Governor Signs Righter Legislation for Newborn Screening

Legislation requiring the Illinois Department of Public Health to provide all newborns with screening tests for the presence of adrenoleukodystrophy (ALD) under a new law sponsored by State Sen. Dale Righter (R-Mattoon) was signed Wednesday by Gov. Bruce Rauner.

“Screening for ALD at such an early stage will help save lives,” Righter said. “All too often, those with ALD are diagnosed too late for treatment to work. It’s a terrible disease, but this law helps us get out in front of it and save our children.”  More...

MD1003 - Biotin European AMN trial ongoing with results expected summer 2016.

MD1003 Update

MD1003 is an experimental medicine that consists of extremely high doses of pharmaceutical-grade biotin.  Whereas daily biotin requirements are in the range of 30 MICROgrams/day (mcg), this agent is dosed at 300 MILLIgrams/day (mg).  There are 1,000 micrograms in ONE milligram.

ALD Connect Work Group 3 had a discussion with Dr. Frederic Sedel, the CEO of MedDay Pharmaceuticals in May 2015.  MedDay is the French biotech that is developing MD1003 in both multiple sclerosis (MS) and adrenomyeloneuropathy (AMN).  Biotin is also known as Vitamin H or Vitamin B7.  Recently, the company reported positive data in Progressive MS, so we wanted to hear Dr. Sedel's advice for AMN patients who might be interested in taking alternative forms of MD1003 off label.  There is a European AMN trial ongoing with results expected next summer (2016).

In spite of a promising outcome in MS, there are concerns unique to AMN that prevent a direct correlation between the two diseases.  In fact, that is one reason the company is advancing the AMN clinical trial.  Here are some specific issues that Dr. Sedel shared with The Stop ALD Foundation:

Possible Impact on AMN

Biotin is a co-factor for several enzymes involved in energy production and as such is believed to be beneficial for AMN patients.  Biotin is also a cofactor of acetyl – CoA carboxylase.  This carboxylase is involved in fatty acid synthesis and may play a role in myelin synthesis.  Due to its role in fatty acid synthesis it is theoretically possible that MD1003 may impact (increase) VLCFA levels.  Although the effects of VLCFA in ALD remain to be fully elucidated, there are a percentage of AMN patients who convert to cerebral onset, which is a more severe disease.  Thus there may be a theoretical increased risk for deleterious effects in the background of increasingly elevated VLCFA’s. However, VLCFA increase has not been observed so far in preliminary studies in one patient treated by MD1003, nor in laboratory experiments using patients’ fibroblasts (skin cells).

Teratogenicity (Birth Defects)

It is still not clear if these very high doses of biotin might be associated with birth defects. Teratogenicity has been observed in one animal species at a dose close to the therapeutic dose used in humans. Given the gravity and seriousness of such a side effect, it is strongly recommended that women of childbearing age avoid this product. It is also not known if any effects might be transmitted through a man's reproductive organs.

Laboratory Abnormalities

Investigators learned from the Progressive MS trial that several patients on MD1003 experienced a series of abnormalities on blood tests.  Although the lab work was indicative of a disease state in 4 patients (hyperthyroidism), it turned out that these were only artifacts because there is something about the high-dosed biotin that interferes with immunoassays.  For example, a person on MD1003 may appear to have thyroid abnormalities to the point where a prescription is provided or even surgery is recommended. The challenge is how to distinguish some of these artifacts from real disease?  The lab work would appear the same, regardless of whether the patient is sick or not, and not all diseases are accompanied by overt clinical symptoms.  This confusion can be the case with dozens of lab tests in a person on high dose biotin.  In contract to the previously mentioned potential for AMN exacerbation and teratogenicity concerns, this is a proven effect associated with the drug, and not just a theoretical or hypothetical happening. Of note, interferences are only observed with certain analysers using biotin as a reagent. The tests can be accurately performed after stopping the treatment with MD1003 for several days (at least one week).

Altered Lab Results Impacting Cortisone Pathways

Given the common incidence of adrenal insufficiency associated with ALD/AMN, and the ongoing and critical need for adrenal replacement, it is essential that patients have accurate ACTH and cortisol monitoring.  These laboratory tests can be confounded in patients on MD1003, which could affect proper steroid dosing, and lead to adrenal crises.

