Cystic fibrosis medications can be tested "in vitro"
Cystic fibrosis-affected pulmonary epithelium to test potential drugs
Researchers at the Massachusetts General Practice Clinic, working under the leadership of Jayaraj Rajagopal, have made a huge step towards developing an effective remedy for the treatment of cystic fibrosis, a hereditary lethal lung disease that claims about 500 lives in the United States every year, with 1,000 new cases registered annually.
At the first stage of the work, they isolated the skin cells of patients with cystic fibrosis and transformed them into induced pluripotent stem cells. After differentiation in the appropriate direction, the cells formed a functioning pulmonary epithelium characteristic of the disease under study. Cells of this tissue capable of unlimited division carry the delta-508 mutation, responsible for 70% of all and 90% of cases of cystic fibrosis in the USA, as well as the G551D mutation, occurring in 2% of patients with this disease. These mutations cause irreversible damage to the pulmonary epithelium, inevitably leading to fatal pulmonary insufficiency.
The recent discovery and official approval of Ivacaftor, a drug that corrects a defect associated with the G551D mutation mentioned above, has become proof of the possibility of treating cystic fibrosis using a traditional molecular approach. This drug was discovered as a result of screening conducted on a cell line that is much inferior to the culture created by the researchers, which is a truly invaluable tool for screening and testing drugs against cystic fibrosis.
Moreover, the cells included in the grown tissue are involved in the pathogenesis of other common lung diseases, including asthma, lung cancer and chronic bronchitis. Therefore, working with cell lines grown from cells of other patients can improve our understanding of the mechanisms underlying the development of these diseases and help in the development of new effective treatments.
Another advantage of the developed model is that the studied cells carry the entire genotype of the patient, which makes it possible to develop highly personalized treatment methods. On the other hand, testing the same experimental drug on cell lines of different patients will allow us to evaluate its effectiveness for the general population.
Article by Hongmei Mou et al. Generation of Multipotent Lung and Airway Progenitors from Mouse ESCs and Patient-Specific Cystic Fibrosis iPSCs is published in the journal Cell Stem Cell.
Evgeniya Ryabtseva
Portal "Eternal youth" http://vechnayamolodost.ru based on the materials of ScienceDaily:
Big Advance Against Cystic Fibrosis: Stem Cell Researchers Create Lung Surface Tissue in a Dish.
06.04.2012