Kua whakaaetia e te NMPA te NDA a pCAR-19B mō te rongoā i te mate toto toto o ngā tamariki.

I te 20 o Hūrae, ka puta te Tono Rongoā Hou(NDA) o te pCAR-19B i whakawhanakehia motuhaketia e Precision Biology i whakaaetia mana e te China National Medical Products Administration (NMPA). Ko tēnei hua te hua CAR-T tuatahi mō te mate pukupuku toto o ngā tamariki i Haina, e whakamahia ana hei rongoā i ngā tūroro 3-21 tau te pakeke me te mate pukupuku toto lymphoblastic acute (ALL) kua hoki mai/koretake te CD19.

Ko te mate pukupuku lymphoblastic acute (ALL) tētahi o ngā pukupuku kino tino noa i roto i ngā tamariki, ā, ko ia tētahi o ngā tino take o te mate i roto i ngā tamariki me ngā taiohi. Kua hurihia e te rongoā pūtau chimeric antigen receptor (CAR-T) te maimoatanga o te ALL pūtau-B kua hoki whakamuri/uaua (R/R), me te hua tuatahi kua whakaaetia e te FDA, arā, ko Kymriah (tisagenlecleucel) i whakawhanakehia e Novartis, ā, i tino whakapai ake i ngā reiti murunga me ngā wā ora.

He hua pūtau CAR-T aunoa te pCAR-19B me tētahi kongakonga taurangi mekameka kotahi (scFv) kua arotauhia hei whakatika i ngā āwangawanga haumaru.

I te Whiringa-ā-rangi 2023, i whakawhiwhia a pCAR-19B ki te "Breakthrough Therapy Designation" e te China National Medical Products Administration mō te rongoā i te B-ALL.

Ngā hua whai huatanga me te haumaru o te pCAR-19B i roto i tētahi whakamātautau haumanu nui e whai wāhi ana ngā tūroro tamariki me ngā taiohi Hainamana me te R/R B-cell ALLi pānuitia i tete Hui-ā-Tau 66 o te American Society of Hematology (ASH).

Ngā Tikanga: He rangahau kotahi-ringa, tuwhera-tapahanga, maha-pokapū tēnei whakamātautau wāhanga 2 (NCT05334823). I whakaurua ngā tūroro ≥ 3 tau te pakeke me te ≤ 21 tau te pakeke i te wā i tirotirohia ai me te CD19+ R/R B-ALL kua whakaūtia. Ko te pito whakamutunga tuatahi ko te reiti murunga whānui (ORR), i tautuhia ko te murunga katoa (CR) me te CR me te whakaoranga toto kāore i oti (CRi) i roto i ngā marama e 3 i muri i te werohanga.

Ngā Hua: I te 18 o Paenga-whāwhā, 2024, e 89 ngā tūroro kua rēhitatia, ā, kua whai leukapheresis, me te 64 ngā tūroro i whiwhi i te werohanga pCAR-19B. Kāore he take o te korenga o te hanga. I roto i ngā tūroro i werohia, 6.25% (4/64) he uaua ki te rongoā, ā, 93.75% (60/64) kua hoki anō ki ngā momo rongoā o mua, ki te whakawhitinga pūtau take allogeneic rānei. Ko te tau toharite he 11 tau (mai i te 3-21), ā, 56.25% (36/64) he tāne. Hei tāpiri, 75% (48/64) o ngā tūroro he iti rawa te kotahi ira mōrearea nui. Ko te taumahatanga waenga o te pupuhi wheua wheua (BM) i te tīmatanga he 58.3% (mai i te 5%-98%) me te taumahatanga pupuhi BM nui (≥50%) i kitea i roto i te 56.25% o ngā tūroro.

I te roanga o te arotakenga toharite mō ngā rā 211 (mai i te 35 ki te 750), ko te ORR pai rawa atu ko te 90.63% (58/64), me te 78.13% o ngā tūroro (50/64) i eke ki te CR, me te 12.5% ​​o ngā tūroro (8/64) i eke ki te CRi, he nui ake i te whai huatanga o ngā hua rongoā CAR-T anti-CD19 arumoni i kōrerotia i roto i te R/R B-ALL. 98.27% (57/58) o ngā tūroro me te ORR i eke ki te whakaoranga iti rawa o te mate toenga (MRD)-kino. Ko te ORR i te 3 marama ko te 76.56% (49/64). Ko te roanga toharite o te urupare (DOR) ko te 10.61 marama (95% wā whakawhirinaki 7.66-20.96). Ko te oranga whānui toharite (OS) ko te 23.92 marama (95% wā whakawhirinaki 9.86-NR).

I puta te mate tukunga cytokine (CRS) me te mate paitini io e pā ana ki ngā pūtau ārai mate (ICANS) i roto i te 98.44% me te 50% o ngā tūroro. I kitea te CRS me te ICANS taumata ≥3 i roto i te 29.69% me te 39.06% o ngā tūroro. Ko te roanga toharite o te CRS me te ICANS taumata ≥3 he 4 rā me te 3 rā. Kāore he mate i pā ki te CRS, ki te ICANS rānei.

Ngā Whakatau: Ahakoa te taumaha o ngā pahūtanga BM me te nui o te mōrearea o ngā rerekētanga ira, i whakaatuhia e te pCAR-19B he nui ngā reiti murunga, i tutuki he reiti teitei o te murunga MRD-kino, he roa ngā urupare, me te āhua haumaru e taea ana te whakamanawanui. Ko tēnei rangahau te whakamātautau haumanu matua tuatahi o te B-ALL o ngā tamariki i roto i te taupori Āhia, e tuku ana i tētahi kōwhiringa maimoatanga hou mō ngā tūroro tamariki me ngā taiohi Hainamana me te R/R B-ALL.

I tēnei wā, he maha atu ngā whakamātautau haumanu CAR-T i Haina, ā, kei te rapu tūroro rātou. Mō te kōrerorero mō ngā rongoā me ngā hangarau hou, ka taea e koe te whakapā atu ki te Tari Ao o te Hōhipera Oncology Rohe Tonga o Beijing.

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Ngā Tohutoro

1.https://www.cde.org.cn/main/xxgk/listpage/9f9c74c73e0f8f56a8bfbc646055026d

2. http://cq.news.cn/20240728/b0a74788930d4be7a45996be2808dd4f/c.html

3.https://mp.weixin.qq.com/s?__biz=MzAxMzgzOTk1OQ==&mid=2650794360&idx=1&sn=f1628888e194d1c2d0fb05894f92a969&chksm=83973240b4e0bb56e67dff6126946d2c9c92d8bc5f436b82552d99dca14072f37f2f360ca567#rd

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