Researchers are reporting in laboratory studies that the anti-cholesterol drug lovastatin shows promise as a potential treatment for a common form of dilated cardiomyopathy, or enlargement of the heart.
Dilated cardiomyopathy decreases the heart’s ability to pump blood, which can lead to irregular heartbeats, blood clots, heart failure, or sudden cardiac death. In this study, researchers obtained stem cells from a family of patients affected by LMNA dilated cardiomyopathy, which accounts for 6% of all familial cases of the disease. The researchers developed the stem cells to resemble endothelial cells, or the tissue lining the blood vessels and the heart.
During the study, the researchers found a link between a mutation in the stem cells and narrowing of the blood vessels, which can contribute to decreased ability to pump blood. They also identified a protein, Krüppel-like Factor 2 (KLF2), that appears to play a role in mediating the observed dysfunction.
Using so-called “clinical trial in a dish” technology, they exposed these stem cells to a variety of FDA-approved drugs in order identify those that could help relieve blood vessel narrowing caused by the mutation. They found that the anti-cholesterol drug lovastatin could reverse damage to blood vessels by increasing expression of the KLF2 protein. When they tested lovastatin on two patients with LMNA dilated cardiomyopathy, the drug caused improvement in their blood vessel health. The study suggests that early use of lovastatin prior to cardiac symptoms could delay onset of LMNA-associated cardiomyopathy, the researchers said. Their study, partly funded by the NHLBI, appeared in Science Translational Medicine.