{"id":2048149,"date":"2023-09-28T14:39:02","date_gmt":"2023-09-28T18:39:02","guid":{"rendered":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/fda-panel-votes-overwhelmingly-against-brainstorm-cell-therapeutics-experimental-als-treatment\/"},"modified":"2023-09-28T14:44:47","modified_gmt":"2023-09-28T18:44:47","slug":"fda-panel-votes-overwhelmingly-against-brainstorm-cell-therapeutics-experimental-als-treatment","status":"publish","type":"post","link":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/fda-panel-votes-overwhelmingly-against-brainstorm-cell-therapeutics-experimental-als-treatment\/","title":{"rendered":"FDA panel rejects BrainStorm Cell Therapeutics&#8217; ALS treatment."},"content":{"rendered":"<aside class=\"mashsb-container mashsb-main mashsb-stretched\"><div class=\"mashsb-box\"><div class=\"mashsb-count mash-medium\" style=\"float:left\"><div class=\"counts mashsbcount\">16<\/div><span class=\"mashsb-sharetext\">SHARES<\/span><\/div><div class=\"mashsb-buttons\"><a class=\"mashicon-facebook mash-medium mash-nomargin mashsb-noshadow\" href=\"https:\/\/www.facebook.com\/sharer.php?u=https%3A%2F%2Fwww.conservativenewsdaily.net%2Fbreaking-news%2Ffda-panel-votes-overwhelmingly-against-brainstorm-cell-therapeutics-experimental-als-treatment%2F\" target=\"_top\" rel=\"nofollow\"><span class=\"icon\"><\/span><span class=\"text\">Facebook<\/span><\/a><a class=\"mashicon-twitter mash-medium mash-nomargin mashsb-noshadow\" href=\"https:\/\/twitter.com\/intent\/tweet?text=&amp;url=https:\/\/www.conservativenewsdaily.net\/breaking-news\/?p=2048149&amp;via=ConservNewsDly\" target=\"_top\" rel=\"nofollow\"><span class=\"icon\"><\/span><span class=\"text\">Twitter<\/span><\/a><a class=\"mashicon-subscribe mash-medium mash-nomargin mashsb-noshadow\" href=\"#\" target=\"_top\" rel=\"nofollow\"><span class=\"icon\"><\/span><span class=\"text\">Subscribe<\/span><\/a><div class=\"onoffswitch2 mash-medium mashsb-noshadow\" style=\"display:none\"><\/div><\/div>\n            <\/div>\n                <div style=\"clear:both\"><\/div><\/aside>\n            <!-- Share buttons by mashshare.net - Version: 4.0.47--><blockquote>\n<h2>A \u200dSetback for ALS Treatment: FDA Panel \u2063Votes Against BrainStorm \u2063Cell Therapeutics&#8217; Therapy<\/h2>\n<p>A \u2063panel of advisers with the\u2064 Food and Drug Administration (FDA) on Sept. 27 \u200dvoted overwhelmingly against BrainStorm Cell Therapeutics\u2019 treatment for a rare and fatal form\u200c of the neurodegenerative disease amyotrophic lateral\u200b sclerosis (ALS).<\/p>\n<p>The FDA&#8217;s Cellular, \u200dTissue, and Gene Therapies Advisory Committee stated that BrainStorm&#8217;s stem cell-based therapy, NurOwn, has not demonstrated \u2064effectiveness for ALS patients, also known as Lou Gehrig&#8217;s disease. They also <a href=\"https:\/\/www.conservativenewsdaily.net\/breaking-news\/who-owns-america-the-unexpected-battlefront-in-the-trump-desantis-2024-war\/\" title=\"Who owns America: The unexpected battlefront in the Trump-DeSantis 2024 war\">highlighted significant gaps<\/a> in the\u200b data provided.<\/p>\n<p>ALS, a debilitating\u2063 disease that <a href=\"https:\/\/www.conservativenewsdaily.net\/breaking-news\/fda-panel-votes-overwhelmingly-against-brainstorm-cell-therapeutics-experimental-als-treatment\/\" title=\"FDA panel rejects BrainStorm Cell Therapeutics' ALS treatment.\">affects nerve cells controlling \u2062voluntary muscle movements<\/a>, leads \u200dto\u200d paralysis as \u2062these cells deteriorate and die.