Lorenzo Odone

Lorenzo Odone, who died on Friday, the day after his 30th birthday, became famous throughout the world with the release, in 1992, of the film Lorenzo's Oil, starring Susan Sarandon and Nick Nolte.

At the time that the film was made, Lorenzo was 14 and had been suffering for eight years from adrenoleukodystrophy (ALD) a genetic disease, affecting mainly boys, which usually results in brain failure and, ultimately, death.

The film dramatised the attempts by his parents, Augusto and Michaela Odone (played by Nolte and Sarandon), to find an effective treatment, a quest which culminated in the invention of "Lorenzo's oil", a derivative of olive and rapeseed oils and the first agent to have demonstrated a therapeutic effect by halting the destruction, by the disease, of the myelin sheathing of the nervous system.

The treatment was patented by Augusto Odone, and a study published in 2005, based on research with 84 boys, demonstrated its efficacy in preventing the onset of the disease's symptoms for a majority of boys diagnosed early with ALD. This discovery came too late for Lorenzo Odone, who was already suffering from the irreversible neurological effects of the disease.

Augusto Odone and his wife went further, however, founding a charity called the Myelin Project, an international scientific body which seeks to promote research into those diseases which destroy myelin, the white matter of the central nervous system without which the brain cannot transmit messages to other parts of the body. The goal is to bring hope to those suffering from conditions such as multiple sclerosis and the leukodystrophies (of which ALD is one).

Lorenzo Michael Murphy Odone was born on May 29 1978 in Washington DC, the son of Augusto Odone, an Italian-born economist with the World Bank and his second wife, Michaela, a linguist. It was clear from his earliest years that Lorenzo was a precocious, bright child, and one who was hungry for knowledge.

By the age of five he was fluent in English, French and Italian, and his parents hoped that he was destined for Harvard.

During his year at kindergarten in Washington he seemed normal and talkative, and it was only when his father was posted abroad, and Lorenzo began classes at a French school on the Comoros Islands in the Indian Ocean, that his teachers reported problems with his behaviour and a deterioration in his attention span. Despite these worrying developments, Lorenzo's appetite for knowledge was undimmed. As his eyesight failed he was taught maths, history, geography and literature by a private tutor; he loved to hear the Greek myths, and the music of Bach and Handel.

But by 1984, when he was six, Lorenzo's behavioural problems had worsened and he began to suffer from blackouts and memory lapses, symptoms akin to those of stroke victims.

At first, Augusto and Michaela Odone believed that their son might have contracted a tropical disease in the Comoros Islands, but a brain scan finally confirmed that Lorenzo was suffering from ALD.

The prevailing medical wisdom decreed that children with ALD typically lived for only a few years beyond diagnosis. But Augusto and Michaela Odone refused to accept this, and began an exhaustive quest for an effective treatment. Despite having no medical training, they contacted specialists and combed the scientific journals.

Lorenzo's father was granted leave by the World Bank to work from the family home outside Washington so that he could help to care for his son. Then, in 1987, having taken early retirement, he invented a treatment, in the form of a vegetable oil, that appeared to arrest the progress of ALD. To help with the oil's development, the Odones enlisted a British scientist, Don Suddaby, who had worked at the Croda Universal chemical company in Hull. He came out of retirement to help produce an edible mixture of olive and rapeseed oil extract, finally sending the Odones a bottle of what would become known as "Lorenzo's oil".

Although the Odones were told that the oil might have a deleterious effect, they decided, in view of the rapid deterioration in their son's condition, to risk administering it. Augusto Odone believes that the treatment was responsible for giving Lorenzo many extra years of life.

By now, however, Lorenzo had become bedridden; he was blind, deaf and almost completely disabled physically. He could signal "yes" and "no" by blinking or moving his fingers.

Rather than relegate their son to an upstairs bedroom, his parents moved to a bungalow, setting up his bed and the equipment he needed in their main living room. It was there that Lorenzo spent the remaining years of his life.

He was dressed for the day and undressed at night, his parents striving to replicate the normal rounds and routines of a growing boy. In an effort to punctuate Lorenzo's daily regime of massages, exercise and physiotherapy, Augusto and Michaela Odone commissioned a special chair in which their son could sit and listen to stories or to music. From time to time his carer would lift Lorenzo out of his chair and take him to bathe in the family's swimming pool at the back of the house.

Occasionally it was possible to discern a nuance in Lorenzo's largely unchanging expression that seemed to signal a response to what was going on around him – for example, he appeared to be able to tell if someone walked into the room – but for the most part he remained inert, a gentle dreaminess about the face disturbed only by the occasional spasm.

His condition required round-the-clock medical care. He was nursed in the family home outside Washington, attended by five nurses working in shifts, as well as by his parents – his mother would spend up to 16 hours at a stretch at her son's bedside. Because he was unable to swallow saliva, he had to be suctioned, sometimes every few minutes. Every bodily function had to be monitored, and he had to be fed five times a day through a tube.

An old family friend called Oumouri, whom the Odones had met during their time in the Comoros Islands, helped with Lorenzo's day-to-day care after Michaela's death in 2000.

Throughout the years of his illness Lorenzo displayed exemplary courage. He had lain paralysed in a darkened room, unable to see or speak, while his body developed normally through childhood and adolescence.

He remained completely unaware of the global celebrity conferred on him by the Hollywood film that told his story. "Michaela once said that the real Lorenzo was locked in his ALD body," Augusto Odone told a British journalist in 2001, "and I believe that is true."

After Michaela Odone's death, Augusto played his son tapes of her voice which she had recorded for him, and Lorenzo appeared to respond to these with conscious and voluntary sounds.

Lorenzo died from complications of aspiration pneumonia at his home in Fairfax, Virginia.

Augusto Odone said his son would be cremated, and his ashes mixed with those of his mother. Now 75, Augusto is preparing a book telling the story of his long quest to cure his son.

Lorenzo Odone is also survived by a half-brother and half-sister from his father's first marriage; his half-sister is the writer, commentator and former editor of The Catholic Herald, Cristina Odone.