{"id":1684,"date":"2012-09-24T22:30:31","date_gmt":"2012-09-24T22:30:31","guid":{"rendered":"https:\/\/www.progeriaresearch.org\/?page_id=1684"},"modified":"2023-09-21T12:18:41","modified_gmt":"2023-09-21T16:18:41","slug":"first-ever-progeria-treatment","status":"publish","type":"page","link":"https:\/\/www.progeriaresearch.org\/de\/first-ever-progeria-treatment\/","title":{"rendered":"Erste Behandlung f\u00fcr Progerie"},"content":{"rendered":"

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Es wurde Geschichte geschrieben: Bei jedem Kind in der allerersten klinischen Arzneimittelstudie zu Progerie zeigte sich eine Verbesserung in einem oder mehreren Bereichen seiner Erkrankung. Damit wurde bewiesen, dass das FTI-Medikament Lonafarnib die erste bekannte, wirksame Behandlung f\u00fcr Kinder mit Progerie ist.<\/p>\n

Klicken Sie hier, um Ihr kostenloses Exemplar des Dokuments mit den Ergebnissen der klinischen Studie herunterzuladen.<\/strong><\/a><\/p>\n

\"\"

Megan und Meghan waren die ersten beiden Kinder, die an der Studie teilnahmen. Die beiden M\u00e4dchen trafen sich zwei Jahre lang alle vier Monate in Boston, um Tests durchzuf\u00fchren, neue Medikamente zu erhalten und zusammen zu spielen! Hier sind sie im Dezember 2008, w\u00e4hrend einer Testpause im Boston Children's Hospital.<\/strong><\/span><\/p><\/div>\n

F\u00dcR MEDIEN:\u00a0 <\/strong>klicken Sie hier<\/a> f\u00fcr die Pressemitteilung, B-Roll und andere Pressedetails.<\/p>\n

Die Ergebnisse von <\/strong>die erste klinische Arzneimittelstudie f\u00fcr Kinder<\/strong><\/a> mit Progerie sind da und es ist offiziell!<\/strong>\u00a0 Lonafarnib, ein Farnesyltransferase-Hemmer (FTI), der urspr\u00fcnglich zur Behandlung von Krebs entwickelt wurde, hat sich bei Progerie als wirksam erwiesen. Jedes Kind zeigte eine oder mehrere der folgenden Verbesserungen: Gewichtszunahme, besseres Geh\u00f6r, verbesserte Knochenstruktur und\/oder, am wichtigsten, erh\u00f6hte Flexibilit\u00e4t der Blutgef\u00e4\u00dfe. Die Ergebnisse der Studie, die finanziert und koordiniert von der Progeria Research Foundation<\/strong>, erschienen am 24. September 2012 in Verfahren der Nationalen Akademie der Wissenschaften<\/a>.<\/em><\/strong><\/p>\n

Gordon et. al., Klinische Studie eines Farnesyltransferase-Hemmers bei Kindern mit Hutchinson-Gilford-Progerie-Syndrom, PNAS, 9. Oktober 2012, Band 109, Nr. 41, 16666-16671<\/em><\/p>\n

\"\"

Natsuki aus Japan mit ihrem Bruder, ihrem Vater und ihrer Mutter, die die Mischung aus Lonafarnib und fl\u00fcssigem S\u00fc\u00dfstoff zubereiten.<\/strong><\/span><\/p><\/div>\n

Ergebnisse zeigen Verbesserungen bei allen Kindern<\/strong>
An der zweieinhalb Jahre dauernden Medikamentenstudie nahmen 28 Kinder aus 16 L\u00e4ndern teil, was 75 Prozent aller weltweit bekannten Progerie-F\u00e4lle zum Zeitpunkt des Studienbeginns entspricht. Die Kinder reisten alle vier Monate nach Boston, um sich im Boston Children's Hospital umfassenden medizinischen Tests zu unterziehen und Medikamente zu studieren. Klinische und translationale Studieneinheit<\/a>. Alle erhielten zwei Jahre lang zweimal t\u00e4glich orales Lonafarnib, ein FTI von Merck & Co., unter der Aufsicht des Leiters der klinischen Studie Mark Kieran, MD, Ph.D., Direktor der p\u00e4diatrischen medizinischen Neuroonkologie am Dana-Farber \/ Children's Hospital Cancer Center, und der Co-Leiterinnen Dr. Monica Kleinman und Dr. Leslie Gordon<\/p>\n

