The Myelin Project RSS Feed

[firstname] [lastname], The Myelin Project Newsletter 18-Dec-09

Candace Root — Fri, 18 Dec 2009 17:50:47 GMT

[menu]

Hello [firstname],

HAPPY HOLIDAYS

It is that time again, time to look back over 2009 and see where we have been and where we are going. Happy 20th Anniversary to the Myelin Project. It was twenty years ago that Augusto and Michaela Odone created The Myelin Project. When their son, Lorenzo, was diagnosed with adrenoleukodystrophy, they were told that nothing could be done. They were told to take him home and wait for him to die. They would not accept that prognosis and so their journey began and they created The Myelin Project.

We are still racing the clock . The single biggest barrier to progress is our scientific research is lack of funding. Won’t you
please help us today with your generous and immediate support. All donations are fully tax deductible.

You may donate online at http://www.myelin.org/en/donations/add.asp

RESEARCH PROGRESS

In the past twenty years, thanks in part to those who have made generous donations to The Myelin Project, we have learned quite a lot about X-linked ALD (X-ALD). While there are still no therapies available to treat the full-blown symptoms of childhood X-ALD, we have found that, if begun before symptoms develop, treatment with Lorenzo’s oil may be able to prevent the onset of symptoms (Arch Neurol. 2005 62:1073-80). Lorenzo’s oil is not always effective. However, if symptoms are detected very early it is possible to perform a bone marrow or cord blood transplant. When successful, this therapy appears to arrest the development of further symptoms. The first successful transplant recipients are just now entering their 20’s, so that it will still be a few years before we know whether they will also escape AMN.

Last month, a great advance in X-ALD treatment was announced. A team headed by Dr. Patrick Aubourg, University Paris-Descartes, have developed a way to transplant the X-ALD gene (Science. 2009 326:818-23). The team was able to remove most of the genes from a virus, and then replace the viral genes with a normal X-ALD gene. The virus retained its ability to get into cells, but lost its ability to cause any disease. The team then took bone marrow cells from an X-ALD patient, and infected them with the modified virus. The virus carried the normal X-ALD gene into the cell, where it began to function. The modified bone marrow cells were given back to the patient. Because they function normally, the modified bone marrow cells were able to restore gene function in the X-ALD patients. This technique will permit treatment of patients for whom a bone marrow donor is unavailable. Even better, because the patient’s own cells are used, the danger of transplant failure is greatly reduced.

Each of these effective treatments relies on identifying carriers of the X-ALD gene very early. Usually, that does not happen unless another family member has been identified. We are working to help change that. Researchers at The Kennedy Krieger Institute of Johns Hopkins University in Baltimore are developing a newborn screening test for the X-ALD gene. When finished, it is anticipated that the test will be widely adopted. Developing this test is important not only because successful treatment relies on early identification, but also because a significant number (perhaps as high as 5%) of X-ALD cases arise from new gene mutations. In those cases, family history is not helpful in identifying at-risk children.

FUND RAISING

The 2009 Got The Nerve? Triathlon held in Mount Gretna, Pennsylvania was a huge success this year with over 675 participants. The Myelin Project would like to thank Chris Kaag, his immediate and extended family, and the over 100 volunteers for all their hard work and efforts put into this well-organized event. Each year this triathlon gets bigger and better. The Myelin Project would also like to thank Got The Nerve? corporate sponsors: Clover Farms, Yuengling Brewery, PRL. Inc, Reading Orthodontic Group, Power Bar, and All Risks. The 2009 Got The Nerve? Triathlon raised over $20,000 for The Myelin Project. The funds raised at this year’s event have been designated to help fund The Augusto Odone New Investigator Award for AMN research.

The Myelin Project would like to express its sincere and deep gratitude to the people of Branford, Connecticut and the surrounding communities who made the 2009 Hammerfest Triathlon a huge success. The 2009 Hammerfest and Brian’s Beachside Boogie raised over $60,000 for The Myelin Project. These funds have been designated to help fund newborn screening for x-linked ALD.

Thank you to the staff of The Owenego Inn, and Jane Rosenthal, John and Pat Bloomquist for hosting the event each year. We also want to thank Race Productions and Race Director, Ron Meneo, together with Michael, Scott and Margit of Race Productions who produce and direct the Hammerfest Triathlon and Brian’s Beachside Boogie each year. In addition to their services, Race Productions also makes a generous financial donation to The Myelin Project each year. Thank you, Race Productions. A special thank you to Molly Phalen, who donated her 16^th birthday to raise money for The Myelin Project and raised over $3,000. The Myelin Project would also like to take this opportunity to thank John, Jean and Brian Kelley, their immediate and extended family for their generous and continued support.

