Researchers believe that cystic fibrosis transmembrane regulator (CFTR) modulators are the drugs to most likely succeed in treating cystic fibrosis (CF) and that CFTR gene modulation holds the key to CF cure, as published in the Journal of Clinical Medicine.

A research team conducted a global web-based survey to ask CF researchers about their opinions on the future of CF treatment. “Our results show that, despite both approaches [ie, gene therapy and gene editing] still being in a pre-clinical development phase, most respondents appeared optimistic about the potential of this new drugs to reduce the CF burden in coming years,” they said.

Most (73.85%) respondents with high or good CF knowledge chose CFTR modulators as the most likely therapeutic option to be successful in treating CF in the next 15 years. For these researchers, correctors and potentiators hold the greater promise.

Most (74.60%) respondents who chose CFTR modulators believe that CF could be cured in the future, with 27.38% indicating a 15-year period and 47.22% more than 15 years to achieve the cure. For the remaining respondents, the cure is either unknown (16.28%) or unlikely (9.12%).

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About 20% of respondents with high or good CF knowledge selected genetic therapies as the most likely therapeutic option to be successful in treating CF in the next 15 years. Among them, transfer RNA-based therapies, adeno-associated viruses (AAVs)-based therapies, messenger RNA-based therapies, lentiviruses-based therapies, and liposome-based therapies were highlighted as the most promising.

Most (90%) respondents who choose genetic therapies believe that CF could be cured in the future, with 65.68% indicating a 15-year period and 22.38% more than 15 years to achieve the cure. For the remaining respondents, the cure is either unknown (7.46%) or unlikely (4.48%).

The opinions were, nevertheless, influenced by the respondents’ knowledge. In the case of CFTR modulators, high-knowledge respondents were more likely to choose correctors or potentiators over stabilizers than good-knowledge respondents.

In the case of genetic therapies, good-knowledge respondents were more likely to choose AAVs-based therapies than high knowledge respondents. A minor percentage (5.90%) of respondents with high or good CF knowledge said to not believe in the success of neither CFTR modulators nor genetic therapies.

The study counted with the participation of more than 500 researchers with high (n=179), good (n=223), or some (n=114) CF background. Of those with high or good knowledge, 391 completed the questionnaire.

Reference

Cabral B, Terlizzi V, Laselva O, Conte Filho C, Mota F. Anticipating new treatments for cystic fibrosis: a global survey of researchers. J Clin Med. 2022;11(5):1283. doi:10.3390/jcm11051283