SCIENTISTS from the Netherlands have revealed that stem cells from intestines of cystic fibrosis patients can predict if a specific medicine will be effective for sufferers of the disease.
Cystic fibrosis is a life-threatening disorder that causes severe damage to the lungs and digestive system. It is an inherited condition and can affect the cells that produce mucus, sweat and digestive juices.
Only half of sufferers will live over the age of 40.
Researchers from at the University Medical Centre Utrecht said the stem cells from the intestines of the patients will enable medical physicians to select the right medicines for optimal treatment of individual patients.
The results have been revealed in the journal Science Translational Medicine today.
Medical biologist Dr Jeffrey Beekman headed the laboratory that developed the cystic fibrosis test.
He said: “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. This offers new perspectives for applying existing medicines as well as for developing new ones.”
Some forms of cystic fibrosis (CF) are so rare that the usual clinical studies to test efficacy of medicines cannot be performed because there are too few patients.
Therefore, physicians are looking for alternative ways to develop new medicines for these patients.
Researchers at UMC Utrecht have now demonstrated in a proof of principle study among a small number of patients that the efficacy of a medicine can be demonstrated for a particular patient using that patient’s intestinal stem cells.
This enables the physician to select the medicine or combination of medicines that is most likely to be efficacious in that particular patient.
Professor Dr Kors van der Ent, head of the cystic fibrosis center at UMC Utrecht, said: “A number of extremely expensive medicines are currently being marketed.
“While their efficacy in clinical research is sometimes disappointing, they may be truly life-saving for individual patients.
“Some of these patients were waiting for lung transplants, but can now be found on the hockey pitch again thanks to the right medication.
“Their lives have completely changed. With our new organoid technology, which we have already used on some 200 CF patients, we are entering a new era of individualised treatment.”
Dr Jacquelien Noordhoek-van der Staaij is director of the Netherlands Cystic Fibrosis Foundation (NCFS) and of CF-Europe, the umbrella organisation for all European organisations for CF patients.
She said the discovery by the Utrecht researchers will be of ‘paramount importance’ for patients worldwide.
“There are many different genetic forms of CF and every patient responds differently,” she said.
“Doctors are struggling with the positioning of new, pioneering medicines, as each patient responds just that little bit differently.
“The entire field of CF patients and practitioners is welcoming this with open arms. We have been waiting for this all along.”
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