Treat using the genes

epictura

Published the 20.10.2017 at 13: 00

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therapy géniquelymphomeparkinson

The Food and Drug Administration (FDA), the very powerful american agency that authorizes the use of drugs in the u.s., has on Wednesday approved the placing on the market of the second gene therapy in the world, the Yescarta, to treat non-hodgkin lymphoma aggressive, a cancer of the blood, very serious. Gene therapy, the most modern therapeutic solutions. It is well of the virtues, including that of may be doing of miracles tomorrow in the treatment of diseases such as Parkinson’s disease, or cancers such as lymphomas, in fact those where a gene is defective ; it is, in particular, all of the ones we talk to the telethon.

… What is gene therapy ? The responses of Dr. Yann Le Douarin

We know gene therapy since when ?

YLD : I make you go back the history of medicine… We are in November 1994 the u.s. congress of the heart, Dr. Jeffrey Isner announced to the applause of thousands of fellow he just regrow the arteries of the heart of a patient, thereby opening a new life to all the victims of myocardial infarction. The reason for this revelation almost miraculous : the first application of a theory brilliant, that of gene therapy.

Gene therapy, if we simply translated, it means ” treatment by the genes “, is it ?

YLD : absolutely ! Behind this name hides in fact a part of Meccano, played by the doctor. Our cells are all controlled by our genes, which form a sort of bar code in which everything is recorded : our past but also our future. And if all men are not the same, this is because the possible combinations of genes are infinite. Some genes are in perfect health ; some people are different from birth ; others are damaged at billions of divisions our cells. This is what happens in Parkinson’s disease, where these genes are damaged preventing the cells secrete the product that prevents it from shaking.

Healing by genes, I understand the principle… But then, it is in the infinitely small. We can not go there with the scalpel…

YLD : This is, in fact, the reflections that are made in the early 90’s, all the researchers. How to act, or there is no tool that can fit it ; how to do gene therapy ? And even using the smallest living organism that can move almost as one pleases in our body : the virus.

This means that all the viruses are not dangerous… some are even very useful…

YLD : Yes, viruses are not all as dangerous as that of the flu or worse, aids. And then we also know, in case of doubt, make it harmless. Rest after in the draw up to the delivery of a piece of a gene to change, and to deliver it to the right place.

In the case of Parkinson’s for example, we inject these viruses repairers to the syringe, in the region of the brain responsible for the disease. Then, they enter the cells to repair them. The proof of this ? These cells recover to produce. This is a true miracle of the intelligence of man.

But how a virus can “repair” ? It is not endowed with intelligence ?

YLD : In fact, it is only used as a carrier. He puts it on the back piece of a gene that is failing, it is that the so-called ” dressage “. It should be injected, and, as he likes to go in the cells to multiply – with flu outbreaks throughout the world can check it out – it quickly finds its target, sneaks into the nucleus of the cell, and entering, brings the piece that is missing. It looks easy, but it is exactly what it is. The more hard it is to find a good virus and put it in the right place.

In what other diseases we are going to use it ?

YLD : Potentially, wherever the genes responsible for the disease have been identified. For example in hemophilia, for the failing hearts, for diseases of which we speak every year at the telethon. “Today marks a new step in the development of a scientific paradigm entirely new for the treatment of serious diseases,” said Dr. Scott Gottlieb, the director of the FDA. The results that led the FDA to approve this treatment are indeed spectacular… On more than 100 patients, six months after treatment, 80 % were still alive. In total, 39 % had no sign of cancer. A very significant result as compared to all other known treatments.