Doctors have for the first time successfully used gene therapy to treat children with a rare disease of the nervous system that gained worldwide recognition thanks to the Hollywood movie "Lorenzo's Oil," a European health group said on Monday.
The European Association for Leukodystrophy (ELA) said the breakthrough was unveiled by French researchers at a congress in Rotterdam, the Netherlands, of the European Society of Gene and Cell Therapy.
The therapy targeted faulty genes that cause adrenoloeukodystrophy (ALD), one of a group of inherited disorders known as leukodystrophies in which the protective myeline sheath protecting nerve cells in the brain becomes damaged.
A team led by Patrick Aubourg, a professor at the Saint Vincent de Paul Hospital in Paris, used a harmless version of the AIDS virus as a Trojan horse, introducing the corrected gene to fix the flawed chromosome in bone marrow taken from the children.
The bone marrow was then reinjected into the patients.
ALD occurs because a flawed gene on the child's X chromosome does not produce a key enzyme to break down an accumulation of so-called very long chain fatty acids in the brain.
Six months after the therapy, the results have been encouraging, showing a pickup in production of this enzyme, ELA said.
"Lorenzo's Oil," a 1992 drama starring Nick Nolte and Susan Sarendon, shows the dogged bid of two parents to devise an oil with fatty acids that can block myelin destruction.