US clears first ‘living drug’ for childhood leukaemia

&Tab;&Tab;<div class&equals;"wpcnt">&NewLine;&Tab;&Tab;&Tab;<div class&equals;"wpa">&NewLine;&Tab;&Tab;&Tab;&Tab;<span class&equals;"wpa-about">Advertisements<&sol;span>&NewLine;&Tab;&Tab;&Tab;&Tab;<div class&equals;"u top&lowbar;amp">&NewLine;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;<amp-ad width&equals;"300" height&equals;"265"&NewLine;&Tab;&Tab; type&equals;"pubmine"&NewLine;&Tab;&Tab; data-siteid&equals;"111265417"&NewLine;&Tab;&Tab; data-section&equals;"1">&NewLine;&Tab;&Tab;<&sol;amp-ad>&NewLine;&Tab;&Tab;&Tab;&Tab;<&sol;div>&NewLine;&Tab;&Tab;&Tab;<&sol;div>&NewLine;&Tab;&Tab;<&sol;div><p>US officials have approved the first treatment that genetically engineers patients&&num;8217&semi; own blood cells to seek and destroy a childhood leukaemia&period;<&sol;p>&NewLine;<p>The CAR-T cell treatment developed by Novartis and the University of Pennsylvania is the first type of gene therapy to hit the US market &&num;8211&semi; and one in a powerful but expensive wave of custom-made &&num;8220&semi;living drugs&&num;8221&semi; being tested against blood cancers and some other tumors&period;<&sol;p>&NewLine;<p>The Food and Drug Administration called the approval historic&period;<&sol;p>&NewLine;<p><i>&&num;8220&semi;This is a brand new way of treating cancer&comma;&&num;8221&semi;<&sol;i> said Dr Stephan Grupp of Children&&num;8217&semi;s Hospital of Philadelphia&comma; who treated the first child with CAR-T cell therapy &&num;8211&semi; a girl who had been close to death&comma; but now is cancer-free for five years and counting&period;<&sol;p>&NewLine;<p><i>&&num;8220&semi;That&&num;8217&semi;s enormously exciting&period;&&num;8221&semi;<&sol;i><&sol;p>&NewLine;<p>CAR-T treatment uses gene therapy techniques not to fix disease-causing genes&comma; but to &&num;8220&semi;turbo-charge&&num;8221&semi; T cells&comma; immune system &&num;8220&semi;soldiers&&num;8221&semi; that cancer can often evade&period;<&sol;p>&NewLine;<p>Researchers filter the cells from a patient&&num;8217&semi;s blood&comma; reprogramme them with a &&num;8220&semi;chimeric antigen receptor&&num;8221&semi; that targets cancer&comma; and grow hundreds of millions of copies&period;<&sol;p>&NewLine;<p>Returned to the patient&comma; the cells can continue multiplying to fight disease for months or years&period;<&sol;p>&NewLine;<p>Novartis did not immediately disclose the therapy&&num;8217&semi;s price&comma; but it is expected to cost hundreds of thousands of dollars&period; It is made from scratch for every patient&period;<&sol;p>&NewLine;<p>&&num;8220&semi;We&&num;8217&semi;re entering a new frontier in medical innovation with the ability to reprogramme a patient&&num;8217&semi;s own cells to attack a deadly cancer&comma;&&num;8221&semi;<&sol;i> said FDA commissioner Scott Gottlieb&period;<&sol;p>&NewLine;<p>This first use of CAR-T therapy is aimed at patients desperately ill with acute lymphoblastic leukaemia&comma; which strikes more than 3&comma;000 children and young adults in the US each year&period;<&sol;p>&NewLine;<p>While most survive&comma; about 15&percnt; relapse despite the current best treatments&comma; and their prognosis is bleak&period;<&sol;p>&NewLine;<p>In a key study of 63 advanced patients&comma; 83&percnt; went into remission&comma; although it is not clear how long the benefit lasts&colon; Some patients did relapse months later&period;<br &sol;>&NewLine;The others still are being tracked to see how they fare long-term&period;<&sol;p>&NewLine;&Tab;&Tab;&Tab;<div style&equals;"padding-bottom&colon;15px&semi;" class&equals;"wordads-tag" data-slot-type&equals;"belowpost">&NewLine;&Tab;&Tab;&Tab;&Tab;<div id&equals;"atatags-dynamic-belowpost-68ed360439f4e">&NewLine;&Tab;&Tab;&Tab;&Tab;&Tab;<script type&equals;"text&sol;javascript">&NewLine;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;window&period;getAdSnippetCallback &equals; function &lpar;&rpar; &lbrace;&NewLine;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;if &lpar; false &equals;&equals;&equals; &lpar; window&period;isWatlV1 &quest;&quest; false &rpar; &rpar; &lbrace;&NewLine;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;&sol;&sol; Use Aditude scripts&period;&NewLine;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;&Tab;window&period;tudeMappings &equals; 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