HIT-CF Europe aims to evaluate the efficacy and safety of three drug candidates in patients with CF and rare mutations, pre-selected by their mini-intestines in the laboratory.
What is HIT-CF Europe?
HIT-CF Europe is a research project which aims to provide better treatment and better lives for people with CF and rare mutations. To achieve this, drug candidates of several companies will first be tested in the laboratory on patient-derived mini-intestines (organoids). Secondly, based on the reaction in the organoids, a smaller group of patients will be assigned to studies (clinical trials) with one of the drug candidates.
All participating centres are part of the ECFS-CTN.
What is cystic fibrosis?
Cystic Fibrosis (CF) is the most common life-threatening inherited disease in Europe. Sticky mucus blocks the respiratory and digestive systems. CF is yet an incurable disease. But early diagnosis, regular follow-up by a multidisciplinary team in a specialized CF clinic, and correct, timely treatment of symptoms can prolong and save lives and improve the quality of life.
A new era of drugs, targeting the basic defect in the cells of people with CF, is arising and will have a big impact on the lives of people with CF if they get access to these drugs.
We take some tissue from the intestine
This tissue is cultured in the lab
The result is an organoid
On this organoid we test medicines
And we bring the correct medicines to the patients
What is the use of organoids?
Organoids are cell cultures that grow in a culture dish, and look similar to the organ from which they are derived. Intestinal organoids can therefore also be called mini-intestines. To make intestinal organoids for the HIT-CF Europe project, rectal tissue samples (biopsies) will be obtained. This procedure is not painful and will take 5-10 minutes. Because organoids are made from stem cells, they contain the same mutations as the person from whom the biopsies are derived. The drug candidates target the basic defect of CF, and the organoids will be used to test on which mutations the drugs have a positive effect.