The Myelin Project

Executive Directors Message

Mon, 21 Jul 2008 21:08:22 GMT

July 17, 2008

Dear Friends:

Augusto wanted me to thank each of you for your cards and letters and kind words of sympathy and encouragement sent to him after Lorenzo's passing. He wishes he could respond to each and every message but that is not possibe due to his health problems. Augusto is doing as well as can be expected and is back to work on his book and hopes that each of you will purchase a copy when it is published.

The 2008 Got the Nerve Triathlon was a huge success. Chris Kaag did a fabulous job of organizing 85 colunteers and 701 participants. Mt. Gretna Lake in Pennsylania is absolutely beautiful and we want to thank them for allowing us to have this event there each year. Chris advised me that he anticipates that this year's event raised $35,000 for The Myelin Project. We cannot begin to tell you how thankful we are for Chris, his family, friends and sponsors for supporting our cause. I want to assure each and every one of you that we will spend this money wisely.

I want you also to know that Chris Kaag has started a new foundation it is called The IM ABLE Foundation and his mission is to enrich the lives of the disabled through raising awareness about physical fitness possibilities for disabled people and encouraging and enabling disabled children to be more active. Please visit the website http://www.im-able.org/

Candace Root

Executive Director

LORENZO ODONE

Mon, 21 Jul 2008 13:38:28 GMT

In Loving Memory of Lorenzo Odone 5/29/1978 to 5/30/2008 It is with great sadness that we announce that Lorenzo Odone finally lost his battle with adrenoleukodystrophy and went to heaven to be with the angels on May 30, 2008. Both his father Augusto and his life-long friend Oumouri Hassane were at his side when he passed away. Lorenzo died one day after his 30th birthday. Lorenzo Odone, whose story was told in the 1992 Hollywood movie Lorenzos Oil, starring Susan Sarandon and Nick Nolte, has died in Fairfax, VA, one day after his 30th birthday. Lorenzo was diagnosed with Adrenoleukodystrophy (ALD) in 1984, aged 6. Doctors told his parents that the neurological disease would swiftly deprive him of all his faculties and lead to his death within a maximum of two years. Odones parents, Augusto and Michaela, despite having no scientific background, decided to research the rare genetic disorder. Their struggle and ultimate triumph, when they found an oil that stopped the...

OTHER WAYS TO DONATE

Thu, 17 Jul 2008 18:54:12 GMT

Ways to Donate at Work:

Combined Federal Campaign –

The Myelin Project is a member organization of the Combined Federal Campaign.   Their CFC number is 11222. Federal employees will find the Myelin Project under the Health and Medical Research Charities Section

Independent Charities of America and Local Independent Charities

The Myelin Project is a member organization of Independent Charities of America and participates in numerous state, city and county charitable campaigns.

United Way

The Myelin Project is a proud participate in the United Way corporate campaigns   If your local United Way Campaign does not list The Myelin Project please write it in
The Myelin Project @ 1400 Wallace, Suite 258, Amarillo, TX 79106

Matching Gift Campaigns

Please check with your employer about matching gifts. Many employers will match your personal donation.

V-DAC

Vehicle Donations to Any Charity

116 Washington Street, Suite E

Pt. Richmond, CA 948801

877-999-8322

Here are some other ways that you can donate to The Myelin Project

Good Search

US Recycling

Donate online:

You may use your credit card to donate on-line http://www.myelin.org/en/donations/add.asp

Our web site is quick, easy and a secure server

By telephone:

You may donate by telephone by calling our toll-free number 800-769-3546

By mail:

You may mail your donation to

1400 Wallace, Suite 258

Amarillo, TX 79106

MESSAGE FROM THE EXECUTIVE DIRECTOR

Thu, 17 Jul 2008 17:16:32 GMT

Lorenzo Odone

Rest in Peace

May 29, 1978 to May 30, 2008

LETTER FROM THE PRESIDENT

Mon, 30 Jun 2008 15:39:43 GMT

March 6, 2008 Dear Friends of The Myelin Project: When the Myelin Project was founded by Augusto and Michaela Odone, the little was known about ALD. Now, twenty years later, we not only know a great deal about the disease, we have treatments that, however imperfect, offer hope of survival for boys who carry the ALD gene. As published in 2005, Lorenzo’s Oil significantly reduces the risk of childhood cerebral ALD in boys carrying the ALD gene. Studies are now underway to discover whether Lorenzo’s oil will delay the onset and progression of adrenomyeloneuropathy in men and women who carry the ALD gene. There is also hope for those boys who carry the gene and develop symptoms of childhood cerebral ALD. When bone marrow transplant was first tried, the results were often very disappointing. Today, though, with advances in matching donors and recipient, and better pre- and post-operative therapies, the...

