31 May 2012

Paving the way for a new medicine

Illustration for article: Paving the way for a new medicine
To develop treatments for rare diseases, AFM-Téléthon has taken an innovative route: using biotherapies based on our knowledge of genes and cells. This strategy is starting to bear fruit.

Cures using biotherapies

Since the 1980s, AFM-Téléthon has been convinced that treatment pathways must be found, based on the origin of these diseases, i.e. genetics. It acquired the means for carrying out this research by devising the French Telethon.

Following up every pathway

Several therapeutic pathways have emerged as knowledge of genes and cells has progressed: gene therapy (use healthy genes as treatment), gene surgery (operate on a sick gene to repair it) or cell therapy (reconstitution of injured organs or tissues using stem cells). To multiply the chances of developing treatments, AFM-Téléthon explores all these pathways, without neglecting research into drugs produced by classic pharmacology.

Clinical trials supported by AFM-Téléthon

A successful strategy. Gene therapy has proved its efficacy on the first rare diseases and trials of new treatments are increasing. AFM-Téléthon supported 33 trials concerning 26 diseases in 2016. Researches in biotherapies initiated by AFM-Téléthon will have very positive effects on common diseases.