The Myelin Project

MESSAGE FROM THE EXECUTIVE DIRECTOR

Fri, 09 May 2008 15:18:01 GMT

Dear Myelin Project Supporters: Thank you for the tremendous response to our new web site. We hope to be able to serve you better and more efficiently with our new web site and welcome your comments and input. We are currently developing a medical/science section of our web site to be used as a reference tool for health care professionals. I would like to take this opportuntity to welcome our new Development Director, Trish Knight. Trish is the founder and President of the Stennis Foundation, a non-profit foundation that raises money to fund leukodsytrophy research. Trish will be developing national fund raising and will also be working with our leukodsytrophy families. If you have a family member with leukodystrophy please contact her at trish.knight@myelin.org She would love to hear from you. She is also compiling a data base of leukodsytrophy patients so please contact her so that she can include you in the data base. If you want to host a fund raiser for The...

Fri, 09 May 2008 15:10:13 GMT

For an update on recent scientific and medical news please see Articles.

Photos of recent events, and find out what's happening in the Myelin Project world.

Photographs of the Hammerfest Triathlon were taken by Kate Berges and were used with permission www.kateberges.com

Photographs of the 12 in 24 Jump for a Cause were taken by Michelle Dobson, our fabulous lone female sky diver.

LETTER FROM THE PRESIDENT

Thu, 08 May 2008 21:42:11 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 success rate is...

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...

DEVELOPMENT

Thu, 08 May 2008 14:08:37 GMT

It’s All About Development

Trish Knight
Director of Development

Development is a new area within The Myelin Project, and this new area is in exactly that stage: the development stage. We are planning our strategies as to how we can further the research of demyelinating diseases, including the Leukodystrophies and Multiple Sclerosis; as

well as bring about the implementation of Newborn Screening for Adrenoleukodystrophy (ALD). For boys who are diagnosed with ALD, treatment with Lorenzo’s Oil and a bone marrow

transplant could slow the progression of the disease. But ultimately, our goal at The Myelin Project is for these diseases to be determined at birth through Universal Newborn Screening. The development of these tests is quite costly; and federal funding is limited. Donations are needed to continue developing these tests, so that children who are diagnosed with ALD or other demyelinating disorders are given a chance at life. It’s all about development.

email Trish.Knight@myelin.org

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 ...

Lorenzos Oil International

Wed, 07 May 2008 19:44:16 GMT

Latin America Alex Gooding email ahgooding@sbinshs.com.ar Tel +54 11 493 24816 Far East Okke Suurenbroek email okkesurrenbroek@nutriciachina.net Tel +8621 5351 6858 Middle East/Africa/India Rosenda Nair email NRose@nutricia.com.ae Tel +971 4 8056736 Argentina ABIN S.A. Cochabamba 3641 1252 Buenos Aires Argentina Tel: +54 11 495 76764 Fax: +54 11 481 36697 Australia Nutricia Australia Pty Ltd P.O. Box 1007 North Ryde Business Centre NSW 1670 Talavera Corporate Centre Level 4, Building D 12-18 Talavera Rd Macquarie Park NSW 2113 Tel: +61 2 8870 0400 Fax: +61 2 9878 4725 Austria Nutricia Nahrungsmittel GmbH & Co.Kg Jochen-Rindt-Strasse 37 A-1230 Vienna Tel: +43 - 1 - 6882626 Fax: +43 - 1 - 6882626 Belgium N.V. Nutricia België Rijksweg 64 B-2880 Bornem Tel: +32 - 3 - 890 22 11 Fax: +32 - 3 - 890 22 12 Brazil Support Products Nutricionais Ltda. Rua Gomes de Carvalho, 1510 - 18° Andar Vila Olimpia São, Paulo, SP Tel: 55 (11)...

