Ka holomua i ka maʻi Autoimmune Pediatric: Hoʻōla ka CAR-T Cell Therapy i ka mea maʻi Lupus

I Iune 2023, ua loaʻa iā Uresa, 15 makahiki, ka lāʻau lapaʻau CAR-T cell ma ka Halemai o ke Kulanui ʻo Erlangen, ʻo ia ka hoʻohana mua ʻana o kēia lāʻau lapaʻau hou e hoʻolohi i ka holomua o ka systemic lupus erythematosus (SLE), kahi maʻi autoimmune koʻikoʻi. I hoʻokahi makahiki ma hope mai, ua olakino ʻo Uresa e like me ka wā ma mua, koe wale nō kekahi mau anu liʻiliʻi.

ʻO Uresa ke keiki mua i mālama ʻia no ka SLE me Hoʻōla lāʻau pale ma ke Kikowaena Kelemania no ka Immunotherapy (DZI) o ke Kulanui ʻo Erlangen. Ua paʻi ʻia ka holomua o kēia lāʻau lapaʻau pilikino ma The Lancet.

Ua wehewehe ʻo Kauka Tobias Krickau, he kauka rheumatologist pediatric ma ka ʻOihana Pediatrics a me ka lāʻau lapaʻau ʻōpio o ka Halemai o ke Kulanui ʻo Erlangen, i ke ʻano kūikawā o ka hoʻohana ʻana i nā pūnaewele CAR-T e mālama ai Nā maʻi AutoimmuneMa mua, ua ʻāpono ʻia ka lāʻau lapaʻau CAR-T no kekahi mau maʻi ʻaʻai koko holomua wale nō.

Ma hope o ka hiki ʻole ʻana o nā lāʻau lapaʻau ʻē aʻe ke kāohi i ka SLE e ʻoi aʻe ana o Uresa, ua kū ka hui noiʻi i kahi hoʻoholo paʻakikī: pono anei e hoʻohana ʻia kēia mau pūnaewele pale ʻenekinia no kahi keiki me kahi maʻi autoimmune? ʻAʻole i ʻike mua ʻia ka pane, ʻoiai ʻaʻohe mea i hoʻāʻo i ka mālama ʻana iā CAR-T no nā maʻi autoimmune pediatric ma mua.

ʻO ka lāʻau lapaʻau CAR-T cell e pili ana i ka unuhi ʻana i kekahi o nā cell immune (T cells) o ka mea maʻi, e hoʻolako iā lākou me nā chimeric antigen receptors (CAR) i loko o kahi lab maʻemaʻe kūikawā, a laila e hoʻokomo hou i kēia mau cell i hoʻololi ʻia i loko o ka mea maʻi. Ke kaʻapuni nei kēia mau cell CAR-T i loko o ke koko, e kuhikuhi ana a luku ana i nā cell B autoreactive (ʻino).

Ua hoʻomaka nā hōʻailona o Uresa i ka hāʻulelau o 2022, me ka migraines, ka luhi, ka ʻeha o nā hono a me nā ʻiʻo, a me ka ʻulaʻula o ka maka—nā hōʻailona maʻamau o ka lupus. ʻOiai ua mālama nui ʻia, ua ʻoi aku ka ʻino o kona kūlana, e hoʻopilikia ana i kona mau puʻupaʻa a me ke kumu o nā pilikia koʻikoʻi.

I ka hoʻomaka ʻana o 2023, ma hope o nā hoʻokipa ʻia ʻana a me nā lāʻau lapaʻau he nui, me ka chemotherapy immunosuppressive a me ka plasma exchange, ua hōʻino ke kūlana o Uresa a hiki i kahi e pono ai ʻo ia e dialysis. Ma muli o ka hoʻokaʻawale ʻia mai nā hoaaloha a me ka ʻohana, ua hāʻule nui kona ʻano ola.

