Sabon Sabbin Jiyya
Ya zuwa yanzu, babu magani don cystic fibrosis (CF), amma masu bincike suna aiki tukuru don samun daya. Masana kimiyya suna kusa yanzu fiye da baya. A cikin shekarun da suka wuce, babban binciken bincike ya haifar da ci gaba da sababbin magungunan da kuma maganin da suka bunkasa ingantaccen rayuwa da kuma rayuwar rayuwa. Dukkan wannan ya bude kofa don sabon bincike wanda zai haifar da magani.
Wadannan sune wasu daga cikin maganin da ake iya nazarin.
Gene Far
A 1989, an gano ginin da ke da alhakin haifar da cystic fibrosis: fadin CFTR . Wannan binciken ya kasance mai ban sha'awa ga ƙungiyar CF. Mutane da yawa sun gaskata cewa binciken zai haifar da magani ta hanyar farfadowa na kwayoyin halitta.
Abin takaici, wannan bai faru ba tukuna, amma ba don rashin ƙoƙari ba. An yi nazari da yawa don gwadawa da gyara nakasar kwayoyin amma babu wani daga cikinsu ya ci nasara. Babban matsala tare da farfadowa na zamani ya zuwa yanzu an gano wani samfurin da zai iya daukar nauyin gyare-gyare a cikin sel.
Har ila yau za'a iya samun bege ga farfadowa na jinsin, ko da yake. A watan Yunin 2009, wani rukuni na masu bincike a Jami'ar North Carolina a Chapel Hill suna da kyakkyawar sakamako ta hanyar amfani da kwayar cutar ta kowacce a matsayin mai ɗaukar hoto don canja wurin jigilar kwayoyin halitta a dakin gwaje-gwajen samfurori. Kungiyar bincike tana aiki a kan hanyar da za ta raunana cutar sanyi don haka ana iya gwada magani a cikin mutane da cystic fibrosis.
VX-770
VX-770 magani ne wanda Vertex Pharmaceuticals yana gwadawa a cikin mutanen da ke da cystic fibrosis wadanda suke da akalla ɗaya kofi na maye gurbin G551D. Da miyagun ƙwayoyi za su iya iya magance lahani a cikin fadin CFTR da kuma mayar da ikonta don buɗe tashar tasirin chloride, don haka izinin gishiri ya gudana cikin kuma daga cikin kwayoyin yadda ya kamata.
Ba kamar tsarin farfadowa ba, VX-770 ba zai maye gurbin mummunar ginin ba. Maimakon haka, idan ya ci nasara, VX-770 zai gyara matsala a cikin jinsin da ke ciki.
VX-809
VX-809 wani magani ne wanda Vertex Pharmaceuticals yana gwadawa a cikin mutanen da suke da nau'i biyu na maye gurbin ΔF508-CFTR. Ya yi kama da VX-770 domin yana iya samun gishiri ta hanyar sel ta dace, amma yana aiki kaɗan. Idan yana aiki da hanyar da masu bincike suke tsammani zai yi, VX-809 zai bude tashar tasirin chloride ta hanyar motsa furotin na CFTR zuwa wurin da yake dacewa a jikin tarin iska.
Miglustat
Miglustat wani magani ne wanda ke aiki da Actelion Pharmaceuticals wanda aka rigaya yayi amfani da su don biyan wasu yanayi, amma an yanzu ana nazarinsa don amfani da mutane tare da cystic fibrosis wanda ke da nau'i biyu na gyaran ΔF508-CFTR. Binciken ya ƙananan (ya ƙunshi masu halartar 15 kawai), amma ya zuwa yanzu an samu sakamakon. Miglustat ya iya canza lahani na CFTR kuma ya mayar da aikin al'ada zuwa sel.
Ataluren
Ataluren, wadda aka kira PTC124, ana binciken shi ne ta hanyar PTC Therapeutics a matsayin magani mai kyau ga mutanen da CF wadanda ke da ma'anar banza. A cikin maye gurɓatattun kalmomi, wani ɓangaren lambar "gibberish" ya bayyana a tsakiyan lambar al'ada a cikin fadin CFTR.
Kalmomin banza suna aiki kamar alamar dakatarwa, yana hana sel daga karanta kowane lambar da ke faruwa bayan shi. Ataluren zai iya gyara wannan matsala ta hanyar taimaka wa sassan don watsi da alamar dakatar da ci gaba da karatun lambar da ke faruwa bayan ta, ta haka ne ya sake dawo da aikin al'ada a cikin sel.
> Sources:
> C. Norez, F. Antigny, S. Noel, C. Vandebrouck, F. Becq. "Ciwon kwantar da cututtuka na CF na jini wanda Miglustat ya biyo baya ya karbi non-CF kamar Phenotype". Jarida na Amirka na Tsarin Cutar Ruwa da Halittun Halittu . Agusta 2009.
> Cibiyar Fibrosis ta Cystic. Yuni 2009. Ciwon Harkokin Drug Development. 24 Yuli 2009.
> Zhang L, Button B, Gabriel SE, Burkett S, Yan Y, et al. 2009 "Samun kyautar CFTR zuwa 25% na Surface Epithelial Cells Ya Koma Kasuwanci na Kasuwancin Gudanar da Harkokin Kasuwancin Fibrosis Airway Epithelium". PLoS Biol 7 (7): e1000155. Doi: 10.1371 / journal.pbio.1000155. 24 Yuli 2009.