Die Gewichtszunahmerate war das wichtigste Ergebnisma\u00df, da Kinder mit Progerie schwere Gedeihst\u00f6rungen erleiden und mit der Zeit eine sehr langsame, lineare Gewichtszunahmerate aufweisen. Die Forscher untersuchten viele andere Bereiche des K\u00f6rpers, darunter die Arteriensteifigkeit (ein Indikator f\u00fcr Herzinfarkt und Schlaganfall), die Knochensteifigkeit (ein Indikator f\u00fcr die Knochenst\u00e4rke) und das Geh\u00f6r. \u201eAls wir mit dieser klinischen Studie begannen, hatten wir keine Ahnung, ob irgendein Aspekt der Progerie reversibel w\u00e4re, da noch nie jemand zuvor eine klinische Behandlungsstudie f\u00fcr Progerie durchgef\u00fchrt hatte. Wir entdeckten, dass sich unter anderem die gro\u00dfen Blutgef\u00e4\u00dfe tats\u00e4chlich verbessern k\u00f6nnen. Dies war eine bahnbrechende Entdeckung f\u00fcr die Kinder, da beschleunigte Herz-Kreislauf-Erkrankungen die Todesursache bei Progerie sind. Obwohl es keine M\u00f6glichkeit gibt, innerhalb einer nur zweij\u00e4hrigen Behandlungsperiode zu wissen, ob wir Schlaganf\u00e4lle oder Herzinfarkte verz\u00f6gert oder die Lebenserwartung erh\u00f6ht haben, zwingen uns diese positiven Ergebnisse, weiterhin nach neuen Behandlungen zu suchen, bis wir unser Ziel von 1999 erreicht haben. Wir m\u00f6chten, dass Kinder mit Progerie 80 Jahre und \u00e4lter werden. Wir m\u00f6chten, dass sie ein erf\u00fclltes und gesundes Leben f\u00fchren\u201c, sagte Dr. Gordon, medizinischer Direktor der PRF und Erstautor der Ver\u00f6ffentlichung zur Behandlungsforschung.<\/p>\n

Rekordtempo des Fortschritts<\/strong><\/p>\n

Die Entdeckung der Behandlungsmethode erfolgte weniger als ein Jahrzehnt, nachdem PRF und der heutige Direktor des National Institutes of Health, Dr. Francis Collins, gemeinsam die Ursache von Progerie identifizierten \u2013 ein beispielloser Zeitrahmen in der Welt der medizinischen Forschung! Doch f\u00fcr PRF und Kinder mit Progerie, die im Durchschnitt nur 13 Jahre alt werden, ist diese Geschwindigkeit von entscheidender Bedeutung, um diesen Wettlauf gegen die Zeit zu gewinnen.<\/p>\n

\"\"

Mateo, Milagros und Fernando strahlen \u00fcber das ganze Gesicht, als sie bei ihrem letzten Besuch Auszeichnungen von PRF erhalten. Auf den Troph\u00e4en steht: \u201eDU HAST ES GESCHAFFT! Du hast den 1. Progerie-Test bestanden \u2013 Du bist EIN SUPERSTAR!\u201c<\/strong><\/span><\/p><\/div>\n

\u201ePRF ist ein gutes Beispiel f\u00fcr eine Organisation, die erfolgreich translationale Forschung erm\u00f6glicht und in beispiellosem Tempo von der Genentdeckung zur klinischen Behandlung \u00fcbergeht\u201c, sagte Dr. Kieran. \u201eVon 1999, als die Organisation gegr\u00fcndet wurde, bis heute hat PRF die genetische Mutation identifiziert, die die Krankheit verursacht, pr\u00e4klinische Forschung finanziert, diese Studie abgeschlossen, eine zweite Studie eingeleitet und arbeitet derzeit mit unserem Team am Boston Children's Hospital an der Planung einer weiteren Studie mit Medikamenten, die wie FTIs spannende Ergebnisse in Progeria-Zellen und Tiermodellen gezeigt haben. Das ist eine beeindruckende Erfolgsbilanz.\u201c<\/p>\n

