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Forget Clearing Cholesterol—What If We Just Stopped Making It?

Researchers at the Medical University of South Carolina used human‑like liver cells derived from induced pluripotent stem cells to screen 130,000 compounds, identifying molecules that block apolipoprotein B (ApoB) and dramatically reduce LDL production. The lead compound, DL‑1, lowered cholesterol and triglycerides in these cells and proved effective in humanized mouse models that carry human liver cells, overcoming the species‑specific failure seen in ordinary mice. By targeting cholesterol synthesis rather than clearance, the approach sidesteps the defective LDL receptors that render statins ineffective for many familial hypercholesterolemia (FH) patients. The findings point to a new therapeutic pathway for the roughly 1 in 200 adults carrying FH‑related gene mutations.