{"id":120684,"date":"2026-11-09T00:21:10","date_gmt":"2026-11-08T23:21:10","guid":{"rendered":"https:\/\/www.infokuryr.cz\/n\/?p=120684"},"modified":"2026-11-08T14:58:08","modified_gmt":"2026-11-08T13:58:08","slug":"nejprve-manipulace-s-daty-nyni-pripady-rakoviny-genova-terapie-novartis-pod-drobnohledem-fda","status":"publish","type":"post","link":"https:\/\/www.infokuryr.cz\/n\/2026\/11\/09\/nejprve-manipulace-s-daty-nyni-pripady-rakoviny-genova-terapie-novartis-pod-drobnohledem-fda\/","title":{"rendered":"Nejprve manipulace s daty, nyn\u00ed p\u0159\u00edpady rakoviny: Genov\u00e1 terapie Novartis pod drobnohledem FDA!"},"content":{"rendered":"
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N\u00e1dory u dvou d\u011bt\u00ed p\u0159\u00edmo souvisej\u00ed s genovou terapi\u00ed vyvinutou spole\u010dnost\u00ed Novartis, jak vypl\u00fdv\u00e1 z vy\u0161et\u0159ov\u00e1n\u00ed americk\u00e9ho \u00da\u0159adu pro kontrolu potravin a l\u00e9\u010div (FDA). To vystavuje l\u00e9k Zolgensma v hodnot\u011b n\u011bkolika milion\u016f dolar\u016f obrovsk\u00e9mu tlaku. Farmaceutick\u00e1 spole\u010dnost byla ji\u017e zodpov\u011bdn\u00e1 za datov\u00fd skand\u00e1l b\u011bhem jeho schvalov\u00e1n\u00ed v roce 2019 \u2013 a v t\u00e9 dob\u011b se trestu vyhnula.<\/span><\/strong><\/p>\n

V\u00edce ne\u017e dva miliony dolar\u016f za jedinou l\u00e9\u010dbu \u2013 a nyn\u00ed v\u011bdci na\u0161li genetick\u00fd materi\u00e1l z podan\u00e9 genov\u00e9 terapie v n\u00e1dorech dvou d\u011bt\u00ed. \u0160v\u00fdcarsk\u00fd farmaceutick\u00fd gigant Novartis je pod tlakem kv\u016fli sv\u00e9mu l\u00e9ku Zolgensma. Americk\u00fd \u00da\u0159ad pro kontrolu potravin a l\u00e9\u010div (FDA) 6. listopadu <\/span>potvrdil<\/span><\/a> , \u017ee DNA z virov\u00e9ho transportn\u00edho syst\u00e9mu pou\u017eit\u00e9ho p\u0159i l\u00e9\u010db\u011b se integrovala do genomu n\u00e1dorov\u00fdch bun\u011bk. Posti\u017een\u00fdm genem je GLI1, jeho\u017e aktivace m\u016f\u017ee podporovat r\u016fst n\u00e1doru. \u00da\u0159ad v\u00fdslovn\u011b uv\u00e1d\u00ed, \u017ee se jedn\u00e1 o p\u0159\u00edpad v\u00fdvoje n\u00e1doru zp\u016fsoben\u00e9ho genovou terapi\u00ed. Pro Novartis je to dal\u0161\u00ed ne\u00fasp\u011bch s l\u00e9kem, jeho\u017e proces schvalov\u00e1n\u00ed byl ji\u017e d\u0159\u00edve poznamen\u00e1n skand\u00e1lem s manipulovan\u00fdmi daty.<\/span><\/p>\n

