Andres is a little boy with ALD which is a form of leukodystrophy, a rare genetic disorder which can destroy the brain. Left untreated, within around two years patients can enter a permanently vegetative state and even die.
Andres however, is undergoing a new treatment in France. Professor Patrick Aubourg and Doctor Nathalie Cartier-Lacave are treating him with stem cells which have been altered by the introduction of inactivated AIDS virus cells. They have already successfully treated other children in this way. Says Nathalie Cartier-Lacave, “We have completely stabilised the evolution of this disease. These children are well, they go to school, they have a social life, a normal family life, and there’s no reason to think that this stabilisation isn’t permanent.” The technique was developed here in the Faculty of Pharmacy in Paris. First, stem cells are harvested from the patient’s soft bone tissue. Then a correcting gene is introduced using inactivated AIDS virus cells. These cells are used because they are the only ones which can penetrate into the heart of the stem cells. Reinjected into the patients, the altered cells navigate straight to the brain where they permanently correct the gene deficiency which causes ALD. The therapy could also work for other more common diseases like hemophilia, thalassemia, Sickle Cell Anaemia, Parkinsons Disease and some cancers. If further research confirms these results, this could become standard treatment in the future – replacing bone transplants which are risky, invasive, and which require a compatible donor to be effective.