Lorenzo oil: a panacea, palliative care, or marketing. Opinion of a neonatologist, pediatrician, and nutritionist

The spectrum of neonatal diagnostic methods for severe hereditary disorders has been expanding since December 31, 2022. In most cases, causal therapy, including diet therapy, can prevent early death and disability in children. X-linked adrenoleukodystrophy (X-ALD) is an inherited disorder of peroxisomal fatty acid oxidation characterized by accumulation of very long-chain saturated fatty acids. There are three clinical forms of this disease. The first one is infantile cerebral form accounting for 48% of all cases. Its onset is usually observed at the age of 2.5–10 years after normal development of a child, manifesting with progressive decortication. The second form is adrenomyelopathy (25% of cases) which starts with adrenal insufficiency at the age of 12–55 years, followed by neurological symptoms. The adult form (3% of cases) manifests after the age of 30 years and combines clinical phenotypes resembling schizophrenia with dementia. A low-fat diet with Lorenzo oil is believed to be effective, because it normalizes plasma levels of C24:0 and C26:0 in X-ALD patients; however, it does not prevent or alleviate neurological symptoms if they have already developed. Diet therapy increases plasma levels of C22:1 and C24:1 fatty acids, although their effect on X-ALD is yet to be elucidated. This review describes the mechanism of Lorenzo oil action, its side effects, case series, and analysis of dietary correction in various groups of patients. A modified diet with dietary fats replaced by Lorenzo oil is effective only in people with asymptomatic X-ALD, as a preventive measure for asymptomatic boys at risk of developing the cerebral form. Lorenzo oil is capable of alleviating and preventing ALD progression in patients without brain lesions. However, in symptomatic patients, the restriction of dietary fats and their replacement by Lorenzo oil is associated with a high risk of life-threatening complications. In this case, nutrition should meet special requirements of a patient, for example, the ability to chew and swallow, while the therapy should be primarily substitutive and symptomatic. Key words: adrenoleukodystrophy, diet therapy, Lorenzo oil