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Wyniki badań nad miejscową terapią genową leczenia ran w przebiegu dystroficznej postaci EB jeszcze w tym roku

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Miejscowa terapia B-VEC dla pacjentów z dystroficzną postacią Epidermolysis bullosa (DEB) polega na dostarczeniu do organizmu kopii genu, który chroni skórę przed powstawaniem pęcherzy i wspomaga zamykanie się ran. Krystal Biotech, firma, która jest twórcą tej terapii, poinformowała, iż wyniki badań będą dostępne jeszcze w obecnym kwartale.

Pod koniec bieżącego roku powinny być dostępne najważniejsze dane z badania klinicznego fazy 3. nad terapią genową leczenia ran B-VEC w przebiegu DEB. Firma Krystal Biotech poinformowała, że ostatni z 31 uczestników badania zakończył 26-tygodniowy okres dawkowania i 30-dniowy okres kontrolny sprawdzający bezpieczeństwo terapii.

– Chcemy wyrazić naszą wdzięczność uczestnikom badania, ich rodzinom, badaczom i koordynatorom za ich udział i zaangażowanie, dzięki czemu możemy terminowo zakończyć badania w tak trudnym roku – podkreślił Suma Krishnan, dyrektor operacyjny Krystal Biotech. – Jesteśmy na dobrej drodze, by ogłosić wyniki badań jeszcze w tym kwartale. Jeżeli okażą się pozytywne, będziemy natychmiast działać nad tym, by zakończyć proces składania wniosków do amerykańskiej Agencji ds. Żywności i Leków (FDA) w celu wprowadzenia terapii genowej dla pacjentów żyjących z dystroficzną postacią EB – dodał dyrektor.

Cotygodniowe stosowanie terapii doprowadziło do całkowitego zamknięcia się ran

Terapia B-VEC dostarcza zdrową kopię genu COL7A1 do komórek w organizmie za pomocą nieszkodliwego wirusa. Mutacje w tym genie są przyczyną dystroficznej postaci EB, prowadząc do wadliwej produkcji białka kolagenowego typu VII, który odpowiada za prawidłowe tworzenie się włókien kotwiczących, umocowujących błonę podstawną do skóry właściwej. Brak genu COL7A1 lub jego mutacje prowadzą do powstawania pęcherzowego oddzielania się naskórka. Skóra i błony śluzowe są wówczas bardzo delikatne i podatne na wszelkie urazy.

Badania kliniczne fazy 3. nad terapią B-VEC rozpoczęły się w 2020 r. w Stanach Zjednoczonych. Celem analiz było udowodnienie bezpieczeństwa i skuteczności terapii u pacjentów cierpiących na DEB w porównaniu z placebo. Udział w badaniu wzięło 31 pacjentów w wieku od 1 do 44 lat. 61 proc. uczestników to pacjenci pediatryczni w wieku poniżej 18 lat. Badanie ukończyło w sumie 29 osób. U każdego z pacjentów badacze zidentyfikowali parę ran – jedna rana została losowo przydzielona do otrzymywania cotygodniowego miejscowego stosowania B-VEC, a druga do placebo. Naukowcy udowodnili, iż stosowanie terapii doprowadziło do całkowitego zamknięcia się rany.

DEB niekiedy prowadzi o powstania śmiertelnych ran

EB jest chorobą genetyczną, która objawia się wybitną wrażliwością skóry na urazy. Główną cechą schorzenia jest oddzielanie się naskórka i występowanie pęcherzy. Poza zmianami skórnymi EB może powodować: próchnicę, defekty szkliwa, przykurcze stawów, zrastanie palców, zwężenie przełyku, atrezję odźwiernika i dystrofię mięśniową. Choroba może również obejmować błonę śluzową jamy ustnej, narządy płciowe oraz odbyt.

DEB jest najczęstszą postacią EB i jest nieuleczalne, a niekiedy prowadzi nawet do powstania śmiertelnych dla pacjenta ran. Powikłaniami dystroficznej postaci Epidermolysis bullosa są niedożywnienie, niedokrwistość, oporny świąd, ból, zakażenia i krytyczna kolonizacja bakterii.

Źródła: epidermolysisbullosanews.com, mp.pl, Krystal Biotech

 

Przeczytaj także: „EB to przede wszystkim ból. Nie ma nawet sekundy, kiedy da się o nim zapomnieć” – historia pacjenta z dystroficzną postacią Epidermolysis bullosa

Przeczytaj bezpłatnie pokrewny artykuł w czasopiśmie „Pielęgniarstwo w Anestezjologii i Intensywnej Opiece ”:

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