In a first, gene therapy halts a fatal brain disease:

In a first, gene therapy halts a fatal brain disease:

Context:

• A new study indicates that gene therapy can hold off Adrenoleukodystrophy (ALD), a rare disorder, without side effects, but only if it is begun when the only signs of deterioration are changes in brain scans.
• The key to making the therapy work is human Immunodeficiency virus (HIV).

Was the study successful?

• The study involved 17 boys aged 4 to 13. All got gene therapy. Two years later, 15 were functioning normally without obvious symptoms.
• The study opened up new avenues for using gene therapy to treat brain diseases.

What is ALD?

• A mutated gene causes a rare disorder, adrenoleukodystrophy, or ALD.
• Nerve cells in the brain die, and in a few short years, children lose the ability to walk or talk.
• They become unable to eat without a feeding tube, to see, hear or think. They usually die within five years of diagnosis.
• The disease strikes about one in 20,000 boys; symptoms first occur at an average age of 7

Is there any cure of the disorder?

• The only treatment is a bone-marrow transplant, if a compatible donor can be found or a transplant with cord blood, if it was saved at birth.
• These transplants are difficult and dangerous therapy, with a mortality rate as high as 20%. Some who survive are left with lifelong disabilities.