We also want to acknowledge the fabulous Lemonade Gang of Branford, CT. This group of young people have raised money and donated selflessly to The Myelin Project. The Lemonade Gang was founded by Greg Nobile and Ryan Bloomquist. The Lemonade Gang began approximately eight years ago. The then very young children wanted to do something to help their dear friend, Brian Kelley, in his heroic fight against adrenoleukodystrophy. The gang has evolved but the core group has stayed intact. While their passion has grown, so too has their committment to the Myelin Project. The Lemonade Gang donated over $6,000 to The Myelin Project this year.

THE AUGUSTO ODONE NEW INVESTIGATOR AWARD

This year The Myelin Project created The Augusto Odone New Investigator Award for AMN (adrenomyeloneuropathy) research. This research award is a collaborative effort with Oliver’s Army and is also co-funded by Oliver’s Army. This research award was created to discover ways to treat adrenomyeloneuropathy (AMN), the forgotten side of X-ALD and also to encourage and keep brilliant, promising young scientists interested in leukodsytrophy research and more specifically AMN research.

Approximately one-third of the males who carry the X-ALD gene develop an inflammatory brain disease in childhood, as was depicted in the movie “Lorenzo’s Oil”. But the men who escape this childhood catastrophe almost invariably develop a different disease in their 20’s or 30’s, adrenomyeloneuropathy (AMN). Even fewer know that about half of women who carry the X-ALD gene also develop AMN, but later in life. The symptoms of AMN may be quite similar to those of multiple sclerosis, although they are quite separate diseases.

We are pleased to announce that the winner of this award is Celia Kassmann of the Max Planck Institute of Experimental Medicine in Goettingen, Germany. Through the generosity of Mark Liley and Oliver’s Army, a second award was made to Dr Aurora Pujole of Institut d'Investigació Biomédica de Bellvitge of Barcelona, Spain, to study AMN. Dr. Pujole intends to study ways to develop rational therapies for AMN, concentrating on some drugs that are already on the market for other purposes.

Drs. Kassmann and Pujole will each receive $150,000 per year for two years to complete their projects.

THANK YOU FOR VISITING OUR WEB SITE

In 2009 our web site had over 176,000 visitors from 185 countries and in 101 languages. In 2009 we opened a branch in Istanbul, Turkey. Turker Aydin is the President and was instrumental in creation of this new Branch and also created the web site which is http://www.myelinturk.org/ Thank you Mr. Aydin and welcome to the Myelin Project family.

WE NEED YOUR HELP

We are still racing the clock . The single biggest barrier to progress is our scientific research is lack of funding. Won’t you please help us today with your generous and immediate support. All gifts to the Myelin project are fully tax deductible.

You may donate online at http://www.myelin.org/en/donations/add.asp

If you are interested in helping The Myelin Project by hosting a fund raising event in your community Jean Kelley, ALD mother and Myelin Project Board member will help you get started and guide you through the process. She has been fantastic fund raiser for the Myelin Project and has offered to share her expertise and work with you to develop a successful event. There is no event or donation too small. Every little bit helps. If you are interested you may email Jean Kelley at jeanakelley@gmail.com

HAPPY NEW YEAR

Stay up to date with our RSS feeds.

18-Dec-09 11:50 AM

[firstname] [lastname], The Myelin Project Newsletter 5-Nov-09

Candace Root — Thu, 05 Nov 2009 20:38:36 GMT

Hello [firstname],

Welcome to the The Myelin Project newsletter here is some very exciting new research news.

Releases

Releases for 6-Oct-09 to 5-Nov-09

Gene therapy technique slows brain disease

Author: Natasha Pinol

Release Date: Thursday 5-Nov-09 2:00 PM

A strategy that combines gene therapy with blood stem cell therapy may be a useful tool for treating a fatal brain disease, French... [More Info]
Posted by: Candace Root

Gene Therapy Advance Saves Two Boys From Rare Brain Disease

Author: Rob Waters

Release Date: Thursday 5-Nov-09 1:00 PM

Nov. 5 (Bloomberg) -- Two seven-year-old boys with a fatal brain disease who couldn’t get bone marrow transplants were saved by... [More Info]
Posted by: Candace Root

Brain Disease Treated by Gene Therapy

Author: Lizzie Buchen

Release Date: Thursday 5-Nov-09 1:00 PM

A treatment based on HIV finds first success in humans. The successful treatment of a brain disease marks a big step forward for gene... [More Info]
Posted by: Candace Root

