2024-fevral: Xitoyning saraton kasalligini davolash bo'yicha ilg'or sa'y-harakatlari kimerik antigen retseptorlari (CAR) tomonidan ishlab chiqilgan T-hujayra (CAR T) terapiyasini joriy etish bilan kuchaydi. ko'p mieloma, aberrant plazma hujayralari bilan tavsiflangan halokatli qon saratoni. Ushbu yangi usul moslashtirilgan tibbiyotda hal qiluvchi momentni ifodalaydi va shu paytgacha alternativalari cheklangan bemorlarga umid beradi.
CAR-T hujayra terapiyasining umumiy ko'rinishi
CAR T-hujayra terapiyasi uses a patient’s own immune cells to identify and target cancer cells that express specific proteins. Engineering these T cells to incorporate artificial receptors known as CARs transforms them into highly targeted weapons capable of destroying shish cells. When someone has multiple myeloma, CAR T cells are changed to target BCMA, a protein that is found in large amounts in patients.
Klinik sinovlar va natijalar
FUCASO
2018 yilda IASO Biotherapeutics and Innovent Biologics ko'p markazli Phase 1/2 tadqiqotlarini baholash uchun boshladi. Equecabtagene autoleucel (FUCASO), Xitoyda birinchi mahalliy ishlab chiqarilgan CAR T-hujayrali davolash. Tadqiqotda ko'p miyeloma bilan og'rigan 79 kishi o'zlarining standart terapiya tanlovlarini tugatgan. Qizig'i shundaki, umumiy javob darajasi (ORR) 94.9% ga erishdi, to'liq javob/qattiq to'liq javob (CR/sCR) darajasi esa 58.2% ni tashkil etdi. O'rtacha tiklanish vaqti va CR/sCR mos ravishda 16 kun va 95 kunni tashkil etdi. Ushbu topilmalar equecabtagene autoleucelning ajoyib samaradorligini va uning mukammal xavfsizlik profilini tasdiqladi.
Ilgari CAR T-hujayrasini davolagan bemorlar uchun ORR 98.5% gacha ko'tarildi, oltitasi CR/sCRga erishdi. Ta'kidlash joizki, sinovda davolangan birinchi bemor 40 oydan ko'proq vaqt davomida qattiq to'liq remissiyani saqlab qoldi.
CILTA-CEL terapiyasi
Cilta-cel, shuningdek, ciltacabtagene autoleucel sifatida ham tanilgan, ko'p miyelom hujayralarida haddan tashqari ifodalangan protein bo'lgan B-hujayra etilish antigenini (BCMA) maqsad qilgan zamonaviy kimerik antigen retseptorlari (CAR) T-hujayralarini davolashdir. Ushbu yangi tibbiyot relapsli va refrakter ko'p miyelomni davolashda katta va'da berdi va an'anaviy davolash muqobil usullaridan charchagan bemorlarga yangi umid beradi.
Cilota-cel bilan davolash miyelom hujayralarida BCMA bilan bog'langan CARlarni ifodalash uchun bemorning T hujayralarini genetik jihatdan o'zgartiradi. Bemorga qaytarilgandan so'ng, bu o'zgartirilgan CAR T hujayralari xavfli hujayralarni muvaffaqiyatli nishonga olishi va yo'q qilishi mumkin, bu esa o'smaning regressiyasiga va ehtimol uzoq muddatli remissiyaga olib keladi.
Klinik samaradorlik
Klinik sinovlar, ayniqsa muhim KARTITUDE-1 Sinov shuni ko'rsatdiki, relapsli yoki refrakter ko'p miyeloma bilan og'rigan, oldindan davolangan bemorlarda Cilta-cel ajoyib samaradorlikka ega. Ushbu sinovlar natijalari 98% ning ajoyib umumiy javob tezligini (ORR) aniqladi, bemorlarning katta qismi qat'iy to'liq javoblarga (sCR) erishdi.
O'rtacha 28 oylik kuzatuvda teri tsellyulozasi barqaror javob berdi, o'rtacha rivojlanishsiz omon qolish hali erishilmadi. Dori-darmon, shuningdek, qo'llab-quvvatlovchi parvarish usullari orqali noxush hodisalar muvaffaqiyatli boshqarilishi bilan oqilona xavfsizlik profillarini namoyish etdi.
