U of M Doctors Discover Treatment for Advanced ALD
Researchers have made a major step forward in treating a devastating disease.
Continuing more than a decade of research, doctors at the University of Minnesota have discovered a treatment to help patients with advanced cases of adrenoleukodystrophy (ALD). ALD is a rare disorder affecting the nerves. The new treatment halted disease progression and allowed patients to survive a bone marrow transplant.
A Rare and Often Fatal Disorder
ALD is a progressive degenerative disorder that affects young boys. With ALD, myelin breaks down over time and causes loss of hearing, sight, mobility, and general nerve function. Myelin is the “insulation” around the nerves. Left untreated, patients with ALD will die, usually within three to five years of diagnosis. There is no cure, but the progression of the disease can be halted with a bone marrow transplant. In very advanced cases, transplant is not recommended.
Anti-Inflammatory Drug Shown to Halt Disease Progression
Doctors gave very advanced ALD patients who would not otherwise be eligible for transplant an anti-inflammatory drug, N-acetyl L-cysteine (NAC). The combination of NAC and transplant halted the disease progression and allowed patients to survive transplant. Post transplant brain scans showed a decrease in inflammation and preservation of myelin.
“We believe that NAC can also help patients with less advanced cases of ALD, and possibly other diseases of inflammation of the myelin,” said Lawrence Charnas, M.D., Ph.D., senior author and pediatric neurologist at the University of Minnesota Children’s Hospital. “This is a major step forward in treating a devastating disease.”
The University of Minnesota Children’s Hospital is a world leader in treating children with ALD and other metabolic storage diseases. This research was funded by the Children’s Cancer Research Fund and the University of Minnesota Department of Pediatrics.