tujuh kunjungan selama periode 2 tahun. Pada kunjungan pertama ini, mereka menjalani tes ekstensif dan dosis pertama obat. Rata-rata dua keluarga telah terbang ke Boston setiap minggu sejak saat itu, dan pada bulan Oktober 2007, uji klinis tersebut telah terdaftar secara penuh. Uji klinis tersebut diharapkan akan berakhir pada bulan Oktober 2009, dengan hasil yang dipublikasikan pada tahun 2010.<\/p>\n<p>Sampai tanggal 1 Oktober 2008, semua kecuali satu anak telah menyelesaikan kunjungan selama seminggu dan 1 tahun.<\/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 dengan bangga mengenakan Medali Uji Coba 1 tahunnya, yang diterimanya di akhir perjalanan terbarunya ke 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, dari 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>\u201cSaya tidak tahu ada penyakit genetik langka lain yang berhasil berkembang dari penemuan gen hingga uji klinis dalam waktu kurang dari empat tahun \u2013 sebuah bukti fenomenal atas kerja keras Yayasan Penelitian 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>Dua puluh delapan (28) anak dari enam belas negara<\/strong> berpartisipasi, berusia 3 hingga 15 tahun. Anak-anak kembali ke Rumah Sakit Anak Boston setiap empat bulan, untuk menjalani tes dan menerima pasokan obat baru, dan tinggal di Boston selama 4-8 hari setiap kunjungan. Saat di rumah, dokter mereka mengawasi anak-anak dengan ketat dan mengirimkan laporan kesehatan bulanan kepada tim peneliti Boston.<\/div>\n<div>\u00a0<\/div>\n<div>Selama masa percobaan, 1-2 anak per minggu akan bepergian ke Boston untuk berpartisipasi.<\/div>\n<div>\u00a0<\/div>\n<div>Anak-anak Berasal dari Negara-negara Berikut:<\/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>Belgia<\/li>\n<li>Kanada<\/li>\n<li>Denmark<\/li>\n<li>Inggris<\/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>\u201cDua Megan\u201d, keduanya berusia 6 tahun, di Boston untuk uji klinis<\/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, dari Belgia dengan Hayley, 9 \u00bd, dari Inggris pada bulan Juni di Rumah Sakit Anak Boston selama kunjungan pertama mereka.<\/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>Jepang<\/li>\n<li>Meksiko<\/li>\n<li>Bahasa Indonesia: Pakistan<\/li>\n<li>Polandia<\/li>\n<li>Portugal<\/li>\n<li>Rumania<\/li>\n<li>Amerika Serikat<\/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>Itu<\/strong> <strong>Uji Klinis Obat Progeria<\/strong><strong>:\u00a0<\/strong><strong>Siapa, Dimana, Kapan, Bagaimana dan Berapa Banyak\u2026<\/strong><\/p>\n<p><strong>Uji klinis ini dipimpin oleh Mark Kieran MD, PhD, <\/strong>Direktur, Neuro-Onkologi Medis Pediatrik, Dana-Farber Cancer Institute dan Rumah Sakit Anak Boston; Asisten Profesor, Departemen Pediatri dan Hematologi\/Onkologi, Harvard Medical School. Dr. Kieran adalah seorang onkolog pediatrik dengan pengalaman luas dengan obat yang sedang diteliti (farnesyltransferase, atau FTI) pada anak-anak.<\/p>\n<div>Uji klinis ini merupakan upaya kolaboratif. Anak-anak tersebut diperiksa oleh dokter di <strong>Rumah Sakit Anak Boston, Institut Kanker Dana-Farber, dan Rumah Sakit Brigham and Women&#039;s,<\/strong> semua institusi Universitas Harvard. Selain itu, dokter dan ilmuwan dari<strong> Sekolah Kedokteran Warren Alpert di Universitas Brown, UCLA, dan NIH<\/strong> membantu menyukseskan uji coba ini. Banyak individu bekerja sama untuk melakukan penelitian ini.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong>Bagaimana kita sampai pada titik ini?<\/strong> Pada tahun 2002, tim peneliti kolaboratif Yayasan Penelitian Progeria <a href=\"https:\/\/www.progeriaresearch.org\/id\/2003\/04\/16\/identification-of-gene-gives-hope-to-children-with-progeria\/\" target=\"_blank\" rel=\"noopener noreferrer\">menemukan gen Progeria.<\/a>\u00a0\u00a0Penemuan ini tidak hanya mengarah pada pemahaman lebih jauh tentang Progeria, tetapi para ilmuwan kini mengetahui bahwa mempelajari Progeria dapat membantu kita mempelajari lebih lanjut tentang penyakit jantung dan proses penuaan normal yang memengaruhi kita semua.