“We deprive children of treatment for commercial reasons”

A week before a European conference which will be devoted, on February 28 in Paris, to the care and innovation pathway for rare diseases, Laurence Tiennot-Herment, the president of the AFM-Téléthon, confides to the JDD her indignation to see French people deprived of access to innovative medicines. In his view, this is due to a European investment ecosystem that is not very favorable to innovation and to the indifference of the public authorities, at a time when the government is nevertheless showing the will to promote therapeutic innovation and to densify the industrial fabric in the country.

Why are you angry ?
I am outraged, as a manager and also as a mother, that gene therapy drugs from French research are not accessible to patients in Europe. The example of Skysona shows how a good story can turn into a nightmare. After ten years of research initiated by French teams and clinical trials, this innovative treatment against X-linked cerebral adrenoleukodystrophy, an ultra-rare and fatal neurodegenerative disease, has been authorized in Europe. But the American laboratory Bluebird Bio, which developed it, announced that it was abandoning the European market to refocus on the American market, for very questionable reasons of commercial strategy, and the hope flew away last October. Gene therapy is no longer accessible to European children who have no other alternative. Will they have to go to the United States tomorrow to benefit from it? How not to be indignant that children are condemned to death in general indifference when there is an effective treatment?

Isn’t this an isolated case?
This also concerns a treatment against Beta-thalassemia, a less rare disease, the marketing of which in Europe has also been abandoned by the same laboratory, Bluebird Bio. With the difference that in this case, there is a therapeutic alternative with very restrictive regular blood transfusions. We who speak for the 3 million French people suffering from rare diseases think that, soon, such aberrations are likely to happen again: other drugs resulting from French research could suffer the same fate. It takes a lot of money between the first laboratory results and the marketing of a gene therapy. From the first stage, developing a drug candidate requires significant funding. It is at this stage, when we realize that it works in mice and in the first patients, that the problems begin. The French ecosystem of funders, devoid of an American-style risk culture, does not ensure the development or even the availability of these treatments, which are often developed here with the support of the Téléthon in particular. So the most creative biotechs cross the Atlantic to be able to raise funds and pursue their growth. It is urgent to encourage the creation of pro-active funds, partly involving the State, with greater means, perhaps on a European scale.

Read also – In Evry, a laboratory incubator in the wake of the Telethon

But, from the start of the pandemic, the government seemed to realize the need to promote therapeutic innovation and drug production in France.
This is undoubtedly true for boxes of paracetamol but not for gene therapies! Between political speeches and facts, there is a huge gap. This is why, since 2018, we have been alerting French leaders: rare diseases are the spearhead of innovation and yet they are left out of current efforts. Our fears were brought to the public square before the problem emerged at the start of the pandemic: in September 2019, we devoted a symposium to the National Assembly to insist on the need to maintain national health independence with the aim of access the gene therapy revolution. Today, Genethon [le laboratoire de pointe du Téléthon] has nineteen drug candidates in its portfolio. Five of them are brought together in a biotech. For several months, we have been trying, without success to date, to raise funds in France. We have approached many investors, alerted the highest level of the State, but nothing is happening. At the same time, American and Asian funds say they are interested in our projects. It’s the world upside down. We are constantly talking about the reindustrialisation of France, but it is possible that in ten years, most gene therapy drugs will be developed and produced elsewhere than here, even though we will have designed them. There is a significant risk of seeing these innovations marketed at exorbitant prices by American companies; and difficult to assume by our health systems. Admittedly, we are a small country, but European sovereignty should be able to get under way in this area.

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Children are condemned to death in general indifference

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Beyond the problem of investments, these drugs have a very high cost. Why, in the case of orphan diseases, has no one ever considered imposing an ex officio licence?
Several months ago, we wrote to Olivier Véran, the Minister of Health, to ask him to activate these mechanisms in the case of Skysona. He has not replied to us to date.

Beyond the human and ethical aspect, why should rare diseases be a public health priority?
Because rare diseases concern 3 million French people and 30 million Europeans. In addition, they are an innovation laboratory for more common diseases, such as cancer and neurodegenerative diseases.

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