Going forward MedDay is developing careful management and educational tools to address the issue of abnormal lab results, which can happen in an estimated 100 different tests.

Purity of Product and Intellectual Property

The product contained in MD1003 is pharmaceutical grade biotin.   This is quite different than supplement-grade products, which do not have the same ongoing requirements of purity and potency.  Supplement content can vary from batch to batch and also may contain impurities. 

That being said, one can likely outsource pharmaceutical-grade manufacturing at an approved and certified facility and obtain pure biotin.  As for any drug in development, it is not recommended that this be done outside the context of a clinical trial before the safety and efficacy data have been fully assessed.

Successful Million Dollar Bike Ride!

We had a great Million Dollar Bike Ride for Rare Disease Resaerch this past Saturday in Philadelphia! Our team was one of 24 total for a variety of rare diseases. We raised over $20k which will be doubled by UPenn to $40k for researchers to apply for ALD research grants. Thanks for everyone's support!

Invitation to UPenn 2015 Rare Disease Million Dollar Bike Ride - ALD

The 2nd annual University of Pennsylvania Million Dollar Bike Ride (MDBR) for Rare Disease Research takes place May 9, 2015 in Philadelphia. For adrenoleukodystrophy (ALD) / adrenomyeloneuropathy (AMN), please help us hit our $50,000 goal by supporting us here.

It would be great if you could also join the ride. Sign up to ride here and select "Stop ALD" as the team you are riding for and "Registration without Fundraising" 

The MDBR will match every dollar we raise so we can turn $50,000 into $100,000 for critical ALD/AMN rare disease research. Last year we raised $25,000 and that was doubled to $50,000. The University of Pennsylvania lead the section process awarding the $50,000 grant to a great ALD/AMN researcher at Mass General in Boston.

Please let us know if you have any questions and thanks in advance for your support!

Sincerely,

Steve Barsh & Amber Salzman
steve@barsh.com
+1.215.888.2101

Leukodystrophies: Making a Difference by Donating Instead of Discarding

The donation of Preimplantation Genetic Diagnosis (PGD) tested and affected embryos to stem cell research at the University of Michigan

For couples with a family history of inherited diseases like Leukodystrophies, that cause pain, suffering, and sometimes death, in vitro fertilization (IVF) treatment with Preimplantation Genetic Diagnosis (PGD) makes it possible to build a family without the risk of passing on the mutant gene and known disease to their children.

Inherent in PGD testing is the production of embryos which test positive for genetic defects. Typically, these disease-affected and abnormal embryos are deemed “not suitable for implantation” and are discarded. Couples now have the opportunity to make a valuable contribution to our understanding of the diseases, many times present in their families such as Adrenoleukodystrophy (ALD), Canavan Disease, Krabbe Disease, and Metachromatic Leukodystrophy, by donating these otherwise discarded embryos to embryonic stem cell research at the University of Michigan’s Stem Cell Program.

This University of Michigan program, called MStem Cell Laboratories, has already produced the world’s first ALD disease-specific human embryonic stem cell line†. Embryos carrying any single-gene disease for which PGD testing is available, (such as those listed above) may be donated to the University of Michigan Institutional Review Board (IRB) approved study.

Is it difficult to donate?

Not at all. Prior to beginning their IVF cycle, couples interested in donation of PGD-tested and disease affected embryos review and sign an informed consent document provided by the University of Michigan. During the consent review, our Study Coordinator will speak by phone with both the female and male interested in embryo donation to answer any questions they may have. Our Study Coordinator also works directly with each couple’s fertility healthcare provider to facilitate the embryo donation process.

Where can I learn more?

Visit the University of Michigan Stem Cell Program website at: www.stemcellresearch.umich.edu/donation/donors.html, or you can call our Study Coordinator at +1-734-649-6557, who will be glad to assist you.
† www.uofmhealth.org/news/archive/201501/stem-cell-success-one-couples-effort-protect-their-son-fatal

* Nakano et al. Cell Stem Cell (2012) Vol: 10 Issue 6 

Penn Orphan Disease Center Announces First Grant Recipients from Million Dollar Bike Ride

Newswise — PHILADELPHIA — The Orphan Disease Center at the Perelman School of Medicine at the University of Pennsylvania, has awarded its inaugural grants funded by proceeds from the 2014 Million Dollar Bike Ride. Thirteen institutions – from academia in the US, Canada, Germany, and Australia – received grants ranging from $35,000 to $60,000 from funds raised by 13 disease-specific cycling teams...

