This article was first published in
Given that he had trained and practised as a physician before turning to filmmaking, it is no surprise that George Miller was so captivated by the story of Michaela and Augusto Odone’s struggle to save their son from a deadly disease that he decided to turn it into a movie.
Lorenzo’s Oil, starring Nick Nolte and Susan Sarandon, became one of 1992’s most successful films and introduced the public to the many nuances of medical research and the contribution that can be made by the sheer doggedness of parents who dedicate their lives to finding a cure for their son stricken with adrenoleukodystrophy (ALD), a devastating disease.
In short, the film is about a young boy afflicted with a horrendous, progressive and incurable disease. His parents immerse themselves in the study of biochemistry and discover an effective treatment that is not recognized by an overcautious and callous medical establishment.
Lorenzo was an exceptionally bright child until in 1984, at the age of six, he began to exhibit changes in his behaviour. His movements became awkward, and he began to have trouble hearing. Then came slurred speech, temper tantrums and difficulty keeping his balance. The diagnosis was terrifying. Lorenzo was a victim of ALD, a genetic disease that typically shows up in boys between the ages of four and 10 and leads to death within a few years.
ALD is the result of very long-chain saturated fatty acids containing more than 22 carbons that build up in blood plasma. These fatty acids are normally produced from dietary shorter-chain fatty acids by enzymes appropriately called “elongases” and are then transported into cells by special transporter proteins. Within cellular organelles known as peroxisomes, the long-chain fatty acids are broken down into smaller molecules that perform a variety of vital functions.
However, in ALD, a mutation in a gene prevents the formation of the transporter protein and results in the buildup of the long-chain fatty acids in blood that end up inactivating nerve cells by stripping them of myelin, their protective sheath.
The Odones did not accept the prevailing medical opinion that no treatment for ALD exists and refused to just wait and watch Lorenzo die. They began to haunt libraries and scoured the medical literature for papers about the disease until one day they came upon a study in a relatively obscure Polish journal describing how the concentration of long-chain fatty acids in the bloodstream of rats can be lowered by feeding the animals a diet of other fatty acids.
It is at this point that the Odones convinced Dr. Hugo Moser (portrayed as Dr. Gus Nikolais by Peter Ustinov in the film,) the world’s leading expert on ALD, to convene the first-ever conference on the disease. Here they learned from Dr. William Rizzo of the Medical College of Virginia about preliminary experiments using olive oil to lower plasma levels of the troublesome long-chain fatty acids. It seems that the elongase enzymes that normally produce these from shorter saturated fatty acids can also interact with unsaturated fatty acids. If they are busy metabolizing these, then the long-chain fatty acids are not produced.
The Odones were quick to start feeding olive oil to Lorenzo, and indeed, the blood levels of his long-chain fatty acids dropped, although not dramatically. They then began a search for oils that might have an even greater affinity for the elongase enzymes and discovered that erucic acid from rapeseed was a candidate. The oil was very expensive to produce, but a British lab agreed to supply it to Lorenzo for experimental purposes.
Feeding Lorenzo a combination of erucic acid and oleic acid from olive oil resulted in a significant reduction in his blood levels of long-chain fatty acids. Unfortunately, by this time Lorenzo was totally paralyzed, could not speak, hear or see, and was only able to communicate somewhat with eye movements.
However, to the surprise of his doctors, not only did Lorenzo manage to evade his predicted demise, but by the age of 14 actually showed some improvement. He was able to swallow, regained some sight and managed to move his head, allowing him to communicate by means of a computer.
The medical community was not thrilled with the movie, claiming that the Odones’ discovery of the oil was overly romanticized and Lorenzo’s improvement overstated. Furthermore, doctors were portrayed as arrogant, close-minded and cold-hearted, unwilling to help in what they considered to be a futile search for a cure.
Augusto Odone underlined this view in the film with his comment that “these scientists have their own agenda, and it is different from ours.” At one point, the character of Dr. Moser declares that “I will have nothing to do with this oil.” That was an unfair attribution as pointed out by Dr. Moser in his review of the film published in the British medical journal, The Lancet. The film, he maintained, invented conflicts between the Odones and the medical establishment that did not exist.
Far from dismissing the nutritional treatment of ALD, Dr. Moser went on to conduct trials with “Lorenzo’s oil” on a large number of ALD patients. The results were disappointing. It seems that Lorenzo with his unusual longevity was an outlier, his survival perhaps due more to the extraordinary care and attention he received from his parents than to the oil administered through his feeding tube. Although Lorenzo’s oil has not turned out to be a cure for ALD, Dr. Moser did find a silver lining in the cloudy story. In boys who have the genetic variant that can lead to ALD, the disease can be prevented if treatment with Lorenzo’s oil is started before symptoms appear.
Although the film’s portrayal of the Odones as lay people who discover a therapy that had eluded the scientific community plays somewhat loose with the facts, profits from the film did allow the Odones to set up the Myelin Project, a foundation to promote research into demyelinating diseases. All the personalities involved in the Lorenzo’s Oil story have since died, but Lorenzo did live to the unprecedented age of 30. Just how much of a life that was is an open question.