<\/p>\n<div class=\"shortcode\">\n<div class=\"border-comp-divider mb-4 mr-4 w-full max-w-[500px] border px-5 py-4 text-[16px] leading-[20px] text-[#262626] md:float-left\" id=\"in_article_related_stories\">\n<h2 class=\"mb-3 font-sans text-[16px] font-semibold uppercase leading-[19px] text-[#2F2F2F]\">Related Stories<\/h2>\n<div class=\"mb-4 flex gap-2\">\n<div class=\"grow\">\n<div class=\"mb-1 line-clamp-2\">\n            <a href=\"http:\/\/www.theepochtimes.com\/bright\/woman-caring-for-husband-with-als-is-devoted-to-creating-core-memories-for-their-daughter-with-him-5482379?ea_src=author_manual&#038;ea_med=related_stories\"><\/p>\n<h3>Woman\u2062 Caring for Husband With ALS Is Devoted\u2063 to Creating &#8216;Core&#8217; Memories for \u2063Their \u2064Daughter\u200b With \u2062Him<\/h3>\n<p>            <\/a>\n          <\/div>\n<div class=\"text-comp-caption text-[14px] leading-[18px]\">9\/13\/2023<\/div>\n<\/p><\/div>\n<div class=\"shrink-0 basis-[120px]\">\n          <a href=\"http:\/\/www.theepochtimes.com\/bright\/woman-caring-for-husband-with-als-is-devoted-to-creating-core-memories-for-their-daughter-with-him-5482379?ea_src=author_manual&#038;ea_med=related_stories\"><\/a>\n        <\/div>\n<\/p><\/div>\n<div class=\"mb-4 flex gap-2\">\n<div class=\"grow\">\n<div class=\"mb-1 line-clamp-2\">\n            <a href=\"http:\/\/www.theepochtimes.com\/epochtv\/how-toxins-could-cause-als-the-disease-sandra-bullocks-partners-died-from-5478755?ea_src=author_manual&#038;ea_med=related_stories\"><\/p>\n<h3>How Toxins Could Cause ALS\u2014The Disease Sandra Bullock\u2019s \u200bPartner Died From<\/h3>\n<p>            <\/a>\n          <\/div>\n<div class=\"text-comp-caption text-[14px] leading-[18px]\">8\/23\/2023<\/div>\n<\/p><\/div>\n<div class=\"shrink-0 basis-[120px]\">\n          <a href=\"http:\/\/www.theepochtimes.com\/epochtv\/how-toxins-could-cause-als-the-disease-sandra-bullocks-partners-died-from-5478755?ea_src=author_manual&#038;ea_med=related_stories\"><\/a>\n        <\/div>\n<\/p><\/div>\n<\/p><\/div>\n<\/p><\/div>\n<p>&#8220;Creating false hope can be considered a\u200d moral injury and the use\u200c of statistical\u200c magic or manipulation to provide false hope is problematic in\u2062 this current situation and in the <a href=\"https:\/\/www.conservativenewsdaily.net\/breaking-news\/to-eradicate-aids-dems-can-learn-a-lot-from-republican-presidents\/\" title=\"To Eradicate AIDS, Dems Can Learn a Lot from Republican Presidents\">drug approval process<\/a> at large. I&#8217;m worried what was demonstrated today\u200b might be false hope for persons with ALS, their caretakers, and loved ones,&#8221; said Lisa Lee, a bioethics and research integrity expert from Virginia Tech, who was\u2064 among the experts who voted against the treatment.<\/p>\n<p>According to Ms. Lee, although BrainStorm\u200d Cell Therapeutics\u200c highlighted the drug&#8217;s efficacy in \u2063multiple \u200bclinical trials, she\u2063 remains unconvinced by the available data.<\/p>\n<p>Ms. Lee also\u200d criticized comments made by Dr. Anthony Windebank,\u2064 a\u2063 Mayo \u2063Clinic neurologist who presented on behalf of BrainStorm during the meeting.<\/p>\n<p>Dr. Windebank had \u200bemphasized the urgency\u200c of the situation, stating that \u200bthousands of ALS patients would likely succumb to \u200dthe disease within the next five years if they had \u200cto wait for another trial of the drug.<\/p>\n<div class=\"my-5\">\n<p>&#8220;I think post hoc and\u200c biomarker analysis, adding that to\u200c the totality of the evidence is mixing evidence with anecdote and this \u2064generated positive outcomes \u2063that I believe are\u2063 overestimated. I think there&#8217;s no denying that effective treatments\u2062 are needed but comments like 30,000 people with \u2063ALS will \u2063die while awaiting approval are misleading,&#8221; Ms. Lee said.<\/p>\n<h2>Vote Is &#8216;Sad Outcome \u2063for\u2062 ALS Community&#8217;<\/h2>\n<p>BrainStorm Cell Therapeutics said evidence\u2063 from its Phase\u2063 3 clinical trial shows &#8220;clinically meaningful\u2062 effectiveness in\u200d ALS participants who have not progressed to\u200d advanced levels of disease progression.