WIR STIMMEN ZU! <\/strong>Wie kam es zu diesem wundervollen Tag?<\/strong>
Nach der Entdeckung der Genmutation, die Progerie verursacht<\/a>identifizierten PRF-finanzierte Forscher FTI<\/a> als m\u00f6gliche medikament\u00f6se Behandlung. Die Progerie verursachende Mutation f\u00fchrt zur Produktion des Proteins Progerin<\/em>, das die Zellfunktion sch\u00e4digt. Ein Teil der toxischen Wirkung von Progerin auf den K\u00f6rper wird durch ein Molek\u00fcl namens \u201eFarnesylgruppe\u201c verursacht, das sich an das Progerinprotein bindet und dabei hilft, die K\u00f6rperzellen zu sch\u00e4digen. FTIs wirken, indem sie die Bindung der Farnesylgruppe an Progerin blockieren und so den von Progerin verursachten Schaden verringern.<\/p>\n

Weitere Einzelheiten zur Studie finden Sie unter <\/strong>Klicken Sie hier f\u00fcr die Pressemitteilung<\/strong><\/a><\/p>\n

Progerie steht im Zusammenhang mit dem normalen Alterungsprozess<\/strong>
Forschung<\/a> zeigt, dass das Progerie-verursachende Protein Progerin<\/em> wird auch in der Allgemeinbev\u00f6lkerung produziert und nimmt mit dem Alter zu. Forscher planen, die Wirkung von FTIs weiter zu erforschen, was Wissenschaftlern helfen k\u00f6nnte, mehr \u00fcber die Herz-Kreislauf-Erkrankung zu erfahren, die Millionen von Menschen betrifft, sowie \u00fcber den normalen Alterungsprozess, der uns alle betrifft.<\/p>\n

\n

\u201eDie Verbindung dieser seltenen Krankheit mit dem normalen Alterungsprozess tr\u00e4gt wichtige Fr\u00fcchte \u2026 durch die Untersuchung seltener Erkrankungen wie Progerie werden wertvolle biologische Erkenntnisse gewonnen. Wir hatten von Anfang an das Gef\u00fchl, dass Progerie uns viel \u00fcber den normalen Alterungsprozess beibringen kann.\u201c<\/p>\n

\u2013 Dr. Francis Collins, Direktor der National Institutes of Health<\/p>\n<\/blockquote>\n

\"\"

Was f\u00fcr ein L\u00e4cheln! Maria hatte w\u00e4hrend einer ihrer Untersuchungspausen im Boston Children's Hospital viel Spa\u00df beim Malen.<\/strong><\/span><\/p><\/div>\n

Helfen Sie uns, alle Kinder mit Progerie zu finden, damit auch sie von unserer Arbeit profitieren k\u00f6nnen<\/strong>
Forscher gehen davon aus, dass zu jedem Zeitpunkt 200 bis 250 Kinder mit Progerie leben. Um unbekannte Kinder zu identifizieren, startete PRF das \u201eFinde die anderen 150\u201c<\/a> Kampagne im Oktober 2009, und seit September 2012 wissen wir von 96 Kindern, die in 35 L\u00e4ndern leben \u2013 eine Steigerung von 83%!! Sie k\u00f6nnen helfen, mehr zu finden, damit sie von der einzigartigen Behandlung und Betreuung profitieren k\u00f6nnen, die PRF bietet. Diese neuen Kinder k\u00f6nnen f\u00fcr zuk\u00fcnftige klinische Studien in Frage kommen. Gehen Sie daher bitte zu <\/em>Finden Sie die anderen 150<\/em><\/a>\u00a0um herauszufinden, wie Sie dazu beitragen k\u00f6nnen, dass dies geschieht.<\/em><\/p>\n

Vielen Dank an ALLE <\/strong>\u2013<\/strong> Ohne Sie h\u00e4tten wir es nicht geschafft!<\/strong><\/a>
Einer der Hauptgr\u00fcnde, warum wir in diesem ersten Versuch bahnbrechende Ergebnisse erzielt haben, sind die gro\u00dfartigen Unterst\u00fctzer, die uns mit Geldern und anderen Mitteln unterst\u00fctzt haben und uns so geholfen haben, unserem ultimativen Ziel \u2013 einer Heilung f\u00fcr Progerie \u2013 einen Schritt n\u00e4her zu kommen. Klicken Sie hier, um eine besondere Hommage an alle zu sehen, die dazu beigetragen haben, dass die Traum<\/em> einer Behandlung ein Wirklichkeit<\/em><\/strong>.<\/p>\n

[\/et_pb_text][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>","protected":false},"excerpt":{"rendered":"