NEWS

Mon, 30 Jun 2008 15:37:51 GMT

IT IS WITH GREAT SADNESS THAT WE ANNOUNCE THAT LORENZO ODONE PASSED AWAY MAY 30, 2008, IN HIS SLEEP, AT HOME IN FAIRFAX, VA. WITH BOTH HIS FATHER, AUGUSTO AND HIS LIFE-LONG FRIEND, OUMOURI HASSANE, AT HIS SIDE.

IF YOU WOULD LIKE TO LEAVE A MESSAGE FOR AUGUSTO YOU MAY DO SO HERE
http://www.myelin.org/en/testimonials/add.asp

IF YOU WOULD LIKE TO MAKE A DONATION IN MEMORY OF LORENZO YOU MAY DO SO HERE

http://www.myelin.org/en/donations/add.asp

Often, we get requests from families for information about bone marrow or cord blood transplants for children with one of the leukodystrophies. I have asked Dr. Charles Peters, of Children’s Mercy Hospital in Kansas City to write a brief article about this of therapy. It is used for adrenoleukodystrophy (ALD), Krabbe Disease (globoid cell leukodystrophy, GLD)) and metachromatic leukodystrophy (MLD). The success rate is encouraging, particularly for ALD and GLD. In both cases, though, success depends on early detection of the disease. This underscores the vital importance of newborn screening. Dr. Peters' article may be found at

http://www.myelin.org/en/cms/?399

DEVELOPMENT

Mon, 30 Jun 2008 15:31:20 GMT

Dear Friends of The Myelin Project: Please allow me to introduce myself. My name is Trish Knight, and I am the newest member of The Myelin Project Executive Staff. I began serving as Director of Development on April 7 of this year, and I must say it is an honor and a privilege to be here! To give you a little bit of history: I am the Founder, President, and Director of The Stennis Foundation. The Stennis Foundation is a 501(c)(3) nonprofit organization, committed to raising awareness of the Leukodystrophies and to raising funds for Leukodystrophies research. I began The Stennis Foundation after my friend, Dr. Sam Stennis, was diagnosed with adult-onset Metachromatic Leukodystrophy (MLD), and nothing was being done to help him, because the Leukodystrophies were such rare diseases. When Dr. Stennis was diagnosed, I began doing my own research, and came across a disease similar to MLD, called Adrenoleukodystrophy (ALD). I also came across a movie called,...

ALEXANDERS DISEASE

Sun, 15 Jun 2008 15:13:35 GMT

ALEXANDER’S DISEASE Margaret Weis, Ph.D. President, The Myelin Project Professor of Pharmaceutical Sciences Texas Tech University Health Sciences Center School of Pharmcy BACKGROUND: Alexander’s disease is characterized by Rosenthal fibers forming in the astrocytes of the brain. Although astrocytes are not nerve cells (neurons), they help regulate the passage of materials from the blood to the neurons of the brain, and may regulate levels of certain important brain chemicals. Alexander’s disease is caused by mutations in the gene coding for glial fibrillary acidic protein (GFAP) important in the function of the astrocytes. It is easy to think of proteins as a long string that must be folded in order to function. It is also easy to see that a long string can be folded in many different ways. Unless the protein is folded in exactly the correct way, it can’t function. Chaperones are in every cell to help proteins fold correctly. The mutation in Alexander’s disease creates a GFAP...

