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Updated: Dec 6, 2025

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
07:04

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

Published on: February 11, 2017

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[Therapy of cystic fibrosis - new drugs give hope]

Patience Eschenhagen1, Carsten Schwarz1

  • 1Sektion Cystische Fibrose, Christiane Herzog-Zentrum Berlin, Charité - Universitätsmedizin Berlin, Klinik für Pädiatrie m. S. Pneumologie, Immunologie und Intensivmedizin, Campus Rudolf Virchow Klinikum, Berlin.

Deutsche Medizinische Wochenschrift (1946)
|October 6, 2020
PubMed

Insights

New modulator therapies offer hope for cystic fibrosis patients, significantly improving lung function and reducing sweat chloride levels. These treatments, including triple combination therapy, are now approved for about 90% of patients with at least one F508del mutation.

Area of Science:

  • Biomedical research
  • Pulmonology
  • Genetics

Background:

  • Cystic fibrosis (CF) is a monogenetic disorder affecting multiple organs.
  • Historically, CF treatments focused on symptom management.
  • Recent advancements include novel modulator therapies targeting the underlying CFTR protein defect.

Purpose of the Study:

  • To evaluate the efficacy of new modulator therapies for cystic fibrosis.
  • To assess the impact of triple combination therapy (elexacaftor/tezacaftor/ivacaftor) on CF patients.
  • To determine the potential of these therapies to alter the disease course.

Main Methods:

  • Clinical studies involving patients with F508del homozygous and heterozygous mutations.
  • Administration of triple combination therapy (elexacaftor/tezacaftor/ivacaftor).
  • Assessment of clinical outcomes including FEV1 and sweat chloride levels.

Main Results:

  • Significant clinical improvement observed in patients treated with triple combination therapy.
  • Rapid reduction in FEV1 and sweat chloride levels detected within 4 weeks.
  • FDA approval granted for the triple combination therapy in the USA.

Conclusions:

  • Elexacaftor/tezacaftor/ivacaftor represents a significant advancement in cystic fibrosis treatment.
  • The therapy is accessible to approximately 90% of CF patients in the USA with at least one F508del mutation.
  • These modulators have the potential to revolutionize cystic fibrosis care.

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