Gene therapy and adrenoleukodystrophy: an interview with Dr. Nathalie Cartier

Written on Wednesday 6 March 2013

Dr. Nathalie Cartier

Dr. Cartier spoke during the Telematin Health chronicle of October 23rd 2012 animated by Brigitte-Fanny Cohen and dedicated to the ELA week and to leukodystrophies.

“The decisive step in the treatment of leukodystrophies was in 2009 when we were able to show for the first time that we could correct sick childrens’ own cells using a viral vector derived from HIV, the virus responsible for AIDS. Of course it was a question of an AIDS virus that was totally inactivated, completely inoffensive that had merely retained the extraordinary property of this virus carrying a therapeutic gene to be able to enter into cells. That was the first successful gene therapy with the help of this virus and we have treated 4 children, with a perspective of up to 6 years for the two first children treated.
The first 2 children treated are very well. Their disease is totally stabilised. The third child treated had a result that was a little less satisfactory as the gene therapy was efficacious but to a level insufficient to allow us to consider it to be a success. The 4th child was treated two years ago and today shows results that are extremely encouraging.
This therapeutic trial allowed us to treat 4 children. Obviously, this is a very low number. It has to be recognised that the trial was extremely cumbersome to set-up. Our aim for 2013 is to carry out a more extensive trial in Europe and the USA, which I think will allow us to treat about 15 children with the same gene therapy and the same vector derived from HIV. If this trial is conclusive, it will be as though we have set up a new drug and it will allow us to propose this treatment on a large scale, for all children affected by leukodystrophy.”

Dr. Nathalie Cartier
Inserm U-745, Paris, France

Source (French):