Cystic Fibrosis Drugs Can Be Tested First Using Patient’s Own Stem Cells in Laboratory

Stem cells obtained from the gut of a patient with cystic fibrosis (CF) and grown in the laboratory could help predict whether a certain treatment will be effective for that particular patient – potentially saving the patient from enduring long and stressful treatments that may never work.

The move is also a step forward in personalized medicine.

In a press release, Dr. Jeffrey Beekman, head of the laboratory that developed the science at University Medical Center (UMC), Utrecht, Netherlands, said testing in the laboratory offers new perspectives for applying existing medicines and developing new ones.

“We are able to grow stem cells into what are known as organoids, in the laboratory. These organoids are genetically identical to the patient and ideal for studying cystic fibrosis. You can tell from them whether the disease will be mild or severe and predict whether or not the patient will respond to certain medicines,” Beekman said.

In a proof of principal study, scientists isolated stem cells from the gut of a small number of CF, then researchers grew the cells in the laboratory to test different drugs. They observed that the efficacy of a drug could be determined using the stem cells, and that the approach could help doctors select the drug, or combination of drugs, most likely to be effective inside the body of the patient.

“With our new organoid technology, which we have already used on some 200 CF patients, we are entering a new era of individualized treatment,” said Dr. Kors van der Ent, head of the CF Center at UMC Utrecht.

CF is a severe genetic condition present from birth. It affects a person’s ability to breath and also makes them more susceptible to lung infections. Many drugs exist to treat CF but they are usually very expensive and do not work for every patient.

Clinical trials to test the efficacy of each CF drug on the right group of patients is difficult and time consuming. Some types of CF are so rare that it is nearly impossible to conduct a meaningful clinical trial.