November 6, 2009
2 Children With Adrenoleukodystrophy Saved Thanks To ELA Association
The ELA Association represents the largest funder for research on this fatal brain disease
The ELA association and Zinedine Zidane, its emblematic ambassador, are proud to announce a world premiere: the results regarding the gene therapy in adrenoleukodystrophy conducted in France have just been published in the prestigious journal Science. Two children have been treated and their diseases have been halted. The children are doing well, which is unexpected for a disease destroying the brain in a few months. This discovery opens up treatment perspectives for numerous widespread diseases.Gene therapy on adrenoleukodystrophy: a world premiere
Adrenoleukodystrophy (ALD) is the most frequent form of leukodystrophies. It affects nearly 30 % of leukodystrophy cases identified by the ELA association. Each year, 35 babies with adrenoleukodystrophy are born in France, but death often occurs during childhood.
Until now, the treatment for ALD relied on bone marrow transplant, an approach which is limited by the scarcity of donors and the risk of serious complications. With this new approach, physicians have chosen to perform an autologous graft combined with gene therapy. For this procedure, the bone marrow stem cells of the patients are harvested, and then corrected in the laboratory before being transplanted back into the patients. Some of these cells will naturally find their way to the patient's brain where they will display their correcting potential. Another innovation: a vector derived from a modified and inactivated HIV virus was used to insert the correct gene into bone marrow cells. HIV represents the only virus able to introduce a therapeutic gene into the nucleus of non-dividing cells, like stem cells and neurons, in order to allow a long-term effect of this gene.
With 7 million Euros invested, ELA is the largest funder for research on ALD
These very large amounts of money have been gathered thanks to the actions undertaken by millions of school children, to the support of the French Ministry of Research, to the generosity of all the donors. Also, the involvement of our partners and ambassadors, led by Zinedine Zidane and Florent Pagny, contribute to this success.
A discovery that will benefit to widespread diseases
Today, ELA is even prouder of this result because this innovative approach opens up treatment perspectives for other types of leukodystrophies and most importantly to other widespread diseases (sickle cell anemia, beta-thalassemia and multiple immunodeficiencies, hemophilia, Parkinson's disease, "¦) which could benefit from a similar gene therapy treatment using a HIV-derived vector.
Founded in 1992 by Guy Alba, parent of a child with ALD, with the encouragement of Pr. Aubourg, chairman of the scientific committee until 2008, ELA brings together European families affected by leukodystrophies, a group of genetic diseases that destroy the myelin (nerve sheath) in the brain. Since its creation, ELA pursues the same goals: support patients, gather families and fund medical research. To accelerate the discovery of a treatment, ELA founded its own research foundation in 2005 with the support of the French Ministry of Research. The foundation's efforts and momentum permitted dedicating 30 million Euros for all kind of leukodystrophies.
Concomitantly, ELA cares for patients and brings special attention to those who may not benefit from research advances. That's why ELA commits 4.3 million Euros for the support of patients and their families needs. With the same motivations, ELA supports the development of similar organizations in Europe (Belgium, Spain, France, Italy, Luxembourg, and Switzerland).
And ELA is planning for more
1. extend the trial in Europe and recruit new patients including adults
2. develop a newborn screening test
3. offer the therapy to all ALD patients
Testimony of Guy Alba, founder and president of the ELA association (ELA) and parent of a child suffering from adrenoleukodystrophy (ALD)
I met Pr. Patrick Aubourg for the first time in 1991, when he diagnosed my 5 year-old son with adrenoleukodystrophy. I knew precisely the damages caused by this horrible disease (loss of motor function, of vision, speech, hearing, memory...) because they were destroying my nephew, 5 years older than my son, prefiguring what will happen to my little boy.
Although petrified by this astounding news, it seemed nevertheless inconceivable to passively wait for the worst to come. I had long conversations with Patrick Aubourg who encouraged me to create a patient organization and accepted to chair its scientific committee. This is how ELA was born in 1992.
17 years later, the present victory over adrenoleukodystrophy bears the marks of all these fighting years, pains, losses, but also of numerous moments of solidarity and happiness. This victory against the disease is due first and foremost to the great research team lead by Pr. Patrick Aubourg and Dr. Nathalie Cartier. We also owe this victory to the tenacity of the highly committed ELA families who held on during all this time. We are furthermore indebted to the thousands of school children involved each year for ELA, to all our donors, all our partners, and if there was only a single one to name, to Zinedine Zidane, given his personal level of involvement and care.
Thanks to him and to them, ELA was able to fund ALD research at both national and European levels, but in the US as well, globally in a quite exceptional manner (7 million Euros invested so far), and to continuously support Pr. Aubourg's team since 1992.
All ELA families were impatiently waiting for this major breakthrough even those who do not expect anything from this discovery because the disease has already taken everything away from them. Today we are even prouder of this result because gene therapy on ALD opens up treatment perspectives for numerous other diseases that are more widespread. The children of ELA have thereby contributed to open the way for a new therapy which, we truly hope, will be useful to many other sick children and adults suffering from many kinds of diseases. It will be our best reward.
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