As a sufferer of the rare nerve disease adrenoleukodystrophy or ALD, Lorenzo Odone was at the centre of some of the most important and well-publicised research into the illness of recent times. The story of his diagnosis, the determination of his parents — Augusto and Michaela — to find a cure and the encouraging results they achieved using an oil developed after diligent research was told in a film, Lorenzo’s Oil, released in 1992. But the presentation in the film of the oil treatment as a miracle cure created controversy; people continued to die of the disease, and it is only relatively recently that the oil’s merits as a preventive measure have been understood.

Lorenzo Odone was born in 1978. Until the age of 6 he was a healthy child, and exceptionally bright. He spoke English, French and Italian, enjoyed stories from Greek mythology, and loved listening to the Brandenburg Concertos and Handel’s Water Music. His Italian-born father Augusto was an economist for the World Bank, and Lorenzo’s early years were spent in Kenya and on the French-speaking Comoros Islands.

In 1984, when Lorenzo was 5, his school reports highlighted the fact that his attention span was becoming poor. He began having violent tantrums, suffered hearing loss and experienced problems with his balance and co-ordination. His mother later recalled: “By January 1985, he could neither speak nor respond in any way. He did not appear to understand us; his sight was impaired and he could not move as much as a finger. He was incontinent; he could not swallow and he had to be fitted with a nasogastic tube.”

A brain scan was recommended and Hugo Moser, Professor of Neurology and Paediatrics at Johns Hopkins University in Baltimore, diagnosed ALD, a genetic condition affecting mainly boys, where the body produces a fatty acid that accumulates in the nerve cells and damages the coating of the nerve fibres. The result is that the nerves are unable to transmit messages to the rest of the body. Doctors informed the Odones that there was no known cure for the condition and suggested that Lorenzo had only two years to live.

The Odones believed that Lorenzo was not in a vegetative state but rather trapped and able to understand what was going on around him. Not ready to accept the bleak prognosis, and despite having no medical or scientific knowledge, they began research to find a treatment. Augusto studied medicine and biochemistry and the connection between brain damage and a build-up of what are called long-chain fatty acids in the blood. He arranged a conference for experts on the subject, which threw up an important piece of information: that oleic acid, an oily liquid, destroys such fatty acids.

In the light of this the Odones developed an oil which combined erucic acid and oleic acid derived from olive and rapeseed oils. Even though many of the people they spoke to about the disease were highly sceptical they phoned scores of medical firms until they found a British chemist working for Croda International who was willing to formulate the oil.

It was found to be more effective at breaking up long-chain fatty acids than any approach yet tried, and Moser put all his ALD patients on it. In 1989 the Odones set up the Myelin Project to fund research into finding a cure for demyelinating diseases, such as ALD and multiple sclerosis, which is much more common.

However, it remained unclear whether the oil was effective as a cure. In the first survey that compared the oil with other therapies, or with doing nothing, Dr William Rizzo, of the Medical College of Virginia, Richmond, treated eight boys, starting in August 1987. Six deteriorated rapidly, while the other two seemed to improve. One relapsed, and the investigators lost contact with the other. Moser studied 70 boys from the time they first showed symptoms. All were given the oil until they lost sight and movement. Moser concluded that the oil had made no difference, and found that 40 per cent of patients treated with the oil could lose blood platelets. However, Moser concluded later, in 2005, that the oil treatment devised by the Odones could prevent the onset of symptoms in 70 per cent of those with ALD.

The movie Lorenzo’s Oil was released in 1992. Starring Nick Nolte and Susan Sarandon, who was nominated for an Oscar, it was directed and co-written by George Miller, whose credits included Mad Max and who was also a medical doctor. Phil Collins included a song about Lorenzo, based on a poem by Michaela, on his 1996 album Dance into the Light.

Lorenzo’s last years were spent in Fairfax County, Virginia, where he received 24-hour nursing care from his family and Omouri Hassane, whom he had known since his childhood in Comoros. He enjoyed having novels read to him, listening to classical music and swimming.

Lorenzo outlived his mother, who died of cancer in 2000, and Moser, who died last year.

Lorenzo Odone was born on May 28, 1978. He died on May 30, 2008, aged 30