Expert opinion on pharmacotherapy · 2025
Advancing cystic fibrosis treatment: investigational agents on the horizon.
СтатьяОбзорНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Santos Lúcia, Camargo Mariana, Terlizzi Vito, Lopes-Pacheco Miquéias
Аннотация
INTRODUCTION: Cystic fibrosis (CF) treatment has been transformed by CF transmembrane conductance regulator (CFTR) modulator therapies, yet significant gaps remain for those with non-responsive or modestly responsive (below therapeutically relevant levels) genotypes and advanced disease. Emerging agents for genetic therapies and targeting symptoms are in development and offer hope for broader, more durable clinical benefits. This review synthesizes the latest clinical advances aiming to shape the future of CF care.
AREAS COVERED: We examine investigational agents in clinical development, including: (1) alternative CFTR modulators (e.g., potentiators, correctors, and amplifiers); (2) gene-based therapies utilizing viral and non-viral vectors; (3) anti-inflammatory approaches; and (4) infection-targeted therapies. Clinical trial data and combination strategies are discussed.
EXPERT OPINION: Despite dramatic improvements in clinical outcomes and survival in people with CF on modulator drugs, further efforts are warranted for ineligible/non-responsive individuals or intolerant to modulators. In addition, it remains vital to identify novel therapeutic strategies to mitigate lung damage and disease progression as infection and inflammation persist even in people with CF receiving 'highly effective' modulator therapy.
AREAS COVERED: We examine investigational agents in clinical development, including: (1) alternative CFTR modulators (e.g., potentiators, correctors, and amplifiers); (2) gene-based therapies utilizing viral and non-viral vectors; (3) anti-inflammatory approaches; and (4) infection-targeted therapies. Clinical trial data and combination strategies are discussed.
EXPERT OPINION: Despite dramatic improvements in clinical outcomes and survival in people with CF on modulator drugs, further efforts are warranted for ineligible/non-responsive individuals or intolerant to modulators. In addition, it remains vital to identify novel therapeutic strategies to mitigate lung damage and disease progression as infection and inflammation persist even in people with CF receiving 'highly effective' modulator therapy.