After screening thousands of compounds for effects on zebrafish genetically altered to model a rare, inherited heart disorder called arrhythmogenic cardiomyopathy (ACM), a team of scientists found a small molecule capable of reducing some key features of the disease (Asimaki A et al. Sci Transl Med. 2014;6:240ra74).
The disorder, which is characterized by frequent cardiac arrhythmias, is a significant cause of sudden death in teens and young adults. The disease is caused by mutations in genes that encode proteins in the desmosome, a specialized complex located between individual heart cells. Although scientists know about these mutations, they have yet to determine the mechanisms involved in cell injury and the development of arrhythmias.
Hampton T. Small Molecule Treats Heart Arrhythmias in Fish. JAMA. 2014;312(5):477. doi:10.1001/jama.2014.9732