Pelizaeus-Merzbacher Disease

Wed, 07 May 2008 16:59:19 GMT

Application of a new progesterone antagonist to reduce PLP overexpression in a mouse model of Pelizaeus-Merzbacher Disease by Klaus-Armin Nave, PhD, Hauke Werner, PhD Max-Planck Institute of Experimental Medicine, Goettingen, Germany Pelizaeus-Merzbacher disease (PMD) is a severe leukodystrophy defined by mutations of the X chromosome-linked gene for proteolipid protein (PLP), the major myelin protein of the central nervous system (CNS). The majority of patients suffer from a PLP-gene duplication and, in consequence, PLP overexpression that is not tolerated by myelin-producing oligodendrocytes. We are testing the hypothesis that PLP overexpression can be pharmacologically lowered to improve the disease course. Although a detailed knowledge of PLP gene regulation in vivo is still lacking, there is indirect evidence to suggest that the steroid hormone progesterone stimulates the PLP gene in oligodendrocytes, similar to the PMP22 gene in Schwann cells (Sereda et al, 2003)....

Glossary

Wed, 07 May 2008 16:35:13 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...

NEWBORN SCREENING TEST

Wed, 07 May 2008 16:01:33 GMT

Dr. Gerald Raymond Reports on Newborn Screening for X-linked Adrenoleukodystrophy and Other Peroxisomal Disorders X-linked adrenoleukodystrophy (ALD) is a genetic disorder occurring in 1 in 17,000 newborns. All ethnic groups are affected about equally. The disease causes a progressive loss of brain white matter and loss of adrenal gland function in boys who have the gene. It strikes previously healthy boys beginning school or young men just starting out in life. Over a third of boys with the biochemical abnormality will show symptoms in childhood. Without treatment, they will be severely disabled within a few months and will die in childhood. There are effective therapies for ALD, including replacement of adrenal hormones, bone marrow therapy for early brain disease, and the investigational use of Lorenzo’s oil. These therapies work best if started before symptoms develop. Until now, diagnosis has relied on the identification of an affected individual and then the systematic...

How To Submit a Grant

Mon, 14 Apr 2008 20:17:50 GMT

Guidelines for Submitting Grant Proposals to The Myelin Project Investigating Novel Treatments for Demyelinating Diseases 1. Purpose. The Myelin Project seeks to fund preclinical or Phase I trials focused on novel treatment strategies for altering the course of, or repairing the damage caused by myelin diseases, including multiple sclerosis and the leukodystrophies. This latter category includes metachromatic leukodystrophy, adrenoleukodystrophy, as well as Refsums, Krabbes, phenylketonuria, Canavans, Pelizaeus-Merzbachers, and Alexanders diseases. 2. Research Objectives. We intend to stimulate novel research on MS and the leukodystrophies, with the twin objectives of stopping their progression and regenerating the myelin sheath, whose loss is these diseases common trait. Our grants will finance the cost of pre-clinical/clinical studies necessary for proof of concept, initial safety and/or efficacy assessments. The review process will be expeditious, lasting at most three...

SCIENTISTS

Mon, 14 Apr 2008 15:15:06 GMT

SCIENTIFIC ADVISORY PANEL 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 Prof. William Blakemore Professor of Neuropathology University of Cambridge Department of Clinical Veterinary Medicine Madingley Road Cambridge CB3 0ES ENGLAND Robin J.M. Franklin, PhD Professor of Neuroscience Cambridge Centre for Brain Repair & Centre for Veterinary Sciences University of Cambridge Madingley Road Cambridge CB3 0ES UNITED KINGDOM Charles ffrench-Constant Professor of Medical Neurology MRC...

Myelin Project Board Members

Wed, 02 Apr 2008 15:34:44 GMT

THE MYELIN PROJECT BOARD OF DIRECTORS OFFICERS Margaret Weis, PhD, President 1400 Wallace Blvd. Suite 259 Amarillo, TX 79106 T: 800-869-3546 F: 806-356-4694 email: Margaret.weis@myelin.org Ms. Diane Graves, Vice President President, Cure ALD Foundation Lincoln, CA 95648 Mrs. Patti Chapman, Secretary Pacific Palisades, CA Mrs. Jean Kelley, Treasurer Branford, CT 06405 DIRECTORS Mr. Hyman Abadi Bogotà, COLOMBIA and Bal Harbour, FL Mr. Neal Andrews (NEAN Enterprises) Birmingham, ALemail: Mr. John Gernhart Kingsley, IA Dr. Jay Gold Florida Psychiatric Consultants, PA Lake Mary, FL Sharon Granetz, PhD Bridgewater, NJ Mr. Eric Hovde (Hovde Financial) Washington, DC Mr. Chris Kaag Got the Nerve? Triathlon, Founder and Director The IM ABLE Foundation, Founder and President Corps Fitness, Inc., President Reading, PA Websites: www.gotthenerve.org / www.corpsfitness.net ...