Ua ʻae ka hui lapaʻau ma ke Kulanui ʻo Erlangen, i alakaʻi ʻia e Polofesa Mackensen, e hana a hoʻohana i nā pūnaewele CAR-T no Uresa ma hope o nā kūkākūkā kikoʻī. Ua hoʻomaka ʻia kēia hoʻohana aloha o ka lāʻau lapaʻau CAR-T ma lalo o ke kānāwai lāʻau lapaʻau a Kelemania a me nā lula hoʻohana aloha.

ʻO ka papahana hoʻōla cell CAR-T ma Erlangen, i alakaʻi ʻia e Polopeka Georg Schett lāua ʻo Polopeka Mackensen, ua mālama nei i nā maʻi me nā maʻi autoimmune like ʻole, me SLE, mai ka makahiki 2021. Ua paʻi ʻia ko lākou holomua me 15 mau maʻi ma ka New England Journal of Medicine i Pepeluali 2024, a ke hana nei lākou i ke aʻo ʻana ʻo CASTLE me 24 mau mea komo, e hōʻike ana i nā hoʻomaikaʻi koʻikoʻi.

I mea e hoʻomākaukau ai no ka lāʻau lapaʻau CAR-T cell, ua hana ʻo Uresa i ka chemotherapy haʻahaʻa e hana i kahi no nā cell CAR-T i loko o kona koko. Ma Iune 26, 2023, ua loaʻa iā Uresa kāna mau cell CAR-T pilikino. I ke kolu o ka pule ma hope o ka mālama ʻana, ua hoʻomaikaʻi ʻia kāna hana puʻupaʻa a me nā hōʻailona lupus, a ua nalowale mālie kāna mau hōʻailona.

Ua pili ke kaʻina hana lapaʻau i ka hoʻonohonoho pono ʻana e hōʻoia i ka pono o ka chemotherapy a me ka pale ʻana i ke koena o ka hana puʻupaʻa. Ua ʻike wale ʻo Uresa i nā hopena ʻaoʻao liʻiliʻi a ua hoʻokuʻu ʻia ʻo ia i ka lā 11 ma hope o ka mālama ʻana.

I ka hopena o Iulai 2023, ua hoʻi ʻo Uresa i ka home, hoʻopau i kāna mau hoʻokolohua, a hoʻonohonoho i nā pahuhopu hou no kona wā e hiki mai ana, me ke kūʻokoʻa a me ka loaʻa ʻana o kahi ʻīlio. Ua hauʻoli ʻo ia i ka launa hou ʻana me nā hoaaloha a hoʻomaka hou i kahi ola ʻōpio maʻamau.

Ua wehewehe ʻo Polofesa Mackensen he nui nō nā pūnaewele CAR-T o Uresa i loko o kona koko, ʻo ia hoʻi, pono ʻo ia i nā infusions antibody i kēlā me kēia mahina a hiki i ka hoʻōla ʻana o kāna mau pūnaewele B. Ua hoʻoikaika ʻo Kauka Krickau ʻo ka holomua o ka mālama ʻana o Uresa ma muli o ka laulima kokoke ʻana o nā ʻano lapaʻau like ʻole ma ke Kikowaena Kelemania no ka Immunotherapy.

ʻAʻole pono hou ʻo Uresa i kekahi lāʻau lapaʻau a i ʻole dialysis, a ua hoʻōla piha ʻia kona mau puʻupaʻa. Ke hoʻolālā nei ʻo Kauka Krickau a me kāna hui i nā haʻawina hou aʻe e ʻimi i ka hiki o nā cell CAR-T i ka mālama ʻana i nā maʻi autoimmune pediatric ʻē aʻe.

Hōʻike kēia hihia koʻikoʻi i ka hiki o ka lāʻau lapaʻau CAR-T cell e hāʻawi i ke kala ʻana no ka wā lōʻihi no nā maʻi pediatric me nā maʻi autoimmune koʻikoʻi e like me SLE. Hōʻike ka holomua o ka lāʻau a Uresa i ke koʻikoʻi o ke komo mua ʻana a me ka laulima multidisciplinary. Pono nā haʻawina lapaʻau hou aʻe e hōʻoia i ka palekana a me ka pono o ka lāʻau lapaʻau CAR-T cell no nā keiki me nā maʻi autoimmune.