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Nevertheless, there's plenty of work ahead before ApoA-1 Milano is certified as an effective treatment for heart disease. For one thing, says Rader, it may be logical to assume that reducing plaque always lowers the risk of heart disease, but "we have no proof of that. That's a big assumption." Besides, he says, "we really have no idea how ApoA-1 Milano did what it did." The prevailing theory is that it works by what he calls the "dump truck" mechanism, binding to cholesterol molecules in plaque and carting them off to the liver, but that's also unproved. "In medicine, whenever you really don't understand a mechanism, you always have to be a little uncertain about what it's really going to mean."
Even if ApoA-1 Milano turns out to be too good to be true, it's just the opening salvo in an entirely new strategy for fighting heart disease. After two decades of focusing on lowering LDL, doctors are now looking hard at raising HDL. Predicts Nissen: "Every pharmaceutical company and a lot of academic researchers and the NIH are going to work on ways to do this."
--Reported by Sora Song/New York