The Stop ALD Foundation Annual Update
December 2009
You helped us achieve a BREAKTHROUGH in 2009! What's next for 2010?
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Greetings!
What great news we were able to deliver in 2009! While working hard in many areas, we are, by far, most proud of our groundbreaking international success in the field of gene therapy. This is the first time ever that a fatal brain disease has been successfully treated by gene therapy, and it was ALD! Our published gene therapy success made news around the world in the scientific and mainstream press. In case you have not seen the details, we'll give a recap below. With your generosity and our hard work, we pulled off what most foundations only dream of -- delivering a safe and effective therapy for a disease. There is still critically important work ahead for 2010, and we'll tell you about our goals and how we would greatly appreciate your continued support.
Gene therapy breakthrough!
[Science on the gene therapy for ALD article] On November 5, 2009, the two French researchers with whom we have worked closely had their pioneering findings published in Science. In this groundbreaking paper, they describe two boys with ALD who received gene therapy treatment and consequently had their brain disease progression halted, and some damage was partially reversed.
This biomedical advance is significant beyond the realm of ALD. We would like to extend our heartfelt thanks and gratitude to you, our supporters and generous donors. By delivering this new treatment, you have helped to make a difference in the lives of children and adults suffering from ALD and AMN globally. In addition, the approaches utilized are applicable in the treatment of many other diseases, such as metachromatic leukodystrophy (MLD), Wiskott-Aldrich Syndrome (WAS), Hurler Syndrome, Krabbe disease, and more. You should be VERY proud.
Goals for 2010
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While we are pleased with our achievements to date, this success has only served to motivate us to expand and refine the next generation of diagnostics and therapies. The knowledge and data acquired to this point have provided a solid and scientifically reliable groundwork for launching and focusing our next series of efforts.
Our goals for 2010 include:
Leverage ongoing efforts to drive forward newborn screening for ALD.Apply the gene therapy approach to a broader population, e.g. adults.Optimize the gene therapy approach to minimize use of toxic medications during the gene therapy procedure.
Drive approaches providing options for advanced-stage ALD boys who are diagnosed too late to benefit from a stem cell transplant or gene therapy. Several cell therapies and drug options are being considered.Drive approaches to provide options for sufferers of AMN. Animal models are being used to examine the application of antioxidants.Continue to support families who are stricken by ALD and need help navigating the way forward.
Please Include The Stop ALD Foundation in Your Year-End Giving
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We realize that times are still economically challenging for all of us. Please know that, more than ever, we deeply appreciate your support and contributions as we build on our current momentum and drive forward our progress. Donations can always be made online by visiting stopald.org and clicking on Donate Now! or please mail your donation to:
The Stop ALD Foundation
500 Jefferson Street
Suite 2000
Houston, Texas 77002-7371
If you are interested in learning more about any of our specific development goals please feel free to contact me directly at amber@stopald.org or call me at +1.610.659.1098.
We look forward to continuing to report on our progress and, in the interim, offer our heartfelt gratitude and best wishes to you for a healthy and happy new year!