What does the evidence say?
There have been several clinical studies looking at whether Lorenzo’s Oil is effective at treating or preventing ALD. The initial excitement surrounding Lorenzo’s Oil came because a number of studies showed that Lorenzo’s Oil can reduce the level of very long chain fatty acids (VLCFAs) in the blood. Unfortunately, further investigation has found that despite these changes Lorenzo’s Oil does not treat or slow the progression of symptoms, such as changes in behaviour and neurological decline, once they have started. It is thought this might be because although Lorenzo’s Oil reduces VLCFAs in the blood, it is not able to penetrate the blood brain barrier and help reduce VLCFA effects in the brain. Having said this, some clinical trials have suggested that Lorenzo’s Oil can prevent or slow the onset of symptoms in boys without symptoms. This has not been backed up by large-scale trials, and therefore although Lorenzo’s Oil MAY be beneficial, it is NOT a proven preventative.
What do the doctors think?
The decision whether to start on the oil is an individual decision, so you may want to discuss the pros and cons with your specialist doctor. A survey of specialist metabolic paediatricians in the UK has shown the majority of doctors do not consider the evidence of its benefit to be strong, but some would be willing to use Lorenzo’s Oil if a parent requested it. All doctors emphasise that Lorenzo’s Oil should not be used as a replacement for regular monitoring, such as blood tests and MRI scans, as it is key that any potential complications of ALD of picked up early so that they can be managed well.
The practicalities: taking Lorenzo’s oil, diet and side effects
Lorenzo’s Oil looks like a bottle of clear liquid at the top with melted fat below. The oil is stored in the fridge and you must mix the two halves at room temperature to make the solution. Most small children are given the oil in a warm drink, like milk. Although it is not very nice to take Lorenzo’s oil, often the younger a child is, the more easily they will be able to deal with it.
For Lorenzo’s Oil to be effective at lowering blood VLCFA levels, patients must go on a very strict low-fat diet, which would have to be worked out by dietician.
Some find this very difficult especially if they start the diet once they have started school or in later life. This also means patients, parents and carers will need to learn how to read the fat content of food labels. Ask to be referred to a dietician, who can help ensure the patient is getting the best possible diet within restrictions. The dietician will also advise on supplements to ensure the patient gets the essential fatty acids he needs.
Lorenzo’s oil normally has side effects- many boys can find it reduces their quality of life, and as any potential positive effect is unproven, the family may opt to stop it. It can reduce the level of platelets in the blood which could result in the blood not clotting as it should. Levels of platelets in the blood are monitored with regular blood tests. The amount of Lorenzo’s oil taken must also be carefully monitored. As patients grow, dietitians will work out how much Lorenzo’s Oil they need to take.
Where do I find Lorenzo’s Oil?
Lorenzo’s Oil is available on NHS prescription throughout the UK but although it is potentially available, it would be up to individual Trusts and Clinical Commissioning Groups ( CCGs) to agree funding and prescribing .In other countries, Lorenzo’s Oil is not always available
The bottom line: four key messages
- Lorenzo’s Oil is NOT effective at TREATING symptoms once they have started.
- Lorenzo’s Oil is NOT a proven PREVENTATIVE, yet it MAY be BENEFICIAL for boys with asymptomatic ALD.
- The decision to start on the oil is an individual decision, but we would recommend seeking advice from your specialist first. If you do start and you feel your son’s quality of life deteriorates it is absolutely fine to stop it.
- Lorenzo’s Oil is should be used as an adjunct, not replacement, to the regular monitoring and clinical management of ALD.
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