Ryoncil ti fọwọsi fun itọju ti sitẹriọdu-refractory acute graft-versus-host disease (SR-aGVHD), ipo idẹruba igbesi aye ti o le waye lati inu isopo sẹẹli stem ẹjẹ ti a ṣe lati rọpo awọn sẹẹli aibuku ti olugba ni awọn oriṣi awọn aarun ẹjẹ kan. , ẹjẹ rudurudu tabi ajẹsara eto. Eyi jẹ ki Ryoncil jẹ itọju ailera MSC akọkọ ti FDA-fọwọsi (Mesenchymal Stem Cell). Ryoncil ni awọn MSC ti o ya sọtọ lati inu ọra inu eegun ti awọn oluranlọwọ eniyan agbalagba ti o ni ilera eyiti o le ṣe iyatọ si ọpọlọpọ awọn iru awọn sẹẹli miiran. Awọn ifun inu iṣan ti awọn sẹẹli stromal mesenchymal (MSCs) pese awọn anfani iwalaaye.
Ní ọjọ́ kejìdínlógún oṣù Kejìlá ọdún 2024, FDA fọwọ́ sí Ryoncil (remestemcel-L-rknd), ìtọ́jú allogeneic (donor) egungun marrow-derived mesenchymal stromal cell (MSC) fún ìtọ́jú àrùn steroid-refractory acute graft-versus-host disease (SR-aGVHD) fún àwọn aláìsàn ọmọdé tí wọ́n wà ní oṣù méjì tàbí jù bẹ́ẹ̀ lọ. Èyí ló mú kí Ryoncil jẹ́ ìtọ́jú àkọ́kọ́ tí FDA fọwọ́ sí fún MSC (Mesenchymal Stem Cell).
Ryoncil ni awọn MSC ti o ya sọtọ lati inu ọra inu eegun ti awọn oluranlọwọ eniyan agbalagba ti o ni ilera eyiti o le ṣe iyatọ si ọpọlọpọ awọn iru awọn sẹẹli miiran.
Sitẹriọdu-refractory acute graft-versus-host disease (SR-aGVHD) jẹ ipo to ṣe pataki ati idẹruba igbesi aye ti o le ni pataki, awọn abajade ilera jakejado, pẹlu ibajẹ si awọn ẹya ara pupọ, dinku didara igbesi aye ati eewu iku ni ipa kan. alaisan. Eyi le waye bi ilolu ti hematopoietic allogeneic hematopoietic (ẹjẹ) stem cell asopo (allo-HSCT).
Ni allo-HSCT, alaisan kan gba awọn sẹẹli hematopoietic hematopoietic lati ọdọ oluranlọwọ ti o ni ilera lati rọpo awọn sẹẹli ti ara wọn ati ṣe awọn sẹẹli ẹjẹ tuntun, ilana ti a ṣe nigbagbogbo gẹgẹbi apakan ti itọju fun awọn iru awọn aarun ẹjẹ kan, awọn rudurudu ẹjẹ tabi awọn eto ajẹsara.
Ifọwọsi ti Ryoncil tẹle igbelewọn ti aabo ati imunadoko rẹ ni ile-iṣẹ lọpọlọpọ, ikẹkọ apa-ẹyọkan ti o kan awọn olukopa ọmọ wẹwẹ 54 pẹlu SR-aGVHD ti o ti ṣe allo-HSCT. Awọn olukopa gba apapọ awọn infusions iṣọn-ẹjẹ mẹjọ ti Ryoncil lẹmeji ni ọsẹ fun ọsẹ mẹrin ni itẹlera. Iwadii ti imunadoko da nipataki lori oṣuwọn ati iye akoko idahun si itọju awọn ọjọ 28 lẹhin ibẹrẹ Ryoncil.
Ninu idanwo miiran fun SR-aGVHD lẹhin allo-HSCT, a rii pe apapo awọn MSCs ati basiliximab (egboogi monoclonal kan ti a lo lati ṣe idiwọ ijusile asopo) yori si idahun pipe ti o dara julọ (CR) ju basiliximab nikan.
O ti ṣe akiyesi ni awọn idanwo ile-iwosan pe awọn infusions IV ti awọn sẹẹli stromal mesenchymal (MSCs) pese awọn anfani iwalaaye.
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To jo:
- Itusilẹ Awọn iroyin FDA – FDA fọwọsi Itọju Ẹjẹ Ẹjẹ Mesenchymal akọkọ lati tọju Sitẹriọdu-refractory Acute Graft-versus-host Arun. Ti firanṣẹ 18 Oṣu kejila 2024. Wa ni https://www.fda.gov/news-events/press-announcements/fda-approves-first-mesenchymal-stromal-cell-therapy-treat-steroid-refractory-acute-graft-versus-host
- Fu, H., Sun, X., Lin, R. et al. Awọn sẹẹli stromal Mesenchymal pẹlu basiliximab mu idahun ti sitẹriọdu-refractory ńlá alọmọ-lapa-ogun arun bi itọju ila-keji: ile-iṣẹ lọpọlọpọ, aileto, idanwo iṣakoso. BMC Med 22, 85 (2024). https://doi.org/10.1186/s12916-024-03275-5
- Kelly, K., Bloor, AJC, Griffin, JE et al. Awọn abajade ailewu ọdun meji ti iPS cell-ti ari awọn sẹẹli stromal mesenchymal ni sitẹriọdu-sooro sitẹriọdu nla ti alọmọ-olodi-ogun. Nat Med 30, 1556-1558 (2024). https://doi.org/10.1038/s41591-024-02990-z
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, a life-threatening condition that may result from blood stem cell transplantation performed to replace recipient's defective stem cells in certain types of blood cancers, blood disorders or immune system disorders. This makes Ryoncil the first FDA-approved MSC (Mesenchymal Stem Cell) therapy. Ryoncil contains MSCs isolated from the bone marrow of healthy adult human donors which can differentiate into multiple other types of cells. Intravenous infusions of mesenchymal stromal cells (MSCs) provide survival benefits.){kind=link}