GENE therapy is coming in from the cold. Two boys treated three years ago with a gene therapy for , the brain disease featured in the film Lorenzo鈥檚 oil, fared so well that doctors have treated a third and are now looking for adult volunteers.
鈥淭hey have normal, family lives,鈥� says of the Descartes University in Paris, France, a member of the team that pioneered the ALD gene therapy.
ALD is caused by a faulty gene that prompts the myelin sheath coating nerves in the brain to wear away, causing impaired speech, movement and eventually death.
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Cartier and her colleagues took blood stem cells from two 7-year-old boys with ALD, infected the cells with a virus carrying a correct copy of the defective gene, then re-injected the stem cells. The boys鈥� symptoms stabilised within 14 months and have not worsened since (Science, ).
Early gene therapy trials were stopped after triggering cancer.