Research on new drug evaluation method in treatment of Long QT Syndrome published in European Heart Journal

A model system based on technology that allows skin cells to be converted into stem cells, and subsequently heart cells, may provide a new way to evaluate drugs in treating Long QT Syndrome, a genetic heart disorder, according to School research published in the European Heart Journal. The study was led by Professor Chris Denning from the Wolfson Centre for Stem Cells, Tissue Engineering and Modelling (STEM).

Long QT Syndrome is a potentially fatal genetic disorder that affects up to 1 in 1000 people. Unfortunately, not all patients benefit from the standard treatments of beta blockers or surgery and new ways are needed to test the potency of new drugs. To address this need, the authors used a new type of technology known as induced pluripotency. This allows skin cells from a patient to be converted into stem cells, then subsequently coaxed into become heart cells (known as cardiomyocytes). Using this approach, the team converted skin cells from a girl with LQTS type 2 into cardiomyocytes. This process was repeated using skin cells from her mother and father, neither of whom has LQTS. Testing these normal (mother and father) or LQTS2 (daughter) stem cell-derived cardiomyocytes in the lab showed that those carrying the LQTS2 mutation developed irregular beat patterns (arrhythmias). Notably, using the patient's own beta blocker treatment blocked the arrhythmias. The researchers then went on to show that much more experimental drugs such as nicorandil and PD118057 were also able prevent arrhythmias. Therefore, this model system may provide a new way to evaluate drugs for the treatment of LQTS.

Reference:

European Heart Journal (2011) 32 (8): 952-962; doi: 10.1093/eurheartj/ehr073.