Hematopoietic Cell Transplantation (HCT) for the Leukodystrophies: Cerebral X-linked Adrenoleukodystrophy (CALD), Globoid-Cell Leukodystrophy (GLD, Krabbe) and Metachromatic Leukodystrophy (MLD)

Sat, 14 Jun 2008 01:33:18 GMT

Charles Peters, MD X-Linked Adrenoleukodystrophy (X-ALD) ALD is an X-linked disorder of very-long-chain fatty acid (VLCFA) metabolism. The childhood form usually starts at about 7 years. About 90% of these boys also have Addison’s disease. The posterior (back) part of the brain is the most common site for demyelination. Neurologic deterioration occurs in all boys. Deficits can include vision/visual processing, hearing/auditory processing, speech, gait, fine motor skills, and activities of daily living. Disability, dementia, death can occur within months to several years after clinical onset. Adult onset adrenomyeloneuropathy is usually diagnosed because of spinal cord problems seen in the 2nd or 3rd decades. Of note, men, like boys, can also develop cerebral disease. A complete evaluation for cerebral X-ALD includes: 1) neurologic examination; 2) neuropsychological assessment; 3) brain MRI. Boys diagnosed with X-ALD due to a family history should have serial monitoring every 6...

AUGUSTO ODONE

Thu, 05 Jun 2008 18:30:42 GMT

I am Augusto Odone, Lorenzo's father and the founder of the Myelin Project. Lorenzo will be celebrating his 30th birthday on May 29, this year. I had promised him to write a book about his story to mark the event. The idea of this book came to me after receiving thousands of letters from high school students and their teachers. Typically these letters were from children who had seen the movie "Lorenzo's Oil," directed by George Miller ("Mad Max," "Happy Feet"). They wanted to know how Lorenzo's parents, unfamiliar with medicine, had discovered it. And, of course, they wanted to know how Lorenzo was doing. The book is now almost completed and will become available next fall. It comes with several pictures illustrating the adventures of Lorenzo in the Comoros Islands, in Kenya and in the States. Anyone interested in the book may inquire about it by emailing me at augusto.odone@gmail.com

LORENZO ODONE PASSED AWAY TODAY IN HIS SLEEP AT HOME.

Sat, 31 May 2008 02:30:01 GMT

It is with great sadness that we announce that Loreno Odone lost his battle with adrenoleukodsytrophy and passed away today in his sleep at home in Fairfax, VA with his father Augusto Odone and his life-long friend Omouri Hassane at his side.

Press Release
Fairfax VA. 5/30/08

Lorenzo Odone, whose story was told in the 1992 Hollywood movie "Lorenzo's Oil", starring Susan Sarandon and Nick Nolte, has died in Fairfax, VA, one day after his 30th birthday. Lorenzo was diagnosed with Adrenoleukodystrophy (ALD) in 1984, aged 6. Doctors told his parents that the neurological disease would swiftly deprive him of all his faculties and lead to his death withing a maximum of two years.

Odone's parents, Augusto and Michaela, despite having no scientific background, decided to research the rare genetic disorder. Their struggle and ultimate triumph, when they found an oil that stopped the disease in its tracks and, in pre-symptomatic boys, prevented the onslaught of ALD, captivated director George Miller, who went on to make 'Happy Feet' and 'The Witches of Eastwick'. He turned the true life story into a film which won Susan Sarandon an Oscar nomination as Best Actress. (Sarandon insisted on bringing Michaela Odone with her to the Oscar ceremonies.)

The film met with criticism from the medical community, who condemned it for setting up false hopes in families of ALD sufferers. The Odones however were amply vindicated by a study published two years ago, which showed that Lorenzo's Oil did indeed stop ALD in pre-symptomatic boys.

The Odones also founded the Myelin Project, a mould-breaking medical research charity which as well as providing leadership in the search for a cure for ALD< also enabled families affected by the disease to meet directly with scientists working in the field "Scientists should never forget that their mission is to relieve human suffering rather than win the Nobel prize" said Augusto Odone when founding the project.

In 1996, Phil Collins wrote the song "Lorenzo", based on lyrics written by Michaela.

Bedridden and unable to communicate from his seventh year, Lorenzo was kept at home by his parents. Nurses cared for him round the clock, as did his indefatigable parents. Lorenzo outlived his mother, who died of lung cancer in 2002. He is survived by his father, Augusto, his brother Francesco and sister and Cristina.