Scientists Halt Brain Disease with New Gene Therapy

Author: Elizabeth Fullerton

Release Date: Thursday 5-Nov-09 1:00 PM

LONDON (Reuters) - Scientists have managed to halt a rare and fatal brain disease with an experimental gene therapy technique using a... [More Info]
Posted by: Candace Root

Duke Studies New Approach in Fetal Transplants for Metabolic Disorders

Author: Joanne Kurtzburg

Release Date: Thursday 15-Oct-09 2:00 PM

October 13th, 2009 --> (PhysOrg.com) -- Researchers say a new development in cord blood transplants for inherited metabolic disorders... [More Info]
Posted by: Candace Root

Stay up to date with our RSS feeds.

5-Nov-09 2:38 PM

Help Someone with a Demyelinating Disease

Kim Hodgson — Wed, 09 Sep 2009 17:00:00 GMT

Untitled Document

Another Wayne Osteen Home Raffle Benefiting The Myelin Project.

Wayne Osteen a local home builder in Amarillo, Texas and is building a new home that will be raffled off as a fund raiser to benefit The Myelin Project.The home is under construction in the new City View addition of Amarillo, Texas. The home will be raffled off in a reverse raffle on Sunday October 18, 2009. Only 4900 tickets will be sold, 100 finalists will be selected and will participate in the reverse raffle.

Win a house in City View and at the same time help someone with a demyelinating disease. Funds raised will be used to fund future research of The Myelin Project.

Raffle tickets are only $50.00!

Acutal image of the house

9-Sep-09 12:00 PM

The Myelin Project Home Giveaway Raffle

Kim Hodgson — Wed, 01 Jul 2009 14:00:00 GMT

Another Wayne Osteen Home Giveaway Benefiting The Myelin Project. Wayne Osteen a local home builder in Amarillo, Texas and is building a new home that will be raffled off as a fund raiser to benefit The Myelin Project. The home is under construction in the new City View addition of Amarillo, Texas. The home will be raffled off in a reverse raffle on Sunday October 18, 2009. Only 4900 tickets will be sold, 100 finalists will be selected and will participate in the reverse raffle for the home giveaway.

Actual image of the house

Make your dreams come true, win a house in City View and at the same time help someone with a demyelinating disease. Funds raised will be used to fund future research of The Myelin Project.

Raffle tickets are only $50.00! Purchase yours online today!

1-Jul-09 9:00 AM

[firstname] [lastname], The Myelin Project Newsletter 19-May-09

Candace Root — Tue, 19 May 2009 15:56:40 GMT

Untitled Document

Hello [firstname],

Welcome to the The Myelin Project newsletter of upcoming events, articles, jobs and more from our membership.

Calendar Events
Articles
Releases

Calendar Events for 19-May-09 to 1-Jul-09

6th Annual Got the Nerve Triathlon
Speaker's Name:
Day: Saturday 23-May-09 8:00 AM
Location: Mt. Gretna Mt. Gretna, PA 17064
Summary: 6TH ANNUAL GOT THE NERVE TRIATHLON Saturday May 23, 2009...
Add to Calendar
Posted by: Candace Root

Lorenzo Odone Memorial
Speaker's Name:
Day: Saturday 30-May-09 9:00 AM
Location:
Summary: May 30, 2009 will be the one year anniversary of Lorenzo Odone's death. Please join us and Augusto in remembering Lorenzo in a moment of silence to honor the memory of Lorenzo and his brave fight with ALD. ...
Add to Calendar
Posted by: Candace Root

Articles for 5-May-09 to 19-May-09

Treating The Other Face of X-ALD -- Adrenomyelineuropahty

Author: Margaret Weis

Release Date: Tuesday 19-May-09 10:30 AM

Treating the Other Face of X-ALD - Adrenomyeloneuropathy It is a great pleasure to announce the launch of the Augusto Odone New Investigator Award, a project co-funded by The Myelin Project and Oliver’s Army http://www.oliversarmy.org/ Young scientists embarking on their research career are invited to submit their plans for a project aimed at finding treatments for adrenomyeloneuropathy (AMN). This debilitating disorder and X-linked adrenoleukodystrophy (X-ALD) are caused by a mutation in... [More Info]
Posted by: Candace Root

Releases for 19-Apr-09 to 19-May-09

NORTHWOOD SCHOOL STUDENTS RAISE MONEY FOR MYELIN DISEASE RESEARCH

Author: Candace Root

Release Date: Thursday 14-May-09 10:00 AM

The Myelin Project Each year the student body raises money for The Myelin Project which was established in 1989 with the aim of... [More Info]
Posted by: Candace Root

MS NEWS - Ex-Salesman Uses Net to Inform, Persuade

Author: Stuart Schlossman

Release Date: Thursday 7-May-09 11:00 AM

You may read the article in its entirety here ... [More Info]
Posted by: Candace Root

Stay up to date with our RSS feeds.