The awards:

Team ALD (Adrenoleukodystrophy)
Florian Eichler, MD
Massachusetts General Hospital / Harvard Medical School
Pilot study on adeno-associated virus serotype 9-mediated gene therapy for adrenomyeloneuropathy

Read the whole article here...

$50,000 pilot grant -- Request for Applications for Adrenomyeloneuropathy (AMN)

As a result of the Million Dollar Bike Ride, the Center for Orphan Disease Research and Therapy (CODRT), announces a Request for Applications (RFA) for a $50,000 a pilot grant for Adrenomyeloneuropathy (AMN).

The RFA and guidelines for application are available on CODRT.  Once on CODRT click on Adrenoleukodystrophy (ALD) or just click here to open the PDF application.

The Timeline for the RFA / grant Program is as follows:

• August 4, 2014 -- RFA Release Posted.
• September 15, 2014 -- Pre-application deadline; documents are to be uploaded no later than 5 pm ET.  Click here for Rare Disease Pilot Grant Pre-application.
• September 18, 2014 -- Invitation to submit a Full-application
• October 15, 2014 -- Full-application deadline (by invitation only).  Documents are to be uploaded no later than 5 pm ET.
• December 1, 2014 -- Anticipated start date.

All applicants must become a member in the Center for Orphan Disease Research and Therapy to participate in this RFA. Please view our membership details.

If you have any questions, please contact: 

Linda Aversa-Caldwell, MS
Associate Director, Center for Orphan Disease Research and Therapy (CODRT)
University of Pennsylvania
laversa@mail.med.upenn.edu
Phone: +1.215.898.6111
Fax: +1.215.573.2273
http://www.med.upenn.edu/orphandisease/

New therapies being investigated for treating ALD/AMN

An ALD Connect workgroup lead by Rachel Salzman, CSO The Stop ALD Foundation, and Kendrick Goss, Scientist II at bluebird bio provided an update at the ALD Connect/ULF meeting in Baltimore July 30 - August 3, 2014. A list of potential treatments were considered and narrowed down to the following:

• Ampyra, a K+ Channel blocker, is said to improve nerve conduction, however no published data exists for ALD/AMN. It is approved for MS.
• MD 1003, an anti-oxidant, is energy producing in neurons and repairs demyelination. Data on one AMN patient was reported and a multi-center AMN trial is being launched in Europe (not available in the US). Contact: jf.dhainaut@ela-fondation.com
• EPI-743, an anti-oxidant, shown to reduce oxidative stress in vitro. An AMN trial is close to being initiated in Italy. Contact: Keith Van Haren at kpv@stanford.edu
• Natalizumab, an anti-integrin, is known to block lymphocyte infiltration in CNS. In-vitro data has been reported but not published. ALD experts have a strong interest in pursuing. It is approved for MS.
• AAV, an in vivo gene therapy treatment. In-vitro and in-vivo mouse data has been shown to lower VLCFAs. More work is ongoing to further prepare for a human study (not prior to 2016).
• Sobiterome, a thyroid agonist that up regulates ABCD2. In-vitro and in-vivo data has been reported but not published. A clinical study is under design.

Team members will continue to consider how to best move forward efforts.

Team members include: 

• Joshua Bonkowsky, Univ of Utah
• Nancy Braverman, McGill
• Marie-Josee Duran, ELA
• Florian Eichler, Mass General
• Ali Fatemi, KKI
• John Fink, Univ of Michigan
• Kendrick Goss, bluebird bio
• Stephan Kemp, Univ of Amsterdam
• Troy Lund, Univ of Minnesota
• Sanjay Magavi, Vertex
• Alex McCampbell, Biogen Idec
• Patricia Musolino, Mass General
• Asif Paker, bluebird bio
• Rachel Salzman, The Stop ALD Foundation
• Keith Van Haren, Stanford. 
• Inna Tzvang, ALD Connect

$44,000 raised for ALD at May 2014 UPenn Rare Disease Bike Ride!

On Saturday, May 3, 2014, the weather was gorgeous and nearly 20 riders on "Team ALD" participated in the first annual University of Pennsylvania Million Dollar Bike Ride for Rare Diseases.

