Yuli 2023: Shaidu daga ƙaramin gwaji na asibiti sun nuna cewa myasthenia gravis, cuta ce mai saurin kamuwa da cuta ta tsarin jijiya, ana iya bi da ita tare da bambancin CAR-T, ingantaccen rigakafin cutar kansar jini. Maganin CAR-T da aka gyara, wanda ke nufin chimeric antigen receptor T-cell, masana kimiyya sun yi amfani da shi. Zai iya rage alamun myasthenia gravis na dogon lokaci kuma bai haifar da wani babban tasiri ba. Binciken, wanda aka buga a cikin The Lancet Neurology, an biya shi ne ta hanyar karamin tallafin kasuwanci daga Cibiyar Kula da Cututtukan Jiki da Shanyewar Jiki (NINDS), wanda ke cikin Cibiyar Kiwon Lafiya ta Kasa. An ba da tallafin ga Gaithersburg, kamfanin Cartesian Therapeutics na Maryland.
Using a groundbreaking therapy like CAR-T to potentially treat a neurological disorder shows how flexible immunotherapy can be when there are few or no other treatment options,” said Emily Caporello, Ph.D., head of the NINDS Small Business Programme.
Myasthenia gravis cuta ce ta autoimmune na dogon lokaci wacce ke faruwa lokacin da tsarin garkuwar jiki ya kai hari ga furotin da aka samu inda ƙwayoyin jijiya ke magana da tsoka. Cutar tana da raunin tsoka wanda ke kara muni idan mutum yana aiki kuma wani lokacin yana samun sauki idan mutum ya huta. Babban burin jiyya da muke da shi yanzu shine don rage bayyanar cututtuka, musamman raunin tsoka.
A cikin binciken, an ba wa mutane 14 da ke da ƙwayar cuta ta myasthenia gravis nau'i daban-daban na Descartes-08, wani nau'i na maganin CAR-T wanda aka gyara wanda ya shafi kwayoyin halitta da ke haifar da ƙwayoyin cuta na myasthenia gravis. An gano mafi kyawun kashi shine kwaya ɗaya sau ɗaya a mako har tsawon makonni shida. Bayanan farko game da yadda maganin ke aiki yana ƙarfafawa, amma ana buƙatar ƙarin gwaje-gwaje na asibiti don sanin yadda yake aiki. Mutane uku da suka ɗauki Descartes-08 sun sami duka ko kusan dukkanin alamun su sun tafi. Wadannan tasirin sun kasance na tsawon watanni shida bayan jiyya. Wasu biyu ba sa buƙatar magani tare da immunoglobulin na ciki wanda ake ba wa wasu mutane masu MG mai tsanani.
Murat V. Kalayoglu, MD, Ph.D., shugaban kasa da kuma Shugaba na Cartesian Therapeutics, ya ce, "Mun ga zurfin, dogon martani ga Decartes-08 wanda ya kasance akalla watanni shida bayan jiyya." "Yanzu mun fara wani babban bazuwar, binciken da ake sarrafa wuribo, wanda shine irinsa na farko don ingantacciyar hanyar maganin ƙwayoyin cuta."
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 cutar kansa, 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.
Yawancin maganin rigakafi, ciki har da CAR-T, na iya haifar da manyan lahani waɗanda, yayin da ake iya jurewa a lokuta na ciwon daji na ci gaba, ya sa ba zai yiwu a yi amfani da shi ba a lokuta na myasthenia gravis da sauran yanayi na dogon lokaci. Kwayoyin T galibi ana canza su ta DNA, wanda ke zama a cikin tantanin halitta kuma ana kwafi duk lokacin da tantanin halitta ya rabu. Wannan na iya sa aikin ya fi ƙarfin kuma ya haifar da illa mai haɗari.
Descartes-08 baya amfani da DNA don canza T-cell saboda DNA tana kwafin kanta lokacin da sel suka rarraba. Madadin haka, yana amfani da manzo RNA (mRNA), wanda baya kwafin kanta lokacin da sel suka rarraba. Sakamakon shine ɗan gajeren lokacin jiyya wanda aka ba da fiye da sau ɗaya maimakon kashi ɗaya, wanda shine yadda tsarin CAR-T na DNA yakan yi aiki. Babban makasudin wannan binciken shine don nemo madaidaicin kashi na Descartes-08 don rage alamun rauni na tsoka yayin da yake haifar da ƙarancin illa kamar yadda zai yiwu.
In a larger gwajin gwaji, 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.