19-May-09 10:56 AM

Treating The Other Face of X-ALD -- Adrenomyeloneuropathy

Margaret Weis — Tue, 19 May 2009 15:30:00 GMT

Treating the Other Face of X-ALD - Adrenomyeloneuropathy

It is a great pleasure to announce the launch of the Augusto Odone New Investigator Award, a project co-funded by The Myelin Project and Oliver’s Army http://www.oliversarmy.org/ Young scientists embarking on their research career are invited to submit their plans for a project aimed at finding treatments for adrenomyeloneuropathy (AMN). This debilitating disorder and X-linked adrenoleukodystrophy (X-ALD) are caused by a mutation in the same gene (known as the ALD gene). While X-ALD has been the focus of much research, very little is known about AMN. This new award is intended to help bridge the gap.

Most of the visitors to this website know something about X-ALD. This genetic disease affects about one-third of boys who carry the X-ALD gene, causing loss of most normal neurologic functions within one or two years. Fewer than 20 years ago, X-ALD was invariably fatal. Thanks, in part, to The Myelin Project, substantial progress has been made in treating X-ALD. The day is near when the final work on a newborn screening test will be complete. The test will enable doctors to identify all boys who carry the X-ALD gene. Treating pre-symptomatic boys with Lorenzo’s Oil significantly reduces the chances that X-ALD will develop. For those boys who develop symptoms despite Lorenzo’s Oil therapy, there remains the option of a bone marrow or cord blood transplant. While still risky, the success rates of bone marrow transplantation have improved significantly in recent years.

Many visitors to this website have not even heard of AMN. This disorder affects the other two-thirds of the males and about one-half of the females who carry the X-ALD gene. AMN is part of the complex of diseases caused by mutations in the ALD gene. In an affected family in which two boys have the same mutation, one may develop ALD and die early, while the other may remain asymptomatic until his 20’s when he develops AMN. X-ALD and AMN have quite different symptoms. AMN is characterized by loss of spinal cord and peripheral nerve function resulting in stiffness, ataxia, weakness to paralysis and sometimes cognitive defects showing that in some men with AMN, the brain is also involved.

In men, symptoms of AMN most often develop between 20 and 30 years of age, when they are just beginning to build a career and start a family. Women become symptomatic later in life, in their 40’s or 50’s, and while the symptoms are usually less severe than men with AMN, they can be serious and debilitating.

If you would like to make a contribution to the Augusto Odone New Investigator Award for AMN Research you may donate online http://www.myelin.org

19-May-09 10:30 AM

The Myelin Project Needs Your Support!

Kim Hodgson — Thu, 16 Apr 2009 16:00:00 GMT

Untitled Document

The Myelin Project needs your support in continuing our mission to end human suffering by those inflicted with demyelinating diseases.

You can show your support by donating to the causes below.

Support research for Leukodystrophies and acquired diseases such as multiple sclerosis Donate online through Facebook. Learn more.

Mission: To raise money to fund accelerated research for myelin disease.

Support Development of a Newborn Screening Test for the Adrenoleukodystrophy (ALD) gene Donate online through Facebook. Learn more.

Mission: To detect ALD gene in newborns so life saving interventions such as Lorenzo’s Oil therapy can be started before clinical symptoms occur.

The Myelin Project is now on Facebook. Become a Fan and stay up to date with the latest Myelin Project news and research updates.

16-Apr-09 11:00 AM

2008 Myelin Project Newsletter

Candace Root — Mon, 29 Dec 2008 18:00:00 GMT

/attachments/wysiwyg/1/2008Newsletter.pdf

29-Dec-08 12:00 PM

How Schwann Cells Might Be Altered So That They Might Be Better Candidates For Myelin Transplantation

Anne Baron-Evercooren, PhD — Wed, 10 Dec 2008 20:00:00 GMT

Dr. Anne Baron-Evercooren, Centre Hospitalier Universitaire, Pitié-Salpêtrière, Paris, presented her investigation of how Schwann cells might be altered so that they might be better candidates for myelin transplantation. By forcing the cells to express an enzyme, sialyltransferase, their ability to migrate was greatly improved.

