Lorenzo’s Oil

Lorenzo's Oil

Lorenzo’s Oil is a combination of a 4:1 mix of oleic acid and erucic acid, extracted from rapeseed oil and olive oil designed to normalize the accumulation of the very long chain fatty acids in the brain thereby slowing the progression of adrenoleukodystrophy (ALD). It is specific to ALD and is NOT designed to be used to treat other leukodystrophies nor multiple sclerosis.  Some US medical insurance companies may provide coverage for the oil, but many do not because it is still considered to be an experimental drug by the Food and Drug Administration (FDA).

If you are the parent of a newly diagnosed ALD patient, click here for important information.

How it Works


The oil, if started early in boys with adrenoleukodystrophy (ALD) showing no symptoms, is now known to have some benefit in preventing the childhood cerebral form of adrenoleukodystrophy. Researchers are still trying to understand the complex relationships among fats in the body, and how Lorenzo’s oil could be further modified to be more effective.  The oil is designed to normalize the accumulation of the very long chain fatty acids in the brain that thereby halting the progression of ALD.

Lorenzo’s Oil is specific to ALD, does not repair myelin, and does not have any known effect on other demyelinating disorders, such as Multiple Sclerosis. In ALD pre-symptomatic boys, however, Lorenzo’s Oil can (but not always) slow down the onset of the disease by stopping the body from producing the very long chain fatty acids, whose buildup leads to demyelination. It is jointly manufactured by Croda International and The Scientific Hospital Supply, and it is distributed by Nutricia Advanced Medical Nutrition. To read an article published by pediatric neurologist Dr. Gerald Raymond about Lorenzo’s Oil and its effect on ALD,  click here.

How to Get It


Lorenzo’s Oil is currently available to patients with ALD for $200.00 USD (+ a $200.00 shipping fee). Given this extremely high cost, The Myelin Project is now offering financial support to assist eligible families in the purchase of the oil. For more information about financial assistance for Lorenzo’s Oil, please contact us.  Shipping of the oil usually takes approximately 7 business days once the documents are received. If you would like to obtain Lorenzo’s Oil, please follow the steps below:

1.  Patient/Family must complete a Patient Request Letter. (Click on the Patient Letter Template below)

Patient Request Letter Template

(Parent name), mother/father of (patient name)

Patient address

 

Date

 

Dear US Customs and FDA Agents,

I wish to import Lorenzo’s Oil for my child, (patient name), for his/her own personal use for a condition he/she has, (condition name). Lorenzo’s Oil is not available in the US which is why I am having to buy it from abroad. I have attached a letter of justification from my child’s physician, which explains why he/she needs this drug, as well as a prescription that the physician has issued for it.

I believe this importation meets the requirements of the guidelines issued by the FDA which can be found at the following address –

https://www.fda.gov/ForIndustry/ImportProgram/ucm173751.htm – and also outlined in the Personal Importation Policy (PIP) document attached. Therefore, the drug and this importation meets the criteria listed below:

  • The drug is unapproved and intended for use in a serious medical condition for which there is no effective treatment domestically available.
  • There will be no commercialization or promotion of the drug in the United States.
  • The drug does not represent an unreasonable health risk to my child as the patient
  • The request is accompanied by an affirmation that the drug is for my child’s use only and I have provided the name and address of my child’s United States-licensed physician responsible for his/her
  • The request is for less than a 3-month supply of the drug. My child needs 40ml of the oil once a day, which is 280ml a week, and a total of 3 bottle for 6 weeks.

I would appreciate if you would exercise your enforcement discretion to permit importation of this drug to help me treat my child’s condition.

Please do not hesitate to contact me should you require any additional clarification either by phone or email.

Yours sincerely,

Signature

Name

Home/office number

Mobile number

Email address

2. Treating Physicians must complete the following: a.) Three-month Prescription b.) the Customs & Border Protection (CBP) Form** c.) a Physician Request Letter (see below). Note: Information on the CBP form must be the physician’s address, SS#, etc. The FDA will NOT allow the oil to be shipped to anyone other than a licensed medical practitioner.

Physician Request Letter Template (New Patient)

Name
Title
Practice name
Practice address
Practice email
Your Email

Date

Dear US Customs and FDA Agents,

I have been treating (patient name) for (condition name) for (X period of time). To date, he/she has been treated with all treatments available in the United States that are known to be effective against (condition name), and none have been fully effective.

(Provide condition-specific information – e.g. X­ ALD is primarily a disorder of the white matter of the nervous system, a disorder of the peroxisomes, and a condition that also often involves deficiencies of the adrenal cortex. Approximately 35% of boys with X-ALD develop the childhood cerebral form, a devastating disease marked by normal early development followed by loss of developmental skills and an early death. Lorenzo’s oil is prescribed to lower the risk of developing cerebral disease. It helps lower very long chain fatty acids in the body, which play a significant role in disease pathogenesis.)

The oil was at one time made available to patients in the US through in the University of Minnesota’s FDA and IRB approved Expanded Access Program. Lorenzo’s Oil is no longer available through this mechanism and Lorenzo’s Oil is no longer available in the US. We are asking that it be imported in order to treat (patient name). Previous research from the Johns Hopkins University showed that Lorenzo’s oil is effective in decreasing the chance of childhood cerebral disease and is safe to use as directed. Please see detailed product information below.

