Dilara Vilic is the first European patient with Cystic Fibrosis (CF) to participate in a unique clinical trial at the Wilhelmina Children’s Hospital in Utrecht. For the clinical trial, patients are selected for the first time with the Utrecht CF organoid model.
Pediatric pulmonologist Kors van der Ent: “Dilara receives a drug candidate that was first tested in the laboratory on intestinal organoids, mini intestines grown from her own intestinal cells. Based on the result in the organoids, we selected Dilara for the CHOICES trial.”
UMC Utrecht’s 030 Lab is coordinating this innovative clinical trial involving 14 centres in 10 different European countries. Thanks to the international collaboration led by Kors van der Ent and the research team of Jeffrey Beekman, Professor of Disease Modelling at UMC Utrecht, 52 CF patients have been selected within Europe. Kors: “It is an example where genetic characteristics are used to choose a matched therapeutic strategy for the right person at the right time. This gives a greater chance of effective treatment with fewer side effects.”
Jeffrey Beekman developed the first clinical application of organoids for people with cystic fibrosis together with Kors van der Ent. By growing organoids from these people and treating them, it is now possible to quickly see whether a particular drug works for this specific person. This is a major benefit in cystic fibrosis, a genetic disease that has many variations and grades and is therefore difficult to treat.
Based on their organoid response, people with CF may participate in the CHOICES study and receive a drug candidate developed in Utrecht by the company FAIR Medicine. With the ultimate goal: to also make a drug available for CF patients with more rare mutations that targets the cause of CF.
Kors van der Ent: “It’s great that the EU now agrees to this, so we can potentially offer these patients with a rare condition better treatment and therefore more perspective.” Jeffrey: “This approach can also help drug development because it gives earlier insight into the success rate of new drugs. The high failure rate of drug candidates is one of the causes of expensive medication.”
Cystic fibrosis (CF) is a serious, congenital disease in which the mucous membranes secrete tough mucus. This causes blockages in the body: in the airways, intestines, pancreas and liver. Over 1,500 people in the Netherlands have CF and 30 children are born with CF every year. Newborns are tested for CF with the heel prick, which detects the disease earlier and ensures that a large proportion of patients can be treated to reduce the impact of the condition.
Cystic fibrosis is caused by an abnormal gene. A child who inherits the abnormal gene from both parents does not make a certain protein. That protein is needed for the transport of chlorine and water through mucosal cells of organs such as lungs, pancreas, liver and intestines. If this does not happen, the mucus in these organs does not get enough moisture and becomes tough.
CF is one of the most common hereditary disorders in the Netherlands; one in 30 people is a carrier of the abnormal CF gene, but is not ill. If parents are both carriers, they have a 25 per cent chance of having a child with CF.