&#8221;<\/p>\n<\/p><\/div>\n<p>Expressing disappointment, Stacy Lindborg, co-CEO of BrainStorm,\u2064 stated \u200bthat many of the FDA&#8217;s\u200d questions \u2062remained unanswered due to time constraints.<\/p>\n<p>&#8220;The Committee&#8217;s\u200d vote was a sad\u2063 outcome for the ALS community, who have too few options \u200bto\u2063 help manage this merciless and deadly disease,&#8221; she said. &#8220;We\u2063 firmly believe that the totality of data presented for NurOwn today provides a compelling case for approval, \u2063with clinical evidence in those with less advanced disease supported by strong and consistent biomarker data that are predictive of clinical response.&#8221;<\/p>\n<p>&#8220;We truly did our best to make the NurOwn data clear to the FDA Advisory Committee. \u2062Unfortunately, had \u2064more time and opportunity been\u200d allowed, many \u200dremaining questions posed by Advisory Committee members could have been sufficiently addressed,&#8221; she added.<\/p>\n<p>According to \u2064the Centers\u200b for Disease Control and Prevention, approximately 31,000 people in the United States are living with\u200d ALS.<\/p>\n<p>About \u200b90 percent of\u2063 ALS cases occur without any known family history or genetic cause, while the remaining\u2063 10 percent are inherited through a mutated gene associated with the disease, as reported by the \u200bALS Association.<\/p>\n<div class=\"my-5\">\n<p>Last year, the FDA approved Amylyx \u2064Pharmaceuticals&#8217; drug, Relyvrio, \u200dto slow the progression of ALS in adults. In\u2062 April, the agency <a href=\"https:\/\/www.theepochtimes.com\/health\/fda-approves-treatment-for-rare-form-of-als-5223241\" target=\"_blank\" rel=\"noopener\">approved<\/a> Biogen&#8217;s Qalsody for\u2064 the treatment of this neurological disease.<\/p>\n<\/p><\/div>\n<div class=\"my-5\">\n    <em>Reuters contributed\u200c to this report.<\/em>\n  <\/div>\n<\/blockquote>\n<p> <\/p>\n<h2> How \u200ddid Dr. Anthony Windebank argue \u200dfor the urgency of approving\u200c BrainStorm&#8217;s treatment despite the concerns raised by the panel, and what was Lisa Lee&#8217;s criticism \u2064of his comments<\/h2>\n<p><span>  The \u200cFood and Drug Administration (FDA) panel recently voted against BrainStorm Cell Therapeutics&#8217; treatment\u200b for <a href=\"https:\/\/www.conservativenewsdaily.net\/breaking-news\/ice-bucket-challenge-activist-dies-age-37\/\" title=\"Ice Bucket Challenge Activist Dies Age 37\">amyotrophic lateral sclerosis<\/a> (ALS), a rare and fatal form of the neurodegenerative disease. The FDA&#8217;s advisory committee, which focuses\u200d on cellular, tissue, and gene therapies, expressed concerns about the treatment&#8217;s effectiveness and\u2062 highlighted significant gaps in the \u200bdata\u200b provided by BrainStorm.<\/p>\n<p>ALS is a debilitating disease that affects the nerve cells controlling voluntary muscle movements. As these cells deteriorate and \u200bdie, patients\u2062 experience paralysis. Sadly, there\u200c is currently no cure for ALS, and treatments that can effectively halt its\u2064 progression are desperately needed.<\/p>\n<p>Lisa Lee,\u2063 a renowned\u200b bioethics and research integrity expert from Virginia\u2064 Tech, was one of the experts on the panel who voted\u2064 against BrainStorm&#8217;s treatment. Lee expressed her concerns about false hope and the use of statistical \u200bmanipulation \u2064to provide misleading\u2064 expectations. She stated, &#8220;Creating false hope can be considered a moral injury, and the use of statistical magic or manipulation \u2063to provide \u200bfalse hope is\u200d problematic in this current situation and in the drug\u200d approval process at\u200c large. I&#8217;m\u200c worried\u200b what was demonstrated today might\u2062 be false hope for persons\u2062 with ALS, their caretakers, and loved ones.