Lorenzo’s Oil Product Information: Lorenzo’s Oil is a mix of oleic acid and erucic acid, extracted from rapeseed oil and olive oil, designed to normalize the accumulation of the very long chain fatty acids in the brain.

Lorenzo’s oil is a combination of glyceryl trierucate (an ester of erucic acid, a 22-carbon monounsaturated fatty acid) and glyceryl trioleate (an ester of oleic acid, an 18-carbon monounsaturated fatty acid), which are generally combined in an approximate ratio of 1:4 (glycerol trierucate:glycerol trioleate) when used for clinical evaluation.

Lorenzo’s Oil is for use in children and adults for the dietary management of X-linked Adrenoleukodystrophy (X-ALD) and Adrenomyeloneuropathy (AMN). Lorenzo’s Oil is usually given in conjunction with a low-fat diet restricting hexacosanoic acid (C26:0).

Lorenzo’s Oil is in liquid form and should be administered orally. The quantity and the dilution administered should be determined by a clinician or a dietitian only. Some treatment protocols suggest that Lorenzo’s Oil should provide 20% of the total calorie intake per day.

The oil should be refrigerated and, once opened, should be used within 1 month. The daily dose should be removed from the bottle every day and stored in a sealed container, in the dark, at room temperature, and should be taken throughout the day as instructed by a clinician or dietitian.

 

This product is produced in the UK for SHS International Ltd. and distributed by Nutricia Advanced Medical Nutrition.

 

SHS International Ltd. address: 100 Wavertree Boulevard Liverpool, United Kingdom

 

Nutricia Advanced Medical Nutrition address: White Horse Business Park,

Newmarket Ave,

Trowbridge BA140XQ, United Kingdom

AES Code-3004 909 140- Central Nervous System

I believe this importation meets the requirements of the guidelines issued by the FDA which can be found at the following address –

https://www.fda.gov/ForIndustry/ImportProgram/ucm173751.htm – and also outlined in the Personal Importation Policy (PIP) document attached. Therefore, the drug and this importation meets the criteria listed below:

 

  • The drug is unapproved and intended for use in a serious medical condition for which there is no effective treatment domestically available.
  • There will be no commercialization or promotion of the drug in the United States.
  • The drug does not represent an unreasonable health risk to the patient
  • The request is accompanied by an affirmation that the drug is for my patient’s use only and we have provided my name and address, as the United States-licensed physician responsible for his/her
  • The request is for less than a 3-month supply of the drug. (Patient name) will need 40ml of the oil once a day, which is 280ml a week, and a total of 3 bottle for 6 weeks.

 

I would appreciate if you would exercise your enforcement discretion to permit importation of this drug to help me treat my patient’s condition.

Please do not hesitate to contact me should you require any additional clarification either by phone or email.

 

Yours sincerely,

Signature

Name

Physician Request Letter Template (Child Patient)

(Parent name), mother/father of (patient name)

Patient address

Date

Dear US Customs and FDA Agents,

I wish to import Lorenzo’s Oil for my child, (patient name), for his/her own personal use for a condition he/she has, (condition name). Lorenzo’s Oil is not available in the US which is why I am having to buy it from abroad. I have attached a letter of justification from my child’s physician, which explains why he/she needs this drug, as well as a prescription that the physician has issued for it.

I believe this importation meets the requirements of the guidelines issued by the FDA which can be found at the following address –

https://www.fda.gov/ForIndustry/ImportProgram/ucm173751.htm – and also outlined in the Personal Importation Policy (PIP) document attached. Therefore, the drug and this importation meets the criteria listed below:

  • The drug is unapproved and intended for use in a serious medical condition for which there is no effective treatment domestically available.
  • There will be no commercialization or promotion of the drug in the United States.
  • The drug does not represent an unreasonable health risk to my child as the patient
  • The request is accompanied by an affirmation that the drug is for my child’s use only and I have provided the name and address of my child’s United States-licensed physician responsible for his/her
  • The request is for less than a 3-month supply of the drug. My child needs 40ml of the oil once a day, which is 280ml a week, and a total of 3 bottle for 6 weeks.

I would appreciate if you would exercise your enforcement discretion to permit importation of this drug to help me treat my child’s condition.

Please do not hesitate to contact me should you require any additional clarification either by phone or email.

Yours sincerely,

Signature
Name
Home/office number
Mobile number
Email address

Physician Request Letter Template (IND Patient)

Name
Title
Practice name
Practice address
Practice email
Your Email

Date

Dear US Customs and FDA Agents,

I have been treating (patient name) for (condition name) for (X period of time). To date, he/she has been treated with all treatments available in the United States that are known to be effective against (condition name), and none have been fully effective.

(Provide condition-specific information e.g. X­ ALD is primarily a disorder of the white matter of the nervous system, a disorder of the peroxisomes,  and a condition that also often involves deficiencies  of the adrenal cortex. Approximately 35% of boys with X-ALD develop the childhood cerebral form, a devastating disease marked by normal early development followed by loss of developmental  skills and an early death. Lorenzo’s oil is prescribed to lower the risk of developing cerebral disease. It helps lower very long chain fatty acids in the body, which play a significant role in disease pathogenesis.)

