{"id":12928,"date":"2023-11-07T13:12:51","date_gmt":"2023-11-07T17:12:51","guid":{"rendered":"https:\/\/afcomunicacion.com\/uncategorized\/cleveland-clinic-performs-first-in-human-gene-therapy-for-hypertrophic-cardiomyopathy\/"},"modified":"2023-11-07T13:12:51","modified_gmt":"2023-11-07T17:12:51","slug":"cleveland-clinic-performs-first-in-human-gene-therapy-for-hypertrophic-cardiomyopathy","status":"publish","type":"post","link":"https:\/\/afcomunicacion.com\/en\/press-room\/cleveland-clinic-performs-first-in-human-gene-therapy-for-hypertrophic-cardiomyopathy\/","title":{"rendered":"Cleveland Clinic performs first-in-human gene therapy for hypertrophic cardiomyopathy"},"content":{"rendered":"\n<ul class=\"wp-block-list\">\n<li><em>The patient in this clinical trial, a 27-year-old woman, received a dose and is doing well.<\/em><\/li>\n<\/ul>\n\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\"><strong>October 2023, Cleveland: <mark style=\"color:#138c00\" class=\"has-inline-color\">Cleveland Clinic<\/mark><\/strong> has administered a new gene therapy to deliver a working gene that addresses the main cause of <strong><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">hypertrophic cardiomyopathy (HCM)<\/mark><\/strong> to the world&#8217;s first patient as part of a clinical trial.<\/p>\n\n\n<div class=\"wp-block-image\">\n<figure class=\"alignleft size-large is-resized\"><img loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"683\" src=\"https:\/\/afcomunicacion.com\/wp-content\/uploads\/2023\/11\/Danielle-Kellner-coordinadora-de-enfermeria-de-la-investigacion-1024x683.jpg\" alt=\"Danielle Kellner, research nurse coordinator\" class=\"wp-image-8504\" style=\"aspect-ratio:1.499267935578331;width:298px;height:auto\" srcset=\"https:\/\/afcomunicacion.com\/wp-content\/uploads\/2023\/11\/Danielle-Kellner-coordinadora-de-enfermeria-de-la-investigacion-1024x683.jpg 1024w, https:\/\/afcomunicacion.com\/wp-content\/uploads\/2023\/11\/Danielle-Kellner-coordinadora-de-enfermeria-de-la-investigacion-300x200.jpg 300w, https:\/\/afcomunicacion.com\/wp-content\/uploads\/2023\/11\/Danielle-Kellner-coordinadora-de-enfermeria-de-la-investigacion-768x512.jpg 768w, https:\/\/afcomunicacion.com\/wp-content\/uploads\/2023\/11\/Danielle-Kellner-coordinadora-de-enfermeria-de-la-investigacion-1536x1024.jpg 1536w, https:\/\/afcomunicacion.com\/wp-content\/uploads\/2023\/11\/Danielle-Kellner-coordinadora-de-enfermeria-de-la-investigacion-2048x1366.jpg 2048w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><figcaption class=\"wp-element-caption\">Danielle Kellner, research nurse coordinator<\/figcaption><\/figure>\n<\/div>\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\">Mutations in the <strong><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">myosin-binding protein C3 (MYBPC3)<\/mark><\/strong> gene are the most common genetic cause of <strong><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">HCM<\/mark><\/strong>. The gene therapy is designed to deliver a working <mark style=\"color:#0b5d8c\" class=\"has-inline-color\"><strong>MYBPC3<\/strong><\/mark> gene to the heart muscle through a one-time infusion of <strong><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">TN-201,<\/mark><\/strong> a first-in-class adeno-associated virus-based gene therapy. The hope is that this new gene will restore normal levels of the protein, which regulates the contraction and relaxation of heart muscle. In preclinical studies, a single dose restored normal protein levels, leading to improvement of the disease.<\/p>\n\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\"><mark style=\"color:#0b5d8c\" class=\"has-inline-color\"><strong>Hypertrophic cardiomyopathy<\/strong> is a complex type of heart disease that causes thickening of the heart muscle, stiffening of the left ventricle and changes to the mitral valve.<\/mark> It is estimated to affect between 600,000 and 1.5 million Americans, or one in 500 people, but many of these patients are not diagnosed until the disease has progressed.