The tale of the determined amateur who proves the professionals wrong is always a compelling one, but with Augusto Odone – who has died aged 80 – it went much deeper. In 1984, his youngest child, Lorenzo, just a month away from his sixth birthday, was diagnosed with adrenoleukodystrophy (ALD), a neurological disorder that causes the degeneration of the brain in young boys. “We were told to go home and watch Lorenzo die,” Odone recalled. Neither he nor his wife, Michaela, was prepared to do that.
And so Augusto, an economist based at the World Bank in Washington DC, who had no scientific training, spent the time that he was not caring for his boy in the library of the George Washington University near his home, trying to understand ALD. It was, he read, linked to the accumulation of Very Long Chain Fatty Acids (VLCFA). How would it be, Odone suggested to various medical experts on the basis of his own reading, if the properties of oleic and erucic acids in combating VLCFA were combined? Might that halt the development of ALD? He organised seminars and conferences to seek expert opinion, but the establishment was sceptical, especially over the use of erucic acid.
Here Odone’s essential character – determined, driven, sometimes even wilful, telephoning colleagues in the middle of the night when an idea occurred to him, so eager was he to translate it into action – took him further than many others in similarly desperate circumstances might have gone. He and his wife simply refused to be defeated by the medical consensus.
They found a retired British chemist, Don Suddaby, willing to make up a batch of the combined oil. And then they set up their own clinical trial, testing it on Michaela’s sister, also a carrier of the ALD gene. When they saw a dramatic drop in her VLCFA levels, with no side effects, they knew they were on to something.
They did not wait for their findings to be peer reviewed, but instead, from 1988, gave what became known as Lorenzo’s Oil to their son every day. It worked. “The ALD serpent that had brought so much grief to our family had been tamed for good,” Odone wrote.
Their story caught the eye of Hollywood and in 1992 Lorenzo’s Oil, starring Nick Nolte and Susan Sarandon, was released, bringing the Odones’ remarkable fight to global attention. They used the film’s success to fund and draw attention to the Myelin Project, a research organisation they had established in 1989 with Augusto as its passionate, unstoppable president, to tackle the next great challenge that faced them – restoring the myelin sheath, the coating on the nerves, that is destroyed in ALD and in multiple sclerosis.
Though Lorenzo’s deterioration had been halted by the oil, it could not reverse the damage already done to his body. Within six months of diagnosis, he had been bedridden, unable to move, speak or swallow, and had to be fed by tube. Both parents cared for him with great tenderness and determination, always conscious that locked inside his body was a fine mind. They made him the calm centre of their lives and their home. It was an extraordinary, exhausting and humbling act of parental love and devotion.
Michaela died from lung cancer in 2000, after which Augusto was joined as principal carer by Oumouri Hassane, who had first become part of the Odone family when Lorenzo was a baby and they were posted to the Comoros in the Indian Ocean by the World Bank.
Progress on myelin regeneration remained painfully slow, though Lorenzo’s medical condition was stable for a quarter of a century, beating off every bout of flu, cold and even the pneumonia that, it had been often predicted, would kill him. However, in 2008 an infection in his feeding tube precipitated his death, the day after his 30th birthday.
Three years earlier, after enduring endless sniping from the medical establishment – the Odones were accused of “preferring a Hollywood ending” – Augusto had been vindicated. An authoritative study by the Kennedy Krieger Institute in Baltimore showed that in young boys with the ALD gene given Lorenzo’s Oil before the onset of symptoms, 74% did not develop the full-blown disease.
Augusto was born in Rome and grew up in the Piedmontese town of Gamalero. His father, Angelo, was a leading figure in the Italian resistance movement, and his mother, Maria, wrote several books on home economics. He studied law at the University of Rome and then went on a Fulbright scholarship to the University of Kansas. In 1969 he moved with his first wife, Ulla Sjostrom, and two children to Washington DC, and joined the World Bank. The marriage ended in divorce, and in 1977 he married Michaela Murphy.
After Lorenzo’s death, he returned to Europe, to the Italian city of Acqui Terme, in the region where he had grown up. He wrote a memoir, Lorenzo and His Parents (2012), allowed himself to be cared for by his grown-up children, and carried on with typical determination during years of poor health. Lorenzo, though, was never far from his thoughts. “I dream of him often. Sometimes he is in his sick bed, but often he is that small boy, running and swimming in the Comoros.”
Augusto is survived by Cristina and Francesco, the children of his first marriage.
• Augusto Odone, economist and medical pioneer, born 6 March 1933; died 24 October 2013
