Ngomhla wama-20 kuJulayi, isicelo esitsha seziyobisi saphunyezwa.(NDA) ye-pCAR-19B eyenziwe ngokuzimeleyo yiPrecision Biology yamkelwa ngokusesikweni yi-China National Medical Products Administration (NMPA). Le mveliso yimveliso yokuqala ye-CAR-T ye-leukemia yabantwana e-China, esetyenziselwa ukunyanga izigulana ezineminyaka eyi-3-21 ezine-CD19-positive relapsed/refractory acute lymphoblastic leukemia (ALL).
I-Acute lymphoblastic leukemia (ALL) yenye yezona zifo zixhaphakileyo ebantwaneni kwaye yeyona nto iphambili ebangela ukufa kubantwana nakwishumi elivisayo. Unyango lweseli ye-chimeric antigen receptor (CAR-T) luye lwaguqula unyango lwe-relapse/refractory (R/R) B-cell ALL, ngemveliso yokuqala evunyiweyo yi-FDA, iKymriah(tisagenlecleucel) eyenziwe yiNovartis, iphucula kakhulu amazinga okukhululwa kunye namaxesha okusinda.
I-pCAR-19B yimveliso yeseli ye-CAR-T ezenzekelayo ene-CD19-specific single-chain variable fragment (scFv) eyenzelwe ukujongana neenkxalabo zokhuseleko.
NgoNovemba ka-2023, i-pCAR-19B inikwe "iNgcali yoNyango oluPhucukileyo" yi-China National Medical Products Administration yokunyanga i-B-ALL.
Iziphumo zokusebenza kakuhle kunye nokhuseleko lwe-pCAR-19B kuvavanyo olubalulekileyo lweklinikhi olubandakanya abantwana baseTshayina kunye nezigulane zabantu abadala abasebatsha abane-R/R B-cell ALLyabhengezwa kwiIntlanganiso Yonyaka yama-66 ye-American Society of Hematology (ASH).
Iindlela: Olu vavanyo lwesigaba sesi-2 (NCT05334823) yayiluphando oluqhutywa ngengalo enye, oluvulelekileyo, noluneziko ezininzi. Izigulane ezineminyaka eyi-≥ 3 kunye ne-≤ 21 ubudala ngexesha lokuhlolwa nge-CD19+ R/R B-ALL eqinisekisiweyo zaqukwa. Isiphelo esiphambili yayilizinga lokuxolelwa lilonke (ORR), elichazwa njenge-complete remission (CR) kunye ne-CR ene-incomplete blood recovery (CRi) kwiinyanga ezi-3 emva kokufakwa.
Iziphumo: Ukusukela nge-18 ka-Epreli, ngo-2024, izigulana ezingama-89 zazibhalisiwe kwaye zenze i-leukapheresis, kwaye izigulana ezingama-64 zifumana i-pCAR-19B infusion. Akukho matyala okusilela kokwenziwa. Phakathi kwezigulana ezifakwe i-infusion, i-6.25% (4/64) yayingavumi kwaye i-93.75% (60/64) yayibuyele kwimigca emininzi yonyango lwangaphambili okanye ukufakelwa kweseli ye-allogeneic stem. Ubudala obuphakathi yayiyiminyaka eli-11 (uluhlu 3-21), kwaye i-56.25% (36/64) yayingamadoda. Ukongeza, i-75% (48/64) yezigulana yayine-gene enye enomngcipheko ophezulu. Umthwalo ophakathi we-bone marrow (BM) blast ekuqaleni yayiyi-58.3% (uluhlu 5%-98%) kunye nomthwalo onzima we-BM blast (≥50%) obonwe kwi-56.25% yezigulana.
Kwi-avareji yokulandelela yeentsuku ezingama-211 (uluhlu lwama-35-750), i-ORR engcono kakhulu yayiyi-90.63% (58/64), apho i-78.13% yezigulane (50/64) zifumene i-CR kunye ne-12.5% yezigulane (8/64) zifumene i-CRi, idlula ukusebenza kakuhle kweemveliso zonyango lwe-anti-CD19 CAR-T ezixeliweyo kwi-R/R B-ALL. I-98.27% (57/58) yezigulane ezine-ORR zifumene i-negative minimal residue disease (MRD)-negative remission. I-ORR kwiinyanga ezi-3 yayiyi-76.56% (49/64). Ubude bexesha eliphakathi lokuphendula (DOR) yayiyi-10.61 iinyanga (95% ixesha lokuzithemba 7.66-20.96). Ukusinda okuphakathi (OS) yayiyi-23.92 iinyanga (95% ixesha lokuzithemba 9.86-NR).
I-Cytokine release syndrome (CRS) kunye ne-immune effector cell-associated neurotoxicity syndrome (ICANS) zenzeke kwi-98.44% kunye ne-50% yezigulane, ngokwahlukeneyo. I-CRS yeBanga ≥3 kunye ne-ICANS zabonwa kwi-29.69% kunye ne-39.06% yezigulane, ngokwahlukeneyo. Ubude obuphakathi be-CRS yeBanga ≥3 kunye ne-ICANS yayiziintsuku ezi-4 kunye neentsuku ezi-3, ngokwahlukeneyo. Akukho kufa kubangelwa yi-CRS okanye i-ICANS.
Izigqibo: Nangona kukho umthwalo onzima wokuqhuma kwe-BM kunye nomngcipheko ophezulu wokwahluka kwezakhi zofuzo, i-pCAR-19B ibonise amazinga aphezulu okuxolelwa, ifikelele kwinqanaba eliphezulu lokuxolelwa okungekuhle kwe-MRD, iimpendulo ezihlala ixesha elide, kunye neprofayili yokhuseleko enokunyamezeleka. Olu phononongo lubonisa uvavanyo lokuqala olubalulekileyo lweklinikhi lwe-B-ALL yobuntwana kubemi baseAsia, lubonelela ngokhetho olutsha lonyango kwizigulane zaseTshayina zabantwana kunye nabantu abadala abancinci abane-R/R B-ALL.
Okwangoku, kukho ezinye iimvavanyo ezininzi zeklinikhi ze-CAR-T eTshayina, kwaye zikhangela izigulane. Ukuze ufumane iingcebiso ngamayeza amatsha kunye netekhnoloji, ungaqhagamshelana neSebe leMicimbi yeZibhedlele ze-Oncology zaseBeijing South Region.
Inombolo yefowuni: 4008803716
I-imeyile：myimmnet@163.com
Iireferensi
1.https://www.cde.org.cn/main/xxgk/listpage/9f9c74c73e0f8f56a8bfbc646055026d
2. http://cq.news.cn/20240728/b0a74788930d4be7a45996be2808dd4f/c.html
4.https://ash.confex.com/ash/2024/webprogram/Paper206408.html
5.https://ashpublications.org/blood/article/140/Supplement%201/4625/491174/The-Safety-and-Efficacy-of-p-CAR-19B-a-Scfv
Ixesha lokuthumela: Novemba-22-2024