Schwann cells (SC) form myelin in the peripheral nerves and are easy to get. Despite their obvious repair potential in the central nervous system (CNS), several studies indicated that SC aren’t effective in repairing CNS myelin. Other cell types (olfactory ensheathing cells, neural stem cells and oligodendrocytes) can repair CNS myelin, but are not very easy to get. Dr. Baron-Van Evercooren’s laboratory has explored some of the differences between SC and these other cell types. All of the myelin forming cells, including the SC, express NCAM, a specific protein, on their surface. However, the NCAM of the CNS myelin forming cells is “decorated” with a specific carbohydrate, while the NCAM of the SCs is “plain”.

To solve this problem, Dr. Baron-Evercooren and her associates have developed a method of getting SCs to produce “decorated” NCAM. After characterizing the modified SC cells in culture, they were transplanted to mice that had spinal cord demyelination. The modified SC were much more efficient at repairing the demyelination than the control SC. These results underline the potential therapeutic benefit of genetically modifying SC to overcome their poor migratory property and promote their repair potential in demyelinating disorders of the CNS. Dr. Baron-Evercoorn’s work has been supported by WFL and INSERM

10-Dec-08 2:00 PM

Identifying Cell Types That Might Be Useful In Re-myelination

Violetta Zujovic, PhD — Wed, 10 Dec 2008 20:00:00 GMT

Violetta Zujovic, Ph.D., Centre Hospitalier Universitaire, Pitié-Salpêtrière, Paris, reported on her work to identify cell types that might be useful in re-myelination.

During development, the entire nervous system begins as a mass of cells called the neural crest. Boundary cap cells (BC) are descended from neural crest cells. The BCs migrate to the boundary between the central (brain and spinal cord) and peripheral (sensory and motor nerves) divisions of the nervous system. BCs are important because they are the ancestors of Schwann cells (the myelin forming cells of the peripheral nerves). In addition, BCs are also the ancestors of some of the nocioceptive (pain-sensing) nerve cells of the dorsal root ganglia (part of the spinal cord).

To gain insights in BC’s behaviour in the demyelinated central nervous system, BCs were isolated from developing mouse brain. When BCs were transplanted to a demyelinated region of a mouse spinal cord, they were able to multiply, thus efficiently repairing the lesion. When grafted at a distance (one vertebra away) from the lesion, the BCs were not only able to multiply, but they and their descendents migrated toward the lesion. The migrating cells colonized and repaired the demyelinated lesion. Interestingly, the BCs were even more efficient at colonizing the demyelinated region than Schwann cells transplanted directly to the lesion.

Thus, there is evidence that boundary cap cells are able to remyelinate central nervous system axons. This evidence strongly indicates that boundary cap cells are of interest as a potential method of central nervous system myelin repair.

10-Dec-08 2:00 PM

Sisters rally genetic researchers to tackle fatal brain disease

noemail@myelin.org — Fri, 06 Nov 2009 17:00:00 GMT

Tom Avril Philadelphia Inquirer (PA) - Nov. 06, 2009 Nov. 6--The busy scientist was about to leave a conference in Washington for his native Hungary when he was buttonholed by a persistent Main Line mother worried about her son's health. Amber Salzman told him he absolutely had to meet a researcher in France who was working on the deadly brain disease that could strike her son. He said, sure, someday. But after boarding the plane to Hungary, he got a surprise message from the flight attendant: Salzman had already set up the meeting, and the French researcher would meet him in Budapest. That fast-track meeting, and plenty of others arranged by Salzman and her sister Rachel, eventually led to pioneering research published today in the journal Science. The authors report that a novel gene therapy seems to stop progression of ALD, the rare disease featured in the movie Lorenzo's Oil. The study describes results from just two patients, and the therapy is...

Gene therapy technique slows brain disease

noemail@myelin.org — Thu, 05 Nov 2009 20:00:00 GMT

A strategy that combines gene therapy with blood stem cell therapy may be a useful tool for treating a fatal brain disease, French researchers have found. These findings appear in the 6 November 2009 issue of the journal Science, which is published by AAAS, the nonprofit science society. In a pilot study of two patients monitored for two years, an international team of researchers slowed the onset of the debilitating brain disease X-linked adrenoleukodystrophy (ALD) using a lentiviral vector to introduce a therapeutic gene into patient's blood cells. Although studies with larger cohorts of patients are needed, these results suggest that gene therapy with lentiviral vectors, which are derived from disabled versions of human immunodeficiency virus (HIV), could potentially become instrumental in treating a broad range of human disorders. This is the first time we were able to successfully use an HIV-derived lentivirus vector for gene therapy in humans, and also the first time that a...

