Ma ka lā 20 o Iulai, ua hoʻopuka ʻia ka Palapala Noi Lāʻau Lapaʻau Hou(NDA) o pCAR-19B i hoʻomohala kūʻokoʻa ʻia e Precision Biology ua ʻae kūhelu ʻia e China National Medical Products Administration (NMPA). ʻO kēia huahana ka huahana CAR-T mua no ka leukemia kamaliʻi ma Kina, i hoʻohana ʻia e mālama i nā poʻe maʻi i piha i nā makahiki 3-21 me ka CD19-positive relapsed/refractory acute lymphoblastic leukemia (ALL).
ʻO ka leukemia lymphoblastic acute (ALL) kekahi o nā puʻupuʻu malignant maʻamau i nā keiki a me ke kumu nui o ka make ʻana i nā keiki a me nā ʻōpio. Ua hoʻololi ka lāʻau lapaʻau cell chimeric antigen receptor (CAR-T) i ka mālama ʻana i ka relapsed/refractory (R/R) B-cell ALL, me ka huahana mua i ʻāpono ʻia e ka FDA, ʻo Kymriah (tisagenlecleucel) i hoʻomohala ʻia e Novartis, e hoʻomaikaʻi nui ana i nā helu hoʻomaha a me nā manawa ola.
He huahana pūnaewele CAR-T autologous ʻo pCAR-19B me kahi ʻāpana loli kaulahao hoʻokahi (scFv) i hoʻoponopono ʻia e like me ke kanaka i hoʻolālā ʻia no ka hoʻoponopono ʻana i nā hopohopo palekana.
I Nowemapa 2023, ua hāʻawi ʻia ʻo pCAR-19B i ka "Breakthrough Therapy Designation" e ka China National Medical Products Administration no ka mālama ʻana iā B-ALL.
ʻO ka hopena pono a me ka palekana o pCAR-19B i loko o kahi hoʻokolohua lapaʻau koʻikoʻi e pili ana i nā maʻi pediatric Kina a me nā ʻōpio makua me R/R B-cell ALLua hoʻolaha ʻia maka Hālāwai Makahiki 66 o ka ʻAhahui Hematology ʻAmelika (ASH).
Nā Hana: ʻO kēia hoʻokolohua pae 2 (NCT05334823) he noiʻi lima hoʻokahi, lepili wehe, multicenter. Ua hoʻokomo ʻia nā maʻi i piha i ka makahiki ≥ 3 a me ≤ 21 mau makahiki i ka nānā ʻana me ka CD19+ R/R B-ALL i hōʻoia ʻia. ʻO ke kumu hope loa ka helu hoʻomaha holoʻokoʻa (ORR), i wehewehe ʻia he hoʻomaha piha (CR) me CR me ka hoʻōla koko piha ʻole (CRi) i loko o 3 mau mahina ma hope o ka hoʻokomo ʻana.
Nā Hualoaʻa: Ma ka lā 18 o ʻApelila, 2024, ua kākau inoa ʻia he 89 mau maʻi a ua hana ʻia ka leukapheresis, me 64 mau maʻi e loaʻa ana i ka hoʻokomo ʻana o ka pCAR-19B. ʻAʻohe hihia o ka hana ʻole ʻana. Ma waena o nā maʻi i hoʻokomo ʻia, he 6.25% (4/64) he paʻakikī ke kūʻē i ka lāʻau lapaʻau a ua hoʻi hou ʻo 93.75% (60/64) i nā laina he nui o ka lāʻau lapaʻau mua a i ʻole ka hoʻoili ʻana o nā cell stem allogeneic. ʻO ka makahiki waena he 11 mau makahiki (pae 3-21), a he 56.25% (36/64) he kāne. Eia kekahi, he 75% (48/64) o nā maʻi i lawe i ka liʻiliʻi o hoʻokahi gene pilikia kiʻekiʻe. ʻO ka ukana pahū iwi waena (BM) ma ka baseline he 58.3% (pae 5%-98%) me ka ukana pahū BM kaumaha (≥50%) i ʻike ʻia ma 56.25% o nā maʻi.
Ma kahi hahai waena o 211 mau lā (pae 35-750), ʻo ka ORR maikaʻi loa he 90.63% (58/64), me 78.13% o nā maʻi (50/64) i hoʻokō i ka CR a me 12.5% o nā maʻi (8/64) i hoʻokō i ka CRi, e ʻoi aku ana ma mua o ka pono o nā huahana lāʻau lapaʻau CAR-T anti-CD19 kalepa i hōʻike ʻia ma R/R B-ALL. 98.27% (57/58) o nā maʻi me ORR i loaʻa ka hoʻomaha maʻi koena liʻiliʻi maikaʻi ʻole (MRD)-negative. ʻO ka ORR ma 3 mau mahina he 76.56% (49/64). ʻO ka lōʻihi waena o ka pane (DOR) he 10.61 mau mahina (95% hilinaʻi waena 7.66-20.96). ʻO ka awelika holoʻokoʻa ola (OS) he 23.92 mau mahina (95% hilinaʻi waena 9.86-NR).
Ua ʻike ʻia ka maʻi hoʻokuʻu Cytokine (CRS) a me ka maʻi neurotoxicity pili i ka cell effector immune (ICANS) ma 98.44% a me 50% o nā maʻi, kēlā me kēia. Ua ʻike ʻia ka Papa ≥3 CRS a me ICANS ma 29.69% a me 39.06% o nā maʻi, kēlā me kēia. ʻO ka lōʻihi waena o ka Papa ≥3 CRS a me ICANS he 4 mau lā a me 3 mau lā, kēlā me kēia. ʻAʻohe make i hoʻopili ʻia i ka CRS a i ʻole ICANS.
Nā Hopena: ʻOiai ke kaumaha o nā pahū BM a me ka nui o ka pilikia o nā ʻano hoʻololi genetic, ua hōʻike ʻo pCAR-19B i nā helu kala kiʻekiʻe, ua loaʻa kahi helu kiʻekiʻe o ke kala ʻana MRD-negative, nā pane paʻa, a me kahi ʻano palekana hiki ke ʻae ʻia. Hōʻike kēia haʻawina i ka hoʻokolohua lapaʻau koʻikoʻi mua o ka B-ALL kamaliʻi i loko o kahi heluna kanaka ʻAsia, e hāʻawi ana i kahi koho lapaʻau hou no nā maʻi pediatric Kina a me nā mākua ʻōpio me R/R B-ALL.
I kēia manawa, nui nā hoʻokolohua lapaʻau CAR-T ʻē aʻe ma Kina, a ke ʻimi nei lākou i nā maʻi. No ke kūkākūkā ʻana i nā lāʻau lapaʻau hou a me nā ʻenehana, hiki iā ʻoe ke hoʻokaʻaʻike aku i ka Beijing South Region Oncology Hospital International Department.
Helu Kelepona：4008803716
Leka uila:myimmnet@163.com
Nā Kuhikuhi
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
Ka manawa hoʻouna: Nov-22-2024