"Lorenzo's oil" therapy for X-linked adrenoleukodystrophy: Rationale and current assessment of efficacy

Journal of Molecular Neuroscience

Volumn 33, Issue 1, 2007, Pages 105-113

  Moser, Hugo W ^a   Moser, Ann B ^a   Hollandsworth, Kim ^a   Brereton, N Hong ^b   Raymond, Gerald V ^a  

^a KENNEDY KRIEGER INSTITUTE   (United States)

^b JOHNS HOPKINS HOSPITAL   (United States)

Author keywords

Adrenoleukodystrophy; Clinical trials; Erucic acid; Fatty acids; Glyceryl trierucate; Glyceryl trioleate; Lorenzo's oil; Newborn screening; Therapy; Very long chain fatty acids

Indexed keywords

CD1B ANTIGEN; CD1D ANTIGEN; CORTICOTROPIN; DOCOSAHEXAENOIC ACID; GANGLIOSIDE ANTIBODY; HYDROCORTISONE; LORENZO OIL; MANGANESE SUPEROXIDE DISMUTASE; MONOUNSATURATED FATTY ACID; OLEIC ACID; POLYUNSATURATED FATTY ACID; TRIOLEIN; TUMOR NECROSIS FACTOR ALPHA; VERY LONG CHAIN FATTY ACID;

ADRENAL CORTEX; ADRENAL FUNCTION; ADRENOLEUKODYSTROPHY; ASTROCYTE; BLOOD BRAIN BARRIER; CHROMOSOME XQ; CLINICAL TRIAL; CYTOLYSIS; DEMYELINATION; DIET RESTRICTION; DISEASE COURSE; DRUG EFFECT; DRUG EFFICACY; FATTY ACID METABOLISM; HEMISPHERE; HUMAN; HYDROCORTISONE RELEASE; LIPID PEROXIDATION; NEUROCHEMISTRY; NEWBORN SCREENING; OLIGODENDROGLIA; OXIDATIVE STRESS; PARAPLEGIA; PATHOGENESIS; PHENOTYPE; REVIEW; SYMPTOMATOLOGY; THROMBOCYTE COUNT; X CHROMOSOME LINKED DISORDER;

ADRENOLEUKODYSTROPHY; DISEASE PROGRESSION; DRUG COMBINATIONS; ERUCIC ACIDS; FATTY ACIDS; HUMANS; TRIOLEIN;

EID: 35348876033     PISSN: 08958696     EISSN: None     Source Type: Journal    
DOI: 10.1007/s12031-007-0041-4     Document Type: Review

References (70)

