Knockout of the risk factor
The APOLLO clinical study conducted by the Cleveland Clinic and presented at the 71st Annual scientific Session of the American College of Cardiology showed that experimental gene therapy reduces the level of genetically determined risk factor for cardiovascular diseases lipoprotein (a) to 98%.
Translated from scientific, "98% reduction" means "up to 2% of the original", i.e. 50 times!
In a double-blind placebo–controlled study, participants received different doses of SLN360, a small interfering RNA (siRNA) that knocks out the gene responsible for the production of lipoprotein (a). The group receiving higher doses showed a 96-98% decrease in lipoprotein (a) levels; after five months, it remained at 71-81% (approximately 3.5 – 5.3 times) below the initial level.
Phase 1 of the APOLLO study showed the safety of experimental treatment.
Lipoprotein (a) is similar to low-density lipoproteins (LDL), "bad cholesterol". It is produced in the liver, where the protein apolipoprotein (a) attaches to a particle similar to LDL. Unlike other lipoproteins, the level of lipoprotein (a) is 80-90% genetically determined. It causes plaque buildup in the arteries that leads to cardiovascular diseases, including heart attack and stroke. There are effective methods to reduce the risk of heart disease by reducing the level of LDL and other lipids, but there are no approved methods to reduce the level of lipoprotein (a), because it is genetically determined, and lifestyle changes, diet or exercise have no effect on it.
The study involved 32 people in five medical centers in three countries. All participants had lipoprotein (a) levels above 150 nmol/L (median 224 nmol/L); 75 nmol/L or less is considered normal. Eight participants received a placebo, and the rest received one of four doses (30, 100, 300 or 600 mg) of SLN360 with a single subcutaneous injection. The participants were monitored for the first 24 hours after the injection, and then periodically examined for five months.
Participants who received 300 mg and 600 mg of SLN360 showed a maximum decrease in lipoprotein (a) levels – by 96% and 98%, and after five months – by 71% and 81% compared to the baseline level. In the placebo group, the level of lipoprotein (a) did not change. High doses of SLN360 also reduced the level of LDL proper by about 20-25%. The most common side effect was soreness at the injection site. The study is not finished: its participants will remain under observation for one year.
The findings suggest that siRNA therapy may be a promising method of preventing coronary heart disease and other cardiovascular diseases in people with high levels of lipoprotein (a), which are estimated to affect 64 million people in the United States and 1.4 billion people worldwide. It is estimated that from 20 to 25% of the world's population has an elevated level of lipoprotein (a).
Article by S.E.Nissen et al. A Single Ascending Dose Study of a Short Interfering RNA Targeting Lipoprotein(a) Production in Individuals With Elevated Plasma Lipoprotein(a) Levels is published in JAMA.
Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru According to the Cleveland Clinic: Cleveland Clinic-Led Trial Finds That Experimental 'Gene Silencing' Therapy Reduces Lipoprotein(a), an Important Risk Factor of Heart Disease, By Up To 98%.