Study of the use of Lorenzo's oil in adults with adrenomyeloneuropathy, the adult form of
This is a double-masked placebo controlled study of glyceryl trioleate-glyceryl trierucate
(Lorenzo's Oil (LO)) therapy in adrenomyeloneuropathy (AMN), the adult form of X-linked
adrenoleukodystrophy (X-ALD). AMN is a slowly progressive distal axonopathy that involves
the long tracts of the spinal cord and differs from the rapidly progressive inflammatory
cerebral forms that most commonly affect boys and adolescents. All forms of X-ALD are
associated with the abnormal accumulation of very long chain fatty acids (VLCFA) in plasma
and tissues. The oral administration of LO normalizes plasma VLCFA levels within 4 weeks.
While previous therapeutic trials of LO therapy in patients with the cerebral forms of X-ALD
have been disappointing, recent studies suggest that it is beneficial in two types of X-ALD:
1) as a preventive of neurological involvement in asymptomatic boys; and 2) in AMN, where
it appears to slow the rate of progression. None of the previous studies have been
controlled, and we are now conducting the first placebo-controlled trial.
The 4-year study will include 120 men with AMN who do not have evidence of cerebral
involvement, and 120 women who are heterozygous for X-ALD and have an AMN-like syndrome. The
rate of progression will be compared in the LO and placebo groups using the Kurtzke EDSS
score as the primary outcome and a variety of secondary outcomes.
Adrenomyeloneuropathy affects about 1 in 40,000 men and 1 in 30,000 women. It is a
progressive disorder that leads to inability to walk and other severe deficits. This study
will provide definitive information whether Lorenzo's Oil therapy can slow progression.
- Males or females age 18 years or older in whom the diagnosis of X-ALD has been
confirmed by VLCFA assay and/or mutation analysis.
- Clinical evidence of spinal cord involvement with EDSS score between 1 and 6.5.
Patients with an EDSS score of 6.5 are severely affected but have retained the
capacity to walk 20 meters with the aid of a walker, crutch, or two canes.
- Either a normal brain MRI, or a type 3 pattern of MRI abnormality in which the
abnormality is considered to represent the centripetal extension of the distal
- Adrenal function assessed by measurement of plasma ACTH and appropriate steroid
replacement if adrenal insufficiency is present.
- Kurtzke EDSS score of >6.5.
- Cognitive or behavioral abnormalities that impair capacity to give informed consent
or carry out procedures that form part of the protocol.
- Current use, or use within 3 months, of Lorenzo's oil or other therapies that may
alter the course of X-ALD. Bone marrow transplant will be a permanent exclusion
- Contraindications for MRI procedure such as subjects with paramagnetic materials in
the body, such as aneurysm clips, pacemakers, intraocular metal or cochlear implants.
- Subjects who are pregnant.
- Allergies to peppermint
- Presence of non-specific conditions that may interfere with clinical assessment or
participation in the protocol.
Gerald V Raymond, M.D.
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.