1. Abdellatif, A. M. (1972). Cardiopathogenic effects of dietary rapeseed oil. Nutrition Reviews, 30, 2-6.
2. Asheuer, M., Bieche, I., Laurendeau, I., Moser, A., Hainque, B., Vidaud, M., et al. (2005). Decreased expression of ABCD4 and BG1 genes early in the pathogenesis of X-linked adrenoleukodystrophy. Human Molecular Genetics, 14, 1293-1303.
3. Assies, J., van Geel, B., Weverling, G. J., Haverkort, E. B., & Barth, P. G. (1994). Endocrine evaluation during dietary therapy for adrenomyeloneuropathy. In H. R. Bhatt, G. H. T. James, G. M. Besser, G. F. Bottazzo, & H. E. Keen (Eds.), Advances in Thomas Addison's diseases (pp. 191-198). Bristol: Journal of Endocrinology.
4. Aubourg, P., Adamsbaum, C., Lavallard-Rousseau, M. C., Rocchiccioli, F., Cartier, N., Jambaque, I., et al. (1993). A two-year trial of oleic and erucic acids ("Lorenzo's oil") as treatment for adrenomyeloneuropathy. New England Journal of Medicine, 329, 745-752.
5. Aubourg, P., Blanche, S., Jambaque, I., Rocchiccioli, F., Kalifa, G., Naud-Saudreau, C., et al. (1990). Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation. New England Journal of Medicine, 322, 1860-1866.
6. Bezman, L., & Moser, H. W. (1998). Incidence of X-linked adrenoleukodystrophy and the relative frequency of its phenotypes. American Journal of Medical Genetics, 76, 415-419.
7. Bezman, L., Moser, A. B., Raymond, G. V., Rinaldo, P., Watkins, P. A., Smith, K. D., et al. (2001). Adrenoleukodystrophy: Incidence, new mutation rate, and results of extended family screening. Annals of Neurology, 49, 512-517.
8. Bizzozero, O. A., Zuniga, G., & Lees, M. B. (1991). Fatty acid composition of human myelin proteolipid protein in peroxisomal disorders. Journal of Neurochemistry, 56, 872-878.
9. Boehm, C. D., Cutting, G. R., Lachtermacher, M. B., Moser, H. W., & Chong, S. S. (1999). Accurate DNA-based diagnostic and carrier testing for X-linked adrenoleukodystrophy. Molecular Genetics and Metabolism, 66, 128-136.
10. Bourre, J. M., Daudu, O., & Baumann, N. (1976). Nervonic acid biosynthesis by erucyl-CoA elongation in normal and quaking mouse brain microsomes. Elongation of other unsaturated fatty acyl-CoAs (mono and poly-unsaturated). Biochimica Et Biophysica Acta, 424, 1-7.
11. Brown, F. R. 3rd, Van Duyn, M. A., Moser, A. B., Schulman, J. D., Rizzo, W. B., Snyder R. D., et al. (1982). Adrenoleukodystrophy: Effects of dietary restriction of very long chain fatty acids and of administration of carnitine and clofibrate on clinical status and plasma fatty acids. Johns Hopkins Medical Journal, 151, 164-172.
12. Choi, J. K., Ho J., Curry, S., Qin, D., Bittman, R., & Hamilton, J. A. (2002). Interactions of very long-chain saturated fatty acids with serum albumin. Journal of Lipid Research, 43, 1000-1010.
13. Cox, C., Dubey P., Raymond, G. V., Mahmood, A., Moser, A. B., & Moser, H. W. (2006). Cognitive evaluation of neurologically asymptomatic boys with X-linked adrenoleukodystrophy. Archives of Neurology, 63, 69-73.
14. Dean, M., Hamon, Y., & Chimini, G. (2001). The human ATP-binding cassette (ABC) transporter superfamily. Journal of Lipid Research, 42, 1007-1017.
15. Di Biase, A., Di Benedetto, R., Fiorentini, C., Travaglione, S., Salvati, S., Attorri, L., et al. (2004). Free radical release in C6 glial cells enriched in hexacosanoic acid: Implication for X-linked adrenoleukodystrophy pathogenesis. Neurochemistry International, 44, 215-221.
16. Dubey, P., Raymond, G., Moser, A. B., Kharkar, S., Bezman, L., & Moser, H. W. (2005). Adrenal insufficiency in asymptomatic adrenoleukodystrophy patients identified by very long chain fatty acid screening. Journal de Pediatria, 146, 528-532.
17. Dubois-Dalcq, M., Feigenbaum, V., & Aubourg, P. (1999). The neurobiology of X-linked adrenoleukodystrophy, a demyelinating peroxisomal disorder. Trends in Neurosciences, 22, 4-12.
18. Fatemi, A., Smith, S. A., Dubey, P., Zackowski, K. M., Bastian, A. J., van Zijl, P. C., et al. (2005). Magnetization transfer MRI demonstrates spinal cord pathology in adrenomyeloneuropathy. Neurology, 64, 1739-1745.