Od schv\u00e1len\u00ed p\u0159\u00edpravku na trh v roce 2019 bylo u d\u0159\u00edve l\u00e9\u010den\u00fdch d\u011bt\u00ed hl\u00e1\u0161eno celkem dev\u011bt p\u0159\u00edpad\u016f vzniku n\u00e1dor\u016f. Ve dvou z t\u011bchto p\u0159\u00edpad\u016f byla souvislost s genovou terapi\u00ed jasn\u011b potvrzena genetick\u00fdm testov\u00e1n\u00edm. Podle spole\u010dnosti Novartis dosud dostalo p\u0159\u00edpravek Zolgensma v\u00edce ne\u017e 6 000 pacient\u016f. Pacientsk\u00e1 organizace SMA Europe nyn\u00ed <\/span>vyz\u00fdv\u00e1 ke<\/span><\/a> komplexn\u00ed informov\u00e1n\u00ed ve\u0159ejnosti. Rodi\u010de, jejich\u017e d\u011bti ji\u017e byly l\u00e9\u010deny, by si m\u011bli b\u00fdt v\u011bdomi potenci\u00e1ln\u00edch rizik, kter\u00e1 jim hroz\u00ed, a toho, zda jsou nutn\u00e9 dal\u0161\u00ed l\u00e9ka\u0159sk\u00e9 prohl\u00eddky.<\/span><\/p>\n

Injekce za milion dolar\u016f, kter\u00e1 naru\u0161\u00ed genom<\/span><\/h3>\n

P\u0159\u00edpravek Zolgensma se pou\u017e\u00edv\u00e1 k l\u00e9\u010db\u011b spin\u00e1ln\u00ed svalov\u00e9 atrofie (SMA). P\u0159i tomto z\u00e1va\u017en\u00e9m d\u011bdi\u010dn\u00e9m onemocn\u011bn\u00ed odum\u00edraj\u00ed nervov\u00e9 bu\u0148ky a svaly postupn\u011b sl\u00e1bnou. Obzvl\u00e1\u0161t\u011b z\u00e1va\u017en\u00e9 formy mohou b\u00fdt v kojeneck\u00e9m v\u011bku fat\u00e1ln\u00ed. Genov\u00e1 terapie si klade za c\u00edl kompenzovat genetickou vadu funk\u010dn\u00ed kopi\u00ed genu. Za t\u00edmto \u00fa\u010delem spole\u010dnost Novartis pou\u017e\u00edv\u00e1 geneticky modifikovan\u00e9 adeno-asociovan\u00e9 viry, kter\u00e9 transportuj\u00ed genetick\u00fd materi\u00e1l do t\u011blesn\u00fdch bun\u011bk.<\/span><\/p>\n

Tato DNA m\u00e1 z\u016fstat p\u0159ev\u00e1\u017en\u011b mimo lidsk\u00e9 chromozomy. M\u016f\u017ee se v\u0161ak tak\u00e9 trvale integrovat do genomu. Pokud naraz\u00ed na nep\u0159\u00edzniv\u00e9 m\u00edsto, mohou se aktivovat geny podporuj\u00edc\u00ed rakovinu. Toto riziko bylo org\u00e1n\u016fm pro regulaci l\u00e9\u010div zn\u00e1mo, ale d\u0159\u00edve bylo v varov\u00e1n\u00edch popisov\u00e1no jako teoretick\u00e9. U dvou d\u011bt\u00ed byly nyn\u00ed z\u00edsk\u00e1ny odpov\u00eddaj\u00edc\u00ed molekul\u00e1rn\u011b biologick\u00e9 d\u016fkazy. T\u0159et\u00ed p\u0159\u00edpad n\u00e1doru se tak\u00e9 t\u00fdk\u00e1 rakovinn\u00e9ho genu GLI1, a\u010dkoli p\u0159esn\u00fd mechanismus je st\u00e1le p\u0159edm\u011btem v\u00fdzkumu. To o to v\u00edce vyvol\u00e1v\u00e1 ot\u00e1zku dlouhodob\u00e9 bezpe\u010dnosti takov\u00e9 l\u00e9\u010dby. Koneckonc\u016f se nejedn\u00e1 o b\u011b\u017en\u00fd l\u00e9k, kter\u00fd lze v p\u0159\u00edpad\u011b probl\u00e9m\u016f jednodu\u0161e vysadit. Zaveden\u00e1 kopie genu m\u00e1 m\u00edt trval\u00fd \u00fa\u010dinek. To je v kone\u010dn\u00e9m d\u016fsledku \u00fa\u010delem l\u00e9\u010dby.<\/span><\/p>\n

