Chikunguru 2023: Humbowo kubva kudiki-diki kiriniki kuyedzwa kunoratidza kuti myasthenia gravis, autoimmune kusagadzikana kwenheyo system, inogona kurapwa nekusiyana kweCAR-T, advanced cancer cancer immunotherapy. Iyo yakagadziridzwa CAR-T kurapwa, iyo inomiririra chimeric antigen receptor T-cell, yakashandiswa nemasayendisiti. Inogona kuderedza zviratidzo zvemyasthenia gravis kwenguva yakareba uye haina kukonzera chero mhedzisiro yakakura. Chidzidzo ichi, chakabudiswa muThe Lancet Neurology, chakabhadharwa nemari yebhizimisi duku kubva kuNational Institute of Neurological Disorders and Stroke (NINDS), iyo iri chikamu cheNational Institutes of Health. Rubatsiro urwu rwakapihwa kuGaithersburg, Maryland-based company Cartesian Therapeutics.
Using a groundbreaking therapy like CAR-T to potentially treat a neurological disorder shows how flexible immunotherapies 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 chirwere chenguva refu che autoimmune chinoitika apo masoja emuviri anorwisa mapuroteni anowanikwa apo masero etsinga anotaura netsandanyama. Chirwere ichi chinoratidzwa nekushaya simba kwetsandanyama kunowedzera kana munhu ari kushanda uye dzimwe nguva anoita nani kana munhu akazorora. Chinangwa chikuru chemishonga yatinayo ikozvino kuderedza zviratidzo, kunyanya kupera simba kwetsandanyama.
Muchidzidzo ichi, vanhu gumi nevana vane generalized myasthenia gravis vakapihwa huwandu hwakasiyana hweDescartes-14, nzira yakagadziridzwa yeCAR-T therapy inonangana nemasero anoita masoja ekudzivirira chirwere anokonzera myasthenia gravis. Mushonga wakanakisisa wakawanikwa kuva piritsi rimwe kamwe pavhiki kwemavhiki matanhatu. Ruzivo rwekutanga nezve kuti kurapwa kunoshanda sei kunokurudzira, asi kumwe kuongororwa kwekiriniki kunodiwa kuona kuti inoshanda sei. Vanhu vatatu vakatora Descartes-08 vane zvese kana zvinenge zvese zviratidzo zvavo zvaenda. Izvi zvakatora mwedzi mitanhatu mushure mekurapwa. Vamwe vaviri havachada kurapwa neintravenous immunoglobulin inopihwa kune vamwe vanhu vane MG yakakomba.
Murat V. Kalayoglu, MD, Ph.D., purezidhendi uye CEO weCartesian Therapeutics, akati, "Takaona mhinduro dzakadzika, dzenguva refu kuDecartes-08 iyo yakatora mwedzi mitanhatu mushure mekurapwa." "Iko zvino takatanga chidzidzo chakakura chisina kujairika, chinodzorwa ne placebo, inova yekutanga yerudzi rwayo yeinjiniya yekugamuchira cell therapy."
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 kanzura yeropa, 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.
Mazhinji ma immunotherapies, kusanganisira CAR-T, anogona kukonzera mhedzisiro yakakura iyo, nepo inoshivirira muzviitiko zvekenza yepamusoro, inoita kuti zvisakwanise kushandiswa mune zviitiko zve myasthenia gravis uye mamwe mamiriro enguva refu. T-masero anowanzo kuchinjwa neDNA, iyo inogara muchitokisi uye inotorwa nguva dzose apo sero rinoparadzana. Izvi zvinogona kuita kuti chiito chive chakasimba uye chinokonzera njodzi yemigumisiro.
Descartes-08 haishandise DNA kushandura T-seli nekuti DNA inozvikopa pachayo kana masero apatsanura. Pane kudaro, inoshandisa mutumwa RNA (mRNA), iyo isingazvikope pachayo kana masero apatsanurana. Mhedzisiro inguva pfupi yekurapa inopihwa kanopfuura kamwe pane imwe dosi, inova iyo DNA-yakarongwa CAR-T therapy inowanzoshanda. Chinangwa chikuru chechidzidzo ichi chaiva chekutsvaga chiyero chakanaka cheDescartes-08 kuderedza zviratidzo zvekushaya simba kwemhasuru apo zvichikonzera migumisiro shoma sezvinobvira.
In a larger kliniki yekuedzwa, 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.
