Rigosertib don RDEB SSC (2022)

An kammala wannan binciken a cikin 2022 kuma ya tabbatar da cewa mutanen da ke da EBS suna matsawa ƙasa da ƙarfi da ƙafafu yayin tafiya fiye da mutanen da ba su da blisters. Masu binciken za su so su ci gaba da aikin su don gwada tasirin motsa jiki na ma'auni da takalma na musamman ko insoles (orthotics) don taimakawa wajen yada matsa lamba a cikin tafin ƙafar ƙafa da kuma yin tafiya cikin kwanciyar hankali.

An buga sakamakon a cikin Jaridar Burtaniya ta ilimin likitanci da Jaridar Binciken Dermatology kuma an gabatar da shi azaman fosta ga Society for Investigative Dermatology.

Jagoran bincike:

Dr Andrew South Mataimakin Farfesa ne a Jami'ar Thomas Jefferson, Philadelphia. Babban abin da ya fi so shi ne fahimtar abubuwan da ke haifar da ci gaba da ci gaba na SCC, musamman ma ciwon daji da ke tasowa a cikin marasa lafiya da ke zaune tare da RDEB. Dr South ya yi aiki a cibiyoyin da ke da tarihin bincike na EB, a London, Scotland, da kuma Philadelphia yanzu, kuma ya himmatu don nemo magunguna ga wannan rukunin cututtuka masu lalacewa ta hanyar aikace-aikacen bincike na kimiyya.

Masu bincike:

Farfesa Johann Bauer shi ne shugaban Sashen Nazarin fata na Asibitin Jami'ar Salzburg. Ya gina kuma ya jagoranci ƙungiyar bincike na EB-House Austria, wanda ya ƙunshi fiye da masana kimiyya 20 da ke aiki akan binciken EB. Babban abin sha'awa shi ne haɓaka ingantaccen magani mai aminci kuma mai dacewa ga kowane nau'in EB. Bugu da ƙari, ƙungiyar a Salzburg tana aiki akan ƙananan hanyoyin tushen kwayoyin don rage alamun EB don haɓaka ingancin rayuwar marasa lafiya da kuma haɓaka hanyoyin kwantar da hankali na RDEB masu alaƙa da SCCs masu tayar da hankali.

Farfesa Jemima Mellero mashawarcin likitan fata ne kuma farfesa a St John's Institute of Dermatology, Guy's da St Thomas' NHS Foundation Trust. Tana da fiye da shekaru 20 tana aiki a asibiti a fannin EB da sauran cututtukan fata na kwayoyin halitta, da kuma bayanan bincike da ke kallon tushen kwayoyin halitta na nau'ikan EB daban-daban, da gwaje-gwajen asibiti zuwa sabbin hanyoyin kwantar da hankali na EB kamar fibroblast da mesenchymal stromal cell. far. Ta sadaukar da ita don ci gaba da wannan aikin don haɓaka ingantattun jiyya ga kowane nau'in EB.

"Aikin da wannan aikin ya fara yana da yuwuwar haifar da ingantaccen magani don maganin ciwon daji na RDEB, burin da Debra UK ta fara da shi a farkon 2000s"

Dr Andrew South

Sunan bayar: "Na farko a cikin EB" gwaji na II na rigosertib na RDEB SCC.

Muna ba da shawarar yin ƙaramin, "na farko a cikin EB" gwajin maganin gwaji da ake kira rigosertib don maganin ciwon daji na EB. Magungunan magani ne mai hanawa na matakai daban-daban waɗanda ke da mahimmanci ga ci gaban ƙwayar cutar kansa. Rigosertib ya kasance a cikin gwaji na asibiti don wasu ciwon daji, musamman myelodysplastic ciwo (ciwon daji na jini) don haka kamfanin da ya kirkiro maganin, Onconova, yana da kwarewa mai kyau na yin amfani da rigosertib a cikin marasa lafiya.

Mun gano cewa a cikin dakin gwaje-gwaje rigosertib yana kashe EB ciwon daji Kwayoyin kuma ba ya cutar da al'ada EB fata Kwayoyin. Wannan aikin zai gwada ko rigosertib zai iya kashe kwayoyin cutar kansa a cikin mai haƙuri na EB kuma ko za a iya jure wa miyagun ƙwayoyi ta marasa lafiya - marasa lafiya na iya ɗaukar rigosertib na baka (kamar allunan, sau biyu a rana) ba tare da tasiri mai mahimmanci ga aikin yau da kullum ba. Idan rigosertib zai iya kashe kwayoyin cutar kansa a cikin mai haƙuri na EB za mu bincika ciwon daji da mai haƙuri don gwadawa da gano wani bangare na ko dai wanda za'a iya amfani dashi don hango ko hasashen ko marasa lafiya na gaba zasu iya amfana daga wannan magani.

Debra ya ba da kuɗin ƙaddamar da gwaji na farko na asibiti na gwajin gwaji don maganin ciwon daji na EB. An kira aikin "Na Farko a EB" gwaji na Phase II na Rigosertib don RDEB SCC. Magungunan mai hanawa ne na matakai daban-daban waɗanda ke da mahimmanci ga ci gaban ƙwayar cutar kansa kuma mun nuna cewa duk ƙwayoyin cutar kansa na RDEB suna amsa wannan magani a cikin dakin gwaje-gwaje. Rigosertib ya kasance a cikin gwaji na asibiti don adadin wasu cututtukan daji kuma ya nuna alamar tasiri mai sauƙi.

Duk da cewa mun sami jinkiri sosai wajen kafa wannan gwaji, wanda ya ruɗe da cutar ta Covid-19, a ƙarshe mun ɗauki majinyacin farko a cikin 2021. Majiyyacin farko da aka yi wa magani (a Austria) ya sami rigosertib a cikin jini kuma ya nuna cikakkiyar amsa, ma'ana kansa kansa. bace da magani. Mara lafiyar ya kasance ba shi da ciwon daji bayan watanni 19 na karbar magani. Kudade daban-daban ya tallafawa jinyar mara lafiya na biyu, wanda ya fara a watan Satumba 2022 kuma wannan lokacin tare da maganin baka. Wannan majiyyaci yana nuna alamun saurin amsawa ga miyagun ƙwayoyi kuma muna da kyakkyawan fata cewa rigosertib na iya zama zaɓi mai inganci don maganin ciwon daji na RDEB. (Daga Rahoton Ci gaban Ƙarshe na 2022.)