Spole\u010dnost Novartis si p\u0159i uveden\u00ed p\u0159\u00edpravku Zolgensma na americk\u00fd trh v kv\u011btnu 2019 \u00fa\u010dtovala katalogovou cenu 2,125 milionu dolar\u016f. Spole\u010dnost tuto obrovskou \u010d\u00e1stku <\/span>zd\u016fvodnila<\/span><\/a> dlouhodob\u00fdmi l\u00e9\u010debn\u00fdmi \u00fasp\u011bchy a potenci\u00e1ln\u00edmi \u00fasporami ve srovn\u00e1n\u00ed s celo\u017eivotn\u00ed farmakoterapi\u00ed. V\u00edce ne\u017e dva miliony dolar\u016f za jednu infuzi \u2013 p\u0159i t\u00e9to cen\u011b by v\u011bdeck\u00e1 dokumentace a bezpe\u010dnostn\u00ed kontroly m\u011bly b\u00fdt alespo\u0148 bezchybn\u00e9. P\u0159esto ani b\u011bhem schvalovac\u00edho procesu nebyla samotn\u00e1 data tak pe\u010dliv\u011b zkoum\u00e1na.<\/span><\/p>\n

Zmanipulovan\u00e1 data \u2013 a FDA nechala Novartis bez trestu<\/span><\/h3>\n

Dne 24. kv\u011btna 2019 z\u00edskala spole\u010dnost Zolgensma registraci v USA. Ve\u0159ejnost v t\u00e9 dob\u011b nev\u011bd\u011bla, \u017ee data ze studi\u00ed na zv\u00ed\u0159atech byla zmanipulov\u00e1na v dce\u0159in\u00e9 spole\u010dnosti Novartis, AveXis. Spole\u010dnost si byla v\u011bdoma nesrovnalost\u00ed ji\u017e p\u0159ed schvalovac\u00edm procesem, ale informovala FDA a\u017e 28. \u010dervna. V t\u00e9 dob\u011b byl l\u00e9k v hodnot\u011b n\u011bkolika milion\u016f dolar\u016f ji\u017e na trhu. Manipulace se t\u00fdkaly studi\u00ed p\u0159edlo\u017een\u00fdch k vyhodnocen\u00ed v\u00fdrobn\u00edho procesu. Kdy\u017e se z\u00e1le\u017eitost v srpnu 2019 dostala na ve\u0159ejnost, FDA <\/span>reagovala<\/span><\/a> ost\u0159e kritizuj\u00edc\u00edm t\u00f3nem. V\u011bdeck\u00e9 \u00fadaje mus\u00ed b\u00fdt \u00fapln\u00e9 a p\u0159esn\u00e9, prohl\u00e1sila agentura. Dokonce pohrozila mo\u017en\u00fdmi ob\u010danskopr\u00e1vn\u00edmi a trestn\u00edmi sankcemi. Nicm\u00e9n\u011b schv\u00e1len\u00ed potvrdila, proto\u017ee podle jej\u00edho hodnocen\u00ed sporn\u00e9 \u00fadaje nezpochyb\u0148ovaly klinick\u00e9 v\u00fdsledky u lid\u00ed.<\/span><\/p>\n

A co se stalo s hroz\u00edc\u00edmi d\u016fsledky? <\/span>Nic<\/span><\/a> . V b\u0159eznu 2020 FDA ukon\u010dila vy\u0161et\u0159ov\u00e1n\u00ed, ani\u017e by spole\u010dnosti Novartis ulo\u017eila jak\u00e9koli sankce. Farmaceutick\u00e1 spole\u010dnost musela opravit sporn\u00e9 dokumenty a bylo j\u00ed povoleno pokra\u010dovat v prodeji sv\u00e9ho p\u0159emr\u0161t\u011bn\u011b drah\u00e9ho l\u00e9ku. Spole\u010dnost v\u011bd\u011bla o zmanipulovan\u00fdch datech p\u0159ed schv\u00e1len\u00edm, teprve pot\u00e9 informovala regula\u010dn\u00ed org\u00e1n pro l\u00e9\u010diva a nakonec vyv\u00e1zla bez sankc\u00ed. Takto prob\u00edhalo p\u0159ezkoum\u00e1n\u00ed l\u00e9ku ur\u010den\u00e9ho pro v\u00e1\u017en\u011b nemocn\u00e9 kojence.<\/span><\/p>\n