DEVEOPMENT OF NEWBORN SCREENING TEST

Thu, 13 Mar 2008 20:43:24 GMT

As you may have heard, all of this could end in only a year or two. Before his death last year, Dr. Hugo Moser and Anne Moser, of the Kennedy Krieger Institute of Johns Hopkins University in Baltimore, was working on developing a test to screen all newborn children for the ALD gene. Anne Moser is continuing the work, and has finished preliminary test development. Work is now in the final stages of validation to determine the false negative and false positive rates. It is anticipated that the test will be ready for widespread use in one to two years. Once at-risk boys are identified at birth, it will become possible to mitigate or prevent entirely, the development of childhood cerebral ALD.

We are pleased to announce that the The Myelin Project has recently contributed $50,000 in funding for development of this test. Unhappily, this sum represents less than 5% of the remaining money needed to finish this important work. It is through the generosity of donors such as yourselves that we will be able to continue to fund this and other important projects to advance the diagnosis and treatment of the leukodystrophies, MS and all of the demyelinating diseases. My sincere thanks for your kind and continuing generosity.

To contribute today, you can securely and easily donate online at: http://www.myelin.org/en/donations/add.asp

Special Events and Recent Activities

Sat, 02 Feb 2008 20:36:10 GMT

View the monthly events and register for The Myelin Project special activities on the Calendar.

2008 Got the Nerve Triathlon

Sat, 02 Feb 2008 20:35:32 GMT

2008 Got the Nerve Triathlon

CURRENT GRANTS

Tue, 06 Nov 2007 22:47:39 GMT

Anne Baron-Van Evercooren, PhD Hopital Salpetriere INSERM, Paris, France Schwann Cell Therapy For Myelin-Repair In Immune-Mediated Demyelinating Disease Of The CNS: A Pre-Clinical Evaluation Funding: 2004: $80,000 Etienne-Emile Baulieu, MD, PhD, Michael Schumacher, PhD, Martine El-Etr, MD, PhD Hopital Bicetre/INSERM, Paris, France The Promyelinating Effects of Progestins, Researcher Antoine Pianos Funding: 2006: $20,000 (Pending) Ian Duncan, PhD, University of Wisconsin-Madison Intraventricular Transplantation Study Funding: 2004: $43,637 Gianvito Martino MD, Angelo Vescovi PhD, Fulvio Mavilio PhD, San Raffaele Scientific Institute, Milan, Italy Stem cells, gene therapy, and growth factors in multiple sclerosis (MS): This study employs a two-pronged approach in an animal model of multiple sclerosis (MS): transplantation (via injection) of neural stem cells, to promote remyelination; and gene therapy using a viral vector, to deliver anti-inflammatory and...

Remembering Michaela

Wed, 24 Oct 2007 20:20:34 GMT

A graduate cum laude from Dunbarton College of the Holy Cross, Michaela Murphy Odone was granted both a French government and a Fulbright scholarship to study at the University of Grenoble (France) and to teach English at a local lycée. In 1966 she made the list of Outstanding Young Women of America. Michaela had an important role both in developing Lorenzos Oil and in setting up the Myelin Project. But one of her major feats was undoubtedly to have kept Lorenzo alive and in good health all these years. She did this by focusing almost exclusively on his care. Giving up all forms of entertainment, she mastered every aspect of the disease—neurological, metabolic, and endocrinological. The care plan she wrote was praised by all doctors who knew the case. For sixteen long years she spent interminable hours at Lorenzos bedside, day in and day out. Her passionate caring for Lorenzo, however, did not prevent her from answering the constant flow of questions from desperate ALD mothers...

