N-acetyl L-cysteine (NAC): New Treatment for Advanced ALD Patients

Filed in archive Studies , Treatment on February 28, 2007

Remember that movie Lorenzo's Oil?

It was a true story of Lorenzo Michael Murphy Odone who is afflicted with adrenoleukodystrophy (ALD) - a rare, progressive degenerative myelin disorder that affects young boys.

ALD has no cure, but since the case of Lorenzo, several treatments for ALD have been found to slow the progression of the disease: diet manipulation, "Lorenzo's Oil" and bone Marrow transplant.

In continuation of more than 10 years of research on this disease, doctors at the University of Minnesota have discovered a treatment to help patients with advanced cases of adrenoleukodystrophy (those who would not be eligible for transplant): a medication called N-acetyl L-cysteine (NAC), an anti-inflammatory drug used to help liver cells recover from Tylenol® (acetaminophen) overdose.

[In these advanced cases, the combination of NAC and transplant halted the disease progression and allowed these patients to survive transplant. Post transplant brain scans showed a decrease in inflammation and preservation of myelin after administering NAC.]

According to Lawrence Charnas, M.D., Ph.D., senior author and pediatric neurologist at the University of Minnesota Children's Hospital:

"We believe that NAC can also help patients with less advanced cases of ALD, and possibly other diseases of inflammation of the myelin. This is a major step forward in treating a devastating disease."

A major step indeed, because ALD patients usually die within three to five years of diagnosis if left untreated.

The results of this new study are published in the late February issue of Bone Marrow Transplantation.

Read the full report.

[article abstract]