19. Feigenbaum, V., Gelot, A., Casanova, P., Daumas-Duport, C., Aubourg, P., & Dubois-Dalcq, M. (2000). Apoptosis in the central nervous system of cerebral adrenoleukodystrophy patients. Neurobiology of Disease, 7, 600-612.
20. Gilg, A. G., Singh, A. K., & Singh, I. (2000). Inducible nitric oxide synthase in the central nervous system of patients with X-adrenoleukodystrophy. Journal of Neuropathology and Experimental Neurology, 59, 1063-1069.
21. Golovko, M. Y., & Murphy, E. J. (2006). Uptake and metabolism of plasma derived erucic acid by rat brain. Journal of Lipid Research, 47, 1289-1297.
22. Hamilton, J. A., Era, S., Bhamidipati, S. P., & Reed, R. G. (1991). Locations of the three primary binding sites for long-chain fatty acids on bovine serum albumin. Proceedings of the National Academy of Sciences of the United States of America, 88, 2051-2054.
23. Ho, J. K., Moser, H., Kishimoto, Y., & Hamilton, J. A. (1995). Interactions of a very long chain fatty acid with model membranes and serum albumin. Implications for the pathogenesis of adrenoleukodystrophy. Journal of Clinical Investigation, 96, 1455-1463.
24. Hubbard, W. C., Moser, A. B., Tortorelli, S., Liu, A., Jones, D., & Moser, H. W. (2006). Combined liquid chromatography-Tandem mass spectrometry as an analytical method for high throughput screening for X-linked adrenoleukodystrophy and other peroxisomal disorders: Preliminary findings. Molecular Genetics and Metabolism, 89, 185-187.
25. Igarashi, M., Belchis, D., & Suzuki, K. (1976a). Brain gangliosides in adrenoleukodystrophy. Journal of Neurochemistry, 27, 327-328.
26. Igarashi, M., Schaumburg, H. H., Powers, J., Kishmoto, Y., Kolodny, E., & Suzuki, K. (1976b). Fatty acid abnormality in adrenoleukodystrophy. Journal of Neurochemistry, 26, 851-860.
27. Ito, M., Blumberg, B. M., Mock, D. J., Goodman, A. D., Moser, A. B., Moser, H. W., et al. (2001). Potential environmental and host participants in the early white matter lesion of adrenoleukodystrophy: Morphologic evidence for CD8 cytotoxic T cells, cytolysis of oligodendrocytes, and CD1-mediated lipid antigen presentation. Journal of Neuropathology and Experimental Neurology, 60, 1004-1019.
28. Kemp, S., Valianpour, F., Denis, S., Ofman, R., Sanders, R. J., Mooyer, P., et al. (2005). Elongation of very long-chain fatty acids is enhanced in X-linked adrenoleukodystrophy. Molecular Genetics and Metabolism, 84, 144-151.
29. Kickler, T. S., Zinkham, W. H., Moser, A., Shankroff, J., Borel, J., & Moser, H. (1996). Effect of erucic acid on platelets in patients with adrenoleukodystrophy. Biochemical and Molecular Medicine, 57, 125-133.
30. Knazek, R. A., Rizzo, W. B., Schulman, J. D., & Dave, J. R. (1983). Membrane microviscosity is increased in the erythrocytes of patients with adrenoleukodystrophy and adrenomyeloneuropathy. Journal of Clinical Investigation, 72, 245-248.
31. Koehler, W., & Sokolowski, P. (1999). A new disease-specific scoring system for adult phenotypes of X-linked adrenoleukodystrophy. Journal of Molecular Neuroscience, 13, 247-252.
32. Koehler, W., & Sokolowski, P. (2005). Clinical phenotypes, diagnosis and treatment of adulthood X-linked adrenoleukodystrophy. In J. Berger, S. Stoeckler & W. Koehler (Eds.), Understanding and treating adrenoleukodystrophy: Present state and future perspectives (pp. 28-60). SPS Verlagsgeseltschaft: Verlag Heilbronn.
33. Kurtzke, J. F. (1983). Rating neurologic impairment in multiple sclerosis: An expanded disability status scale (EDSS). Neurology, 33, 1444-1452.
34. Moser, H. W., Aubourg P., Cornblath, D., Borel, J., Wu, Y.-W., Bergin, A., et al. (1991). Therapy for X-linked adrenoleukodystrophy. In R. J. Desnick (Ed.), Treatment of genetic disease (pp. 111-129). New York: Churchill Livingstone.
35. Moser, H. W., & Brereton, N. H. (2005) Adrenoleukodystrophy and other peroxisomal disorders. In S. W. Ekvall & V. K. Ekvall (Eds.), Pediatric nutrition in chronic diseases and developmental disorders: Prevention, assessment and treatment (pp. 312-317). New York: Oxford Univ. Press.