I jin\u00e9 genov\u00e9 terapie jsou podez\u0159el\u00e9, \u017ee zp\u016fsobuj\u00ed rakovinu<\/span><\/h3>\n

Ned\u00e1vn\u00e9 p\u0159\u00edpady n\u00e1dor\u016f nyn\u00ed vyvol\u00e1vaj\u00ed obavy i ohledn\u011b dal\u0161\u00edch genov\u00fdch terapi\u00ed. FDA poukazuje na dva dal\u0161\u00ed v\u011bdecky zdokumentovan\u00e9 p\u0159\u00edpady, kdy se DNA z pou\u017eit\u00fdch syst\u00e9m\u016f pro pod\u00e1v\u00e1n\u00ed vir\u016f integrovala do lidsk\u00e9ho genomu a p\u0159isp\u011bla k rozvoji n\u00e1doru. To znamen\u00e1, \u017ee probl\u00e9m se neomezuje pouze na l\u00e9k spole\u010dnosti Novartis v hodnot\u011b n\u011bkolika milion\u016f dolar\u016f.<\/span><\/p>\n

Jeden takov\u00fd p\u0159\u00edpad byl <\/span>publikov\u00e1n<\/span><\/a> v kv\u011btnu 2026 v \u010dasopise New England Journal of Medicine . P\u011btilet\u00e9mu chlapci se \u010dty\u0159i roky po experiment\u00e1ln\u00ed genov\u00e9 terapii pro z\u00e1va\u017enou metabolickou poruchu vyvinul n\u00e1dor na mozku. V\u011bdci v n\u00e1doru nalezli genetick\u00fd materi\u00e1l z podan\u00e9ho virov\u00e9ho vektoru, kter\u00fd se integroval do genu PLAG1 souvisej\u00edc\u00edho s rakovinou. N\u00e1dor byl chirurgicky odstran\u011bn. Dal\u0161\u00ed publikovan\u00fd p\u0159\u00edpad se t\u00fdk\u00e1 n\u00e1doru v p\u00e1te\u0159i, kde byla rovn\u011b\u017e prok\u00e1z\u00e1na vazba na integrovanou virovou DNA.<\/span><\/p>\n

L\u00e9\u010div\u00fd p\u0159\u00edpravek Itvisma od spole\u010dnosti Novartis, schv\u00e1len\u00fd teprve v listopadu 2025, je nyn\u00ed tak\u00e9 pod drobnohledem. Pou\u017e\u00edv\u00e1 stejn\u00fd virov\u00fd vektor a genov\u00fd design jako Zolgensma, ale pod\u00e1v\u00e1 se odli\u0161n\u011b a je ur\u010den pro star\u0161\u00ed pacienty. U p\u0159\u00edpravku Itvisma dosud nebyly hl\u00e1\u0161eny \u017e\u00e1dn\u00e9 p\u0159\u00edpady rakoviny. FDA nicm\u00e9n\u011b pova\u017euje riziko rakoviny u obou l\u00e9k\u016f za relevantn\u00ed vzhledem k jejich spole\u010dn\u00e9mu mechanismu \u00fa\u010dinku a monitoruje tak\u00e9 dal\u0161\u00ed programy genov\u00e9 terapie.<\/span><\/p>\n

