February 2011 Stop ALD Foundation 2010 Accomplishments and 2011 Plans

The Stop ALD Foundation (SALD) had a very busy and productive 2010 which is reflected in the advances we wish to share with you as we pursue our ongoing mission to improve therapies and develop cures for adrenoleukodystrophy (ALD). Tragically, ALD continues to be a dreadful diagnosis, often accompanied by a fatal course in young boys and severe debilitation in adult men. We are working hard to create the possibility of a different result.

I'd like to share some of our highlights from 2010.

In terms of the Foundation's long-term commitment to progressing gene therapy in ALD, we previously reported on the landmark scientific paper published late last year

by our partners in this novel clinical trial. The clinical trial continues, and we are pleased to share that two more children have been treated! At the annual American Society of Cell & Gene Therapy meeting, which was held in Washington, D.C. this past May, Dr. Nathalie Cartier reported updated results in three boys. These children were selected for gene therapy because they had no donor match and were otherwise facing decline leading to death. At the end of her very well received presentation she publicly acknowledged the important role that SALD has played in this project.

We should all feel grateful that these boys, whose prognoses were uniformly fatal, are all back with their families and attending school. Our donors and supporters played a crucial role in rescuing these children. Savor this. It isn't every day that one can literally save a life.

The gene therapy program continues to grow and the academic success has been leveraged into the biotechnology industry. SALD actively advocates on behalf of this initiative with regulatory agencies. In December of 2010, the Foundation attended a series of FDA Advisory Committee hearings where issues critical to our mission were being reviewed. We are fortunate that members of this expert committee are well aware of the ALD gene therapy strategy, and our interactions with them have uniformly been positive.

Our Foundation has also been active in advancing research into treatments foradrenomyeloneuropathy (AMN). We have co-sponsored a clinical trial based in The Netherlands in which men afflicted with AMN were enrolled and treated with bezofibrate, a medicine which has been approved and used in treating disorders other than ALD and AMN. This drug is being administered in increasing doses and the subsequent levels of VLCFAs will be measured. This study has moved forward with extreme efficiency thanks to the dedication of the Dutch team of investigators. We are pleased that when they encountered promising data in the lab they approached us for financial sponsorship. Our thorough, yet fully vetted and streamlined process was able to negotiate terms quickly. We look forward to receiving top line data in the first months of 2011. If the results are promising the Dutch medical system will take over sponsorship of a much larger multi million dollar trial.

In addition to the bezofibrate study, SALD has also been active in assembling an international team that will address AMN from another angle. Preliminary investigations into the effect of oxidative stress have been met with promising results in mouse models. Following oral therapy affected mice have demonstrated marked improvement in tests evaluating their motor skills. We would like to follow up on these laboratory data with a trial designed for patients. This will require a coordinated multidisciplinary effort and a development paradigm which is SALD's specialty.

These exciting developments are just but a few of the highlights of 2010. Due to your continued and steady support, SALD has been able to be a key contributor to several innovative and worthy efforts. This has resulted in medical interventions which would not otherwise be possible. In the case of the three boys who received gene therapy, their families would now be facing terminal and end-of-life decisions for their beloved children, rather than celebrating the encouraging outcomes which they are now experiencing. We hope the same will hold true for the fourth boy who was recently treated, but for whom it is still too early to know results.

Leading The Stop ALD Foundation is a profound privilege and a great responsibility that continues to motivate me deeply on a daily basis. I reflect that my nephew, Oliver, of beloved memory, would have been starting college this year if not for ALD. I do not want any more children to be robbed of their futures. I do not want any more parents to helplessly witness the decline of their sons.

Our important work continues thanks to your dedicated and generous support. Due to the rarity of this disease and the current economic climate, it is only too easy for the desperate needs of those suffering from ALD to be ignored. The affected families who are relying on us, however, have no options. Let us not let them down.

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.