July 2023: Evidence from a small-scale clinical trial shows that myasthenia gravis, an autoimmune disorder of the nervous system, could be treated with a variation of CAR-T, an advanced blood cancer immunotherapy. The modified CAR-T treatment, which stands for chimeric antigen receptor T-cell, was used by scientists. It could reduce myasthenia gravis symptoms for a longer time and didn’t cause any major side effects. The study, which was published in The Lancet Neurology, was paid for by a small business grant from the National Institute of Neurological Disorders and Stroke (NINDS), which is part of the National Institutes of Health. The grant was given to Gaithersburg, Maryland-based company Cartesian Therapeutics.
Using a groundbreaking therapy like CAR-T to potentially treat a neurological disorder shows how flexible imunoterapi 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 nyaéta kasakit otoimun jangka panjang anu lumangsung nalika sistim imun awak nyerang protéin kapanggih dimana sél saraf ngobrol jeung otot. Kasakit dicirikeun ku kelemahan otot nu jadi parah lamun jalma aktip sarta kadang meunang hadé mun jalma rests. Tujuan utama tina perlakuan urang ayeuna nyaéta pikeun ngurangan gejala, utamana kelemahan otot.
Dina pangajaran, 14 jalma anu ngagaduhan myasthenia gravis digeneralisasi dibéré jumlah anu béda tina Descartes-08, bentuk modifikasi terapi CAR-T anu nargétkeun sél anu ngadamel antibodi anu nyababkeun myasthenia gravis. Dosis anu pangsaéna nyaéta hiji pél saminggu sakali salami genep minggu. Inpormasi awal ngeunaan kumaha jalanna perlakuan anu nyorong, tapi langkung seueur tés klinis diperyogikeun pikeun nangtoskeun kumaha jalanna. Tilu jalma anu nyandak Descartes-08 ngagaduhan sadayana atanapi ampir sadayana gejalana ngaleungit. épék ieu lumangsung salila genep bulan sanggeus perlakuan. Dua anu sanés henteu peryogi deui perawatan sareng immunoglobulin intravena anu dipasihkeun ka sababaraha urang anu ngagaduhan MG serius.
Murat V. Kalayoglu, MD, Ph.D., Présidén jeung CEO Cartesian Therapeutics, ceuk, "Urang nempo jero, réspon lila-langgeng mun Decartes-08 nu lumangsung sahenteuna genep bulan sanggeus perlakuan." "Kami ayeuna parantos ngamimitian studi acak anu langkung ageung, dikawasa plasebo, anu mangrupikeun anu munggaran pikeun terapi sél adopsi anu direkayasa."
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 kangker getih, 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.
Seueur immunotherapies, kalebet CAR-T, tiasa nyababkeun efek samping utama anu, sanaos tiasa ditolerir dina kasus kanker maju, teu mungkin dianggo dina kasus myasthenia gravis sareng kaayaan jangka panjang anu sanés. Sél T biasana dirobih ku DNA, anu tetep dina sél sareng ditiron unggal waktos sél ngabagi. Ieu tiasa ngajantenkeun tindakan langkung kuat sareng nyababkeun efek samping anu bahaya.
Descartes-08 henteu ngagunakeun DNA pikeun ngarobah T-sél sabab DNA nyalin sorangan nalika sél ngabagi. Gantina, éta ngagunakeun messenger RNA (mRNA), anu henteu nyalin diri nalika sél ngabagi. Hasilna mangrupikeun periode perawatan anu pondok anu dipasihkeun langkung ti sakali tibatan dosis tunggal, nyaéta kumaha terapi CAR-T anu diprogram DNA biasana jalanna. Tujuan utama ulikan ieu pikeun manggihan dosis katuhu Descartes-08 pikeun ngurangan gejala kelemahan otot bari ngabalukarkeun saloba mungkin efek samping.
In a larger sidang klinis, 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.
