XML Feed for RxPG News   Add RxPG News Headlines to My Yahoo!   Javascript Syndication for RxPG News

Research Health World General
 
  Home
 
 Latest Research
 Cancer
 Psychiatry
 Genetics
  X Chromosome
  Genetic Disorders
   Brachydactyly
   Huntington's
   Fragile X Syndrome
   Progeria
   MSUD
  Cloning
 Surgery
 Aging
 Ophthalmology
 Gynaecology
 Neurosciences
 Pharmacology
 Cardiology
 Obstetrics
 Infectious Diseases
 Respiratory Medicine
 Pathology
 Endocrinology
 Immunology
 Nephrology
 Gastroenterology
 Biotechnology
 Radiology
 Dermatology
 Microbiology
 Haematology
 Dental
 ENT
 Environment
 Embryology
 Orthopedics
 Metabolism
 Anaethesia
 Paediatrics
 Public Health
 Urology
 Musculoskeletal
 Clinical Trials
 Physiology
 Biochemistry
 Cytology
 Traumatology
 Rheumatology
 
 Medical News
 Health
 Opinion
 Healthcare
 Professionals
 Launch
 Awards & Prizes
 
 Careers
 Medical
 Nursing
 Dental
 
 Special Topics
 Euthanasia
 Ethics
 Evolution
 Odd Medical News
 Feature
 
 World News
 Tsunami
 Epidemics
 Climate
 Business
Search

Last Updated: Nov 17th, 2006 - 22:35:04

Genetic Disorders Channel
subscribe to Genetic Disorders newsletter

Latest Research : Genetics : Genetic Disorders

   DISCUSS   |   EMAIL   |   PRINT
Lorenzo's oil (LO) reduced the risk of developing severe X-linked adrenoleukodystrophy (ALD)
Jul 12, 2005, 12:26, Reviewed by: Dr.

Sixty-six patients (74 percent) were well at last follow-up. Twenty-one patients (24 percent) developed MRI abnormalities and 10 patients (11 percent) developed neurological abnormalities. The researchers found a significant association between the development of MRI abnormalities and an increase in the levels of the saturated very long chain fatty acid C26:0. "Patients who had a neurological abnormality had significantly higher weighted average C26:0 levels than those who did not have an abnormality, suggesting that an LO-induced decrease in the C26:0 level could protect against the inflammatory cerebral disease," the authors report.

 
Treatment of boys with X-linked adrenoleukodystrophy (ALD) with Lorenzo's oil (LO) reduced their risk of developing the severe debilitating form of the disease, according to a study in the July issue of the Archives of Neurology, one of the JAMA/Archives journals.

Individuals with ALD accumulate high levels of saturated very long-chain fatty acids (VLCFA) in their brains. The course of the disease results in a number of different manifestations [phenotypes], according to background information in the article. The rapidly progressive cerebral ALD (CERALD) type typically begins between ages four and eight and progresses rapidly to total disability within a few years. An adult form is non-inflammatory, progresses slowly and is far less disabling. Children who do not develop abnormalities as measured by magnetic resonance imaging (MRIs) by age seven or clinical symptoms by age 10, have greatly diminished risk of developing cerebral ALD.

In 1989, one of the authors of this study, Augusto Odone, pioneered a treatment (Lorenzo's oil), which was shown to normalize the levels of saturated very long-chain fatty acids within four weeks in most patients with ALD. "The striking effect of LO on plasma C26:0 [a saturated very long-chain fatty acid] levels engendered the hope that it would be of clinical benefit for patients with ALD," the authors write. However, previous clinical trials led to the conclusion that Lorenzo's oil did not alter the rate of progression of the disease in patients who already had neurological symptoms.

Hugo W. Moser, M.D., of the Kennedy Kreiger Institute, Baltimore, and colleagues treated 89 boys with ALD who had no neurological symptoms and normal brain MRIs with moderate dietary fat restriction and Lorenzo's oil between 1989 and 2002. Sixty-four of the patients were younger than seven years old when they began treatment and all were followed up for an average of approximately seven years. Because of the devastating nature of cerebral ALD, and the hope that the striking reduction of very long chain fatty acid levels would lead to clinical benefit, none of the boys were given placebo. Fatty acids blood levels were assessed every month for the first six months after enrollment in the study and every three to six months thereafter. Neurological examinations and MRIs were scheduled every six to 12 months.

