ʻO ka lāʻau lapaʻau CAR-T no ka Myasthenia Gravis (MG)

ʻO Myasthenia Gravis (MG) kahi maʻi neuromuscular autoimmune mau loa i hoʻokumu ʻia e nā autoantibodies (ʻo ia hoʻi nā anti-acetylcholine receptor antibodies, AchRAb) e hoʻopilikia ana i ka kamaʻilio ʻana ma waena o nā aʻalolo a me nā 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 e hāʻule ana i nā immunosuppressants maʻamau, IVIG, FcRn inhibitors, a me nā complement inhibitors, ua kaupalena ʻia nā koho lapaʻau.

ʻO ka lāʻau lapaʻau dual-target CD19/BCMA CAR-T a BIOOCUS ke hōʻike nei i kahi lāʻau lapaʻau autologous cell holomua e hoʻopau pololei i nā cell B pathogenic a me nā cell plasma ke kuleana no ka hana ʻana o autoantibody, e hāʻawi ana i ka hiki ke kala hohonu a mau loa i ka MG paʻakikī ke mālama.

ʻO ka lāʻau lapaʻau CAR-T no MG he immunotherapy holomua a pilikino kahi i hana ʻia ai nā pūnaewele T ponoʻī o ka mea maʻi e hōʻike i nā mea loaʻa antigen chimeric (CAR) e kuhikuhi ana i ka CD19 (ma nā pūnaewele B) a me BCMA (ma nā pūnaewele plasma). Ma hope o ka hoʻonui ʻia ʻana i loko o ka hale hana, ua hoʻokomo hou ʻia kēia mau pūnaewele CAR-T pālua i loko o ka mea maʻi, kahi e hoʻopau koho ai lākou i nā pūnaewele pale autoreactive e hoʻokele ana iā MG, no laila e hōʻemi ana i nā pae AchRAb a hoʻihoʻi i ka hana neuromuscular.

Hoʻoponopono kēia ala pālua-pahuhopu i nā ʻāpana cellular a me humoral o ka pane autoimmune, e hāʻawi ana i kahi hoʻonohonoho hou ʻana o ka ʻōnaehana pale i hoʻohālikelike ʻia me nā hoʻōla pākahi-pahuhopu a i ʻole nā ​​​​​​lāʻau lapaʻau kuʻuna.

• Hoʻomaikaʻi wikiwiki a hoʻomau i nā hōʻailona: Ka emi nui ʻana o ka luhi o nā ʻiʻo, ptosis, diplopia, dysarthria, a me ka nāwaliwali o nā lālā.
• Ka hoʻopau hohonu ʻana o nā cell B a me nā cell plasmaʻO ka emi ʻana i ʻike nui ʻia a mau loa hoʻi ma ka helu ʻana o AchRAb a me ka huina helu B-lymphocyte.
• Nā helu lapaʻau i hoʻomaikaʻi ʻiaNā loaʻa nui ma nā helu Quantitative Myasthenia Gravis (QMG) a me nā hana o ke ola o kēlā me kēia lā (ADL).
• ʻIke palekana maikaʻiHoʻomanawanui maikaʻi ʻia me ka ʻike ʻole ʻia o ka cytokine release syndrome (CRS) a i ʻole neurotoxicity i nā hihia i mālama ʻia.
• Ka hiki ke kala ʻia no ka wā lōʻihiHoʻēmi a hoʻopau paha i ka pono no ka pale ʻana i ka maʻi pale mau loa.
• Hoʻopilikino ʻia a pololeiHoʻohana i nā pūnaewele ponoʻī o ka mea maʻi, e hoʻemi ana i ka pilikia o ka hōʻole ʻia.

1. Leukapheresis– Ka hōʻiliʻili ʻana o nā pūnaewele T o ka mea maʻi ma o ka apheresis koko peripheral.
2. ʻEnekinia Genetic– Hoʻololi ʻia nā pūnaewele T e hōʻike i nā CD19 a me BCMA CARs a hoʻonui ʻia i loko o nā hale GMP.
3. Hoʻomaʻamaʻa (inā pono)– ʻO ka lymphodepletion pōkole e hoʻomaikaʻi i ka engraftment CAR-T.
4. Hoʻokomo ʻia ʻo CAR-T– Hoʻokomo ʻia hoʻokahi o CD19 CAR-T (0.5×10⁶/kg) + BCMA CAR-T (1×10⁶/kg).
5. Nānā a me ka Hahai ʻana– Loiloi mau ʻia o ka hoʻonui ʻana o CAR-T, nā pae AchRAb, nā helu o nā cell-B, nā helu QMG/ADL, a me ka hana o nā ʻōkana a hiki i 12+ mahina.

• Ka paepae CD19/BCMA CAR-T pālua ʻoi loa i hoʻonohonoho pono ʻia no nā maʻi autoimmune.
• ʻO ka hui hana GMP papa honua a me ka hui immunotherapy akamai me ka ʻike loea i nā malignancies hematologic a me nā kūlana autoimmune refractory.
• Kākoʻo piha no nā mea maʻi honua: ka hoʻonohonoho ʻana i nā ʻōlelo he nui, ka hoʻoili ʻana ma ke kahua mokulele, ka noho ʻana, a me ka hahai ʻana i ka wā lōʻihi.
• Ka moʻolelo i hōʻoia ʻia ma CAR-T no nā maʻi autoimmune (me ka SLE) a me ka hoʻolālā mālama pilikino.
• Mālama piha-ʻano mai ka loiloi mua-lāʻau a hiki i ka nānā ʻana ma hope o ka hoʻokomo ʻana.