36. Moser, A. B., Jones, D. S., Raymond, G. V., & Moser, H. W. (1999a). Plasma and red blood cell fatty acids in peroxisomal disorders. Neurochemical Research, 24, 187-197.
37. Moser, A. B., Kreiter, N., Bezman, L., Lu, S., Raymond, G. V., Naidu, S., et al. (1999b). Plasma very long chain fatty acids in 3,000 peroxisome disease patients and 29,000 controls. Annals of Neurology, 45, 100-110.
38. Moser, A. B., & Moser, H. W. (1999). The prenatal diagnosis of X-linked adrenoleukodystrophy. Prenatal Diagnosis, 19, 46-48.
39. Moser, H. W., Moser, A. B., Kawamura, N., Murphy, J., Suzuki, K., Schaumburg, H., et al. (1980). Adrenoleukodystrophy: Elevated C26 fatty acid in cultured skin fibroblasts. Annals of Neurology, 7, 542-549.
40. Moser, H. W., Raymond, G. V., & Dubey, P. (2005). Adrenoleukodystrophy: New approaches to a neurodegenerative disease. JAMA, 294, 3131-3134.
41. Moser, H. W., Raymond, G. V., Koehler, W., Sokolowski, P., Hanefeld, F., Korenke, G. C., et al. (2003). Evaluation of the preventive effect of glyceryl trioleate-trierucate ("Lorenzo's oil") therapy in X-linked adrenoleukodystrophy: Results of two concurrent trials. Advances in Experimental Medicine and Biology, 544, 369-387.
42. Moser, H. W., Raymond, G. V., Lu, S. E., Muenz, L. R., Moser, A. B., Xu, J., et al. (2005). Follow-up of 89 Lorenzo's oil treated asymptomatic adrenoleukodystrophy patients. Archives of Neurology, 62, 1073-1080.
43. Mosser, J., Douar, A. M., Sarde, C. O., Kioschis, P., Feil, R., Moser, H., et al. (1993). Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters. Nature, 361, 726-730.
44. Odone, A., & Odone, M. (1989) Lorenzo's oil: A new treatment for adrenoleukodystrophy. Journal of Pediatric Neuroscience, 5, 55-61.
45. Oya, Y., Nakayasu, H., Fujita, N., Suzuki, K., & Suzuki, K. (1998). Pathological study of mice with total deficiency of sphingolipid activator proteins (SAP knockout mice). Acta Neuropathologica (Berl.), 96, 29-40.
46. Oz, G., Tkac, I., Charnas, L. R., Choi, I. Y., Bjoraker, K. J., Shapiro, E. G., et al. (2005). Assessment of adrenoleukodystrophy lesions by high field MRS in non-sedated pediatric patients. Neurology, 64, 434-441.
47. Paintlia, A. S., Gilg, A. G., Khan, M., Singh, A. K., Barbosa, E., & Singh I. (2003) Correlation of very long chain fatty acids accumulation and inflammatory disease progression in childhood ALD: Implications for potential therapies. Neurobiology of Disease, 14, 425-439.
48. Peters, C., Charnas, L. R., Tan, Y., Ziegler, R. S., Shapiro, E. G., DeFor, T., et al. (2004). Cerebral X-linked adrenoleukodystrophy: The international hematopoietic cell transplantation experience from 1982 to 1999. Blood, 104, 881-888.
49. Poulos, A., Gibson, R., Sharp, P., Beckman, K., & Grattan-Smith, P. (1994). Very long chain fatty acids in X-linked adrenoleukodystrophy brain after treatment with Lorenzo's oil. Annals of Neurology, 36, 741-746.
50. Powers, J. M., DeCiero, D. P., Cox, C., Richfield, E. K., Ito, M., Moser, A. B., et al. (2001). The dorsal root ganglia in adrenomyeloneuropathy: Neuronal atrophy and abnormal mitochondria. Journal of Neuropathology and Experimental Neurology, 60, 493-501.
51. Powers, J. M., DeCiero, D. P., Ito, M., Moser, A. B., & Moser, H. W. (2000). Adrenomyeloneuropathy: A neuropathologic review featuring its noninflammatory myelopathy. Journal of Neuropathology and Experimental Neurology, 59, 89-102.
52. Powers, J. M., Liu, Y., Moser, A. B., & Moser, H. W. (1992). The inflammatory myelinopathy of adreno-leukodystrophy: Cells, effector molecules, and pathogenetic implications. Journal of Neuropathology and Experimental Neurology 51, 630-643.
53. Powers, J. M., Pei, Z., Heinzer, A. K., Deering, R., Moser, A. B., Moser, H. W., et al. (2005). Adrenoleukodystrophy: Oxidative stress of mice and men. Journal of Neuropathology and Experimental Neurology 64, 1067-1079.
