Project name: Project category: Kind of research: Country: Principle investigator:

SAPPHIRE study: A national validation study of an intestinal stem cell model to predict and to improve the long-term efficacy of CFTR modulating therapy in individual subjects with CF

CFTR
New Therapies
Clinical research Netherlands Prof. Kors van der Ent - Prof. Jeffrey Beekman
Start date: 2017
End date: 2021
Status: Ongoing
Abstract:

Newly developed CF therapies target the CFTR protein directly, increasing the number of channels (Lumacaftor) in the cell membrane and/or improving their function (Ivacaftor or Lumacaftor/Ivacaftor). Patient variations influence the response to these drugs, resulting in a highly variable therapy response in clinical practice that currently cannot be predicted. This affects the cost-effectiveness of these treatments and therefore their pricing and reimbursement by insurers. This project aims to validate a new diagnostic model that can predict and optimize CF therapy response on an individual level. The model uses organoid stem cell cultures, obtained from patient biopsies, and will analyze the predictive value of organoids for short term and long term clinical responses to newly developed CF therapeutics. This allows individual prediction and optimised therapy responses as well as increased cost-effectiveness. Additionally, the model can be used to test any new drugs, since patient materials are stored in a bio-bank for future use.

Website URL: https://www.ncfs.nl/onderzoek/lopend/hitcf
Researcher: Dr. Marne Hagemeier
Institute: UMC Utrecht
City: Utrecht
Project amount (€): 100.000

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