Naukowcy opracowali żel zawierający zmodyfikowane wirusy opryszczki pospolitej przeznaczony do leczenia ran u pacjentów z dystroficzną, recesywną postacią pęcherzowego oddzielania się naskórka. Herpeswirusy dostarczają gen kolagenu typu VII do skóry, co zapobiega powstawaniu pęcherzy.
Naukowcy ze Stanford University School of Medicine od ponad dekady pracują nad skuteczną terapią genową w leczeniu epidermolysis bullosa (EB) – pęcherzowego oddzielania się naskórka. Obecnie badają żel do miejscowej aplikacji, podczas cotygodniowej zmiany opatrunku w warunkach ambulatoryjnych. Produkt dostarcza do skóry wirusa opryszczki pospolitej, który został genetycznie zmodyfikowany tak, by nie mógł się replikować ani rozprzestrzeniać na inne obszary ciała. Herperwirus dostarcza kopię kolagenu na powierzchnię skóry.
U większości badanych pacjentów rany zamknęły się po 3 miesiącach
Badanie kliniczne zostało przeprowadzone z udziałem dziewięciu pacjentów, wśród których troje było dziećmi. Pacjenci cierpieli na recesywną, dystroficzną postać pęcherzowego oddzielania się naskórka (RDEB), która charakteryzuje się mutacją w genie kodującym białko kolagenowe typu VII. Oznacza to, że produkcja kolagenu jest obniżona i zaburzona, co prowadzi do powstawania pęcherzy. Skóra u pacjentów z RDEB jest wyjątkowo delikatna, a nawet najmniejszy dotyk może spowodować powstanie rany podatnej na infekcję.
Każdy z pacjentów miał leczone dwie rany – jedną z pomocą żelu, a drugą placebo. Po trzech miesiącach eksperci ocenili, w jaki sposób żel wspomógł gojenie się urazów, a później monitorowali pacjentów, by upewnić się, że rany nie otworzyły się ponownie.
U większości pacjentów rany, które potraktowano żelem opracowanym przez naukowców, zagoiły się w ciągu 3 miesięcy. Wyjątkiem było dwóch uczestników. Jeden z pacjentów miał ranę, która utrzymywała się od 10 lat i pokrywała większą część ciała chorego. Zastosowanie żelu pozwoliło na 70 proc. zagojenie się u razu. Z kolei u innego pacjenta, który miał 5-letnią rozległą ranę na stopie, leczenie musiało być przeprowadzone w dwóch cyklach, ale ostatecznie rana zagoiła się w przeciągu 8 miesięcy.
Terapia poprawia jakość życia pacjentów
Dr Peter Marinkovich, dyrektor Stanford Medicine’s Blistering Disease Clinic, wyjaśnia, że terapia wzmacnia skórę, dzięki czemu rany goją się szybciej i pozostają przez długi czas zamknięte. Eksperci zaobserwowali, że już w 9 dniu od leczenia skóra zaczęła wytwarzać kolagen VII, a ekspresja utrzymywała się przez kolejne 100 dni. Wirus nie wnika jednak głęboko w skórę, więc nie zapobiega całkowicie powstawaniu pęcherzy. Oznacza to, że terapia musi być stosowana regularnie. Zdaniem dr. Marinkovicha leczenie to znacznie poprawia jednak jakość życia pacjentów.
Naukowcy planują teraz opublikować wyniki badań i przesłać je do FDA w celu zatwierdzenia leku. W przyszłości zostaną również przeprowadzone badania nad skutecznością terapii w leczeniu ran na przełyku, w rogówce i okolicach odbytu u pacjentów z EB.
Źródło: med.stanford.edu
Przeczytaj także: Wyniki badań nad miejscową terapią genową leczenia ran w przebiegu dystroficznej postaci EB jeszcze w tym roku
Przeczytaj bezpłatnie pokrewny artykuł w czasopiśmie „Chirurgia Plastyczna i Oparzenia”:
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