The world's first drug that prevents the body from producing "bad" cholesterol has been created
Monash University researchers have developed and tested a groundbreaking, world-first oral drug muvalaplin that lowers lipoprotein(a) levels.Researchers have developed the world's first oral drug to tackle a form of cholesterol that has previously been untreatable and is largely due to genetics. This makes it difficult to control its levels through exercise, diet or other lifestyle factors. For the same reason, the "bad" cholesterol is badly affected by special drugs - statins. We are talking about lipoprotein (a) or Lp(a). Lp(a) is a type of low-density lipoprotein (LDL) cholesterol. It is sticky, so it increases the risk of blockages and blood clots (atherosclerosis) in the arteries.
To tackle the problem, researchers at Monash University have developed and tested a pioneering, world-first oral drug, mulvalaplin (mulvalaplin), which interrupts the body's ability to produce Lp(a).
Lp(a) is composed of one molecule of LDL particle apolipoprotein B100 (apo B100) and one molecule of glycoprotein, apolipoprotein(a) or apo(a). Mulvalaplin disrupts the interaction between apoB100 and apo(a), resulting in lower Lp(a) levels.
A total of 114 participants were enrolled in the study; 89 received muvalaplin and 25 received placebo. The experiment was conducted in two phases. The first part evaluated the effect of a single dose of muvalaplin alone in increasing increments from 1 mg to 800 mg in healthy participants with a mean age of 29 years. The second part evaluated the effect of a single daily dose in increasing increments of 30 mg to 800 mg. Healthy participants took the drug for 14 days, the mean age of the study subjects was 32 years and they had elevated plasma Lp(a) levels.
Baseline levels of Lp(a) and LDL cholesterol were measured at the beginning of the study and the effects of muvalaplin on its levels were observed. The researchers observed a decrease in Lp(a) levels from baseline as early as the second day when participants received multiple doses. The reduction in Lp(a) ranged from 64% to 65% at doses of 100 mg or more on days 14 and 15. No deaths or serious adverse events were reported.
Researchers note the limitations of the study, namely that this was a small phase 1 study. Larger and longer clinical trials in more diverse populations will be needed.
The study is published in the journal JAMA Network.