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18 Jun 2012

Helping research in many ways

Illustration for article: Helping research in many ways
AFM-Telethon supports research by financing young researchers’projects. In 2015, 285 programmes and young researchers were financed by the association.

Genethon Bioprod, a unique centre in the world

 

Genethon Bioprod is the biggest center in the world that is completely dedicated to the production of the first gene therapy treatments for clinical trials on human beings concerning rare diseases.
 
Genethon Bioprod offers a great hope for patients suffering from rare diseases that, up to now, couldn’t be cured.
 
Genethon Bioprod’s goal is to issue 20 clinical  vector batches per year, which will be used for clinical trials on human beings in France but also abroad. Genethon Bioprod will help accelerate the pace of clinical trials on human beings. Hence, new treatments will be available even more quickly for patients in the interest of all.
 
In 2012, thanks to donors’ generosity, Genethon Bioprod has already purchased two “production suites”. Qualification and certification steps for building and equipment are being processed. The path is now open for the batch production of drugs in late 2012, early 2013, that will be used for human clinical trials for 4 diseases.
 
  • Immunodeficiency: Wiskott-Aldrich syndrome 

This immune system disease provokes hemorrhage, eczema, cancer and leads to death, most of the time before the age of 15.
This new treatment is also an indirect source of hope for leukemia, AIDS, sickle cell anemia, hemophilia.
 
  • Blood diseases: chronic granulomatous disease

The disease is responsible for severe infection and hyper-inflammation.
This new treatment is also an indirect source of hope for leukemia, AIDS, sickle cell anemia, hemophilia.
 
  • Muscle diseases: Duchenne muscular dystrophy

Duchenne dystrophy’s symptoms are typical: progressive and irreversible muscular weakness, complete loss of the capacity to walk, usually before the age of 15, as well as the other muscle functions (arms and legs, diaphragm, heart) leading to the patient’s death.
This new treatment is also an indirect source of hope for other types of muscle weakness due to aging, AIDS and cancer.
 
  • Eye disease: Leber hereditary optic neuropathy

The symptom is characteristic: the patient suddenly loses his central vision.
This new treatment is also an indirect source of hope for age-related macular degeneration and diabetes-related blindness
 

What is gene therapy?

Gene therapy is a real medical revolution. Its efficiency was proven with the first clinical trials on various diseases: immunodeficiency, rare brain and blood diseases.
The innovative technique consists of introducing a healthy gene (or gene-drug) into the patient’s body in order to correct the genetic anomaly that is responsible for the disease. 
 

How to get involved?

Take part into the production of the first batches of gene therapy treatments dedicated to human clinical trials for rare diseases.
€ 400,000 to € 500,000 (€ 160,000 to € 200,000 after tax deduction) = production cost for 1 drug batch.
 

Helping young researchers imagine tomorrow's medicine

By funding research grants, you help young researchers stay and work in France. You also help in increasing France’s scientific influence worldwide.
 
Every year, AFM-Telethon organizes a “young researchers’ seminar”. There are several aims: to share information about ongoing research, to promote and to honor the best projects.
 

How to get involved?

You can help us fund those jobs.
 
For example…
  • The yearly “cost” of a post-doc student is € 50,000 (€ 20,000 after tax deduction)
  • The organization of a young researchers’ seminar costs € 40,000