<\/div>\n<div>\u00a0<\/div>\n<div>Sejak penemuan gen tersebut, dukungan dari para peneliti, dokter, dan keluarga anak-anak penderita Progeria telah membawa kita ke persimpangan lain dalam pencarian pengobatan. Para peneliti telah mengidentifikasi pengobatan obat yang potensial untuk anak-anak penderita Progeria, yang disebut penghambat farnesyltransferase (FTIs), dan telah melakukan penelitian di laboratorium yang mendukung uji coba obat tersebut pada manusia.\u00a0<a href=\"https:\/\/www.progeriaresearch.org\/id\/2006\/02\/16\/three-studies-released-that-bring-us-closer-than-ever-to-understanding-progeria-and-to-disease-treatment\/\">klik disini<\/a>\u00a0untuk rincian lebih lanjut tentang penelitian ini.<\/div>\n<div>\u00a0<\/div>\n<div>\n<div><strong>Bagaimana obat ini bekerja pada Progeria?<\/strong><\/div>\n<div>Protein yang kami yakini bertanggung jawab atas Progeria disebut progerin. Untuk memblokir fungsi sel normal dan menyebabkan Progeria, molekul yang disebut &quot;kelompok farnesyl&quot; harus dilekatkan pada protein progerin. FTI bekerja dengan menghalangi (menghambat) perlekatan kelompok farnesyl pada progerin. Jadi, jika obat FTI dapat menghalangi perlekatan kelompok farnesyl ini pada anak-anak penderita Progeria, maka progerin dapat &quot;dilumpuhkan&quot; dan Progeria membaik.<\/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>Sel progeria menjadi normal saat FTI diterapkan. Capell et al., PNAS, 2005. Sel normal. Sel progeria. Sel progeria setelah diobati dengan FTI<\/strong><\/em><\/div>\n<div>\u00a0<\/div>\n<div>\n<div><strong>Berapa biaya persidangan PRF? <\/strong>\u00a0Kami memperkirakan uji coba ini akan menelan biaya PRF $2 juta dolar. Biaya ini akan digunakan untuk pengujian klinis, penerjemah, penerbangan, makanan, penginapan, dan biaya medis tertentu. <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\" \/>selama periode waktu 2 \u00bd tahun tersebut.<\/div>\n<div><strong>\u00a0<\/strong><\/div>\n<div><strong><em>Donasi Anda akan membantu mewujudkan uji coba ini.<\/em><\/strong><\/div>\n<div>\n<p><em>PRF perlu mengumpulkan sekitar $2 juta dolar untuk mendanai uji coba ini, dan hingga Juli 2009, kami telah mengumpulkan $1,9 juta!<\/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>Kita <a href=\"https:\/\/www.progeriaresearch.org\/id\/circle-of-hope-3\/\" target=\"_blank\" rel=\"noopener noreferrer\">Lingkaran Harapan<\/a> telah diperluas\u2026<\/strong><\/p>\n<\/div>\n<div>\u00a0<\/div>\n<div>Pada tahun 2006, kampanye Circle of Hope dibuat untuk mengumpulkan $100.000 per tahun selama 5 tahun agar Bank Sel, Pengujian Diagnostik, dana hibah penelitian, dan program lainnya tetap berjalan dengan kecepatan penuh. Memenuhi target pengumpulan dana ini akan memungkinkan kami untuk mengimbangi meningkatnya minat terhadap penelitian Progeria, dan untuk melanjutkan laju kemajuan kami. Siapa yang dapat membayangkan bahwa kurang dari setahun kemudian, kami akan berada di tengah-tengah kampanye untuk mengumpulkan $2 juta untuk mendanai uji coba obat untuk mengobati Progeria?! Kampanye Circle of Hope kami sekarang mencakup usaha ini.<\/div>\n<div>\u00a0<\/div>\n<div>Tolong bantu kami menjaga <strong>Lingkaran <\/strong>utuh, jadi <strong>harapan<\/strong> pengobatan menjadi <strong>realitas.\u00a0<\/strong><a href=\"https:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR\">Menyumbangkan<\/a> Hari ini.<\/div>\n<div><em>\u00a0<\/em><\/div>\n<div><strong>Inilah saatnya untuk menemukan pengobatan untuk Progeria<em>.<\/em><\/strong><\/div>\n<div>\u00a0<strong>Bersama-sama, kita\u00a0<em>AKAN<\/em>\u00a0temukan obatnya!<\/strong><\/div>\n<\/div>\n<p>[\/et_pb_teks][\/et_pb_kolom][\/et_pb_baris][\/et_pb_bagian]<\/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\/id\/2008\/05\/16\/first-ever-progeria-clinical-drug-trial-surpasses-half-way-mark\/\" \/>\n<meta property=\"og:locale\" content=\"id_ID\" \/>\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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