Minidarms predicts the use of drugs for cystic fibrosis

Researchers at UMC Utrecht have succeeded in reproducing organs of patients with CF. They did this by growing a small piece of intestinal tissue in the "mini-arms" in the laboratory.

Next, with the aid of these mini-arms, they studied the effects of three types of drugs in 24 patients with various forms of cystic fibrosis. Successfully: the effects of drugs on airway function and sweat in patients could be clearly related to the effects of the drug on minidarms.

Testing

The mini-arms do not look like the intestine seen under the microscope. (you can not see them with the naked eye). "But they have all the features," says Kors van der Ent of the UMC, a specialist in lung diseases and pediatricians. "All the cells we have in our own intestines are also in this miniature arm."

The great advantage of this evolution is that separate drugs can be tested for each patient. "Without loading the patient, we can test all available drugs," says Van der Ent. "And from each patient separately, we can see how they react to the drug."

Jill (25 years old) has cystic fibrosis

Cystic fibrosis (cystic fibrosis) affects Jill's daily life properly. At the age of two and a half, the doctors discovered that she was suffering from cystic fibrosis. "You are often sick with CF.I have a chronic fever, you can imagine that you do not feel comfortable with that."

She also lies often with pulmonary infections in the hospital. "My body no longer responds to the antibiotics you can take at home." She can do a good thing every day. "With my friends in town, or something sporty or on my horse, even more energy."

Hope

She hopes the research done by UMC Utrecht will change that. Three years ago she was a piece of her intestines; now she is waiting and hoping to find a medicine that will work for her.

"Research gives hope," she says. "I love it so much that it's a search for everyone, not just for the regular mutations of cystic fibrosis." Four years ago, I thought, "I will not find a cure for cystic fibrosis in my life." Now, I hope for the first time.

Appropriate Drug

The results of the research are particularly hopeful for people with a rare mutation in cystic fibrosis. "With them, it was very difficult to never participate in research," says Vincent Gulmans, head of research and quality care at the Dutch Cystic Fibrosis Foundation. "There are drugs available, but not for these people."

Thanks to the mini-arms, it is also possible to search for a suitable medication for these patients. Gulmans is happy with that. "You can predict if people will react to the drug, you can reach a much larger group and you can also take a closer look at what works and what does not work."

Function of Sweat

Good The patient can live longer and the quality of life is much better. This has already proved this research. Gulmans: "We have examples of people who, for example, were on a lung transplant list and were able to get rid of it, who were hospitalized every year and are not hospitalized now."