Mini guts for Cystic Fibrosis


With the aid of mini guts (organoids) it may be predicted if a (combination of) medicine(s), aimed at the cause of CF inside the cellular structure, will result in a positive effect in the patients concerned.

Researchers and companies could now test in mini guts if candidate medicines are effective or not, before a study will be done with actual groups of patients. Besides, patients showing rare mutations can be examined this way. Generally speaking, they cannot participate in a study needing larger groups of patients.

Also, new medicines could be tested in mini guts, in order to obtain the optimal mix of medicines for every single patient.

Research mini guts

The Utrecht Medical Centre, together with the Hubrecht Institute, developed a method to isolate ‘stem cells’ from intestinal cells in patients with CF, in the lab. These stem cells can be stored in the lab for a very long time. By cultivating stem cells to become mini guts (organoids), lab tests can be effectuated, in order to find out whether or not patients will react well to new medicines. This research is part of the larger research program HIT CF. For more information, please look at www.cfonderzoek.nl/hitcf.

This testing is not very annoying to patients. Once-only, a bit of gut tissue will be removed from the rectum (thermometer method). This part of the intestines has no pain sensors. The rest of the research will be done in the lab.

Mini guts in a bio-bank

The years of 2015 and 2016 will be spent investigating if testing in these mini guts are showing the very same results as when the medicines will be applied directly to the patient. For this, tissue from patients with CF will be used that already was stored in a so-called bio-bank. In addition to this, tissues will be added from as many people as possible, who (during 2015) are (going to be) treated with medicines aimed at the problem of the CFTR protein inside the cells. This includes the following studies: ABBA, TicTac or TRIO. Tissues from patients with CF who, during the second half of 2015, will start with Kalydeco (ivacaftor) treatment are welcomed as well.

The aim is to gather as many tissues as possible, during the period of second half of 2015 and beginning of 2016, from people with rare CF mutations. Right now, it is being clarified if it will be possible to enter tissues from all Dutch patients in the bio-bank, during the following two years. By doing so, no later than by the end of 2018, the bio-bank should be completed with mini guts of all Dutch patients with CF.

Patients who are having their intestinal stem cells taken, will sign an agreement for having the mini-guts stored, and for the use thereof for research reasons.

Do you have any questions about when your or your child’s tissue can be taken? Please contact your paediatrician or pneumonologist, or discuss this matter at your next check-up.

organoids.jpg

Mini guts
Source: Hubrecht Organoid Technology

Adopt a mini gut

Organizing a bio-bank with mini guts from patients with Cystic Fibrosis is very costly. Would you like to financially support the research of the mini-guts for CF? Please adopt a mini-gut at www.adopteereenminidarm.nl.