A ranar 20 ga Yuli, Sabon Amfani da Magani(NDA) na pCAR-19B wanda aka ƙirƙiro ta hanyar Precision Biology wanda Hukumar Kayayyakin Lafiya ta Ƙasa ta China (NMPA) ta amince da shi a hukumance. Wannan samfurin shine samfurin CAR-T na farko don cutar sankarar bargo ta yara a China, wanda ake amfani da shi don magance marasa lafiya masu shekaru 3-21 waɗanda ke da cutar sankarar bargo ta jini mai tsanani ta CD19 (ALL) da ta sake dawowa/tabbatacce.
Cutar sankarar lymphoblastic mai tsanani (ALL) tana ɗaya daga cikin cututtukan da suka fi yawa a cikin yara kuma babbar sanadin mace-mace a cikin yara da matasa. Maganin ƙwayoyin chimeric antigen receptor (CAR-T) ya kawo sauyi ga maganin sake dawowa/ragewa (R/R) B-cell ALL, tare da samfurin farko da FDA ta amince da shi, Kymriah (tisagenlecleucel) wanda Novartis ya ƙirƙira, wanda ya inganta yawan gafara da lokutan tsira sosai.
pCAR-19B samfurin ƙwayoyin CAR-T ne mai sarrafa kansa tare da ingantaccen ɓangaren canza sarkar guda ɗaya na CD19 (scFv) wanda aka tsara don magance matsalolin tsaro.
A watan Nuwamba na 2023, Hukumar Kayayyakin Lafiya ta Ƙasa ta China ta ba da izinin pCAR-19B don magance B-ALL.
Inganci da sakamakon aminci na pCAR-19B a cikin wani muhimmin gwaji na asibiti wanda ya shafi yara da matasa 'yan ƙasar Sin waɗanda ke ɗauke da ƙwayoyin R/R B DUKan sanar da shi aTaron Shekara-shekara na Ƙungiyar Nazarin Jini ta Amurka (ASH) karo na 66.
Hanyoyi: Wannan gwajin mataki na 2 (NCT05334823) wani bincike ne da aka gudanar a hannu ɗaya, wanda aka buɗe, kuma aka yi shi a wurare da yawa. An haɗa da marasa lafiya 'yan shekara ≥ 3 da ≤ shekaru 21 a lokacin gwajin tare da tabbatar da CD19+ R/R B-ALL. Babban abin da aka fi mayar da hankali a kai shi ne yawan gafarar da aka samu (ORR), wanda aka bayyana a matsayin cikakken gafarar da aka samu (CR) da kuma CR tare da cikakken murmurewa daga jini (CRi) cikin watanni 3 bayan allurar.
Sakamako: Ya zuwa ranar 18 ga Afrilu, 2024, an yi wa marasa lafiya 89 rajista kuma an yi musu gwajin cutar leukapheresis, tare da marasa lafiya 64 da aka yi wa allurar pCAR-19B. Babu wani lamari na gazawar masana'anta. Daga cikin marasa lafiyar da aka yi wa allurar, 6.25% (4/64) ba su da ƙarfin hali kuma 93.75% (60/64) sun sake komawa ga jiyya da yawa a baya ko dashen ƙwayoyin halitta na allogeneic. Matsakaicin shekaru shine shekaru 11 (tsakanin 3-21), kuma 56.25% (36/64) maza ne. Bugu da ƙari, 75% (48/64) na marasa lafiyar suna ɗauke da aƙalla kwayar halitta ɗaya mai haɗari. Matsakaicin nauyin fashewar ƙashi (BM) a farkon shine 58.3% (tsakanin 5%-98%) tare da nauyin fashewar BM mai nauyi (≥50%) da aka lura a cikin 56.25% na marasa lafiya.
A matsakaicin bin diddigin kwanaki 211 (tsakanin 35-750), mafi kyawun ORR shine 90.63% (58/64), tare da 78.13% na marasa lafiya (50/64) sun cimma CR da 12.5% na marasa lafiya (8/64) sun cimma CRi, wanda ya zarce ingancin samfuran maganin CAR-T na kasuwanci da aka ruwaito a cikin R/R B-ALL. 98.27% (57/58) na marasa lafiya da ORR sun sami ƙarancin raunin da ya rage (MRD) - koshin lafiya. ORR a cikin watanni 3 shine 76.56% (49/64). Matsakaicin tsawon lokacin amsawa (DOR) shine watanni 10.61 (tazara ta amincewa 95% 7.66-20.96). Matsakaicin rayuwa gabaɗaya (OS) shine watanni 23.92 (tazara ta amincewa 95% 9.86-NR).
Ciwon sakin Cytokine (CRS) da kuma ciwon neurotoxicity da ke da alaƙa da ƙwayoyin cuta masu tasiri ga garkuwar jiki (ICANS) sun faru a cikin kashi 98.44% da 50% na marasa lafiya, bi da bi. An lura da CRS na aji ≥3 da ICANS a cikin kashi 29.69% da 39.06% na marasa lafiya, bi da bi. Matsakaicin tsawon lokacin CRS na aji ≥3 da ICANS shine kwana 4 da kwana 3, bi da bi. Babu mace-mace da aka danganta da CRS ko ICANS.
Kammalawa: Duk da nauyin fashewar BM da kuma babban haɗarin bambancin kwayoyin halitta, pCAR-19B ya nuna yawan gafara, ya sami babban adadin gafarar MRD-negative, martani mai ɗorewa, da kuma bayanin aminci mai jurewa. Wannan binciken ya wakilci gwaji na farko na asibiti na B-ALL na yara a cikin al'ummar Asiya, yana ba da sabon zaɓi na magani ga yara da matasa 'yan ƙasar Sin waɗanda ke fama da R/R B-ALL.
A halin yanzu, akwai wasu gwaje-gwajen asibiti da yawa na CAR-T a China, kuma suna neman marasa lafiya. Don neman shawara kan sabbin magunguna da fasahohi, zaku iya tuntuɓar Sashen Kasa da Kasa na Asibitin Oncology na Yankin Kudu na Beijing.
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Nassoshi
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
Lokacin Saƙo: Nuwamba-22-2024