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[text on screen] An Interview with Professor Patrick Aubourg, MD. Hopital Saint-Vincent de Paul University, INSERM in Paris. A successful gene therapy trial to treat ALD
[text on screen] What is particular to adrenoleukodystrophy (ALD) and the current standard of care that drove you to choose gene therapy as a way to treat ALD?
Dr. Patrick Aubourg: Twenty years ago we showed that bone marrow transplantation, which was from using cells from a donor, is able to arrest the cerebral disease provided the procedure is performed at the early stage of the disease.
But it is not always possible to make bone marrow transplantation because of the lack of donor of cord blood.
And in addition, bone marrow transplantation is associated with significant risk, particularly in adults.
So, the idea was to use the own bone marrow cells of the patient's and introduce a normal copy of the gene using a new vector therapy.
[text on screen]: What were the results of the clinical trial?
Dr. Patrick Aubourg: Up to now we have treated two boys who were candidate for bone marrow transplantation but without donor, and overall we can say with confidence that more than two years after gene therapy, their brain disease has been arrested in an identical way as it could have been arrested from bone marrow transplantation.
That is, the brain lesion progressed a little bit after gene therapy and is the same after bone marrow transplantation and were free fourteen months after gene therapy, the brain lesion has been arrested and I am quite confident that it will be arrested for life now.
[text on screen] What would have happened to these 2 boys if they did not receive this gene therapy treatment? 0:01:56.000,0:02:23.000 Dr. Patrick Aubourg: If it could not have been possible to do gene therapy in these two boys. The disease would have progressed markedly and they would be now in very severe condition with loss of vision, loss of all motor function, a lot of walking in the late stage of the disease, and they're very at risk to die after a few months of, say, a couple of years.
[text on screen]: What does this mean for children and adults suffering from this often fatal disease?
Dr. Patrick Aubourg: The meaning for the ALD family is that we can expect, now, in the very near future, that this gene therapy approach could be available for all patients who are candidates to a bone marrow transplantation in a blood-brain disease.
Even, first, for those with no matched donor; no matched donor or perfectly matched cord blood, and this in particular concerns the adult, which lack a very fine donor because it is necessary to have the right number of cells.
In addition, we can also anticipate, more later, that this gene therapy approach could become primary therapy for all patients with cerebral disease. In particular in the adult, it because the risk of bone marrow transplantation is very high, close to 30%.
And, even, we have still to work on that, later on, we think the patient will have only the spinal cord form of the disease which is called adrenomyelneuropathy, which affects, as you know, adult males but also heterozygote (woman) carrier, might also benefit from this therapy.
[text on screen]: How did you overcome many of the challenges you encountered?
Dr. Patrick Aubourg: It has been a long way. In fact, with my collaborator, Nathalie Cartier, we started to work on this gene therapy program in the early '90s, specifically in '93.
But at the end of the '90s we moved to use a new therapy; new gene therapy which is derived from a *** virus, and I must say that The Stop ALD Foundation and particularly Amber and Rachel Salzman have played a pivotal role since early 2000 in helping us to contact the right persons in the U.S. to make possible this trial, in particular to make the vector and without their help this would not have been possible and I must thank them very much on behalf of all ALD patients.
[text on screen] Produced by The Stop ALD Foundation, www.stopald.org. For more information please contact Amber Salzman, ph: +1.610.659.1098 * amber@stopald.org