Perseverance pays

Learning from Lorenzo's oil

 
By Vibha Varshney
Published: Sunday 07 June 2015

Perseverance pays

-- Neurologist Hugo Moser's unveiling of a 10-year-long study into the effects of Lorenzo's oil has shown that perseverance and logic are a powerful substitute to conventional research. When Lorenzo, the son of Augusto and Michaela Odone, was diagnosed with adrenoleukodystrophy (ald), there was no cure for the disease. Unable and unwilling to let their child die, they worked hard at understanding biochemistry. They pored through textbooks, pestered scientists and came up with Lorenzo's oil -- a mixture of oleic and erucic acids -- that could prevent some of the damage the disease causes.

The Odones did not have things easy. The first step was to understand the disease, and the nature of fatty acid synthesis. Essentially, ald is characterised by destruction of myelin, which insulates the nerves and helps in conducting impulses from one part of the body to another.

ald could be caused by either hereditary or acquired diseases. Demyelation, or the loss of myelin, occurs due to the accumulation of very long chain fatty acids (vlcfa) in parts of the body, such as the brain. These fatty acids accumulate because the function of certain enzymes that can oxidise the vlcfa gets impaired. While conventional therapies aim at increasing the levels of this oxidising enzyme, the Odones' approach was to reduce synthesis of vlcfa.

Augusto and Michaela Odone's initial experiments with oleic acid on Lorenzo showed that it could reduce vlcfa levels in the blood. This led them to further experiments with a combination of other fatty acids to find the most suitable mixture. A combination of olive oil and rapeseed oil was found to be the most effective. This was purified to ensure that only fatty acid chains of certain length were given to the patient. The oil is now manufactured by Croda International in Britain. Six bottles of the oil can be obtained for us $40; each bottle lasts for 10 to 14 days.

It has taken the medical community 10 years to fight deeply ingrained biases against what they saw as a 'do-it-yourself' remedy, and validate this concoction. There were, initially, vehement articles in respected magazines and journals denouncing the Odones' research. It is likely that people working on ald for a very long time found it difficult to digest the fact that a layperson had beaten them to it.

In the late 1980s, two teams, one led by Moser at the Kennedy Krieger Institute in Baltimore, Maryland, and the other based in Europe, studied 104 boys under the age of six, who suffered from ald. While some boys scrupulously followed the Lorenzo's oil regime, others did not. In the first group of boys, levels of fatty acids became nearly normal. In the second group, vlcfa remained abnormally high. A comparison between these groups showed that boys who followed the Lorenzo's oil regime had a lower risk of developing the symptoms of ald. "It's not a final cure for the disease, but it does give some additional time for scientists and doctors to come up with alternative therapies," says Ronald Wanders, Laboratory for Genetic Metabolic Diseases, Emma Children's Hospital, University of Amsterdam.

"You have to be proactive when you have a disease in the family or yourself," says Odone. You cannot wait for doctors to tell you what remedies are available; you contact people who might have a cure, he says.

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