Scientists Halt Brain Disease with New Gene Therapy

noemail@myelin.org — Thu, 05 Nov 2009 19:00:00 GMT

LONDON (Reuters) - Scientists have managed to halt a rare and fatal brain disease with an experimental gene therapy technique using a deactivated version of the AIDS virus, a study published on Thursday showed. The international team used a disabled form of human immunodeficiency virus (HIV) to deliver working genes to two boys with the brain disease X-linked adrenoleukodystrophy (ALD). Their success may help shape future treatment. Patrick Aubourg of Inserm-University Paris Descartes, who led the study, said it was the first time scientists had successfully used an HIV-derived delivery technique for gene therapy in humans, and the first time gene therapy had been used effectively in a severe brain disease. Up to now we have treated two boys ... and we can say with confidence that more than two years after gene therapy their brain disease has been arrested, he said. Featured in the 1992 movie Lorenzo's Oil, ALD is a rare hereditary condition caused by a deficiency of a protein...

Brain Disease Treated by Gene Therapy

noemail@myelin.org — Thu, 05 Nov 2009 19:00:00 GMT

A treatment based on HIV finds first success in humans. The successful treatment of a brain disease marks a big step forward for gene therapy.Getty Researchers have halted a fatal brain disease by delivering a therapeutic gene to the stem cells that mature into blood cells. The gene was transferred using a virus derived from HIV, a technique that researchers have pursued for more than a decade but has not been successful in humans until now. Together with his colleagues, paediatric neurologist Patrick Aubourg at INSERM — France's main biomedical research agency — and at the Saint-Vincent de Paul Hospital in Paris, developed the system to treat X-linked adrenoleukodystrophy (ALD), a neurodegenerative disease that affects young males. ALD results in severe degeneration of the myelin sheath, a structure that is crucial for brain-cell function. The disease is caused by mutations in a gene encoding the ALD protein, which has an important role in cells that make up the myelin sheath...

Gene Therapy Advance Saves Two Boys From Rare Brain Disease

noemail@myelin.org — Thu, 05 Nov 2009 19:00:00 GMT

Nov. 5 (Bloomberg) -- Two seven-year-old boys with a fatal brain disease who couldn’t get bone marrow transplants were saved by scientists whose gene therapy technique may let doctors treat other incurable disorders. Doctors in Paris delivered the gene into the boys’ bodies using HIV, the virus that causes AIDS. The virus, stripped of genetic material that makes it toxic, integrates permanently into the DNA of cells it enters, scientists said. That means the modified gene remains in the blood-forming stem cells for the life of the patient, according to a report in the journal Science. Two years after the experimental treatment, the neural damage has been halted or reversed and the two boys attend school and lead normal lives, said Nathalie Cartier, the study’s lead author. The treatment was cited as an example of a comeback for gene therapy, after years of setbacks, in an editorial in the journal. Their disease is completely stabilized, they are fine and there’s no reason this...

Duke Studies New Approach in Fetal Transplants for Metabolic Disorders

noemail@myelin.org — Thu, 15 Oct 2009 19:00:00 GMT

October 13th, 2009 --> (PhysOrg.com) -- Researchers say a new development in cord blood transplants for inherited metabolic disorders may be curative for some babies who are treated while still in the womb. Joanne Kurtzberg, MD, professor of pediatrics and pathology and director of the Pediatric Blood and Marrow Transplant Program at Duke University Medical Center, says the new approach uses a small, select number of therapeutic stem cells that have been treated to hasten and improve the process of engraftment. Kurtzberg is formally studying the procedure in a pilot trial open to newly pregnant mothers known to be at risk for having children with lethal metabolic disorders. Metabolic disorders -- also known as lysosomal storage disorders -- include several dozen rare, inherited genetic diseases marked by the absence of specific enzymes the body needs to break down and get rid of metabolic byproducts of energy production. Left untreated, metabolic disorders can lead to bone,...

Pioneering HHS HRSA Grant Funds Establishment of First National Newborn Screening Clearinghouse

noemail@myelin.org — Thu, 24 Sep 2009 18:30:00 GMT

WASHINGTON – September 16, 2009 – The nation’s first Newborn Screening Clearinghouse (NBSC), connecting millions of parents and healthcare providers with resources and information relevant to more than four million newborns screened annually, will be created through a $3.75 million cooperative agreement from the U.S. Health and Human Services, Health Resources and Services Administration (HRSA), Genetic Services Branch. The project will span five years. Genetic Alliance and the National Newborn Screening and Genetics Resource Center (NNSGRC) at the University of Texas Health Science Center at San Antonio, will develop the NBSC with the Genetics and Newborn Screening Regional Collaborative Groups, March of Dimes, the Association of Public Health Laboratories and many other partners. For more than 40 years infants born in the U.S. have been screened for an increasing number of congenital conditions, yet parents are often unaware of the number and quality of screening their children...

