Ukwelashwa kwe-BioOCUS Medical Group's Dual CD19/BCMA CAR-T Kuletha Imiphumela Ethembisayo ku-Refractory Myasthenia Gravis: Uhambo Lwesiguli Esisesincane
I-Myasthenia Gravis (MG) iyisifo esingapheli se-autoimmune neuromuscular esibangelwa ama-autoantibodies (ikakhulukazi ama-anti-acetylcholine receptor antibodies, i-AchRAb) aphazamisa ukuxhumana kwezinzwa nemisipha, okuholela ekubuthakathakeni okushintshashintshayo, i-ptosis, i-dysphagia, kanye nasezimweni ezimbi kakhulu ukwehluleka kokuphefumula okusongela impilo. Ezigulini ezine-MG engaphikisiyo ezingazange zisabele kuma-immunosuppressants avamile, ama-IVIG, ama-FcRn inhibitors, noma ama-complement inhibitors, izinketho zokwelapha zilinganiselwe—kuze kube manje.
Ukwelashwa kwe-BIOOCUS Medical Group okuqondiswe kabili kwe-CD19/BCMA CAR-T kumelela ukwelashwa kwamaseli e-autologous okuphumelelayo okuqeda ngokunembile amaseli e-B abangela izifo kanye namaseli e-plasma abangela ukukhiqizwa kwama-autoantibody. Le ndlela yokuqondiswe kabili inikeza ithuba lokuxolelwa okujulile nokuhlala isikhathi eside ngokubhekana nezingxenye zamaseli kanye nezama-humoral zempendulo yokuzivikela komzimba.
Icala elimele liqokomisa isithembiso sokwelashwa. Isiguli sesifazane esineminyaka engu-20 ubudala, esibizwa ngokuthi i-LYN, satholakala sine-Myasthenia Gravis engaphikisi (i-CNGIIIa, i-AchRAb-positive) kanye ne-B3-type thymoma/thymic hyperplasia yangemva kokuhlinzwa. Sasinezinkinga ezihambisana naso ezihlanganisa i-osteoporosis, ikhanda le-femoral elihlangene kanye ne-left proximal tibial avascular necrosis, kanye ne-iron-deficiency anemia. Naphezu kwezindlela eziningi zokwelapha kusukela ngo-2019 kuya ku-2025—kufaka phakathi ama-steroid anesilinganiso esiphezulu kanye ne-azathioprine, i-tacrolimus, i-cyclosporine, i-IVIG ephindaphindwayo, i-efgartigimod, i-eculizumab kanye ne-telitacicept, kanye ne-rituximab (500 mg)—isimo sakhe asizange silawulwe.
NgoNovemba 2025, kwaqalwa inqubo ye-CAR-T futhi i-cyclosporine yamiswa okwesikhashana. Amaseli egazi angaphandle aqoqwe ngoDisemba 1, 2025. NgoDisemba 15, 2025, wathola ukumnika i-CD19 CAR-T (0.5×10⁶/kg) kanye ne-BCMA CAR-T (1×10⁶/kg). Azikho izehlakalo ezimbi ezabonwa ngemuva kokumnika.
NgoMashi 2026, ukuhlolwa kokulandelela kwabonisa ukwanda okusheshayo kwe-CAR-T ngaphakathi kwe-vivo, kanye ne-CD19 CAR-T ephezulu ngosuku lwe-10 (7.62%, 4.622×10⁷/L) kanye ne-BCMA CAR-T ngosuku lwe-10 (17.17%, 1.0414×10⁸/L), okuqhubeke kuze kube yinyanga yesi-3. Ukuthuthuka kwelabhorethri kwakuhlanganisa ukwehla okukhulu kwamazinga e-AchRAb kanye nokubalwa kwe-B-lymphocyte iyonke, ngokubalwa kwegazi okuzinzile, ukusebenza kwesibindi nezinso. Ngokwezokwelapha, izinzuzo eziphawulekayo zabhalwa ku-Quantitative Myasthenia Gravis (QMG) kanye ne-Activities of Daily Living (ADL). Izimpawu ezifana ne-diplopia, ukuqina kobuso, i-dysarthria, kanye nokukhathala kwemilenze kwabonisa ukuxazulula noma ukuthuthuka okuphawulekayo.
Leli cala libonisa amandla okuguqula ukwelashwa kwe-BIOOCUS Medical Group okuqondiswe kabili kwe-CD19/BCMA CAR-T ekufinyeleleni ukuvuselelwa okujulile komzimba kanye nenzuzo yezokwelapha enenjongo ku-MG engaphikisi. Ukwelashwa kubekezelelwa kahle, kungekho CRS noma ubuthi be-neurological obubonakalayo, futhi kunesithembiso sokuphumula kwesikhathi eside ngenkathi kunciphisa isidingo sokucindezelwa kwamasosha omzimba okungapheli.