October 2011 -- Stop ALD update on driving forward research on faster therapies for children in immediate need and research on anti-oxidant effectiveness on AMN symptoms.

Dear Stop ALD Foundation Supporter,

To date, the Foundation has played a key role in finding a therapy for ALD patients whose disease has just started to progress. Looking forward to the fourth quarter of 2011, we will turn our attention to two different and, for us, very significant goals:  (1) Driving forward a therapeutic approach for patients whose disease has progressed beyond the ability of current therapies to be effective, and (2) determining whether anti-oxidant treatments are of benefit to help treat AMN patients.

The gene therapy work which has been done to date works with the patient's own stem cells and, even when successful, takes at least 12 to 18 months to halt the disease. Because so many ALD families experience their first diagnosis after the disease has started to progress, their children do not have the luxury of 18 months of time before a successful therapy may be given a chance - they need a therapy which begins to work much more quickly in order to save these children. To address this critical need, we will work with physicians and scientists to investigate using a different gene therapy approach which targets brain cells and which can go into the brain immediately and stop the disease in a much shorter time frame. In order to progress this approach, we have to test this theory in laboratory mice, and our aim is to have this initiative launched in mice by year end. If this therapy proves to be successful in mice, we will immediately initiate the additional pre-clinical steps necessary before the therapy would be ready to be tested in humans. 

Looking to our second goal, in order to properly explore whether various anti-oxidant compounds may be effective in treating symptoms of AMN, the compounds will similarly need to be tested first in mice. Toward that end, and because AMN mice need to age before they exhibit AMN symptoms, we will work with a lab to begin breeding and aging the mice as the therapeutic agents are investigated and the approach is formally defined. Our goals is to launch these activities by year end as well.

As we pursue these two near-term primary goals, we of course will continue to support and work with others to find additional promising therapeutic approaches to ALD and AMN, to implement newborn screening in as many states as will permit it, to find ways to accelerate ongoing initiatives managed by others working in this field, and to continue to help families faced with managing ALD and AMN patients.

Please Include The Stop ALD Foundation in Your Giving
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.