# GLYCEROL-TRIERUCATE THERAPY OF ADRENOLEUKODYSTROPHY

> **NIH NIH R01** · HUGO W. MOSER RES INST KENNEDY KRIEGER · 1994 · $183,270

## Abstract

The key biochemical abnormality in X-linked adrenoleukodystrophy (ALD) is
the accumulation of saturated very long chain fatty acids (VLCFA) in
tissues and body fluids, due to a genetic defect of a peroxisomal enzyme
that normally degrades these substances.  ALD affects mainly the adrenal
cortex, nervous system white matter and testis.  While the endocrine
deficits can be helped by steroid replacement, the neurological disability
cannot be treated and often leads to severe disability and premature death.

Neurological involvement ranges from rapid cerebral demyelination with mean
age of onset at age 7.2 + 1.7 (SD) years, to much more slowly progressive
spinal cord and peripheral nerve involvement in adults
(adrenomyeloneuropathy).  Our laboratory conducts a national program for
the diagnosis of X-linked ALD and peroxisomal disorders, and through this
program has identified more than 700 males with X-linked ALD, by far the
largest group of patients with this disorder anywhere.

Recent studies have demonstrated that a new dietary approach can normalize
the levels of saturated VLCFA in the plasma of patients with ALD within 2-4
weeks.  The regimen involves the oral administration of a glycerol
trierucate (GTE) oil together with certain other dietary modifications.
Diagnosis ALD can be achieved in the early postnatal period (as well as
prenatally), at least several years before the onset of neurological
disability in untreated patients.  The striking biochemical effect of the
new regimen for the first time offers the opportunity to test whether early
normalization of saturated VLCFA can prevent or ameliorate the neurological
disability in ALD.  The proposed study has three components:  1) a double-
blinded trial involving adults with adrenomyeloneuropathy and
neurologically involved ALD heterozygotes; 2) a prevention trial for
neurologically asymptomatic boys with the biochemical defect of ALD; and 3)
a study of neurologically symptomatic boys in whom the rate of neurological
progression will be compared with that in more than 100 untreated childhood
ALD patients who had previously been diagnosed in our laboratory.

## Key facts

- **NIH application ID:** 2199915
- **Project number:** 5R01HD026371-05
- **Recipient organization:** HUGO W. MOSER RES INST KENNEDY KRIEGER
- **SAM.gov UEI:** DKMDCB5HNBL7
- **Principal Investigator:** HUGO W MOSER
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 1994
- **Award amount:** $183,270
- **Award type:** 5
- **Project period:** 1990-01-01 → 1994-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/2199915

## Citation

> US National Institutes of Health, RePORTER application 2199915, GLYCEROL-TRIERUCATE THERAPY OF ADRENOLEUKODYSTROPHY (5R01HD026371-05). Retrieved via MORVS 2026-10-25 from https://api.morvs.ai/grant/nih/2199915. Licensed CC0.

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