If the term wasn't quite overused, I would venture a definition for Inclisiran: it is a sort of cholesterol vaccine. Partly due to its characteristics, partly due to the methods of administration.
After being adopted (exactly a year ago) from the UK health service, where 300.000 beneficiaries are expected, comes today also approved in Italy, from Aifa.
How does Inclisiran, the drug that halves cholesterol, work?
I'll start by saying that it is not a cough syrup, and it is not suitable for everyone. Inclisiran, which is given twice a year as an injection, is prescribed primarily to patients who genetically suffer from high cholesterol, those who have suffered a heart attack or stroke, and those who do not respond to other cholesterol-lowering drugs.
This obviously does not take anything away from the proclamations that are being heard in these hours. The recent approval of the drug was a cause for great celebration, due to its potential benefits and innovative therapeutic approach. The "gene silencing" exploited by Inclisiran fights against the causes of high cholesterol, it is not limited to the symptoms. It aims to target a specific gene and hinder its functioning.
What is gene silencing
Messenger RNA (mRNA) is a type of RNA that copies and transmits genetic instructions from DNA and generates particular proteins based on these instructions. It is a fundamental role for our body. In the case of “gene silencing” applied to cholesterol, the target is a protein called PCSK9. People with high levels of “bad” cholesterol (LDL) have excess of it. It must be prevented from being produced, and for this reason we try to "destroy" the instructions that serve to produce it.
It is the first time that this technology has not been used for the treatment of rare diseases but of a much more widespread condition.
Future treatments
Inclisiran is an important test bed: after cholesterol, in the future we could use this weapon against other enemies such as Alzheimer's disease, cancer and more. Several other gene-silencing drugs are currently being studied to treat one variety of other ailments: kidney transplant rejection, the glaucoma, even the scars.
Each of these treatments gene silencing would use techniques similar to those of other currently existing drugs, targeting a specific gene or protein and turning it off. In the case of cancer, the matter is more complex, because it will be necessary to target different proteins: for this we will need more clinical studies.
This challenge, combined with that of keeping costs low, will keep researchers busy in the near future: but those who wonder (too) impatiently why so many discoveries never reach practical application should know that this happens. In due time and manner.
The potential benefits of therapies using gene silencing are vast and could have a significant impact on many different diseases. The future of gene silencing therapies is very promising and we can only wait to see what new treatments are developed. Let's start with cholesterol!