The Myelin Project Scientists

Mon, 22 Oct 2007 20:23:48 GMT

SCIENTIFIC ADVISORY PANEL 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 Prof. William Blakemore Professor of Neuropathology University of Cambridge Department of Clinical Veterinary Medicine Madingley Road Cambridge CB3 0ES ENGLAND Ian Duncan, PhD Professor, Neurology Department of Medical Sciences School of Veterinary Medicine University of Wisconsin-Madison 2015 Linden Drive West Madison, WI 53706-1102 USA Robin J.M. Franklin, PhD Senior Lecturer in Experimental Neurology Cambridge Centre for Brain Repair & Centre for Veterinary Sciences University of Cambridge Madingley Road Cambridge CB3 0ES UNITED KINGDOM Charles ffrench-Constant, PhD Professor of Neurological Genetics University of Cambridge Department of Pathology ...

MISSION STATEMENT

Thu, 20 Sep 2007 19:14:22 GMT

The Myelin Project is an international organization aimed at acclerating clinical and translatinal research focused on myeln repair. Myelin, the insulating sheath surrounding nerve cells, can be destroyed by hereditary neurodegenerative disorders such as the leukodystrophies, and by acquired diseases such as multiple sclerosis. All together, demyelinating diseases affect more than two million people worldwide. The Myelin Project was established in 1989 with the aim of funding research on the demyelinating diseases, such as the leukodystrophies and multiple sclerosis. The organization was founded by Augusto Odone and his late wife, Michaela. Their son, Lorenzo, suffers from X-linked adrenoleukodystrophy (X-ALD), the most common of the leukodystrophies. The story of the Odones struggle was dramatized in the film Lorenzos Oil, starring Nick Nolte and Susan Sarandon, released by Universal Studios in 1992. A multinational gathering of families joined the Odones in their...

Lorenzo

Wed, 19 Sep 2007 22:09:55 GMT

Lorenzo’s Oil: Advances in the Treatment of Neurometabolic Disorders INHERITED NEUROMETABOLIC DISORDERS ARE INdividually rare and have many causes. Many are progressive disorders with treatment options limited to symptomatic care. X-linked adrenoleukodystrophy (ALD) is a rare disorder first described almost 100 years ago. As a result of intense basic science and clinical research, remarkable progress has been made in elucidating the biochemical irregularities, molecular genetics, diagnostic testing, and pathogenesis of this disorder. 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. It is a progressive disorder characterized by Addison disease, childhood dementia, and neurologic deterioration to a vegetative state. A key discovery was the identification of lipid inclusions consisting of cholesterol esters and an excess of very longchain ...

Archive 2004 and Earlier

Wed, 19 Sep 2007 21:45:18 GMT

Research was advanced in these areas in 2004 and earlier.

Archive 2005

Wed, 19 Sep 2007 21:44:11 GMT

Research continued in these areas in 2005

Archives 2006

Wed, 19 Sep 2007 21:42:29 GMT

Research continued in these areas during 2006.

Current Grants

Wed, 19 Sep 2007 20:53:51 GMT

These organizations and individuals are being supported in their research.

Early Detection Called Key to Effectiveness

Mon, 03 Sep 2007 13:06:12 GMT

Early detection called key to effectiveness By Carey Goldberg, Globe Staff | July 12, 2005 New research offers scientific vindication for the parents whose fight to save their ailing son inspired the 1992 movie Lorenzos Oil. It comes too late to help the real-life Lorenzo, who is nearly totally paralyzed, but suggests that the oil could help save future generations of boys from the same, fatal nerve disorder. The paper, published yesterday in the Archives of Neurology, found that the oil was often effective at fending off symptoms in young boys who had the genetic disease that has devastated Lorenzo Odone. Among the 89 boys who started taking the oil before symptoms appeared, only 11 percent went on to develop them, compared with the usual 35 percent. In a prototypical clash between activist parents and resistant doctors, the medical establishment has been extremely skeptical about the effectiveness of Lorenzos oil. Among those skeptics was Dr. Hugo W. Moser, a professor at Johns...