Novartis by m\u011bl kone\u010dn\u011b poskytnout jasno<\/span><\/h3>\n

Je\u0161t\u011b p\u0159ed ned\u00e1vn\u00fdm ozn\u00e1men\u00edm FDA po\u017eadovala pacientsk\u00e1 organizace SMA Europe od spole\u010dnosti Novartis v\u011bt\u0161\u00ed transparentnost. Rodiny by nem\u011bly muset shroma\u017e\u010fovat informace o potenci\u00e1ln\u00edch rizic\u00edch rakoviny z f\u00e1m a soci\u00e1ln\u00edch m\u00e9di\u00ed. Spole\u010dnost mus\u00ed jasn\u011b vysv\u011btlit, co je o p\u0159\u00edpadech n\u00e1dor\u016f zn\u00e1mo, jak\u00e1 vy\u0161et\u0159ov\u00e1n\u00ed prob\u00edhaj\u00ed a jak\u00e9 jsou d\u016fsledky pro d\u011bti, kter\u00e9 jsou ji\u017e l\u00e9\u010deny.<\/span><\/p>\n

Spole\u010dnost Novartis uji\u0161\u0165uje, \u017ee ve v\u0161ech p\u0159\u00edpadech v\u010das informovala regula\u010dn\u00ed org\u00e1ny pro l\u00e9\u010div\u00e9 p\u0159\u00edpravky. Dv\u011b d\u011bti, u kter\u00fdch byla potvrzena genetick\u00e1 souvislost, byly l\u00e9\u010deny na sv\u00e9 n\u00e1dory a nad\u00e1le jsou l\u00e9ka\u0159sky sledov\u00e1ny. Potenci\u00e1ln\u00ed riziko vzniku n\u00e1doru v d\u016fsledku integrace virov\u00e9 DNA bylo ji\u017e zm\u00edn\u011bno i v informac\u00edch o p\u0159\u00edpravku.<\/span><\/p>\n

\u00da\u0159ad pro kontrolu potravin a l\u00e9\u010div (FDA) vy\u0161et\u0159uje, zda je t\u0159eba zp\u0159\u00edsnit varov\u00e1n\u00ed pro p\u0159\u00edpravky Zolgensma a Itvisma. Prob\u00edhaj\u00edc\u00ed dlouhodob\u00e9 studie jsou rovn\u011b\u017e p\u0159ezkoum\u00e1v\u00e1ny. \u00da\u0159ad chce zjistit, zda je sou\u010dasn\u00e9 monitorov\u00e1n\u00ed dostate\u010dn\u00e9 k detekci tvorby n\u00e1dor\u016f u l\u00e9\u010den\u00fdch pacient\u016f v ran\u00e9 f\u00e1zi. L\u00e9ka\u0159i a administr\u00e1to\u0159i studi\u00ed byli pov\u011b\u0159eni, aby takov\u00e9 p\u0159\u00edpady pe\u010dliv\u011b sledovali a hl\u00e1sili. Zva\u017euj\u00ed se dal\u0161\u00ed regula\u010dn\u00ed opat\u0159en\u00ed proti t\u011bmto l\u00e9k\u016fm.<\/span><\/p>\n

\"\"<\/p>\n

 <\/p>\n","protected":false},"excerpt":{"rendered":"

N\u00e1dory u dvou d\u011bt\u00ed p\u0159\u00edmo souvisej\u00ed s genovou terapi\u00ed vyvinutou spole\u010dnost\u00ed Novartis, jak vypl\u00fdv\u00e1 z…<\/p>\n","protected":false},"author":2,"featured_media":120685,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":[],"categories":[7],"tags":[245,895,12014,1366,46],"_links":{"self":[{"href":"https:\/\/www.infokuryr.cz\/n\/wp-json\/wp\/v2\/posts\/120684"}],"collection":[{"href":"https:\/\/www.infokuryr.cz\/n\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.infokuryr.cz\/n\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.infokuryr.cz\/n\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.infokuryr.cz\/n\/wp-json\/wp\/v2\/comments?post=120684"}],"version-history":[{"count":0,"href":"https:\/\/www.infokuryr.cz\/n\/wp-json\/wp\/v2\/posts\/120684\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.infokuryr.cz\/n\/wp-json\/wp\/v2\/media\/120685"}],"wp:attachment":[{"href":"https:\/\/www.infokuryr.cz\/n\/wp-json\/wp\/v2\/media?parent=120684"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.infokuryr.cz\/n\/wp-json\/wp\/v2\/categories?post=120684"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.infokuryr.cz\/n\/wp-json\/wp\/v2\/tags?post=120684"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}