See the photo gallery here.

• ~20 riders for Team ALD.
• 540 total riders in the event.
• Team ALD raised $22,000 for ALD research and the University of Pennsylvania doubled that (since we met our $20,000 goal) and a total of $44,000 will be directed toward ALD research!
• Overall, the event raised $1.4 million for rare disease research at the University of Pennsylvania!

Can't wait for the 2nd time the ride occurs in 2015!  Check here for more info.

Success of Aidans Law leads to national push for testing

(WZZM) - The success of Aidans Law in New York is leading to a new push for national screening of infants for Adrenoleukodystrophy (ALD). The law is named for Aidan Seeger a seven year old New York boy who died from the disease. Continues...

A Brooklyn Boy Who Lost A Life, But Helped Save Others

Amazing NPR story about the family who lost their son Aidan to ALD, but have done so much to make sure this never happens to other families by getting "Aidan's Law" passed.  Now ALD newborn screening is rolling out, albeit slowly, state-by-state.

California Assembly Health Committee voted 19 to 0 to move forward with the legislation for ALD NBS!

Success! We got a unanimous pro vote to move ALD newborn screening forward from all Assembly members today at the Capital after Gina Cousineau. Patti Chapman and I testified. Woowoo!

Great legislation proposed for ALD Newborn Screening in California!

(Sacramento) -- Doctor Richard Pan, Democratic Assemblymember from Sacramento, has authored legislation to add Adrenoleukodystrophy, or ALD, to the list of genetic disorders all California infants are screened for when they're born. Dr. Pan says Assembly Bill 1559 can save a lifetime of debilitating problems for a child by properly diagnosing and treating ALD before its symptoms appear. Dr. Pan says other states already screen for ALD and a federal advisory committee has recommended ALD be added to the newborn screening list in all states. Here's more in this Assembly Access video. http://www.asmdc.org/pan

HRSA's Secretary Advisory Committee (SAC) unanimously voted in favor of moving ALD newborn screening forward to an external evidence review committee

On January 16, HRSA's Secretary Advisory Committee (SAC) unanimously voted in favor of moving ALD newborn screening forward to an external evidence review committee. Dr Jerry Raymond, Pediatric Neurologist at University of Minnesota Medical Center, Fairview, and Dr. Amber Salzman, President of The Stop ALD Foundation, spoke at the meeting's public comment section. Over the next 2 years, the External Review Committee will review the disease, review the newborn screening test, understand what happens when a newborn would be identified to have ALD, and look at the therapies. A recommendation will then be made to the SAC whether ALD should be added to the Recommended Uniform Newborn Screening Panel (RUSP). The Stop ALD Foundation initiated the process to get ALD added to the RUSP starting September 2012 understanding this was a lengthy and complex procedure. We remain committed to ensuring that all newborns are screened for ALD so treatment can be applied early, saving countless children and families from suffering from a treatable disease that most often is diagnosed too late.

New York State to screen newborns for Adrenoleukodystrophy

New York State to screen newborns for Adrenoleukodystrophy

BY: Metro Source Staff | April 03, 2013

ALBANY - In approving the 2013 budget, Governor Cuomo has approved funding for Aidan’s Law, a law that adds Adrenoleukodystrophy (ALD) to New York’s Newborn Screening panel.

The law is named for Aidan Seeger, a boy from Brooklyn, NY who suffered from the disease. Seeger passed away from ALD in 2012 at seven years old. ALD is a Leukodystrophy, as is Krabbe, the disease that Jim Kelly’s son, Hunter, suffered from.

ALD is an inherited metabolic disorder, which affects the nervous system and is usually fatal if not treated before symptoms appear. This devastating disease has an estimated incidence of 1 in 17,000 in boys. If untreated, those affected lose all voluntary muscle function, as well as vision and hearing, and eventually die.  Read more >>

“Quote test”

Dr. Cartier-Lacave, INSERM - Paris, talks her work with gene therapy as a cure for ALD in this video interview

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.

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.

December 2009 Stop ALD Foundation Update and 2010 Goals

The Stop ALD Foundation Annual Update

December 2009
You helped us achieve a BREAKTHROUGH in 2009!  What's next for 2010? 
~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~

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
~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~

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
~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~~
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!