Mona Lisa’s Hands Reveal Potential Breakthrough in High Cholesterol Treatment
A recent discovery involving Leonardo da Vinci’s iconic painting, the Mona Lisa, has brought new hope for the treatment of high cholesterol. Researchers at the Medical University of South Carolina (MUSC) have found that the hands of Mona Lisa show signs of a condition called xanthomas, which is associated with familial hypercholesterolemia (FH). This genetic disorder affects around 1 in 200 adults and can lead to elevated levels of bad cholesterol, putting individuals at a higher risk of developing cardiovascular disease.
Traditionally, statins have been prescribed to treat high cholesterol by increasing the levels of low-density lipoprotein receptor (LDLR), which helps regulate cholesterol. However, these medications are not effective for all patients, especially those with specific FH mutations. This has motivated researchers to explore alternative treatment options.
The team at MUSC took a unique approach to tackle FH. They screened a collection of around 130,000 chemical compounds to identify molecules that could decrease the secretion of apolipoprotein B (apoB) from liver cells. ApoB is a major protein found in LDL particles. By testing these compounds, they were able to find a group of molecules that not only reduced apoB secretion but also lowered cholesterol levels. These findings have the potential to open doors for new treatments for FH.
To conduct their research, the scientists generated liver-like cells from induced pluripotent stem cells (iPSCs) derived from skin or blood samples. This method allowed them to test a large number of compounds quickly, which ultimately led to the discovery of a unique class of compounds that could potentially treat FH.
Though the compounds showed promising results in the laboratory, they proved ineffective when tested on mice. However, the researchers found a solution by using Avatar mice, which are mice with livers made from human cells. This innovative model enabled the compounds to work effectively.
Stephen Duncan, a professor and SmartState Endowed Chair at MUSC, emphasized the significance of this research, stating, Showing that you can use these human stem cells as a system to model disease, complete a drug discovery process, and find a drug that could potentially be used to treat a patient – that is the epitome of personalized medicine. This shows there is a very feasible way to do drug discovery using a human system.
Despite the promising results, there is still more research to be done. The next step is to understand the drug target and the mechanism of action. Duncan stressed that this is a top priority for future studies.
The groundbreaking findings of this study offer hope to those suffering from FH and provide insights into a potential treatment option. As researchers delve deeper into understanding the compounds’ therapeutic effects and unravel the mechanism of action, advancements in personalized medicine may pave the way for improved high cholesterol treatments and a brighter future for patients at risk of cardiovascular disease.
