“We are facing a positive wave of clinical trials and new treatments”, declared on franceinfo Marc Peschanski, scientific director of the Istem institute, the Institute of stem cells for the treatment of diseases, on the occasion of the launch of the Telethon 2021 on Friday 3 December.
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“What has advanced dramatically is gene therapy” which makes it possible to cure more and more children, explained the researcher. This progress is enabled by “people’s generosity” and donations on the occasion of this great operation.
franceinfo: Is this 35th edition of the Telethon important when we know that the mobilization was more complicated in 2020?
Marc Peschanski: It is extremely important, because we are facing a great positive wave of clinical trials, treatments, new ideas that we are experimenting with patients who are waiting for them. This is something that should absolutely not be missed, it is the realization of these decades during which the Telethon funded our research. It is really on the basis of the generosity of people that what we can consider as the beginning of success has been built.
In 35 years of Telethon, what concrete scientific progress has already been made and what remains to be done?
We are dealing with 7,000 diseases, so we still have a few left. 40 years ago, when I finished my medical studies, all these diseases were considered incurable, it was a word that was used to students. When I got into this kind of research, my colleagues said I was just a “stamp collector” because there would never be a cure. The Telethon is the idea that no, there is no inevitability.
“What has advanced dramatically is gene therapy. We see children living when the disease condemned them after 18 months, two years. It affects hundreds and hundreds of children around the world. ! “
Marc Peschanski, scientific director of the Istem instituteto franceinfo
So there is a concretization, a variety of diseases for which gene therapy can be applied. Then there is the cell theory, which is more recent but which is starting to give the first results, on the retina, on diabetes. The path was the right one, scientists must have the means to work over several years.
There are still many diseases to discover, to decipher, treatments to find?
The priority now is not the diseases to be discovered, because we know most of the diseases, we have the means to know. We are going to name new diseases that we already know. It is at the level of treatments that we must look. Today, the most optimistic estimates say that only 5% of genetic diseases have a start of treatment, 95% have nothing. When I say that we have nothing, it is not that we are doing nothing. On the contrary, we are doing a great deal because we all have this perspective in front of us of the therapeutic application.