<\/p>\n\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\">The cause of <strong><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">hypertrophic cardiomyopathy<\/mark><\/strong> may be unknown or attributed to genetics, high blood pressure or ageing, making it difficult to identify a high-risk population. Symptoms include chest pain, palpitations, shortness of breath, fatigue and syncope (fainting). Most people with <strong><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">hypertrophic cardiomyopathy<\/mark><\/strong> have a low risk of sudden cardiac death. However, the condition is the most common cause of sudden cardiac death in people under 30.<\/p>\n\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\"><em>&#8220;Our understanding of HCM has advanced, and we are excited to be part of combining that knowledge with technology such as gene therapy to advance even further,&#8221;<\/em> <strong><mark style=\"color:#138c00\" class=\"has-inline-color\">said Milind Desai, M.D., M.B.A., director of the Hypertrophic Cardiomyopathy Center at Cleveland Clinic and vice chair of Cleveland Clinic&#8217;s Heart, Vascular &amp; Thoracic Institute.<\/mark><\/strong> <strong><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">Dr. Desai is an investigator in the phase 1b MyPeak-1 clinical trial.<\/mark><\/strong> <em>&#8220;We look forward to continuing to study this therapy in a patient population that is often not properly diagnosed and can suffer debilitating symptoms and sudden death.&#8221;<\/em><\/p>\n\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\"><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">Currently, there is only one specific medicine to treat hypertrophic cardiomyopathy. <\/mark>Mavacamten, a medicine approved by the FDA in April 2022 following a trial led by <strong><mark style=\"color:#138c00\" class=\"has-inline-color\">Cleveland Clinic<\/mark><\/strong>, is used to treat the obstructive form of HCM and reduce the need for invasive procedures. <mark style=\"color:#0b5d8c\" class=\"has-inline-color\">Surgical options include septal myectomy, in which a surgeon removes a small amount of the thickened septal wall to widen the outflow tract (the path blood takes) from the left ventricle to the aorta. <\/mark>Another option is alcohol septal ablation, a cardiac catheterisation procedure in which a small amount of pure alcohol is injected into the septum, causing it to shrink to a more normal size and widen the path for blood flow. However, few high-volume centres perform these procedures, which can limit patient access and require repeat interventions.<\/p>\n\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\">The patient in this clinical trial, a 27-year-old woman, received a dose and is doing well. The <strong><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">phase 1b MyPeak-1 clinical trial <\/mark><\/strong>is a multicentre study designed to assess the safety and clinical efficacy of this one-time infusion. The trial aims to enrol at least six symptomatic adults who have been diagnosed with non-obstructive MYBPC3-associated HCM and who have implantable cardioverter defibrillators.<\/p>\n\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\"><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">The trial is sponsored by <strong>Tenaya Therapeutics Inc.<\/strong><\/mark><\/p>\n\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\"><mark style=\"color:#0b5d8c\" class=\"has-inline-color\">Dr. Desai is a paid consultant for <strong>Tenaya Therapeutics Inc. and Bristol Myers Squibb.<\/strong><\/mark><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\" \/>\n\n\n\n<p class=\"wp-block-paragraph\"><img loading=\"lazy\" decoding=\"async\" width=\"300\" height=\"47\" src=\"https:\/\/afcomunicacion.com\/wp-content\/uploads\/2022\/03\/Cleveland_Clinic_logo_color-300x47.png\" alt=\"\"><\/p>\n\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\"><strong>About Cleveland Clinic<\/strong><\/p>\n\n\n\n<p class=\"has-text-align-justify has-black-color has-text-color wp-block-paragraph\"><strong><a href=\"https:\/\/my.clevelandclinic.org\"><mark style=\"color:#138c00\" class=\"has-inline-color\">Cleveland Clinic<\/mark><\/a><\/strong> is a nonprofit multispecialty academic medical centre that integrates clinical and hospital care with research and education. Located in <strong><mark style=\"color:#138c00\" class=\"has-inline-color\">Cleveland<\/mark><\/strong>, Ohio, it was founded in 1921 by four renowned physicians with a vision of providing outstanding patient care based on the principles of