Gene therapy using HIV seen halting degenerative brain disease
A deactivated form of HIV has been used by scientists in a successful gene therapy trial of 17 boys with fatal degenerative brain disease adrenoleukodystrophy (ALD), according to a study published in the New England Journal of Medicine. The disabled form of the virus was used as a means of inserting genes into cells. Trial sponsor Bluebird Bio of Massachusetts revealed that after two years, 15 of the boys exhibited no obvious symptoms of ALD. The company hopes to market a gene therapy for ALD, but did not say how much it would cost.

For the first time, doctors have used gene therapy to stave off a fatal degenerative brain disease, an achievement that some experts had thought impossible.

The key to making the therapy work? One of medicine’s greatest villains: HIV.

The patients were children who had inherited a mutated gene causing 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. 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.