Gene Therapy for Fatal Brain Disorder 'Just the Beginning'

THURSDAY Nov. 5, 2009 -- The reported success of gene therapy in treating two children with adrenoleukodystrophy (ALD) brings hope to patients with the potentially crippling and fatal brain disorder and their families, says a nonprofit group that supports ALD research.

"These gene therapy results are exciting, but they are just the beginning," Eve Lapin, a founding member of the Stop ALD Foundation, said in a news release from the group. "Time is of the essence in finding the safest and most effective therapies. Every day, more children become afflicted with ALD, and their chances of surviving depend on the success of trials such as this."

ALD is a genetic disorder that affects one in 18,000 people. The most serious form of the disease destroys the myelin sheath of the brain's neurons and generally affects boys between the ages of 4 and 10. They suffer a steady decline that leads to permanent disability and death within two to five years of diagnosis.

In a new report, French doctors detail the cases of two boys with ALD who underwent gene therapy. Genetically defective bone marrow blood cells were removed from the boys and "corrected" by insertion of properly functioning genes. The treated cells were injected back into the patients. The therapy halted the progression of ALD, and the boys' conditions remain stable more than two years later. No adverse effects of the gene therapy have been seen by the doctors.

The report is published in the Nov. 6 issue of Science.

The Stop ALD Foundation supported this gene therapy initiative through funding and by bringing together experts who provided crucial scientific and biomedical contributions. The foundation is now pushing for a larger, international study.

This "pioneering work in gene therapy brings hope to those stricken not only by ALD but many other serious diseases as well," foundation president Amber Salzman said in the news release.

A form of ALD called adrenomyeloneuropathy (AMN) can affect male and female adults but tends to be less severe than ALD in boys.

More information

The U.S. National Institute of Neurological Disorders and Stroke has more about ALD.

Posted: November 2009


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