54. Powers, J. M., Schaumburg, H. H., Johnson, A. B., & Raine, C. S. (1980). A correlative study of the adrenal cortex in adrenoleukodystrophy - evidence for a fatal intoxication with very long chain saturated fatty acids. Investigative & Cell Pathology, 3, 353-376.
55. Rasmussen, M., Moser, A. B., Borel, J., Khangoora, S., & Moser, H. W. (1994). Brain, liver, and adipose tissue erucic and very long chain fatty acid levels in adrenoleukodystrophy patients treated with glyceryl trierucate and trioleate oils (Lorenzo's oil). Neurochemical Research, 19, 1073-1082.
56. Rizzo, W. B., Leshner, R. T., Odone, A., Craft, D. A., Jennings, S. S., & Jaitly, R. (1990). X-linked adrenoleukodystrophy: Biochemical and clinical efficacy of dietary erucic acid therapy. In G. Uziel, R. J. A. Wanders, & M. E. Cappa (Eds.), Adrenoleukodystrophy and other peroxisomal disorders (pp. 149-162). Amsterdam: Excerpta Medica.
57. Rizzo, W. B., Leshner, R. T., Odone, A., Dammann, A. L., Craft, D. A., Jensen, M. E., et al. (1989). Dietary erucic acid therapy for X-linked adrenoleukodystrophy. Neurology, 39, 1415-1422.
58. Rizzo, W. B., Phillips, M. W., Dammann, A. L., Leshner, R. T., Jennings, S. S., Avigan J., et al. (1987). Adrenoleukodystrophy: Dietary oleic acid lowers hexacosanoate levels. Annals of Neurology, 21, 232-239.
59. Rizzo, W. B., Watkins, P. A., Phillips, M. W., Cranin, D., Campbell, B., & Avigan, J. (1986). Adrenoleukodystrophy: Oleic acid lowers fibroblast saturated C22-26 fatty acids. Neurology, 36, 357-361.
60. Sauer, F. D., & Kramer, J. K. G. (1983). The Problems Associated with the Feeding of High Erucic Acid Rapeseed Oils and Some Fish Oils to Experimental Animals. In J. K. G. Kramer, F. D. Sauer, & W. J. Pigden (Eds.), High and low erucic acid rapeseed oils (pp. 253-292). Toronto: Academic Press.
61. Schaumburg, H. H., Powers, J. M., Raine, C. S., Suzuki, K., & Richardson, E. P. Jr. (1975). Adrenoleukodystrophy. A clinical and pathological study of 17 cases. Archives of Neurology, 32, 577-591.
62. Shapiro, E., Krivit, W., Lockman, L., Jambaque, I., Peters, C., Cowan, M., et al. (2000). Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy. Lancet, 356, 713-718.
63. Tagawa, Y., Laroy, W., Nimrichter, L., Fromholt, S. E., Moser, A. B., Moser, H. W., et al. (2002). Anti-ganglioside antibodies bind with enhanced affinity to gangliosides containing very long chain fatty acids. Neurochemical Research, 27, 847-855.
64. Tsuji, S., Suzuki, M., Ariga, T., Sekine, M., Kuriyama, M., & Miyatake, T. (1981). Abnormality of long-chain fatty acids in erythrocyte membrane sphingomyelin from patients with adrenoleukodystrophy. Journal of Neurochemistry, 36, 1046-1049.
65. Uziel, G., Bertini, E., Bardelli, P., Rimoldi, M., & Gambetti, M. (1991). Experience on therapy of adrenoleukodystrophy and adrenomyeloneuropathy. Developmental Neuroscience, 13, 274-279.
66. Valianpour, F., Selhorst, J. J. M., van Lint, L. E. M., van Gennip, A. H., Wanders, R. J. A., & Kemp, S. (2003). Analysis of very long chain fatty acids using electrospray ionization mass spectrometry. Molecular Genetics and Metabolism, 79, 189-196.
67. van Geel, B. M., Assies, J., Haverkort, E. B., Koelman, J. H., Verbeeten, B. Jr., Wanders R. J., et al. (1999). Progression of abnormalities in adrenomyeloneuropathy and neurologically asymptomatic X-linked adrenoleukodystrophy despite treatment with "Lorenzo's oil". Journal of Neurology, Neurosurgery and Psychiatry, 67, 290-299.
68. Vargas, C. R., Wajner, M., Sirtori, L. R., Goulart, L., Chiochetta, M., Coelho, D., et al. (2004). Evidence that oxidative stress is increased in patients with X-linked adrenoleukodystrophy. Biochimica et Biophysica Acta, 1688, 26-32.
69. Whitcomb, R. W., Linehan, W. M., & Knazek, R. A. (1988). Effects of long-chain, saturated fatty acids on membrane microviscosity and adrenocorticotropin responsiveness of human adrenocortical cells in vitro. Journal of Clinical Investigation, 81, 185-188.
70. Zackowski, K., Dubey, P., Raymond, G. V., Mahmood, A., Moser, A. B., & Moser, H. W. (2006). Relating sensorimotor function and axonal integrity in adrenomyeloneuropathy. Archives of Neurology, 63, 74-80.