Millions of people share a fatal genetic heart risk. This experimental medication could change the situation

An experimental drug could change the situation for millions of Americans with higher genetic risk of cardiovascular disease. In clinical trial data published this weekend, Eli Lilly’s Lépodisiran has considerably reduced the levels of people of a dangerous cholesterol called lipoprotein (A) or LP (A).

Eli Lilly announced the results evaluated by the peers of his phase 2 test of Lepodisiran on Sunday, which involved more than 300 people genetically predisposed to the high LP (A). People taking the highest doses of Lepodisiran experienced a 94% drop in LP levels (A) to a year later. The results indicate that Lepodisiran could soon become the first treatment of gender for this common genetic risk factor.

Our body cholesterol is transported through several types of lipoproteins. Lipoprotein with low density, or LDL, is often known as the bad type of cholesterol, because too much can increase the risk of reinforcement of the plate in our arteries (atherosclerosis), which then increases the chances of having heart lines, features and other cardiovascular problems.

LP (A) is a form of LDL which can also increase the risk of plaque accumulation. But unlike the classic LDLs, LP (A) levels are largely determined by our genetics, not in lifestyle. It is estimated that around one in five people in the world is genetically predisposed to have a high LP (a), and there is no existing intervention which can reduce LP (A) – at least for the moment.

Our genes contain the necessary instructions for cells to produce proteins, but this only happens when the genes are expressed. Our bodies sometimes use a specific form of RNA to silence this expression of genes, called small interferring RNA (Sirna). In recent years, scientists have developed Sirna -based drugs that have been working on the same principle, such as Lepodisiran. Lepodisiran inhibits the production of the apolipoprotein liver (A), a key component of LP (A).

In the phase 2 alpaca trial, 320 people with high LP (A) were randomly assigned to five conditions; Three groups received two subcutaneous injections from Lepodisiran at the start of the study and six months later at variable doses. Another group received the highest dose of Lepodisiran at the start of the study and a placebo six months later. And the last group received only a placebo.

Everyone received the Lépodisiran experienced a reduction in the LP (A) compared to the placebo. But those in the highest dose saw a drastic reduction of 94% of the LP (A) to six months. People received only one highest injection of the dose experienced a slight rebound in LP (A) a year later (an overall reduction of 88%), while people received two injections underwent a 95% reduction in LP (A) a year later. The results were published in the New England Journal of Medicine.

Phase 2 trials are mainly used to further test the safety of a drug and find its optimal dose. It will therefore take more research to confirm these results. But associated with previous data, Lepodisiran generally seems to be safe, without serious adverse events reported linked to the treatment in this last trial. And given the dramatic of these results, external experts are naturally enthusiastic about the future of drugs.

“This is remarkable,” said NBC NBC News, director of preventive cardiology at the Cardiovascular Center at the University of Michigan, Eric Frankel Cardiovascular Center in Ann Arbor, who was not involved in the study. “These drugs have the potential to eliminate this lipoprotein almost.”

Eli Lilly has already started to register volunteers for her phase 3 test of Lepodisiran. If the drug continues to function as hoped, it could become the last realization for therapies based on Sirna. In 2021, the Food and Drug Administration approved Alnylam Pharmaceuticals and Leqvio de Novartis as a lodbed treatment for people with certain genetic or poorly controlled atherosclerosis conditions.