Luulyo 2023: Caddaynta tijaabada caafimaad ee cabbirka yar waxay muujinaysaa in myasthenia gravis, cillad difaaca jidhka ah ee habdhiska neerfaha, lagu daweyn karo kala duwanaanshiyaha CAR-T, immunotherapy kansarka dhiigga horumaray. Daawaynta CAR-T ee wax laga beddelay, oo u taagan unug-cell-cell-kimeric antigen, waxa isticmaalay saynisyahannadu. Waxay yarayn kartaa calaamadaha gravis-ka 'myasthenia gravis' muddo dheer mana aysan keenin waxyeelo weyn. Daraasaddan oo lagu daabacay wargeyska The Lancet Neurology, ayaa waxaa lagu bixiyay deeq ganacsi oo yar oo ka timid Machadka Qaranka ee Dhibaatooyinka Neerfaha iyo Stroke (NINDS), kaas oo qayb ka ah Machadyada Caafimaadka Qaranka. Deeqda waxaa la siiyay Gaithersburg, oo ah shirkadda Cartesian Therapeutics ee fadhigeedu yahay Maryland.
Using a groundbreaking therapy like CAR-T to potentially treat a neurological disorder shows how flexible daawoyinka difaaca jirka 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 waa cudur difaaca jirka oo muddo dheer socda kaasoo dhaca marka habka difaaca jirku uu weeraro borotiin laga helo halka unugyada dareemayaasha ay kula hadlaan murqaha. Cudurka waxaa lagu gartaa daciifnimo murqaha ah oo ka sii dara marka uu qofku firfircoon yahay oo mararka qaar ladnaado marka qofku nasto. Hadafka ugu weyn ee daawaynta aan hadda haysano waa in la yareeyo calaamadaha, gaar ahaan daciifnimada muruqa.
Daraasadda, 14 qof oo qaba xanuunka 'myasthenia gravis' ayaa la siiyay xaddi kala duwan oo ah Descartes-08, oo ah qaab la bedelay ee CAR-T therapy kaas oo bartilmaameedsada unugyada sameeya unugyada difaaca jirka ee keena gravis myasthenia. Qiyaasta ugu fiican waxaa la ogaaday in uu yahay hal kiniin todobaadkii hal mar muddo lix toddobaad ah. Macluumaadka hore ee ku saabsan sida wanaagsan ee daawadu u shaqeyso waa mid dhiirigelin leh, laakiin baaritaanno caafimaad oo badan ayaa loo baahan yahay si loo go'aamiyo sida ugu wanaagsan ee ay u shaqeyso. Saddex qof oo qaatay Descartes-08 ayaa dhammaan ama ku dhawaad dhammaan calaamadahoodu ka baxeen. Saamayntani waxay socotay muddo lix bilood ah daawaynta ka dib. Laba qof oo kale ayaan hadda u baahnayn daawaynta immunoglobulin-xididka ee la siiyo dadka qaarkiis ee qaba MG halis ah.
Murat V. Kalayoglu, MD, Ph.D., madaxweynaha iyo maamulaha Cartesian Therapeutics, ayaa yiri, "Waxaan aragnay jawaabo qoto dheer, jawaabo waara Decartes-08 oo socday ugu yaraan lix bilood ka dib daaweynta." "Waxaan hadda bilownay daraasad ballaaran oo aan kala sooc lahayn, oo lagu xakameynayo placebo, taas oo ah tii ugu horreysay ee nooceeda ah ee daaweynta unugyada korsashada."
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 kansarka dhiigga, 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.
Daawooyinka difaaca jirka oo badan, oo ay ku jiraan CAR-T, waxay sababi karaan waxyeelooyin waaweyn, iyadoo loo dulqaadan karo kiisaska kansarka sare, waxay ka dhigayaan mid aan suurtagal ahayn in la isticmaalo xaaladaha myasthenia gravis iyo xaaladaha kale ee muddada dheer. Unugyada T-ga waxaa badiyaa beddela DNA-da, kaas oo ku dhex jira unugga oo la koobiyeeyo mar kasta oo unugu kala qaybiyo. Tani waxay ka dhigi kartaa ficilka mid xoogan oo keeni kara saameyno khatar ah.
Descartes-08 uma isticmaasho DNA si ay u bedesho unugyada T-unugyada sababtoo ah DNA-da lafteeda ayaa koobiysa marka unugyadu qaybsadaan. Taa beddelkeeda, waxay isticmaashaa Messenger RNA (mRNA), taas oo aan lafteeda koobiyn marka unugyadu qaybsadaan. Natiijadu waa muddo daawaynta gaaban oo la bixiyo wax ka badan hal mar halkii laga siin lahaa hal qiyaas, taas oo ah sida daawaynta CAR-T ee DNA-du u shaqeyso sida caadiga ah. Hadafka ugu weyn ee daraasaddan ayaa ahayd in la helo qiyaasta saxda ah ee Descartes-08 si loo yareeyo calaamadaha daciifnimada muruqa iyada oo sababeysa waxyeellooyin yar intii suurtagal ah.
In a larger tijaabada caafimaadka, 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.
