Scientists find unlikely comrade in gene therapy for fatal disease

The surprise ingredient: They disabled the HIV virus so it couldn’t cause AIDS, and then used it to transport the healthy new genetic material.

The experiment marks the first time researchers have tried that long-contemplated step in people — and the first effective gene therapy against a severe brain disease, said lead researcher Dr. Patrick Aubourg of the University Paris-Descartes.

Although it’s a small, first-step study, it has "exciting implications” for other blood and immune disorders that had been feared beyond gene therapy’s reach, said Dr. Kenneth Cornetta, president of the American Society of Gene and Cell Therapy.

Adrenoleukodystrophy, or ALD, is a rare genetic disease that, in its most devastating form, destroys the coating of nerve fibers in boys’ brains. Without that coating, called myelin, the neurological system breaks down. The disease often strikes between the ages of four and 10.

Two years later, the boys have shown no sign of worsening brain damage and are functioning well with 15 percent of their blood cells producing the healthy protein, said Aubourg.

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