Imagine that we can get the body itself to develop the necessary medications. Genetic technology called RNA-therapy may play a huge role in the treatment of many diseases. Norwegian scientists are trying to develop a cure for one of the most common types of cancer — breast cancer.
“Although we started only in October 2019, we can say that the prototype is ready, we will soon begin testing on animals,” says researcher Sven even Borgos (Sven Even Borgos).
He headed the direction, which in the framework of the European project for the development of treatment against this dangerous type of cancer like triple-negative breast tumor, is the Norwegian Agency for industrial and technical research SINTEF. Project EXPERT at its disposal as much as 150 million kroner, as well as scientists from 11 countries.
The Central role research plays in the matrix ribonucleic acid, or mRNA.
An mRNA referred to as a copy of the functional segments of DNA. Cells use it as instructions for making proteins.
Now scientists are developing a cure based on mRNA. The idea is to give your body artificially created instructions so that it itself could produce important proteins, the lack of which causes disease.
The object of gene therapy — the body’s own proteins
How important are genes for protein production, can be seen on example of hemophilia. Those who suffer from it, missing one of the genes responsible for the production of proteins, by which the blood coagulates.
“Many people probably remember the debate in 2018 about a very expensive medication “Spinrite” that effectively helps some patients with spinal muscular atrophy. This disease slowly but surely destroys the muscles of a person. And the action “Spinnaz” based just on related technologies”.
“”Spinrate” costs for each patient in the many millions because software companies want to regain the money spent on long and expensive investigations,” says the scientist of SINTEF.
According to him, this shows how important state funding for such medical research. Medicines, funded by the company, become available to this society.
Namely, it is the aim of the project EXPERT and scientists involved in the development of drugs against breast cancer based on mRNA.
But how mRNA works as a medicine?
“Drugs based on the mRNA so effective because they work the same way as our genes. The gene contains some codes that tell the body what proteins to produce, and these instructions must pass through mRNA”, explains Borges, a physicist with a PhD in molecular genetics.
Proteins, in turn, responsible including for the exchange of nutrients, degradation of pollutants and the renewal of the body cells.
Thus, thanks to the new method, the body will produce exactly one protein, the information on which it will report the mRNA.
To create the synthetic segment of the mRNA, scientists should be the very DNA with which to make a functional copy. Also need an enzyme that can read DNA code and build a copy.
“This is one of the most important processes for any kind of life,” he says.
But this time everything happens in the test tube. The reaction mixture is purified using a special washing technique, by which genetic components are separated from each other. This allows you to choose exactly that part, which is then aktiviziruyutsya.
No side effects
Proteins, which will work as a cure, naturally adapted to the patient, unlike medication, produced industrially.
Most conventional drugs act by altering the function of various proteins. Often these changes are inaccurate. Causes side effects from minor to life-threatening. In addition, we all react differently to medications, so the medication appeared effective for the mother, it may not work on daughter with the same disease.
An mRNA consists of four components, which are abbreviated as A, U, C and G. the order is read by cells, how the letters in the recipe. If the “recipe” mRNA body is not read, it and any effect will not have, and thus side effects will not. Otherwise, the body will produce exactly one protein, which is required only in the right place.
“For the production of medicines and vaccines it opens up fantastic opportunities,” says the researcher.
However, mRNA is a very fragile structure. If she gets lost, for example, once in the bloodstream, the body itself destroy it. Therefore, scientists have developed a valuable component of a container: it is wrapped in nanoparticles, which typically consist of fats such as cholesterol.
In this fat “sleeping bag” from the nanoparticles mRNA survives in our blood.
Traveling through the body, it finds its way to the cells. There fat also has a special feature: thanks to the cholesterol cells easier to let nanoparticles. The cage surrounds the “packaging” of medicine in its membrane, releasing the mRNA and begins producing the desired protein.
The liver pharmaceutical factory
“However, it is difficult to target nanoparticles directly in the target cells. Very often they get into the liver,” says Borgos.
According to him, the liver works as a repair station, which absorbs everything that’s not supposed to be in the blood. But proteins it can produce. If the liver actively takes mRNA containing nanoparticles, it starts to produce a lot of protein, which the body needs, and send it into the blood. Thus the liver becomes a kind of pharmaceutical factory of the body.
But working on a new cure for cancer, scientists need to put a drug that triggers the immune cells directly into the tumor.
And then it is very important that protective fat container load optimally. It quality and testing is now Norwegian team SINTEF. Her scientists already have years of experience with testing of medical nanoparticles.
The many ways to use
Use of mRNA as a drug can be many different ways. In addition, it activates their own immune system to fight cancer, it also works very well as a vaccine against genetic diseases.
Project EXPERT scientists considering to use this medication for the treatment of heart attack.
Solutions of nanoparticles intended for drug delivery to the desired location, the convenience of having a component of mRNA can be easily replaced, without changing essentially the rest of the mixture, and it will not affect its distribution in the body. This means that when new information about the disease, the way to effective cure will be short.
“Medicine based on the mRNA you can even type directly into the spinal cord to hit it in the head. Thus it will overcome the blood-brain barrier. This almost impenetrable barrier protects the brain, but greatly complicates the treatment of his diseases”, the scientist explains.