Sixty-six patients (74 percent) were well at last follow-up. Twenty-one patients (24 percent) developed MRI abnormalities and 10 patients (11 percent) developed neurological abnormalities. The researchers found a significant association between the development of MRI abnormalities and an increase in the levels of the saturated very long chain fatty acid C26:0. "Patients who had a neurological abnormality had significantly higher weighted average C26:0 levels than those who did not have an abnormality, suggesting that an LO-induced decrease in the C26:0 level could protect against the inflammatory cerebral disease," the authors report.

"We recommend that LO therapy be offered to male patients with ALD who are neurologically asymptomatic, have normal brain MRI results, and are at risk of developing CERALD," the authors conclude. "This recommendation is based on strongly suggestive, albeit not fully definitive, evidence of a preventive effect combined with our awareness of the severe prognosis of the untreated patients with CERALD. ...The patients who are younger than seven years represent prime candidates for this therapy. We hypothesize that intensive LO therapy during the ages at which the risk for CERALD is greatest may protect against this phenotype until they reach the ages at which the risk for CERALD diminishes."

In an editorial accompanying the article, Raymond Ferri, M.D., Ph.D. and Phillip F. Chance, M.D., of the University of Washington, Seattle, write "In recent years, extraordinary progress has also been made in developing effective treatments, and ALD serves as an excellent model for the treatment of neurometabolic diseases. ...Current treatment includes hematopoietic stem cell transplantation (HSCT) to stabilize neurologic progression, steroid therapy for adrenal insufficiency, and symptomatic treatments. The article by Moser et al in this issue may establish new standards for the treatment of this degenerative disorder."

"Therefore, Moser and colleagues propose LO therapy for all asymptomatic patients with biochemical evidence of ALD to slow the progression of disease and to prevent symptoms until the child is past the age for the development of the childhood cerebral form of the disorder," the authors write. "Successful implementation of this practice requires early identification of at-risk patients. However, because almost 20 percent of the patients are either asymptomatic or have Addison disease only, at-risk children may not be identified. As also mentioned in the article, neonatal screening would identify more at-risk patients at a very early age. This would allow for further studies to examine very early treatment with LO for affected children, and dietary therapy can be studied in other ALD phenotypes [manifestations]. Also, this study can be extended to follow patients for an even greater duration to establish the full treatment effects of LO."

"X-linked ALD is a rare, progressive neurometabolic disorder, but coordinated, worldwide research efforts have made it a treatable disease," the authors conclude. "Dietary therapy started early in life and HSCT have markedly improved the longevity and quality of life for affected people, and new standards for treatment have been established."
 

- July issue of the Archives of Neurology
 

Arch Neurol. 2005;62:1073-1080

 
Subscribe to Genetic Disorders Newsletter
E-mail Address:

 

This study was supported by grants from the Johns Hopkins University School of Medicine General Clinical Research Center, from the National Center for Research Resources/National Institutes of Health, Bethesda, Md., the National Institutes of Health, Bethesda, Md., the Office of Orphan Drug Products of the Food and Drug Administration, Rockville, Md., and the Myelin Project, Dunn Loring, Va.

Related Genetic Disorders News

Williams Syndrome, the brain and music
Exploring genetics of congenital malformations
FDA Approves Idursulfase As First Treatment for Hunter Syndrome
PARP1 inhibitors can protect Huntington's disease affected cells from damage
Gene therapy protects neurons in Huntington's disease
Huntingtin cleavage is caused by caspase-6
The risk of transmission of genetic disorders through donor's sperm
Liver transplants provide metabolic cure for maple syrup urine disease
Spinocerebellar ataxia type 5 (SCA5) gene pinpointed
Scientists probe connection between regulatory DNA and disease


For any corrections of factual information, to contact the editors or to send any medical news or health news press releases, use feedback form

Top of Page

 

© Copyright 2004 onwards by RxPG Medical Solutions Private Limited
Contact Us