Manje singakwazi ukuhlela kabusha imizimba yethu
I-FDA ivume ukwelashwa kwe-geni yokwelashwa okokuqala-ukunyakaza kwemlando kumlando wezokwelapha. Ukwelashwa okusha- I-Kymriah (tisagenlecleucel) -ishintsha ama-T-cell ukulwa ne-leukemia.
Ukwamukelwa kukaKymriah kusebenza njenge-harbinger yokwamukelwa kwamanye amafomu we-gene therapy okuzolwa nomdlavuza nezinye izifo ezibucayi ngokusebenzisa indlela enomlando nokuthakazelisayo: ukuhlela kabusha umzimba ngokusebenzisa izindlela zokufakelwa.
I-Gene-Based Based Therapy
Ukwelashwa kwegciwane elisuselwe kumakhalekhukhwini kubhekisela kumkhuba wokwethulwa kwezinto ezintsha zofuzo zibe esitokisini esithile bese usufaka kabusha la maseli alungisiwe emzimbeni. Ngesinye isikhathi emzimbeni, la maseli amasha angakhipha ama-protein asiza ukulwa nomdlavuza noma ezinye izifo ezimbi.
I-Kymriah ihilela ukwethulwa kwe-T-cell-gene-modified-type of lymphocyte, noma i-white cell cell-emzimbeni. Lawa maseli T-modified aqaphela futhi ahlasela amangqamuzana e-leukemia.
I-Gene thérapie ingcono ukwelashwa ngamakhemikhali ngoba izakhi zofuzo ziyingozi kakhulu. I-Chemotherapy ithinta umzimba wonke futhi ibangele imiphumela emibi yomzimba wonke. Ngama-gene based based therapy, amangqamuzana avuselelwe kabusha afakwa endaweni yangakini futhi alwe nama-tumor ngokuqondile.
Ngaphezu kokuba uhlobo lwe-gene therapy, i-Kymriah nayo i-agent e-immunotherapeutic. I-Immunotherapy ihlanganisa amandla omzimba omzimba ukuphatha izifo.
I-Kymriah ichazwa
Ngokwezindlela zobuchwepheshe, i-Novartis's Kymriah iyindlela yokwelashwa ye-T-cell ye-anti-receptor (CAR) ye-T-cell esetshenziselwa ukuphulukisa okubuyiselwayo noma ukuphikisana (okungukuthi, ukumelana nokwelapha) I-B-cell acute lymphoblastic leukemia.
I-Kymriah uhlobo olumisiwe lwe-immunotherapy lapho i-T-cells yegciwane isuke iqoqwe kuqala bese ithunyelwa esikhungweni sokukhiqiza.
Ama-T-seli avunwa esebenzisa inqubo ebizwa nge-lephapharesis.
Esikhungweni sokukhiqiza, lawa maseli e-T aguqulwa ukuze afake isakhi esenza amakhodi wezwi le-CAR. Lawa maseli alungisiwe abuye abuyele esibhedlela. Ngesinye isikhathi emzimbeni, ama-CAR ahlose i-antigen ethiwa i-CD19 etholakala phezu kwamangqamuzana e-leukemia futhi abulale la maseli anomdlavuza. Kanye egazini, lawa maseli T-modified akhule futhi anwebe ukuqeda umdlavuza phakathi kwamasonto amabili noma amathathu.
Ochwepheshe base-FDA basekela ukuvuma kwabo kwe-Kymriah engxenyeni eyodwa yesivivinyo sesigaba sesibili sokutholwa kwengqondo lapho abantwana abangu-63, intsha, nabasha abasebekhulile abanomdlavuza we-B-cell obhekene ne-B-cell obhekene ne-acute lymphoblastic leukemia bathola umthamo owodwa wokwelapha. Kulaba bahlanganyeli, amaphesenti angama-83 afika ekuxoxweni emva kwezinyanga ezintathu. Ngaphezu kwalokho, amaphesenti angu-75 ahlala engenazo izifo emva kwezinyanga eziyisithupha. Okubaluleke kakhulu, uKymriah wanikezelwa ukuHlola okuPhambili kanye nokuThuthukiswa kokuPhatha kwe-FDA.
I-leukemia ye-lymphoblastic acute ingumdlavuza we-bone lomthambo negazi. Idala umzimba ukuba wenze i-lymphocyte engavamile. Lezi zingqamuzana zegazi ezingavamile ngokushesha zixosha amakhemikhali egazi enempilo emnothweni wethambo. Sidinga amaseli ethu egazi ukuze asinde.