Opexa Reports Additional Favorable Data with Tovaxin for Multiple Sclerosis

noemail@myelin.org — Thu, 24 Sep 2009 18:00:00 GMT

Opexa Reports Additional Favorable Data with Tovaxin® for Multiple Sclerosis THE WOODLANDS, Texas (September 8, 2009) – Opexa Therapeutics, Inc. (NASDAQ: OPXA), a company developing Tovaxin®, a personalized T-cell immunotherapy for multiple sclerosis (MS), today announced results from further analysis of the double-blind, placebo-controlled, 52-week Phase IIb TERMS clinical study of 150 patients with Relapsing Remitting MS (RRMS). This analysis evaluated patients with an annualized relapse rate of one or greater at study entry (ARR≥1). More than 83% of the Tovaxin-treated group (n=85) remained relapse free at one year and the annualized relapse rate after treatment decreased to 0.20, a 42% reduction compared to placebo. The results of this expanded analysis confirm those found in the previously-reported per-protocol analysis of patients in the TERMS study with ARR>1. This post-hoc analysis which represents 86% of the total patient population in the TERMS study...

Opexa Announces Stem Cell Agreement with Leading

noemail@myelin.org — Thu, 10 Sep 2009 16:00:00 GMT

Opexa Announces Stem Cell Agreement with Leading Global Pharmaceutical Company THE WOODLANDS, Texas (August 7, 2009) – Opexa Therapeutics, Inc. (NASDAQ: OPXA), a company developing a novel T-cell immunotherapy for multiple sclerosis (MS), today announced that it has entered into an exclusive agreement with Novartis, one of the world’s largest pharmaceutical companies, for the further development of Opexa’s novel stem cell technology. This technology, which has generated preliminary data showing the potential to generate monocyte derived islet cells from peripheral blood mononuclear cells, was in early preclinical development at Opexa. Under the terms of the agreement, Novartis will acquire the stem cell technology from Opexa and Novartis will have full responsibility for funding and carrying out all research, development and commercialization activities. Opexa will receive an upfront cash payment of $3 million, plus an additional $1 million as a technology transfer fee to be...

American Brain Coalition Newsletter Highlights The Myelin Project

noemail@myelin.org — Fri, 17 Jul 2009 14:00:00 GMT

/attachments/wysiwyg/1/AmericanBrainCoalition.htm

NEWS

Wed, 13 Jan 2010 21:18:39 GMT

MYELIN PROJECT BOARD MEMBER OPENING

Have you or has someone you love been affected by a demylinating disease such as MS, ALD, AMN,
Krabbe, Alexander's Disease. Would you like to make a contribution and a difference in the direction of myelin repair research? This is not a paid position but an opportunity to serve on a forward-thinking, prestigious Board. Candidates should have a passion for finding a cure, time and/or resources to do successful fundraising, willingness to participate in four Board meetings per year - three via conference call and one on location for the Annual Myelin Project Symposium where scientific information and the latest cutting edge research in myelin repair is presented. Please submit your resume or biography by e-mail to Margaret Weis margaret.weis@myelin.org stating your interest and qualifications.

LETTER FROM THE PRESIDENT

Sat, 12 Dec 2009 18:33:12 GMT

December 12, 2009 A Letter from the President: There Has Been Progress. In the last twenty years, thanks in part to those who have made generous donations to the Meylin project, we have leared quite a lot about X-linked ALD (X-ALD). While there are still no therapies available to treat the full-blown symptoms of childhood X-ALD, we have found that, if begun before symptoms develop, treatment with Lorenzo’s oil may be able to prevent the onset of symptoms (Arch Neurol. 2005 62:1073-80). Lorenzo’s oil is not always effective. However, if symptoms are detected very early it is possible to perform a bone marrow or cord blood transplant. When successful, this therapy appears to arrest the development of further symptoms. The first successful transplant recipients are just now entering their 20’s, so that it will still be a few years before we know whether they will also...