New Class of Drugs For Multiple Sclerosis

Wed, 21 May 2008 15:17:53 GMT

I have placed this abstract on our website because it summarizes information on fingolimod. This is the first member of what may be a new class of drugs for the treatment of multiple sclerosis. Without becoming overly technical, sphingosine-1-phosphate, or S1P, is a type of lipid that seems to be important in immune signaling. By binding to specific target molecules (receptors), S1P helps to regulate the immune system. Fingolimod acts at S1P receptors and may prevent immune-system damage in the central nervous system. The drug has progressed to Phase III clinical trials, meaning that it met expectations for safety (Phase I) and showed promise of efficacy in a small group of patients (Phase II). The Phase III trial will test the drug in a larger group of patients, and will take some time to complete. We will all watch this trial with great interest. Prog Drug Res. 2008;66:361, 363-81. Ascomycete derivative to MS therapeutic: S1P receptor modulator FTY720. Hiestand PC, Rausch M,...

HAPPY 30TH BIRTHDAY LORENZO

Mon, 19 May 2008 15:17:02 GMT

Happy Birthday, Lorenzo! To some, a young man turning 30 years old might not be such a big deal. But for Lorenzo Odone, this is a tremendous milestone. The first five and a half years of his life, Lorenzo was an active boy who spoke three languages. He had lived in the Comoros Islands in East Africa for three years with his parents Augusto and Michaela. When Lorenzo was five, his teachers noticed behavioral changes and spoke with Augusto and Michaela about them. It was not long until Lorenzo was diagnosed with Adrenoleukodystrophy (ALD) in 1984. Adrenoleukodystrophy (ALD) is one of a group of genetic disorders called the leukodystrophies that cause damage to the myelin sheath, an insulating membrane that surrounds nerve cells in the brain. People with ALD accumulate high levels of saturated, very long chain fatty acids (VLCFA) in the brain and adrenal cortex because they do not produce the enzyme that breaks down these fatty acids in the normal manner. The loss of...

BEST IN AMERICA CERTIFIED CHARITY

Wed, 14 May 2008 17:27:49 GMT

Learn More

The Independent Charities Seal of Excellence is awarded to the members of Independent Charities of America and Local Independent Charities of America that have, upon rigorous independent review, been able to certify, document, and demonstrate on an annual basis that they meet the highest standards of public accountability, program effectiveness, and cost effectiveness.
These standards include those required by the US Government for inclusion in the Combined Federal Campaign, probably the most exclusive fund drive in the world.
Of the 1,000,000 charities operating in the United States today, it is estimated that fewer than 50,000, or 5 percent, meet or exceed these standards, and, of those, fewer than 2,000 have been awarded this Seal.

Northwood School

Tue, 13 May 2008 22:14:57 GMT

NEWS 10 NOW Students raise money for myelin disease research 05/10/2008 06:37 PM By: Katie Morse LAKE PLACID, N.Y. -- Students at Northwood School spent their Saturday afternoon raising money for a disease many people have never even heard of. Picture electrical cord. The rubber thats on the cord insulates those wires, well the myelin sheath insulates all our nerves in our body, said Karen Fleming, Northwoods school nurse. People with a damaged myelin may develop multiple sclerosis or other myelin diseases The students raised money for disease research, and held a tennis tournament, concert and three-mile walk. We get really into activities like this, and really do our part in charity work and hosting projects like this, said Emily Simmons, Student Council Presidet. Students raise money for myelin disease research High schoolers in Lake Placid spent their weekend raising money for a good cause. The students were out in the community to...

Glossary

Mon, 12 May 2008 23:19:13 GMT

A Adrenoleukodystrophy (ALD): A rapidly progressive X-linked genetic disorder marked by the accumulation of saturated very long chain fatty acids (VLCFA) in all tissues and body fluids, with a preference for the brain and the adrenal glands. This buildup leads to inflammation of the brain and erosion of the white matter of the central nervous system and the adrenal glands. It affects only boys; while there is a neonatal form, it typically strikes between the ages of 6 and 10. Adrenomyeloneuropathy (AMN): The adult form of ALD, milder and more slowly progressive than the childhood form; it affects the spinal cord and peripheral nervous system. Arylsulfatase: The lack of this enzyme results in the toxic accumulation of sulfatide deposits in metachromatic leukodystrophy, leading to demyelination of the CNS. Astrocyte: See Glia. Asymptomatic: Having a disease without manifestation of symptoms. Axon: Myelin-ensheathed tail of the neuron that is responsible for carrying...

