GENE THERAPY SUCCESS IN SEVERE BRAIN DISORDER APPLAUDED BY THE STOP ALD
FOUNDATION
Affected Families Achieve Goal of Saving Children. Exciting Results Reported in
Science.
You can download a copy of The Stop ALD Foundation press release in PDF format
here.
Houston, TX -- November 5, 2009 -- The Stop ALD Foundation today applauded the
investigators who are reporting in the
current issue of Science successful results from the pioneering use of gene
therapy for adrenoleukodystrophy (ALD), a potentially crippling and fatal brain
disorder in young boys.
“As an organization founded by families affected by ALD, we know too well the
ravages that this disorder inflicts on its victims and the heartbreak it brings
to those who love them,” said Amber Salzman, president of The Stop ALD
Foundation. “We are deeply thankful to Drs. Cartier and Aubourg and the many
other scientists and physicians whose achievement is reported in Science, and we
look forward to continuing to work with them to build on their success. Their
pioneering work in gene therapy brings hope to those stricken not only by ALD
but many other serious diseases as well.”
The Stop ALD Foundation has been involved in this gene therapy initiative since
2001 by providing direct funding and by bringing together parties in the US and
Europe who provided critical scientific and biomedical contributions. Given the
encouraging results reported in Science, the foundation will continue to stay
involved, assisting in driving forward a larger, international study including
U.S. patients. It is anticipated that this next study may be open to a more
diverse ALD population, including adult men who suffer from the same genetic
disorder.
The promise of gene therapy in ALD is that it will enable each patient to serve
as his own stem cell donor, obviating the need to find matching donors and
avoiding the serious risks and sometimes lethal side effects of stem cell
transplantation. The paper in Science details the cases of two boys who
underwent gene therapy at Saint Vincent de Paul Hospital in Paris. The boys were
born with a genetic mutation that by the time of their hospital admission had
already resulted in early brain lesions. Their therapy began with removal of
some of their own bone marrow stem cells. These genetically defective cells were
then corrected via a laboratory procedure whereby functioning genes were
inserted. Last, the treated cells were injected back into the young patients.
This therapy arrested the progression of ALD, and over two years later the boys’
conditions have stabilized. No adverse effects of the gene therapy have been
noted to date.
“I know the urgency of boys stricken by ALD and the pain of their parents,” said
Eve Lapin, a founding member of The Stop ALD Foundation. One of her sons died of
ALD, and another is confined to a wheelchair as a consequence of graft versus
host disease following a stem cell transplant. “These gene therapy results are
exciting, but they are just the beginning. Time is of the essence in finding the
safest and most effective therapies. Every day more children become afflicted
with ALD, and their chances of surviving depend on the success of trials such as
this.”
About ALD Adrenoleukodystrophy (ALD) is a genetic disorder estimated to affect 1 in 18,000
people. Its most devastating form destroys the myelin sheath of the brain’s
neurons generally affecting boys between the ages of four and ten years. At
first they show behavioral problems, such as withdrawal or difficulty
concentrating commonly misdiagnosed as attention deficit disorder (ADD) or
attention deficit hyperactivity disorder (ADHD). Gradually their symptoms grow
worse and may include blindness, deafness, seizures, loss of muscle control, and
progressive dementia. This relentless decline leads to permanent disability and
death within two to five years from diagnosis. Accurate and early diagnosis is
essential to an opportunity for effective therapy. The disorder can also affect
adults, women as well as men, in which case it is known as adrenomyeloneuropathy
(AMN). AMN tends to be less severe in adults than in boys, though some men also
develop fatal demyelination in the brain.
About The Stop ALD Foundation The Stop ALD Foundation is a registered not-for-profit medical research
organization that drives research into new therapies and advances understanding
of ALD. More information is available at www.stopald.org.
Citation: “Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in
X-Linked Adrenoleukodystrophy.” By Nathalie Cartier, Salima Hacein-Bey-Abina,
Cynthia C. Bartholomae, Gabor Veres, Manfred Schmidt, Ina Kutschera, Michel
Vidaud, Ulrich Abel, Liliane Dal-Cortivo, Laure Caccavelli, Nizar Mahlaoui,
Véronique Kiermer, Denice Mittelstaedt, Céline Bellesme, Najiba Lahlou, François
Lefrère, Stéphane Blanche, Muriel Audit, Emmanuel Payen, Philippe Leboulch,
Bruno l’Homme, Pierre Bougnères, Christof Von Kalle, Alain Fischer, Marina
Cavazzana-Calvo, Patrick Aubourg. Science, Vol. 326 No. 5954, November 5, 2009.
For press inquiries please contact: Amber Salzman, Ph.D. President, The Stop ALD Foundation www.stopald.org Ph: +1.610.659.1098 amber@stopald.org
