Screening experiments have revealed several small molecules that might help treat cardiac amyloid diseases that are caused by aggregates of the protein transthyretin, analogous to the β-amyloid deposits seen in the brains of individuals with Alzheimer disease (Alhamadsheh MM et al. Sci Transl Med. 2011;3:97ra81).
Cardiac amyloidosis is a chronic and progressive condition that can lead to arrhythmias, heart failure, and death. Cardiac deposits of transthyretin can be found at autopsy in 10% to 15% of individuals older than 65 years.
Hampton T. Molecules Block Heart Amyloid. JAMA. 2011;306(13):1431. doi:10.1001/jama.2011.1392