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News from SALD '11

Patricia Message
14 Mar 2011, 06:19 PM

Hi, I’ve received this mail from the Stop ALD Foundation that I want to share with you: “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 for adrenomyeloneuropathy (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.”
josepanton Message
10 Apr 2011, 06:09 PM

Hi Patricia and all the other members, I can give you all the "link": about the study you talk in your topic. In my opinion there are a good group of people working to find out a solution for our disease. Hope they find a solution in few time. Best wishes
SallyBuckner Message
11 Apr 2011, 08:35 PM

This is certainly good news for oth ALD and AMN sufferers. (I'm the latter; my father had it, my elder son died from complications dealing with it, my younger son is fighting it with exercise and diet, my sister has been wheelchair bound for a dozen years and I am using a power chair most of the time now (so much for labeling women as merely "carriers"). So I am anxious for these trials to succeed!!! Thank you for letting us know. Best wishes to all.