[et_pb_section fb_built=\u201d1\u2033 _builder_version=\u201d4.16\u2033 global_colors_info=\u201d{}\u201d][et_pb_row _builder_version=\u201d4.16\u2033 background_size=\u201dinitial\u201d background_position=\u201dtop_left\u201d background_repeat=\u201drepeat\u201d global_colors_info=\u201d{}\u201d][et_pb_column type=\u201d4_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text _builder_version=\u201d4.16\u2033 background_size=\u201dinitial\u201d background_position=\u201dtop_left\u201d background_repeat=\u201drepeat\u201d global_colors_info=\u201d{}\u201d] Es wurde Geschichte geschrieben: Jedes Kind in der allerersten klinischen Arzneimittelstudie zu Progerie zeigte eine Verbesserung in einem oder mehreren Bereichen seiner Erkrankung. Dies beweist, dass das FTI-Medikament Lonafarnib die erste bekannte, wirksame Behandlung ist [\u2026]<\/p>","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"

History has been made, with every child in the first-ever Progeria clinical drug trial showing improvement in one or more areas of their condition, proving that the FTI drug lonafarnib is the first known, effective treatment for children with Progeria.<\/p>

Click here for your free copy of the clinical trial results paper<\/strong><\/a><\/p>[caption id=\"\" align=\"alignleft\" width=\"300\"]\"\" Megan and Meghan were the first two children to enroll in the trial. The two girls met in Boston every four months for two years, for testing, to receive new drug supply, and to play together! Here they are in December 2008, on a break from tests at Boston Children\u2019s\u2019 Hospital.<\/strong><\/span>[\/caption]

FOR MEDIA:\u00a0 <\/strong>click here<\/a> for the press release, b-roll and other press details.<\/p>

The results of <\/strong>the first-ever clinical drug trial for children<\/strong><\/a> with Progeria are in and it\u2019s official!<\/strong>\u00a0 Lonafarnib, a type of farnesyltransferase inhibitor (FTI) originally developed to treat cancer, has proven effective for Progeria. Every child showing improvement in one or more of four ways: gaining additional weight, better hearing, improved bone structure and\/or, most importantly, increased flexibility of blood vessels. Results of the study, which was funded and coordinated by The Progeria Research Foundation<\/strong>, were published September 24, 2012 in Proceedings of the National Academy of Sciences<\/a>.<\/em><\/strong><\/p>

Gordon et. al., Clinical Trial of a Farnesyltransferase Inhibitor in Children with Hutchinson-Gilford Progeria Syndrome, PNAS, October 9, 2012 vol. 109 no. 41 16666-16671<\/em><\/p>[caption id=\"\" align=\"alignleft\" width=\"287\"]\"\" Natsuki, from Japan, with her brother, father and mother who prepares the lonafarnib-liquid sweetener mixture.<\/strong><\/span>[\/caption]

Results Yield Improvements in All Children<\/strong>
Twenty-eight children from sixteen countries participated in the 2 \u00bd year drug trial, representing 75 percent of known Progeria cases worldwide at the time the trial began. The children traveled to Boston every four months to receive comprehensive medical testing and study medications through Boston Children\u2019s Hospital\u2019s Clinical and Translational Study Unit<\/a>. All received oral lonafarnib, an FTI supplied by Merck & Co., twice-a-day for two years, under the supervision of clinical trial chair Mark Kieran, M.D., Ph.D.<\/a>, Director of Pediatric Medical Neuro-oncology at the Dana-Farber \/ Children\u2019s Hospital Cancer Center, and co-chairs Dr. Monica Kleinman<\/a> and Dr. Leslie Gordon<\/a><\/p>

Rate of weight gain was the primary outcome measure, because children with Progeria experience severe failure to thrive, with a very slow linear rate of weight gain over time. The researchers examined many other areas of the body, including arterial stiffness (a predictor of heart attack and stroke), bone rigidity (an indicator of bone strength) and hearing. \u201cWhen we started this clinical trial we had no idea whether any aspect of Progeria would be reversible, because no one had ever conducted a clinical treatment trial for Progeria before.\u00a0 We discovered that, among other things, the major blood vessels can actually improve.\u00a0 This was a breakthrough discovery for the children, since accelerated cardiovascular disease is the cause of death in Progeria. Though there is no way to know whether we have delayed strokes, heart attacks, or increased longevity within just a 2-year treatment period, these positive results compel us to continue pushing for new treatments until we accomplish what we set out to do in 1999. We want children with Progeria to live until they\u2019re 80 and beyond. We want them to live full, healthy lives,\u201d said Dr. Gordon, PRF\u2019s Medical Director and first author of the treatment discovery paper.<\/p>

