Rigosertib ya RDEB SSC (2022)

Kafukufukuyu adamalizidwa mu 2022 ndikutsimikizira kuti anthu omwe ali ndi EBS amakankhira pansi mwamphamvu ndi mapazi awo poyenda kusiyana ndi anthu opanda matuza opweteka. Ofufuzawa akufuna kupitiriza ntchito yawo kuyesa zotsatira za masewera olimbitsa thupi ndi nsapato zapadera kapena insoles (orthotics) kuti athandize kufalitsa kupanikizika pamtunda wa phazi ndikupangitsa kuyenda momasuka.

Zotsatira zidasindikizidwa mu Briteni Journal of Dermatology ndi Journal of Investigative Dermatology ndipo idaperekedwa ngati poster kwa a Society for Investigative Dermatology.

Wofufuza wamkulu:

Dr Andrew South ndi Pulofesa Wothandizira ku Thomas Jefferson University, Philadelphia. Zokonda zake zazikulu ndikumvetsetsa zochitika zomwe zimatsogolera ku chitukuko ndi kupita patsogolo kwa SCC, makamaka makhansa omwe amabwera kwa odwala omwe amakhala ndi RDEB. Dr South wagwirapo ntchito m'mabungwe omwe ali ndi mbiri yolimba ya kafukufuku wa EB, ku London, Scotland, ndipo tsopano Philadelphia, ndipo akudzipereka kupeza machiritso ku gulu lowononga la matenda pogwiritsa ntchito kafukufuku wofunikira wa sayansi.

Ofufuza nawo:

Prof Johann Bauer ndi mkulu wa dipatimenti ya Dermatology pa University Hospital Salzburg. Anamanga ndipo watsogolera gulu lofufuza la EB-House Austria, lomwe lili ndi asayansi oposa 20 omwe amagwira ntchito pa kafukufuku wa EB. Cholinga chake chachikulu ndikukhazikitsa njira yotetezeka komanso yothandiza ya jini yamitundu yonse ya EB. Kuphatikiza apo, gulu ku Salzburg limagwiritsa ntchito njira zazing'ono zamamolekyu kuti achepetse zizindikiro za EB kuti apititse patsogolo moyo wa odwala komanso kupanga chithandizo chamankhwala a RDEB okhudzana ndi ma SCC ankhanza.

Prof Jemima Mellerio ndi katswiri wa dermatologist komanso pulofesa ku St John's Institute of Dermatology, Guy's ndi St Thomas' NHS Foundation Trust. Ali ndi zaka zoposa 20 akugwira ntchito zachipatala m'munda wa EB ndi matenda ena a khungu lachibadwa, komanso kafukufuku wofufuza momwe maselo amitundu yosiyanasiyana a EB amakhalira, komanso mayesero azachipatala kukhala mankhwala atsopano a EB monga fibroblast ndi mesenchymal stromal cell. chithandizo. Amadzipereka kupitiliza ntchitoyi kuti apange chithandizo chamankhwala chamitundu yonse ya EB.

"Ntchito yomwe idayambika ndi polojekitiyi imatha kubweretsa chithandizo chovomerezeka cha khansa ya RDEB, cholinga chomwe Debra UK adayamba nacho kumayambiriro kwa zaka za m'ma 2000"

Dr Andrew South

Mutu wa Grant: "Choyamba mu EB" gawo II kuyesa kwa rigosertib kwa RDEB SCC.

Tikufuna kuyesa pang'ono, "choyamba mu EB" chamankhwala oyesera otchedwa rigosertib pochiza khansa ya EB. Mankhwalawa ndi oletsa njira zosiyanasiyana zomwe ndizofunikira pakukula kwa maselo a khansa. Rigosertib wakhala akuyesa zachipatala za khansa zina zingapo, makamaka myelodysplastic syndrome (khansa yamagazi) chifukwa chake kampani yomwe idapanga mankhwalawa, Onconova, ili ndi chidziwitso chabwino chogwiritsa ntchito rigosertib kwa odwala.

Tazindikira kuti mu labotale rigosertib imapha ma cell a khansa ya EB ndipo siyivulaza ma cell akhungu a EB. Pulojekitiyi idzayesa ngati rigosertib ikhoza kupha maselo a khansa mwa wodwala EB komanso ngati mankhwalawa amatha kulekerera odwala - kodi odwala angatenge rigosertib oral (monga mapiritsi, kawiri pa tsiku) popanda kukhudzidwa kwakukulu pazochitika za tsiku ndi tsiku. Ngati rigosertib ikhoza kupha ma cell a khansa mwa wodwala EB tiwona khansayo ndi wodwalayo kuyesa kuzindikira mbali ina yomwe ingagwiritsidwe ntchito kulosera ngati odwala amtsogolo angapindule ndi mankhwalawa.

Debra adathandizira kukhazikitsidwa kwa kuyesa kwachipatala koyamba kwa chithandizo choyesera kuchiza khansa ya EB. Ntchitoyi idatchedwa "First in EB" Phase II kuyesa kwa Rigosertib kwa RDEB SCC. Mankhwalawa ndi oletsa njira zosiyanasiyana zomwe ndizofunikira pakukula kwa maselo a khansa ndipo tawonetsa kuti maselo onse a khansa ya RDEB amayankha mankhwalawa mu labotale. Rigosertib adakhalapo m'mayesero azachipatala kwa makhansa ena angapo ndipo adawonetsa zotsatira zoyipa kwambiri.

Ngakhale tidakhala ndi kuchedwa kwakukulu pakukhazikitsa kuyesaku, kusokonezedwa ndi mliri wa Covid-19, tidalemba wodwala woyamba mu 2021. Wodwala woyamba kuthandizidwa (ku Austria) adalandira rigosertib m'mitsempha ndikuwonetsa kuyankha kwathunthu, kutanthauza kuti khansa yawo. mbisoweka ndi chithandizo. Wodwalayo amakhalabe wopanda khansa pambuyo pa miyezi 19 akulandira chithandizo. Ndalama zodzipatula zathandizira chithandizo cha wodwala wachiwiri, womwe unayamba mu Seputembara 2022 ndipo nthawi ino ndi mankhwala amkamwa. Wodwala uyu akuwonetsa zizindikiro zofananira za kuyankha mwachangu kwa mankhwalawa ndipo tikukhalabe ndi chiyembekezo kuti rigosertib ikhoza kukhala njira yabwino yothandizira khansa ya RDEB. (Kuchokera Lipoti Lomaliza la 2022.)