Lorenzo's Oil Adrenoleukodystrophy: Male Genetic Disorder

Before the release of the film Lorenzo's oil, few people will have heard of the rare genetic disease Adrenoleukodystrophy (ALD). The movie helped to raise awareness of the condition by following the struggle of one family to find a cure for this debilitating neurological disease. After two years of careful research and pushing the specialists in the field to reveal information, the family of young Lorenzo Odone finally invented an oil that, when taken, had an effect on the fatty acids that are the cause of the problem. The oil, extracted from a combination of rapeseed and olive oil has had its therapeutic effects verified by a 10 year study.
Adrenoleukodystrophy affects only males.
Research & Adrenoleukodystrophy
Research on Lorenzo's oil was conducted over a 10-year trial on 104 boys with the ALD gene. All the boys were under the age of six and symptom free on commencement of the trial. Dr Hans Moser, the lead researcher, found that the boys who were not given Lorenzo's oil scrupulously were nearly three times as likely to develop symptoms as those who were given it as prescribed. However, one possible problem is that the study is a small and ran with no placebo group. It is still possible therefore that the boys could develop ALD symptoms in the adult form of the disorder.
What is Adrenoleukodystrophy?
Adrenoleukodystrophy is transmitted as an X-linked trait. Its incidence is estimated at 1 out of 20,000 to 1 in 50,000 and affects all races. ALD is a metabolic defect in the accumulation of long chain fatty acids in the nervous system, adrenal gland, and testes, where the accumulated material disrupts normal activity. What results is impairment and eventual loss of neural impulses throughout the body. Bodily functions deteriorate and eventually brain damage occurs. Without treatment many boys are bedridden and they die before reaching their teens. The adult form of the disease becomes evident in late teens and early 20s, it progresses more slowly.
Treatment for Adrenoleukodystrophy
The main treatment remains bone marrow transplantation and this is still seen as the best hope of survival. Lorenzo's oil can be said at present to delay symptoms and further trials are required . Dr Moser is now recommending that all presymptomatic boys with Adrenoleukodystrophy be given Lorenzo's oil.
Lorenzo Odone was 28 years old on May 29, 2006 and severely disabled. Sadly the discovery his parents made was too late to prevent severe and progressive damage but his father believes Lorenzo's oil has prolonged his life.