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A hope for patients with cystic fibrosis

A hope for patients with cystic fibrosis

Olomouc - Scientists from the Institute of Molecular and Translational Medicine, which has come into being as part of the European project BIOMEDREG at the Medical Faculty of Palacký University in Olomouc, have made a significant breakthrough in the fight against a fatal disease - cystic fibrosis. Olomouc doctors, in collaboration with a team of Canadian scientists from McGill University in Montreal, found out that fenretinide, which is used in the treatment and prevention of some cancers, can effectively prevent the infection of Pseudomonas aeruginosa, which is frequently a cause of death of patients suffering from cystic fibrosis. Based on this finding, the American Bureau of Food and Drug Administration (FDA) has now licensed the initiation of clinical trials in patients. "Something like this happens only every two or three years. If all goes well, the results of the clinical trials could be known within 16 to 24 months," explains Associate Professor Marián Hajdúch,Ph.D, Director of the Institute of Molecular and Translational Medicine of the Medical Faculty of Palacký University Olomouc, who leads a team of Czech scientists in the Canadian project.

There are around 70,000 patients with cystic fibrosis worldwide. In the Czech Republic, this fatal genetic disease has affected approximately 200 patients. "Nowadays, the patients live till they reach adult age, but even so their lives are dramatically shortened. They often die of chronic infections that will wear them down, the most common type being Pseudomonas  aeruginosa. This disease is accompanied by a severe metabolic defect that causes even a dysfunction of the immune system. When we were studying a preparation that is used for treating tumours, we discovered that fenretinide, which is a derivative of vitamin A, is able to regulate precisely the lipid defect," says Professor Frantisek Kopřiva, Ph.D., the chief physician of the Pulmonary Ward in Children's Clinic of Olomouc University Hospital. His colleague, Associate Professor Hajdúch, mentions the cystic fibrosis research they are conducting in Olomouc and adds that they have just completed a clinical study with another derivate of vitamin A, which is used as a regular dietary supplement. "It is lycopene, a red pigment contained in tomatoes, and we are anxiously waiting for the result of testing this derivative of retinol."

The decision of the American agency FDA to authorize the initiation of a clinical trial in this case has a significant effect for the subsequent development of the drug, which has been awarded the status of “orphan-drug “. This is the type of drug that most large pharmaceutical companies are not predominantly concerned with, because there are very few patients (as is the case with cystic fibrosis), and therefore it is not from their point of view sufficiently commercially attractive. “There are special arrangements for these situations to help the patients who have the misfortune to suffer from such a rare disease. The FDA and the European Medicines Agency (EMEA) apply in these cases a simplified and shortened development phase and registration, because it is often impossible to carry out clinical trials on, for example, three thousand patients. It is a special process of approval, which is shortened and dramatically discounted, so the costs of developing the drug are not so prohibitive and the approval process runs much faster,” Associate Professor Hajdúch explains the approval process of the "orphan drugs". A similar approval process applying for the status of an "orphan drug" to be granted to fenretinide is currently running at the European Medicines Agency, and it is expected that the European branch of the study, which should take place in the Czech Republic, should open for our patients next year.

The international research project focused on treating cystic fibrosis is lead by the Canadian Professor Dr. Danuta Radzioch from McGill University in Montreal; in addition to Olomouc specialists from the Institute of Molecular and Translational Medicine LF UP Olomouc, there are also scientists involved from the Central University of Venezuela in Caracas, under the direction of Professor Juan De Sanctis.

The BIOMEDREG project (www.biomedreg.eu), which provides an umbrella for the establishment of the Institute of Molecular and Translational Medicine at the Faculty of Medicine of Palacký University in Olomouc is financed by the European Regional Development Fund and the state budget of the Czech Republic through the Operational Programme Research and Development for Innovation.