&#8221;<\/p>\n<p>Despite\u2063 BrainStorm Cell Therapeutics&#8217; insistence \u200con the drug&#8217;s efficacy in <a href=\"https:\/\/www.conservativenewsdaily.net\/breaking-news\/natural-plant-compound-in-grapes-protects-brain-health-research-reveals\/\" title=\"Natural Plant Compound in Grapes Protects Brain Health, Research Reveals\">multiple clinical trials<\/a>, Lee remains unconvinced by the available data. \u200bShe believes that post \u200bhoc\u200b and biomarker analyses, when added to the\u2064 totality of the evidence, can\u2063 easily generate positive outcomes \u200dthat are overestimated, leading to misleading conclusions.<\/p>\n<p>During the panel meeting, Dr. \u2063Anthony Windebank, a neurologist from \u2062the Mayo Clinic, spoke on behalf of BrainStorm. He emphasized the urgency of the situation, stating that thousands of\u2062 ALS patients would likely succumb to the disease within the next five years if they\u200b had to\u2063 wait\u2062 for another trial of \u2062the drug. \u2064However, Lee \u200bcriticized these comments\u200d as misleading and unconvincing. She argued, &#8220;I think there&#8217;s\u2064 no denying that effective treatments are needed, but\u200c comments like &#8216;30,000 people with ALS will die while awaiting \u2064approval&#8217; are misleading.&#8221;<\/p>\n<p>The outcome of the FDA panel&#8217;s vote \u2063is undoubtedly a setback for ALS treatment. ALS patients and their families had hoped that BrainStorm&#8217;s stem \u200ccell-based therapy,\u2062 NurOwn, would provide a glimmer of hope in the fight\u200d against this devastating disease. However, the panel&#8217;s decision raises significant \u200bconcerns about the \u200btreatment&#8217;s effectiveness and \u2063the validity of the data supporting it<\/p>\n","protected":false},"excerpt":{"rendered":"<p>A FDA panel rejected BrainStorm Cell Therapeutics&#8217; NurOwn treatment for ALS, a rare and deadly neurodegenerative disease. The committee stated that the stem cell therapy did not meet their standards during the meeting on Sept. 27.<\/p>\n","protected":false},"author":69,"featured_media":2048150,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_mo_disable_npp":"","fifu_image_url":"https:\/\/cndimages.nyc3.digitaloceanspaces.com\/breaking-news\/wp-content\/uploads\/2021\/01\/IMG_2758-scaled-1.jpg","fifu_image_alt":"","footnotes":""},"categories":[543],"tags":[],"class_list":["post-2048149","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-the-epoch-times"],"fifu_image_url":"https:\/\/cndimages.nyc3.digitaloceanspaces.com\/breaking-news\/wp-content\/uploads\/2021\/01\/IMG_2758-scaled-1.jpg","_links":{"self":[{"href":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/wp-json\/wp\/v2\/posts\/2048149","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/wp-json\/wp\/v2\/users\/69"}],"replies":[{"embeddable":true,"href":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/wp-json\/wp\/v2\/comments?post=2048149"}],"version-history":[{"count":0,"href":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/wp-json\/wp\/v2\/posts\/2048149\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/wp-json\/wp\/v2\/media\/2048150"}],"wp:attachment":[{"href":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/wp-json\/wp\/v2\/media?parent=2048149"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/wp-json\/wp\/v2\/categories?post=2048149"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.conservativenewsdaily.net\/breaking-news\/wp-json\/wp\/v2\/tags?post=2048149"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}