ALD (Adrenoleukodystrophy)
ALD is a rare genetically acquired disease that affects young boys typically between the ages of 4-10 years old. The disease was the focus of the movie Lorenzo's Oil, 1992, starring Susan Sarandon and Nick Nolte.
- ALD is a degenerative neurological disorder that causes damage to the nerve cells of the brain.
- Approximately 1 in 20,000 boys are born with the disorder.
- Currently, the only known treatment is a stem cell transplant that can be highly effective if performed at an early stage before young boys become symptomatic.
- Unless a family history of ALD is known, the disease is rarely detected at birth. As a result, most young boys appear completely healthy during their early childhood years and remain undiagnosed until later in life (approximately age 4-10) when they start to show initial symptoms - frequently too late to stop the progression of this devastating disease.
- As a result, death is unfortunately the most common outcome in the majority of young boys born with ALD that are not diagnosed early in life.
- Initial symptoms are typically inattentiveness and lack of focus in areas such as school work and following instructions. Given the nature of these symptoms, ALD is frequently mis-diagnosed as Attention Deficit Hyperactivity Disorder (ADHD).
- This is tragic, because precious time is lost during which treatment could have otherwise been initiated.
- Newborn screening provides the opportunity to detect the disease shortly after birth, allowing treatment to be initiated at an early stage, when it can still be effective at halting the progression of this deadly disease.
- By simply detecting the disease early in life, most young boys that receive a stem cell transplant can go on to live normal, healthy lives.
- Promising research in the field of gene therapy is currently ongoing that offers the potential to dramatically improve existing treatment options. However, even with gene therapy, the key to achieving a good outcome is early detection.
Additional Links on ALD - Sourced from Stop ALD.org
SITES THAT DISCUSS ADRENOLEUKODYSTROPHY
- Stop ALD.org
- ALD Connect
- The ALD Foundation
- ALD Life
- The Myelin Project
- Oliver's Army
- Orpha.Net -- European Rare Diseases Organization and Their Discussion on ALD and many great links & resources
- The United Leukodystrophy Foundation
- US ClinTrial.gov -- Listing of ongoing US ALD clinical trials
- WikiPedia on ALD
- X-Linked ALD Database
- FightALD.org
- The Blood and Marrow Transplant Information Network
- Bone Marrow Donors Worldwide
- Duke University's Pediatric Stem Cell Transplant Program
- National Marrow Donor Program
- University of Minnesota: Fairview University
- Related Donor Cord Blood Program. For families impacted by a life-threatening disease where a cord blood transplant could be a cure, the cord blood from a new family baby can be collected and stored at no cost to the family and then may be used to treat an affected biological sibling or parent who has a diagnosed disease.
