Frequently Asked Questions

Frequently Asked Questions

GENERAL QUESTIONS

WHAT IS THE MYELIN PROJECT?


We are a 501(c)3 non-profit organization focused on improving the quality of life for those living with ALD and AMN through research,advocacy and family support. We believe we can accomplish our mission by pursuing the following initiatives to: fund medical research to advance treatment and to find a cure, raise awareness, promote education, & advocate for newborn screening of ALD, and to provide financial support to low-income ALD families in need.

The Myelin Project was established in 1989 by Augusto and Michaela Odone for their son Lorenzo who suffered from adrenoleukodystrophy (ALD), a rare genetic myelin disease that deteriorates myelin in the brain and central nervous system. Although not medical doctors, the Odones developed a treatment,“Lorenzo’s Oil” now adopted throughout the world. The story of the Odones’ struggle against ALD was dramatized in the 1992 Universal Studios release “Lorenzo’s Oil” starring Nick Nolte and Susan Sarandon.

WHAT IS ALD?


Adrenoleukodystrophy (ALD) is an x-linked metabolic disorder, characterized by progressive neurological deterioration due to demyelination of the cerebral white matter. Brain function declines as the protective myelin sheath is gradually stripped from the brain’s nerve cells. Without that sheath, the neurons cannot conduct action potentials—in other words, they stop telling the muscles and other elements of the central nervous system what to do. This sequence of events appears to be related to an abnormal accumulation of saturated very-long-chain fatty acids (VLCFAs) in the serum and tissues of the central nervous system, which sets off an abnormal immune response that leads to demyelination. It is unclear exactly how this chain of events works, but scientists do know that it has its roots in genetics. Learn more.

WHAT IS AMN?


Adrenomyeloneuropathy (AMN) is a genetic neuro-degenerative disease. It is the adult onset of adrenoleukodystrophy (ALD). In about half of the boys who inherit the mutated ALD gene, symptoms of the disease do not develop until young adulthood, and in general, they progress more slowly. Beginning in their 20s and 30s, these young men exhibit neurological based motor lesions in their extremities. These lesions progress over many years and are inevitably accompanied by moderate to severe handicap. In approximately one third of AMN patients, the central nervous system can also become involved. These AMN patients undergo the same mental and physical deterioration as boys with the childhood form of the disease, adrenoleukodystrophy (ALD). The progression of the disease is slower, usually declining to a vegetative state and/or death.

WHAT IS MYELIN?


Myelin is an insulating layer, or sheath, that forms around nerves, including those in the brain and spinal cord. It is made up of protein and fatty substances. The purpose of the myelin sheath is to allow electrical impulses to transmit quickly and efficiently along the nerve cells. If myelin is damaged, the impulses slow down which is what occurs in adrenoleukodystrophy (ALD).

LORENZO'S OIL QUESTIONS

WHAT IS LORENZO'S OIL?


Lorenzo’s Oil is a combination of a 4:1 mix of euric acid and oleic acid, extracted from rapeseed oil and olive oil.  It used in the treatment of pre-symptomatic patients with ALD.  The oil was formulated by Augusto and Michaela Odone after their son Lorenzo was diagnosed withe disease in 1984, at the age of five. Lorenzo’s Oil is specific to ALD, does not repair myelin, and does not have any known effect on other demyelinating disorders. In ALD pre-symptomatic boys, however, Lorenzo’s Oil often (but not always) prevents the onset of the disease by stopping the body from producing the very long chain fatty acids, whose buildup leads to demyelination.

HOW DOES IT WORK?


In ALD pre-symptomatic boys, Lorenzo’s Oil can slow down the onset of the disease by stopping the body from producing the very long chain fatty acids, whose buildup leads to demyelination.

HOW CAN I GET IT?


Lorenzo’s Oil is currently available to patients with adrenoleukodystrophy (ALD) aged 18 months to 18 years that meet the criteria: normal MRI, and positive very long chain fatty acids (VLCFA)s. To obtain Lorenzo’s Oil, please contact: Dr. Gerald Raymond gvraymon@umn.edu (612) 625-5859 at the University of Minnesota, or Dr. Ali Fatemi fatemi@kennedykrieger.org (443) 923-2750 at the Kennedy Krieger Institute.


 

  • Lorenzo’s Oil is specific to ALD and is NOT designed to be used to treat other leukodystrophies nor Multiple Sclerosis.
  • Initial  start and consent to use the oil needs to go through Dr. Gerald Raymond (see contact information).
  • Some insurance companies will provide coverage for the oil, but others do not because it is still considered to be an experimental drug by the Food and Drug Administration (FDA).
  • The Johns Hopkins University Study of Lorenzo’s Oil’s effect on ALD will cease evaluations as of November 1st, 2014.

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CAN I USE THE OIL FOR MS?


Unfortunately, no. There is no evidence that Lorenzo’s Oil has any affect on any other demyelinating diseases.  Lorenzo’s Oil is specific to ALD, does not repair myelin, and does not have any known effect on other demyelinating disorders.  For information on reliable MS treatment options, please click here to be directed to The National Multiple Sclerosis Society website.

ADDITIONAL RESOURCES

SITES THAT DISCUSS ADRENOLEUKODYSTROPHY

ALD Connect
The ALD Foundation
ALD Life
The Myelin Project
Oliver’s Army
Orpha.Net — European Rare Diseases Organization and Their Discussion on ALD and many great links & resources
The United Leukodystrophy Foundation
US ClinTrial.gov — Listing of ongoing US ALD clinical trials
WikiPedia on ALD
X-Linked ALD Database
FightALD.org


FOR PHYSICIANS, SCIENTISTS & HEALTHCARE WORKERS

Avoiding the Misdiagnosis of Adrenoleukodystrophy: Distinguishing ALD from ADD/ADHD


STEM CELL (CORD BLOOD / BONE MARROW) TRANSPLANTS RESOURCES

The Blood and Marrow Transplant Information Network
Bone Marrow Donors Worldwide
Duke University’s Pediatric Stem Cell Transplant Program
National Marrow Donor Program
University of Minnesota: Fairview University
The Cord Blood Banking Center – A service to help expecting mothers donate their cord blood or bank for their own purposes.
Related Donor Cord Blood Program.  For families impacted by a life-threatening disease where a cord blood transplant could be a cure, the cord blood from a new family baby can be collected and stored at no cost to the family and then may be used to treat an affected biological sibling or parent who has a diagnosed disease.