cooperation, compassion and innovation. <strong><mark style=\"color:#138c00\" class=\"has-inline-color\">Cleveland Clinic<\/mark><\/strong> has pioneered many medical breakthroughs, including coronary artery bypass surgery and the first face transplant in the United States. &nbsp;<\/p>\n\n\n\n<p class=\"has-text-align-justify has-black-color has-text-color wp-block-paragraph\"><em>U.S. News &amp; World Report<\/em> consistently names <strong><mark style=\"color:#138c00\" class=\"has-inline-color\">Cleveland Clinic<\/mark><\/strong> as one of the nation&#8217;s best hospitals in its annual &#8220;America&#8217;s Best Hospitals&#8221; survey. Among <strong><mark style=\"color:#138c00\" class=\"has-inline-color\">Cleveland Clinic&#8217;s<\/mark><\/strong> 72,500 employees worldwide are more than 5,050 salaried physicians and researchers, and 17,800 registered nurses and advanced practice providers, representing 140 medical specialties and subspecialties. &nbsp;<strong><mark style=\"color:#138c00\" class=\"has-inline-color\">Cleveland Clinic<\/mark><\/strong> is a 6,500-bed health system that includes a 173-acre main campus near downtown Cleveland, 21 hospitals, more than 220 outpatient facilities, and locations in northeast Ohio; southeast Florida; Las Vegas, Nevada; Toronto, Canada; Abu Dhabi, UAE; and London, England. <\/p>\n\n\n\n<p class=\"has-text-align-justify has-black-color has-text-color wp-block-paragraph\">In 2021, there were 10.2 million outpatient visits, 304,000 hospital admissions and observations, and 259,000 surgeries across <strong><mark style=\"color:#138c00\" class=\"has-inline-color\">Cleveland Clinic&#8217;s<\/mark><\/strong> health system. Patients came for treatment from every state and 185 countries. &nbsp;<\/p>\n\n\n\n<p class=\"has-text-align-justify has-black-color has-text-color wp-block-paragraph\">Visit us at <mark style=\"color:#138c00\" class=\"has-inline-color\"><span style=\"text-decoration: underline\">clevelandclinic.org<\/span><\/mark>. Follow us at twitter.com\/<mark style=\"color:#138c00\" class=\"has-inline-color\"><span style=\"text-decoration: underline\">ClevelandClinic<\/span><\/mark>. News and resources available at<mark style=\"color:#138c00\" class=\"has-inline-color\"><span style=\"text-decoration: underline\"> newsroom.clevelandclinic.org<\/span><\/mark>.<\/p>\n\n\n\n<p class=\"has-black-color has-text-color wp-block-paragraph\"><em>Find out more about <mark><a href=\"https:\/\/afcomunicacion.com\/nosotros\/\"><mark style=\"background-color:#ffffff;color:#0b5d8c\" class=\"has-inline-color\"><span style=\"text-decoration: underline\">AF Comunicaci\u00f3n Estrat\u00e9gica<\/span><\/mark><\/a><mark style=\"background-color:#ffffff\" class=\"has-inline-color\"><a href=\"https:\/\/afcomunicacion.com\/nosotros\/\"> <\/a>and&nbsp;<\/mark><a href=\"https:\/\/afcomunicacion.com\/nuestros-clientes\/\"><mark style=\"background-color:#ffffff;color:#0b5d8c\" class=\"has-inline-color\"><span style=\"text-decoration: underline\">its clients<\/span><\/mark><\/a><\/mark><\/em><\/p>\n","protected":false},"excerpt":{"rendered":"<p>October 2023, Cleveland: Cleveland Clinic has administered a new gene therapy to deliver a working gene that addresses the main cause of hypertrophic cardiomyopathy (HCM) to the world&#8217;s first patient as part of a clinical trial. Mutations in the myosin-binding protein C3 (MYBPC3) gene are the most common genetic cause of HCM. The gene therapy [&hellip;]<\/p>\n","protected":false},"author":12,"featured_media":8504,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[486],"tags":[3291,3292,3293,3294,3295,3296,3297],"class_list":["post-12928","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-press-room","tag-1b-mypeak-1-en","tag-c3-fijadora-de-miosina-mybpc3-en","tag-desfibriladores-cardioversiones-en","tag-fda-2-en","tag-miocardiopatia-hipertrofica-mch-en","tag-miotomia-septal-en","tag-tn-201-en"],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v28.6 (Yoast SEO v28.6) - https:\/\/yoast.com\/product\/yoast-seo-premium-wordpress\/ -->\n<title>Cleveland Clinic performs first-in-human gene therapy for hypertrophic cardiomyopathy<\/title>\n<meta name=\"description\" content=\"Cleveland Clinic has administered a new gene therapy to deliver a working gene to 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