EVERY LIFE ART CONTEST - Empowering Artists Affected by Rare Disease

Fri, 20 Nov 2009 15:50:58 GMT

Due to several inquiries asking for more time to complete their submission we are extending the deadline to 12/31/09. Please call Julia Jenkins at 415.884.0223 for more information. The Kakkis EveryLife Foundation invites artists to participate in the first annual EveryLife Art Contest. PRIZES: Two Grand Prizes will be awarded in each age group. Age Groups: Children 5-11: $100 Visa Gift Card Teens 12-17: $250 Visa Gift Card Adults 18+: $500 Visa Gift Card Each Grand Prize winner will also receive a Flip Video camera. Winners will use the Flip Video to record their story about being an artist affected by a rare disease. The artists' video stories will be posted on the Kakkis EveryLife Foundations Website. Awards for Outstanding Art: All contestants with works of outstanding art will receive a $25 Visa Gift Card. ELIGIBILITY: The EveryLife Art Competition is open to all artists affected by a Rare Disease ages...

How To Submit a Grant

Tue, 27 Oct 2009 16:04:52 GMT

The Myelin Project is not currently accepting any new research grant applications at this time. 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 Refsum's, Krabbe's, phenylketonuria, Canavan's, Pelizaeus-Merzbacher's, and Alexander's 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...

Wed, 07 Oct 2009 22:07:33 GMT

Demyelinating Diseases In Brief

Wed, 23 Sep 2009 15:02:57 GMT

Demyelinating diseases are those in which myelin is the primary target. They fall into two main groups: acquired diseases (i.e., multiple sclerosis) and hereditary neurodegenerative disorders (i.e., the leukodystrophies). Although their causes and etiologies are different, they have the same outcome: CNS demyelination. Without myelin, nerve impulses are slowed or stopped, leading to a constellation of neurological symptoms. Axons with normal myelin Demyelinated axons: Nerve impulse conduction slows or stops completely Acquired Diseases The most common of these is multiple sclerosis (MS), which usually manifests itself between the 20th and 50th years of life. Current estimates are that approximately 2.5 million people worldwide have MS, with between 250,000 and 350,000 cases in the United States, 50,000 cases in Canada, 130,000 cases in...

Augusto Odone New Investigator Award

Tue, 08 Sep 2009 18:49:52 GMT

Name of Award: Augusto Odone New Investigator Award Purpose: To encourage promising young scientists to pursue a career in research aimed at investigating the pathogenesis and treatment of adrenomyeloneuropathy. Preference will be given to projects that are translational/clinical in nature. Funding: The award will be co-funded by The Myelin Project and Oliver's Army http://www.oliversarmy.org/ Applicant Description: 1. Applicants must have an independent academic or research track position at a junior level, by the time of award activation. The applicant must meet institutional requirements for submission of an extramural, peer reviewed grant proposal. 2. Applicants may have no more than seven years of active faculty/staff appointment prior to award activation. 3. Postdoctoral research fellows are ineligible for this award unless they will have faculty/staff appointment at the time of activation. 4. Applicants may have no more than $175,000 per year in other extramural...

Myelin Project Board Members

Tue, 28 Jul 2009 12:29:47 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. John Gernhart Kingsley, IA Dr. Jay Gold Florida Psychiatric Consultants, PA Lake Mary, FL Sharon Granetz, PhD Bridgewater, NJ 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

LORENZO ODONE

Wed, 08 Jul 2009 21:21:36 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 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 within 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...

Science and Society Gala Sponsorship Information

Tue, 09 Jun 2009 18:15:10 GMT

For more information on Gala Sponsorship and price packages
/attachments/wysiwyg/1/GalaSponsorshipPkg2009.pdf

Lorem ipsum survey

noemail@myelin.org — Thu, 09 Aug 2007 20:40:38 GMT

Objectives:

Lorem ipsum dolor sit amet, consectetuer adipiscing elit, sed diem nonummynibh euismod tincidunt ut lacreet dolore magna aliguam erat volutpat. Ut wisis enim ad minim veniam, quis nostrud exerci tution ullamcorper suscipit lobortis nisl ut aliquip ex ea commodo consequat. Duis te feugifacilisi.

Duis autem dolor in hendrerit in vulputate velit esse molestie consequat, vel illum dolore eu feugiat nulla facilisis at vero eros et accumsan et iusto odio dignissim qui blandit praesent luptatum zzril delenit au gue duis dolore te feugat nulla facilisi.

Ut wisi enim ad minim veniam, quis nostrud exerci taion ullamcorper suscipit lobortis nisl ut aliquip ex en commodo consequat. Duis te feugifacilisi per suscipit lobortis nisl ut aliquip ex en commodo consequat.Lorem ipsum dolor sit amet, consectetuer adipiscing elit, sed diem nonummy nibh euismod tincidunt ut lacreet dolore magna aliguam erat volutpat.