Hāʻawi ka lāʻau lapaʻau CD19/BCMA CAR-T pālua a BIOOCUS Medical Group i nā hopena hoʻohiki i ka Myasthenia Gravis Refractory: He huakaʻi a kahi maʻi ʻōpio
ʻO Myasthenia Gravis (MG) kahi maʻi neuromuscular autoimmune mau loa i hoʻokumu ʻia e nā autoantibodies (ʻo ia hoʻi nā antibodies receptor anti-acetylcholine, AchRAb) e hoʻopilikia ana i ka pilina o ke aʻalolo-ʻiʻo, e alakaʻi ana i ka nāwaliwali loli, ptosis, dysphagia, a i nā hihia koʻikoʻi e hoʻoweliweli ana i ka hanu ʻana. No nā poʻe maʻi me ka MG refractory i pane ʻole i nā immunosuppressants maʻamau, IVIG, FcRn inhibitors, a i ʻole nā mea hoʻohui complement, ua kaupalena ʻia nā koho lapaʻau - a hiki i kēia manawa.
ʻO ka lāʻau lapaʻau dual-target CD19/BCMA CAR-T a BIOOCUS Medical Group e hōʻike ana i kahi holomua autologous cell therapy e hoʻopau pololei ana i nā cell B pathogenic a me nā cell plasma kuleana no ka hana autoantibody. Hāʻawi kēia ʻano dual-target i ka hiki ke hoʻihoʻi hohonu a mau loa ma o ka hoʻoponopono ʻana i nā ʻāpana cellular a me humoral o ka pane autoimmune.
Hōʻike kahi hihia hoʻohālikelike i ka ʻōlelo hoʻohiki a ka lāʻau lapaʻau. Ua ʻike ʻia kahi maʻi wahine he 20 makahiki, i kapa ʻia ʻo LYN, me ka Myasthenia Gravis refractory (CNGIIIa, AchRAb-positive) a me ka post-surgical B3-type thymoma/thymic hyperplasia. Loaʻa iā ia nā maʻi like ʻole e like me ka osteoporosis, ka bilateral femoral head a me ka left proximal tibial avascular necrosis, a me ka iron-deficiency anemia. ʻOiai he nui nā laina lāʻau lapaʻau mai 2019 a 2025—me nā steroids kiʻekiʻe me ka azathioprine, tacrolimus, cyclosporine, repeated IVIG, efgartigimod, eculizumab me ka telitacicept, a me rituximab (500 mg)—ua mau kona kūlana me ka ʻole o ka hoʻomalu ʻia.
I Nowemapa 2025, ua hoʻomaka ʻia ke kaʻina hana CAR-T a ua hoʻōki ʻia ka cyclosporine. Ua hōʻiliʻili ʻia nā hunaola mononuclear koko peripheral ma Dekemaba 1, 2025. Ma Dekemaba 15, 2025, ua loaʻa iā ia kahi infusion o CD19 CAR-T (0.5 × 10⁶ / kg) me BCMA CAR-T (1 × 10⁶ / kg). ʻAʻohe hanana maikaʻi ʻole i ʻike ʻia ma hope o ka infusion.
Ma Malaki 2026, ua hōʻike nā loiloi hahai i ka hoʻonui wikiwiki ʻana o CAR-T in-vivo, me ka piko CD19 CAR-T i ka lā 10 (7.62%, 4.622 × 10⁷ / L) a me BCMA CAR-T i ka lā 10 (17.17%, 1.0414 × 10⁸ / L), i hoʻomau ʻia a hiki i ka mahina 3. ʻO nā hoʻomaikaʻi ʻana o ka hale hana i hoʻokomo i ka emi nui ʻana o nā pae AchRAb a me ka helu B-lymphocyte holoʻokoʻa, me nā helu koko paʻa, ka hana ate a me nā puʻupaʻa. Ma ke ʻano lapaʻau, ua hoʻopaʻa ʻia nā loaʻa koʻikoʻi ma Quantitative Myasthenia Gravis (QMG) a me nā helu Activities of Daily Living (ADL). ʻO nā hōʻailona e like me ka diplopia, ka ʻoʻoleʻa o ka maka, ka dysarthria, a me ka luhi o nā lālā i hōʻike i ka hoʻonā ʻana a i ʻole ka hoʻomaikaʻi nui ʻana.
Hōʻike kēia hihia i ka hiki ke hoʻololi i ka lāʻau lapaʻau dual-target CD19/BCMA CAR-T a BIOOCUS Medical Group i ka hoʻokō ʻana i ka hoʻonohonoho hou ʻana o ka pale kino a me ka pono lapaʻau koʻikoʻi i ka MG refractory. Ua ʻae maikaʻi ʻia ka lāʻau lapaʻau, me ka ʻike ʻole ʻia o CRS a i ʻole neurotoxicity, a paʻa i ka hoʻohiki no ke kala ʻana no ka wā lōʻihi me ka hōʻemi ʻana i ka pono no ka immunosuppression mau loa.