Record Pace of Progress<\/strong><\/p>

The treatment discovery comes less than a decade after PRF and now National Institutes of Health Director Dr. Francis Collins joined forces to identify the cause of Progeria \u2013 an unheard of timeline in the world of medical research!\u00a0 But for PRF and children with Progeria, who live to an average age of just 13 years, such speed is vital to win this race against time.<\/p>[caption id=\"\" align=\"alignleft\" width=\"250\"]\"\" Mateo, Milagros and Fernando are all smiles upon receiving awards from PRF at their final visit. The trophies say, \u201cYOU DID IT! You finished the 1st Progeria Trial \u2013 You\u2019re A SUPERSTAR!\u201d<\/strong><\/span>[\/caption]

\u201cPRF is a good example of an organization successfully enabling translational research, moving from gene discovery to clinical treatment at an unprecedented pace,\u201d said Dr. Kieran. \u201cFrom 1999, when the organization was founded, to today, PRF has identified the genetic mutation that causes the disease, funded preclinical research, completed this trial, initiated a second trial, and is currently working with our team at Boston Children\u2019s Hospital to plan yet another trial with drugs that, like FTIs, have shown exciting results in Progeria cells and animal models. That\u2019s an awesome track record of accomplishment.\u201d
WE AGREE!<\/strong>How Did We Get to This Wonderful Day?<\/strong>
Following the 2003 discovery of the gene mutation that causes Progeria<\/a>, PRF-funded researchers identified FTIs<\/a> as a potential drug treatment. The Progeria-causing mutation leads to the production of the protein progerin<\/em>, which damages cell function. Part of progerin\u2019s toxic effect on the body is caused by a molecule called a \u201cfarnesyl group,\u201d which attaches to the progerin protein and helps it damage the body\u2019s cells. FTIs act by blocking the attachment of the farnesyl group onto progerin, reducing the harm progerin causes.<\/p>

For more study details, <\/strong>CLICK HERE for the press release<\/strong><\/a><\/p>

Progeria Linked to Normal Aging Process<\/strong>
Research<\/a> shows that the Progeria-causing protein progerin<\/em> is also produced in the general population and increases with age. Researchers plan to continue exploring the effect of FTIs, which may help scientists learn more about the cardiovascular disease that affects millions, as well as the normal aging process that affects us all.<\/p>

\"Connecting this rare disease and normal aging is bearing fruit in an important way...valuable biological insights are gained by studying rare disorders such as Progeria. Our sense from the start was that Progeria had a lot to teach us about the normal aging process.\"<\/p>

- Dr. Francis Collins, Director of the National Institutes of Health<\/p><\/blockquote>[caption id=\"\" align=\"alignright\" width=\"350\"]\"\" What a smile! Maria really enjoyed painting during one of her breaks from testing at Boston Children\u2019s Hospital.<\/strong><\/span>[\/caption]

Help Us Find All Children With Progeria so They, Too, Can Benefit From Our Work<\/strong>
Researchers believe that at any given time, there are 200-250 children living with Progeria. To identify unknown children, PRF launched the \u201cFind the Other 150\u201d<\/a> campaign in October 2009,\u00a0and as of September 2012, we know of 96 children living in 35 countries- an 83% increase!! You can help find more so they can benefit from the unique treatment and care that PRF provides. These new children may be eligible for future clinical trials, so please go to <\/em>Find the other 150<\/em><\/a>\u00a0to find out how you can help make that happen.<\/em><\/p>

What a smile! Maria really enjoyed painting during one
of her breaks from testing at Boston Children\u2019s Hospital.<\/strong><\/span><\/p>

Thank you ALL <\/strong>-<\/strong> We Couldn\u2019t Have Done it Without You!<\/strong><\/a>
One of the main reasons we achieved breakthrough results in this first trial is because of the tremendous supporters who provided funding and other support, helping to get us one step closer to achieving our ultimate goal \u2013 a cure for Progeria. Click here to see a special tribute to all those who helped make the dream<\/em> of a treatment a reality<\/em><\/strong>.<\/p>","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"class_list":["post-1684","page","type-page","status-publish","hentry"],"yoast_head":"\ntreatment | 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\/first-ever-progeria-treatment\/\" \/>\n<meta property=\"og:locale\" content=\"de_DE\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"treatment | The Progeria Research Foundation\" \/>\n<meta property=\"og:description\" content=\"[et_pb_section fb_built=”1″ _builder_version=”4.16″ global_colors_info=”{}”][et_pb_row _builder_version=”4.16″ background_size=”initial” background_position=”top_left” 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