Unyango lwe-BioOCUS Medical Group's Dual CD19/BCMA CAR-T luzisa iziphumo ezithembisayo kwi-Refractory Myasthenia Gravis: Uhambo lwesigulana esiselula
I-Myasthenia Gravis (MG) sisifo esingapheliyo se-autoimmune neuromuscular disorder esibangelwa zii-autoantibodies (ngokuyintloko ii-anti-acetylcholine receptor antibodies, i-AchRAb) eziphazamisa unxibelelwano lwemithambo-luvo nezihlunu, nto leyo ekhokelela ekuguquguqukeni kobuthathaka, i-ptosis, i-dysphagia, kwaye kwiimeko ezinzima ukusilela kokuphefumla okusongela ubomi. Kwizigulana ezine-MG enganyangekiyo ezingakhange ziphendule kwii-immunosuppressants eziqhelekileyo, ii-IVIG, ii-FcRn inhibitors, okanye ii-complement inhibitors, iindlela zonyango bezilinganiselwe—kude kube ngoku.
Unyango lwe-BIOOCUS Medical Group olujolise kwi-CD19/BCMA CAR-T luyindlela entsha yonyango lweeseli ze-autologous oluphelisa ngokuchanekileyo iiseli ze-B ezibangela izifo kunye neeseli ze-plasma ezinoxanduva lokuvelisa ii-autoantibodies. Le ndlela yokujongana neeseli ezimbini inika ithuba lokukhululwa okunzulu nokuhlala ixesha elide ngokujongana nenxalenye yeseli kunye neye-humoral yempendulo ye-autoimmune.
Ityala elimeleyo ligxininisa ithemba lonyango. Isigulana esibhinqileyo esineminyaka engama-20 ubudala, esibizwa ngokuba yi-LYN, safunyaniswa sine-Myasthenia Gravis enganyangekiyo (CNGIIIa, AchRAb-positive) kunye ne-B3-type thymoma/thymic hyperplasia emva kotyando. Wayenezifo ezichaphazela umzimba eziquka i-osteoporosis, intloko ye-femoral yamacala omabini kunye ne-left proximal tibial avascular necrosis, kunye ne-iron-deficiency anemia. Nangona unyango luninzi ukususela ngo-2019 ukuya ku-2025—kuquka ii-steroids ezinedosi ephezulu kunye ne-azathioprine, i-tacrolimus, i-cyclosporine, i-IVIG ephindaphindwayo, i-efgartigimod, i-eculizumab kunye ne-telitacicept, kunye ne-rituximab (500 mg)—imeko yakhe ayizange ilawuleke.
NgoNovemba ka-2025, kwaqaliswa i-CAR-T protocol kwaye i-cyclosporine yayekwa. Iiseli zegazi ezingaphandle kwe-peripheral mononuclear zaqokelelwa nge-1 kaDisemba ka-2025. Nge-15 kaDisemba ka-2025, wafumana i-CD19 CAR-T (0.5×10⁶/kg) kunye ne-BCMA CAR-T (1×10⁶/kg). Akukho ziganeko zimbi ezibonwe emva kokufakwa.
NgoMatshi 2026, uvavanyo olulandelayo lubonise ukwanda ngokukhawuleza kwe-CAR-T kwi-in-vivo, kunye ne-CD19 CAR-T ephezulu ngosuku lwe-10 (7.62%, 4.622×10⁷/L) kunye ne-BCMA CAR-T ngosuku lwe-10 (17.17%, 1.0414×10⁸/L), eqhubekekayo ukuya kwinyanga yesi-3. Uphuculo lwelabhoratri luquka ukwehla okukhulu kwamanqanaba e-AchRAb kunye nokubalwa kwe-B-lymphocyte iyonke, kunye nokubalwa kwegazi okuzinzileyo, ukusebenza kwesibindi kunye nezintso. Ngokwezonyango, ukuzuza okuphawulekayo kwarekhodwa kwi-Quantitative Myasthenia Gravis (QMG) kunye ne-Activities of Daily Living (ADL) scores. Iimpawu ezifana ne-diplopia, ukuqina kobuso, i-dysarthria, kunye nokudinwa kwamalungu kwabonisa ukuphuculwa okanye ukuphucuka okuphawulekayo.
Eli tyala libonisa amandla okuguqula indlela yonyango lwe-BIOOCUS Medical Group olujolise ngayo kwi-CD19/BCMA CAR-T ekufezekiseni ukubuyiselwa komzimba okunzulu kunye nenzuzo ebalulekileyo yeklinikhi kwi-MG enganyangekiyo. Olu nyango lunyanyezelwa kakuhle, kungekho CRS okanye ubuthi be-neurotoxicity obubonwayo, kwaye lunesithembiso sokukhululwa ixesha elide ngelixa kunciphisa isidingo sokucinezelwa kwamajoni omzimba okungapheliyo.