Outreach Letter to ALD/ALM Families

Thu, 08 May 2008 21:22:28 GMT

Dear ALD/AMN Families: I am writing to let you know about a study of the treatment of adrenomyeloneuropathy (AMN) which is now in progress at the Kennedy Krieger Institute and Johns Hopkins Hospital, both located in Baltimore, Maryland. Adult males and females with AMN who experience symptoms but who are, at minimum, able to walk 20 yards with crutches, canes, or a brace are eligible to enroll. The primary purpose of the study is to determine whether Lorenzo’s Oil (a 4:1 mixture of glyceryl trioleate and glyceryl trierucate) helps patients with AMN. This part of the study is placebo-controlled, that is, half of the patients receive Lorenzos Oil, while the other half receives a placebo oil. The other part of the study is an individualized physical therapy program, customized to address each patient’s needs based upon a detailed analysis of their muscle strength, degree of spasticity, sensation, balance and gait. This comprehensive analysis is conducted using state-of-the-art...

GOOD NEWS FOR MLD PATIENTS

Thu, 08 May 2008 21:16:26 GMT

There is some very good news today for metachromatic leukodystrophy (MLD) patients and their families. The U.S.. Food and Drug Administration (FDA) has granted Orphan Drug status to Zymenex for Metazym, and approved the start of Phase II Clinical Trials. This step forward is particularly good news for The Myein Project. Through the British Trust for The Myelin Project, our branch in the United Kingdom, The Myelin Project has helped to fund development of this MLD treatment. Metazym is a preparation of arylsulfatase A, the enzyme that is not working correctly in MLD. By giving patients the enzyme, it is hoped that further destruction of myelin will be stopped. I would like to extend my sincere thanks to our generous donors whose contributions helped make this step possible. What is an Orphan Drug? An orphan drug is one that is developed for the treatment of a rare disease; that is, a disease affecting fewer than 200,000 persons. The U.S. Congress passed the Orphan Drug Act in...

How Can I Get Lorenzo's Oil?

Wed, 07 May 2008 20:58:19 GMT

In the U.S., Lorenzo's Oil is currently only available to patients taking part in a clinical trial under the direction of Dr. Gerald Raymond of the Kennedy Krieger Institute. He can be reached at (800) 873-3377. Dr. Raymond has assumed the work of the late Dr. Hugo Moser. We are working with the FDA to obtain approval of Lorenzo's Oil as a drug, so that it can be widely available.

In the U.S., Lorenzo's Oil can only be obtained through prescription by Kennedy Krieger authorized physicians. A 500ml bottle costs approximately $56.00. Some insurance companies will provide coverage for the oil, but others do not because it is still considered an experimental drug by the FDA.

The oil is jointly manufactured by Croda International of Britain and SHS International (Scientific Hospital Supply.

If you live outside the United States, please see Lorenzo's Oil International

Scientific Advisory Panel

Wed, 07 May 2008 20:08:59 GMT

Ian Duncan, PhD Chair of Scientific Advisory Panel Professor, Neurology Department of Medical Sciences School of Veterinary Medicine University of Wisconsin-Madison 2015 Linden Drive West Madison, WI 53706-1102 USA Email Duncan@svm.vetmed.wisc.edu Dr. Annik Baron-Van Evercooren Director, Unit on Disorders of Myelin and Muscle Ion Channels Faculté Pitié-Salpêtrière Laboratoire des Pathologies de la Myéline INSERM CJF 97-11 105 boulevard de l’Hôpital 75634 Paris Cedex 13 FRANCE William Blakemore< Ph.D. Professor of Neuropathology University of Cambridge Department of Clinical Veterinary Medicine Madingley Road Cambridge CB3 0ES ENGLAND Wolfgang Bruck, M.D. Professor of Neuropathology Institut fur Neuropathologie Universitatsmedizin Gottingen Robert-Koch-Strabe 40 37075 Gottingen GERMANY Robin J.M. Franklin, PhD Professor of Neuroscience Cambridge Centre for Brain Repair & Centre for Veterinary Sciences ...