Julayi 2023: Ubufakazi obuvela esivivinyweni somtholampilo esincane bubonisa ukuthi i-myasthenia gravis, ukuphazamiseka kwe-autoimmune yesistimu yezinzwa, ingelashwa ngokuhlukahluka kwe-CAR-T, ukwelapha okuthuthukisiwe komdlavuza wegazi. Ukwelashwa okuguquliwe kwe-CAR-T, okumele i-chimeric antigen receptor T-cell, kwasetshenziswa ososayensi. Kunganciphisa izimpawu ze-myasthenia gravis isikhathi eside futhi akuzange kubangele noma yimiphi imiphumela emibi kakhulu. Lolu cwaningo, olwashicilelwa kuThe Lancet Neurology, lukhokhelwe uxhaso lwamabhizinisi amancane oluvela kuNational Institute of Neurological Disorders and Stroke (NINDS), eyingxenye yeNational Institutes of Health. Isibonelelo sinikezwe iGaithersburg, inkampani ezinze eMaryland iCartesian Therapeutics.
Using a groundbreaking therapy like CAR-T to potentially treat a neurological disorder shows how flexible ama-immunotherapies can be when there are few or no other treatment options,” said Emily Caporello, Ph.D., head of the NINDS Small Business Programme.
I-Myasthenia gravis yisifo se-autoimmune sesikhathi eside esenzeka lapho amasosha omzimba ehlasela iphrotheni etholakala lapho amangqamuzana ezinzwa ekhuluma nemisipha. Lesi sifo sibonakala ngokuba buthaka kwemisipha okuba kubi kakhulu uma umuntu ematasa futhi kwesinye isikhathi uba ngcono uma umuntu ephumule. Umgomo oyinhloko wezokwelapha esinazo manje ukunciphisa izimpawu, ikakhulukazi ubuthakathaka bemisipha.
Ocwaningweni, abantu abayi-14 abane-myasthenia gravis ejwayelekile banikezwe amanani ahlukene e-Descartes-08, uhlobo olushintshiwe lokwelapha i-CAR-T oluqondise kumaseli enza amasosha omzimba abangela i-myasthenia gravis. Umthamo ongcono kakhulu watholakala ukuthi iphilisi elilodwa kanye ngesonto amasonto ayisithupha. Ulwazi lwangaphambi kwesikhathi mayelana nokuthi ukwelashwa kusebenza kahle kangakanani luyakhuthaza, kodwa ukuhlolwa okwengeziwe komtholampilo kuyadingeka ukuze kutholwe ukuthi kusebenza kahle kangakanani. Abantu abathathu abathathe i-Descartes-08 babenazo zonke noma cishe zonke izimpawu zabo zaphela. Le miphumela yahlala izinyanga eziyisithupha ngemva kokwelashwa. Abanye ababili babengasakudingi ukwelashwa nge-intravenous immunoglobulin enikezwa abantu abathile abane-MG eyingozi.
UMurat V. Kalayoglu, MD, Ph.D., umongameli kanye ne-CEO yeCartesian Therapeutics, uthe, "Sibone izimpendulo ezijulile, ezihlala isikhathi eside ku-Decartes-08 ezithathe okungenani izinyanga eziyisithupha ngemuva kokwelashwa." "Manje sesiqale ucwaningo olukhudlwana olungahleliwe, olulawulwa yi-placebo, okungelokuqala lwayo uhlobo lokwelashwa kwamaseli okutholwa okunjiniyela."
In CAR-T therapy, a patient’s T-cells are reprogrammed to fight a specific target. T-cells are a key part of the immune system that can find and kill invading pathogens. With umdlavuza wegazi, the cancer itself is now the new target. For myasthenia gravis, the goal is to kill the bad cells that make the antibodies that cause damage.
Ama-immunotherapies amaningi, kuhlanganise ne-CAR-T, angabangela imiphumela emibi kakhulu, nakuba ibekezelelwa ezimweni zomdlavuza ophakeme, yenza kube nzima ukusebenzisa ezimweni ze-myasthenia gravis nezinye izimo zesikhathi eside. Ama-T-cell ngokuvamile ashintshwa yi-DNA, ehlala kuseli futhi ikopishwa njalo lapho iseli lihlukana. Lokhu kungenza isenzo sibe namandla futhi kubangele imiphumela emibi eyingozi.
I-Descartes-08 ayisebenzisi i-DNA ukushintsha ama-T-cell ngoba i-DNA iyazikopisha lapho amaseli ehlukana. Kunalokho, isebenzisa i- messenger RNA (mRNA), engazikopishi lapho amaseli ehlukana. Umphumela uba isikhathi esifushane sokwelashwa esinikezwa izikhathi ezingaphezu kwesisodwa esikhundleni somthamo owodwa, okuyindlela ngokuvamile yokwelapha ye-CAR-T ehlelwe yi-DNA. Umgomo oyinhloko walolu cwaningo kwakuwukuthola umthamo olungile we-Descartes-08 ukunciphisa izimpawu zobuthakathaka bemisipha ngenkathi kubangela imiphumela emibi embalwa ngangokunokwenzeka.
In a larger isivivinyo somtholampilo, Descartes-08 therapy is now being tried to see if it can help reduce the symptoms of myasthenia gravis. Importantly, there will also be a placebo group in this study. This is an important control to make sure that any improvement seen is due to the treatment and not something else.
