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05 Dec 2012

Telethon 2012 : New drug trials in 2013

Illustration for article: Telethon 2012 : New drug trials in 2013
Young patients with immunodeficiencies, rare diseases of the blood and brain, have already benefited from innovative therapies stemming from research driven by the Telethon. Thirty-six trials are in progress or in preparation for nearly 30 diseases.

The AFM-Telethon is a pioneer in the development of innovative treatments for gene therapy. In 2011, Genethon began a trial for Wiskott-Aldrich syndrome (in French), a severe immunodeficiency. Genethon is also launching a European gene therapy trial for another immunodeficiency, Chronic Granulomatous Disease (CGD). At the end of 2012, clinical teams based in France, Germany, the United Kingdom and Switzerland, will include 20 patients in a trial of a gene therapy treatment produced at Genethon.

Production of gene therapy treatments

To carry out trials in humans, Genethon created Genethon Bioprod. This centre is dedicated to the production of gene therapy vectors - carriers of the gene treatment - in sufficient quantity and quality for clinical trials. To treat a man, 3500 times more vectors are needed than to treat a mouse. And the cost of a batch of vectors for one patient is several hundred thousand euros. Genethon has a major role in the acceleration of therapeutic innovation and was awarded with the 2012 French Galien Prize.

Trials for vision disorders

At the end of 2011, researchers and clinicians in Nantes began the first French gene therapy trial in humans for an eye disease (in French), Leber's congenital amaurosis. Nine patients are currently being recruited. Other vision disorders are also on fast track for human trials. Genethon and The Institute of Vision will launch a new trial for Leber’s Hereditary Optic Neuropathy in 2013.

Biological dressings for drepanocytic ulcers

After having reconstituted an epidermis from embryonic stem cells, the I-Stem laboratory, co-founded by the AFM-Telethon, is continuing its efforts to propose this unlimited resource of cells for patients with rare skin diseases, severe burns and for people with ulcers associated with different diseases.

A cell therapy trial for the treatment of skin ulcers associated with sickle cell disease (a genetic disorder of the blood) is due to start in 2013. The ulcerations will be treated with a biological dressing consisting of completely reconstructed, standardised and laboratory controlled epidermis. If the trial is successful, this type of dressing may also be appropriate in other diseases.

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