(Patient name) has been taking Lorenzo’s oil since (month, year) and has done very well. (Patient name) was previously enrolled in the University of Minnesota’s FDA and IRB approved Expanded Access Program. Lorenzo’s Oil is no longer available through this mechanism and Lorenzo’s oil is no longer available in the US. We are asking that it be imported in order to treat (patient name). Previous research from the Johns Hopkins University showed that Lorenzo’s oil is effective in decreasing the chance of childhood cerebral disease and is safe to use as directed. Please see detailed product information below.

Lorenzo’s Oil Product Information:

Lorenzo’s Oil is a mix of oleic acid and erucic acid, extracted from rapeseed oil and olive oil, designed to normalize the accumulation of the very long chain fatty acids in the brain.

Lorenzo’s oil is a combination of glyceryl trierucate (an ester of erucic acid, a 22-carbon monounsaturated fatty acid) and glyceryl trioleate (an ester of oleic acid, an 18-carbon monounsaturated fatty acid), which are generally combined in an approximate ratio of 1:4 (glycerol trierucate:glycerol trioleate) when used for clinical evaluation.

Lorenzo’s Oil is for use in children and adults for the dietary management of X-linked Adrenoleukodystrophy (X-ALD) and Adrenomyeloneuropathy (AMN). Lorenzo’s Oil is usually given in conjunction with a low-fat diet restricting hexacosanoic acid (C26:0).

Lorenzo’s Oil is in liquid form and should be administered orally. The quantity and the dilution administered should be determined by a clinician or a dietitian only. Some treatment protocols suggest that Lorenzo’s Oil should provide 20% of the total calorie intake per day.

The oil should be refrigerated and, once opened, should be used within 1 month. The daily dose should be removed from the bottle every day and stored in a sealed container, in the dark, at room temperature, and should be taken throughout the day as instructed by a clinician or dietitian.

This product is produced in the UK for SHS International Ltd. and distributed by Nutricia Advanced Medical Nutrition.

SHS International Ltd. address: 100 Wavertree Boulevard Liverpool, United Kingdom
Nutricia Advanced Medical Nutrition address: White Horse Business Park,
Newmarket Ave,
Trowbridge BA140XQ, United Kingdom

AES Code-3004 909 140- Central Nervous System

I believe this importation meets the requirements of the guidelines issued by the FDA which can be found at the following address –

https://www.fda.gov/ForIndustry/ImportProgram/ucm173751.htm – and also outlined in the Personal Importation Policy (PIP) document attached. Therefore, the drug and this importation meets the criteria listed below:

  • The drug is unapproved and intended for use in a serious medical condition for which there is no effective treatment domestically available.
  • There will be no commercialization or promotion of the drug in the United States.
  • The drug does not represent an unreasonable health risk to the patient
  • The request is accompanied by an affirmation that the drug is for my patient’s use only and we have provided my name and address, as the United States-licensed physician responsible for his/her
  • The request is for less than a 3-month supply of the drug. (Patient name) will need 40ml of the oil once a day, which is 280ml a week, and a total of 3 bottle for 6 weeks.

I would appreciate if you would exercise your enforcement discretion to permit importation of this drug to help me treat my patient’s condition.

Please do not hesitate to contact me should you require any additional clarification either by phone or email.

Yours sincerely,

Signature

Name

3.  Please email your completed documents to Max Goodman at mgoodman@wepclinical.com. For more information, please contact us.

History


Failing to find a doctor capable of treating their young son Lorenzo’s rare disease, Augusto and Michaela Odone, founders of The Myelin Project, sought their own cure. They set out on a mission to find a treatment to save their child. In their quest, the Odones clashed with doctors, scientists, and support groups, who were skeptical that anything could be done about Adrenoleukodystrophy (ALD), much less by laypeople. But they persisted, setting up camp in medical libraries, reviewing animal experiments, badgering researchers, questioning top doctors all over the world, and even organizing an international symposium about the disease.

Despite research dead ends, the horror of watching their son’s health decline, and being surrounded by skeptics (including the coordinators of the support group they attended), they persisted until they finally hit upon a therapy involving adding a certain kind of oil (actually containing two specific long chain fatty acids, isolated from rapeseed [canola] oil and olive oil) to their son’s diet.

They contacted over 100 firms around the world until they found an elderly British chemist (Don Suddaby) working for Croda International who was willing to take on the challenge of distilling the proper formula. It proved successful in normalizing the accumulation of the very long chain fatty acids in the brain that had been causing their son’s steady decline, thereby halting the progression of the disease. There was still a great deal of neurological damage remaining which could not be reversed until new treatments were found to regenerate the myelin sheath (a lipid insulator) around the nerves.

For information about ALD diagnosis and treatment options, please contact the Kennedy Krieger Institute or call toll-free (844) 334-3211. To contact International Care Management Coordinators, please call (888) 554-2080 or e-mail international@kennedykrieger.org.