Real Health

The adrenoleukodystrophy is the most common form in France of leukodystrophy, a rare genetic disease gradually destroys the brain. For the first time, 2 children were rescued by autologous bone marrow whose genes have been repaired.

The adrenoleukodystrophy (ALD) representsesent nearly 30% of cases of leukodystrophy identified by ELA (European Association against leukodystrophies). This association strongly mediated through Zinedine Zidane is the main funder of research on this disease through public donations.

The ALD is a rare but fatal disease characterized by progressive destruction of myelin, the protective sheath of neurons in the brain. So far,only a bone marrow transplant was possible to limit the effects of ALD, treatment related to the existence of a compatible donor and with many potential complications. But today, the gene therapy opens new perspectives, with the announcement of the treatment of two children by their own stem cells without using a donor extérieust

To succeed in this world premiere, the team of Prof. Dr. Aubourg and Cartier (Hôpital Saint Vincent de Paul, INSERM) collected stem cells, bone and put them in contact with a vector comprising a functional version of the gene deficient in ALD. This vector "medicine" is a derivative modifieded and completely inactivated the AIDS virus, it allows to enter the therapeutic gene in the nuclei of stem cells. These do not divide, allowing a therapeutic effect long term.

Then these genetically modified cells were reinfused to 2 children, Angel (9 years) and Andres (8 years). They then recolonized their bone marrow and some of them, by a natural mechanism, havemigrated to the brains of patients to exercise their corrective role. Result, the disease progression was arrested several months after treatment, without particular side effects so far.

This new therapeutic option, obtained through the support of an association of parents who fought for 17 years, is very promising and should be followed by an expansion of testing or screening systématiq ue of the disease at birth, to treat before the onset of neuronal damage.

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These results also consider the future use of other drugs, viruses, especially for treating genetic diseases much more common as the beta-thalassemia, the5_cytose.htm "target =" _blank "> sickle cell anemia, the Hemophilia and even Parkinson's disease.

Jean-Philippe Riviere

Source: ELA, press release, November 5, 2009
Photo: ELA