Lapho inani lamaseli ejwayelekile egazi linciphisa ngokuphawulekayo, ukufa kungagcina.
Nakuba abadala asebekhulile bangakha i-ALL, ikakhulukazi isifo sezingane. Cishe amaphesenti angama-90 ezingane ne-NONKE aya ekuxoxweni ngemuva kokwelashwa. I-Kymriah ivunyelwe ukuba isetshenziswe kwiziguli ezineminyaka engaphansi kwengu-25 ubudala futhi zibe nomdlavuza obuye wabuyela noma umdlavuza ongenamathekisthi.
Ukukhathazeka NgoKymriah
Omunye ngokuphathelene nomthelela omubi womuthi wanoma yiluphi uhlobo lwe-monoclonal antibody therapy yilona i-cytokine release syndrome, impendulo yokusabela okubizwa nangokuthi isiphepho se-cytokine. Ama-Cytokines amaphrotheni amancane afihliwe ngamangqamuzana futhi anomphumela kwamanye amaseli.
Kubantu abaningi, izimpawu ze-cytokine release syndrome zincane noma zilinganiswa futhi zingaphathwa kalula. Lezi zimpawu zihlanganisa:
- umfutho wegazi ophansi
- ukukhathala
- tachycardia
- ikhanda
- umkhuhlane
- isiyaluyalu
- izikhukhula
- ukushayeka
- emqaleni
- ukukhathazeka ukuphefumula
Kodwa-ke, iziguli ezinegazi lomdlavuza wegazi-njenge-ALL-zisengozini ephakeme yokuthuthukisa isiphepho esinamandla se-cytokine nezimpawu ezisongela ukuphila. Ngenxa yalesi sizathu, ukucindezela kudingeka kwenziwe ezindaweni eziphezulu zevolumu kanye nabachwepheshe abaqeqeshwe ukuphatha izinkinga ezinkulu ezifana nesiphepho se-cytokine.
Okokugcina, uKymriah kulindeleke ukuthi idle cishe u-$ 500,000. Noma kunjalo, ukwelashwa nge-Kymriah kushibhile kunokwakheka kwamathambo, okuvame ukusetshenziselwa ekwelapheni i-leukemia.
Izwi elivela
Ukuphazamisa noma ukuphinda uvuselele KONKE yisifo esibulalayo. Ukwamukelwa kukaKymriah kunikeza ithemba kubantu abasha abangamakhulu amaningana e-United States abangenakho ukwelashwa okusele. Kuye kwafakazela ukuthi iphumelele ekuvivinyweni kwemitholampilo. Noma kunjalo, ukuvivinywa kokwelashwa kwalinganiselwe isikhathi eside, futhi kusazobonakala ukuthi uKymriah ingenza yini ukukhipha impilo yonke.
Ngamazwi amaningi, imvume ye-FDA yendlela yokwelashwa kwezakhi zofuzo ngokokuqala ngqangi isebenzisa iminyaka emisha yemithi: unyaka lapho singakwazi khona ukuhlela kabusha amaseli ethu ukulwa nomdlavuza nezinye izifo ezibulalayo.
> Imithombo:
> I-Breslin S. Cytokine-release syndrome: ukuhlolwa okubukeziwe kanye nokwelapha. I-Clinical Journal of Oncology Nursing. 2007; 11: 37-42.
> Colwell J. Panel OKs I-CAR T Therapy for Leukemia. Ukutholakala kweCanscer. Ngo-July 27, 2017.
> I-FDA News Release. Ukugunyazwa kwe-FDA kuletha ukwelashwa kokuqala kwegenesheni e-United States. Agasti 30, 2017.
> High KA. I-Gene Therapy kuMithi Wezokwelapha. Ku: Kasper D, Fauci A, Hauser S, Longo D, Jameson J, Loscalzo J. eds. Izimiso zikaHarrison ze-Internal Medicine, 19e eNew York, NY: McGraw-Hill; 2014.
> Klepin HD, i-Powell BL. Ukuhlukana Kwamaselula Omhlophe. Ku: Halter JB, Ouslander JG, Studenski S, High KP, Asthana S, Supiano MA, Ritchie C. eds. I-Geriatric Medicine and Gerontology, i-7e eNew York, i-NY: iMcGraw-Hill.