New hope in fight against 'Lorenzo's Oil' disease

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The Independent Online

A dramatic breakthrough in the possible treatment of childhood diseases of the nervous system – including the disorder featured in the film Lorenzo's Oil – was announced by scientists in America yesterday.

For the first time, researchers appear to have cured a similar disease in mice by the injection of human stem cells designed to re-build the sheath of fatty tissue surrounding nerve cells that is lost in inherited diseases such as adrenoleukodystrophy, which last week claimed the life of Lorenzo Odone, whose battle against the disease was dramatised in the film.

The myelin sheath performs the vital role of insulating nerve impulses; the possibility of re-building it offers hope to thousands of children with a host of gene disorders such as Tay-Sachs and Krabbe disease, as well as adults suffering from multiple sclerosis.

Although the research is still at the earliest stages, the scientists believe its results are a further demonstration of the power of stem-cell treatments.

The laboratory mice in the study all suffered from an inherited condition that would normally kill them within about 20 weeks of birth. However, some of the mice that received the treatment not only lived for several months after that, but actually improved to the point of seeming to be cured.

"It's extremely exciting to think about not only treating but actually curing a disease," said Professor Steven Goldman, of the University of Rochester Medical Centre in New York, who led the research team. "Unfortunately, right now, we can do little more besides tell parents to prepare for their kids to die," said Professor Goldman, whose study is published in the journal Cell Stem Cell.