Qiyinchiliklar va imkoniyatlar
Despite the promising results, significant challenges remain. Most klinik tadqiqotlar in China are small-scale and single-center, with insufficient rigor and coordination among institutions. To address these restrictions, firms must aggressively participate in the commercialization of CAR T therapy, ensuring that it is widely available to patients.
Furthermore, improved manufacturing methods, faster regulatory channels, and increased patient access are required to ensure that CAR T-hujayra terapiyasi is successfully translated from laboratory bench to bedside. Collaborations between academia, industry, and government agencies are critical for driving innovation and improving patient care.
Ko'p miyelomli bemorlarda CAR T-Cell terapiyasining xavflari qanday?
The side effects of CAR T-Cell therapy in multiple myeloma are similar to those in leukemia and limfoma.
Sitokinlarni chiqarish sindromi (CRS) isitma, past qon bosimi, nafas olish qiyinlishuvi, ko'ngil aynishi va toshmalar kabi alomatlarni keltirib chiqaradigan CAR T-hujayrali terapiyasining eng keng tarqalgan yon ta'siridan biridir. Ko'pgina hodisalar engil, ammo og'ir holatlar hayot uchun xavfli bo'lishi mumkin.
Nevrologik nojo'ya ta'sirlarOrientatsiya, tutilishlar yoki gapirish va tushunishda qiyinchiliklar og'ir bo'lishi mumkin va tez tibbiy aralashuvni talab qiladi.
Boshqa jiddiy yon ta'sirlar: Bularga infuziya paytida allergik reaktsiyalar, infektsiya xavfini oshiradigan immunitet tizimining zaiflashishi, qon hujayralari sonining pastligi va uzoq muddatli nevrologik tizimning potentsial oqibatlari kiradi.
To summarize, while CAR T-cell therapy provides significant benefits in terms of high response rates and personalized treatment for multiple myeloma patients, it also carries risks such as cytokine release syndrome and neurological side effects, which must be carefully monitored and managed by healthcare providers. Close monitoring after therapy is required to maintain patient safety and optimize treatment natijalar.
Kelajak istiqbollari
Xitoy biomedikal tadqiqotlarga katta sarmoya kiritishda davom etar ekan, CAR T-hujayralarini davolash sohasi keskin o'sishi kutilmoqda. Xitoy Milliy Tibbiy Mahsulotlar Boshqarmasi (NMPA) equecabtagene autoleuselni ham yutuq terapiyasi, ham etim dori sifatida belgilash orqali CAR T-hujayralarini davolash va'dasini tan oldi. Ushbu ma'qullash Xitoy hukumatining ilg'or tibbiy yutuqlarni rivojlantirishga sodiqligini ko'rsatadi.
Shuningdek, ekkabtagen autoleucel neyromielit optik spektr kasalligi uchun mumkin bo'lgan davolash usuli sifatida ko'rib chiqilmoqda. Bu shuni ko'rsatadiki, CAR T-hujayrali terapiya saratondan tashqari boshqa narsalar uchun ham qo'llanilishi mumkin.
With continuous investment in infrastructure, education, and collaborative relationships, China is well-positioned to lead the way in CAR T-hujayra terapiyasi for multiple myeloma and other life-threatening diseases.
Xitoyda ko'p miyelom uchun CAR T-Cell terapiyasining narxi qancha?
Ko'p miyelom uchun CAR T-Cell terapiyasining narxi tanlangan CAR T Cell terapiyasi turiga va tanlangan shifoxonaga bog'liq. Turli shifoxonalarda FUCASO davolash narxi taxminan 200,000 200 AQSh dollarini tashkil qiladi. CILTA-CEL terapiyasining narxi ham 250,000-XNUMX XNUMX AQSh dollari orasida o'zgarib turadi. Biroq, bemorlar ham tanlashlari mumkin CAR T-Cell terapiyasining klinik sinovlari 60-80,000 XNUMX AQSh dollari orasida turadi.
