Kev Kho Mob Hauv Pediatric Autoimmune Disease: CAR-T Cell Therapy Kho Lupus Patient

Thaum Lub Rau Hli 2023, 15-xyoo-laus Uresa tau txais CAR-T cell therapy ntawm Erlangen University Tsev Kho Mob, kos npe rau thawj zaug siv qhov kev kho tshiab no kom qeeb kev loj hlob ntawm cov kab mob lupus erythematosus (SLE), kab mob autoimmune hnyav. Ib xyoos tom qab, Uresa xav tias noj qab nyob zoo li qub, ib yam li ob peb tus mob khaub thuas me.

Uresa yog thawj tus menyuam kho SLE nrog kev tiv thaiv kab mob ntawm Erlangen University's German Center for Immunotherapy (DZI). Kev vam meej ntawm qhov kev kho tus kheej no tau luam tawm hauv The Lancet.

Dr. Tobias Krickau, kws kho mob rheumatologist ntawm Erlangen University Tsev Kho Mob Department of Pediatrics thiab Adolescent Medicine, piav qhia txog qhov txawv ntawm kev siv CAR-T hlwb los kho cov kab mob autoimmune. Yav dhau los, CAR-T kho tau pom zoo tsuas yog rau qee yam mob qog noj ntshav siab.

Tom qab tag nrho lwm cov tshuaj tsis tuaj yeem tswj hwm Uresa qhov kev ua phem rau SLE, pab pawg tshawb fawb tau ntsib qhov kev txiav txim siab nyuaj: puas yuav siv cov tshuaj tiv thaiv kab mob no rau menyuam yaus uas muaj kab mob autoimmune? Cov lus teb tsis tau muaj dua, vim tsis muaj leej twg tau sim kho CAR-T rau cov kab mob autoimmune rau menyuam yaus ua ntej.

CAR-T cell therapy suav nrog kev rho tawm qee tus neeg mob lub cev tiv thaiv kab mob (T hlwb), muab lawv nrog chimeric antigen receptors (CAR) hauv chav kuaj tshwj xeeb huv, thiab tom qab ntawd rov ua cov cell hloov kho rau hauv tus neeg mob. Cov hlwb CAR-T no ncig hauv cov ntshav, tsom thiab rhuav tshem autoreactive (tshuaj lom) B hlwb.

Uresa cov tsos mob pib thaum lub caij nplooj zeeg xyoo 2022, suav nrog migraines, qaug zog, mob pob qij txha thiab leeg, thiab pob khaus ntawm lub ntsej muag - cov cim qhia ntawm lupus. Txawm hais tias kev kho mob hnyav, nws tus mob hnyav zuj zus, cuam tshuam rau nws lub raum thiab ua rau muaj teeb meem loj.

Thaum ntxov 2023, tom qab ntau lub tsev kho mob thiab kev kho mob, suav nrog kev siv tshuaj tua kab mob tiv thaiv kab mob thiab kev sib pauv ntshav ntshav, Uresa tus mob tsis zoo mus rau qhov uas nws xav tau kev lim ntshav. Kev cais tawm ntawm cov phooj ywg thiab tsev neeg, nws lub neej zoo poob qis.

Pab neeg kho mob ntawm Erlangen University, coj los ntawm xibfwb Mackensen, pom zoo tsim thiab siv CAR-T hlwb rau Uresa tom qab kev sib tham ntxaws. Txoj kev hlub tshua no siv CAR-T txoj kev kho tau pib raws li lub teb chaws Yelemees txoj cai lij choj siv tshuaj thiab kev siv siab hlub.

CAR-T cell therapy program ntawm Erlangen, coj los ntawm xibfwb Georg Schett thiab xibfwb Mackensen, tau kho cov neeg mob uas muaj ntau yam kab mob autoimmune, suav nrog SLE, txij li xyoo 2021. Lawv txoj kev vam meej nrog 15 tus neeg mob tau luam tawm hauv New England Journal of Medicine thaum Lub Ob Hlis. 2024, thiab tam sim no lawv tab tom ua txoj kev tshawb fawb CASTLE nrog 24 tus neeg koom, txhua tus qhia txog kev txhim kho tseem ceeb.

Txhawm rau npaj rau CAR-T cell therapy, Uresa tau siv tshuaj kho mob qis los ua kom muaj chaw rau CAR-T hlwb hauv nws cov ntshav. Thaum Lub Rau Hli 26, 2023, Uresa tau txais nws tus kheej CAR-T hlwb. Los ntawm lub lim tiam thib peb tom qab kev kho mob, nws lub raum ua haujlwm thiab cov cim lupus zoo tuaj, thiab nws cov tsos mob maj mam ploj mus.

Cov txheej txheem kev kho mob koom nrog kev sib koom tes ua tib zoo kom ntseeg tau tias kev siv tshuaj khomob zoo thiab kev tiv thaiv lub raum ua haujlwm ntxiv. Uresa tsuas muaj kev mob tshwm sim me me thiab raug tso tawm rau hnub 11 tom qab kho.

Thaum lig Lub Xya Hli 2023, Uresa tau rov qab los tsev, ua tiav nws qhov kev xeem, thiab teem lub hom phiaj tshiab rau nws lub neej yav tom ntej, suav nrog kev ywj pheej thiab tau txais tus dev. Nws zoo siab rov sib txuas nrog cov phooj ywg thiab rov pib lub neej hluas li qub.

Xib fwb Mackensen piav qhia tias Uresa tseem muaj cov CAR-T hlwb tseem ceeb hauv nws cov ntshav, uas txhais tau hais tias nws xav tau cov tshuaj tiv thaiv kab mob txhua hli kom txog thaum nws cov B hlwb rov zoo. Dr. Krickau tau hais ntxiv tias kev ua tiav ntawm Uresa txoj kev kho mob yog vim muaj kev sib koom tes ntawm ntau yam kev kho mob ntawm German Center for Immunotherapy.

Uresa tsis xav tau tshuaj lossis lim ntshav, thiab nws lub raum tau zoo lawm. Dr. Krickau thiab nws pab neeg tab tom npaj cov kev tshawb fawb ntxiv los tshawb txog lub peev xwm ntawm CAR-T hlwb hauv kev kho lwm yam kab mob autoimmune rau menyuam yaus.

Cov ntaub ntawv pov thawj no qhia tau tias muaj peev xwm ntawm CAR-T cell therapy los muab kev tso cai mus sij hawm ntev rau cov neeg mob uas muaj kab mob autoimmune hnyav xws li SLE. Kev vam meej ntawm Uresa txoj kev kho mob qhia txog qhov tseem ceeb ntawm kev cuam tshuam ntxov thiab kev sib koom tes ntau yam. Cov kev tshawb fawb soj ntsuam ntxiv yog xav tau kom paub tseeb tias kev nyab xeeb mus sij hawm ntev thiab kev ua tau zoo ntawm CAR-T cell therapy rau cov menyuam yaus uas muaj kab mob autoimmune.