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[firstname] [lastname], The Myelin Project Newsletter 26-Sep-11

Greg Benton - noemail@myelin.org — Tue, 27 Sep 2011 03:08:02 GMT

Untitled Document

Hello [firstname],

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

Articles

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Articles for 28-Jul-11 to 26-Sep-11

Mice Stem Cells Guided into Myelinating Cells by the Trillions

Author: Greg Benton

Release Date: Monday 26-Sep-11 1:00 PM

Scientists at Case Western Reserve University School of Medicine found a way to rapidly produce pure populations of cells that grow into the protective myelin coating on nerves in mice. Their process opens a door to research and potential treatments for multiple sclerosis, cerebral palsy and other demyelinating diseases afflicting millions of people worldwide. The findings will be published in the online issue of Nature Methods, Sunday, Sept. 25, at 1 p.m. EST. The mouse cells... [More Info]
Posted by: Greg Benton

Clinical Successes and New Technologies Revive Gene Therapy

Author: Greg Benton

Release Date: Friday 9-Sep-11 1:00 PM

***Click the link below to read the article.*** http://www.genengnews.com/analysis-and-insight/clinical-successes-and-new-technologies-revive-gene-therapy/77899451/
Posted by: Greg Benton

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26-Sep-11 8:08 PM

Mice Stem Cells Guided into Myelinating Cells by the Trillions

Greg Benton - noemail@myelin.org — Mon, 26 Sep 2011 20:00:00 GMT

The findings will be published in the online issue of Nature Methods, Sunday, Sept. 25, at 1 p.m. EST.

"The mouse cells that we utilized, which are pluripotent epiblast stem cells, can make any cell type in body," Paul Tesar, an assistant professor of genetics at Case Western Reserve and senior author of the study, explained. "So our goal was to devise precise methods to specifically turn them into pure populations of myelinating cells, called oligodendrocyte progenitor cells, or OPCs."

Their success holds promise for basic research and beyond.

"The ability of these methods to produce functional cells that restore myelin in diseased mice provides a solid framework for the ability to produce analogous human cells for use in the clinic," said Robert H. Miller, vice dean for research at the school of medicine and an author of the paper.

Tesar worked with CWRU School of Medicine researchers Fadi J. Najm, Shreya Nayak, and Peter C. Scacheri, from the department of genetics; Anita Zaremba, Andrew V. Caprariello and Miller, from the department of neurosciences; and with Eric. C. Freundt, now at the University of Tampa.

Myelin protects nerve axons and provides insulation needed for signals to pass along nerves intact. Loss of the coating results in damage to nerves and diminished signal-carrying capacity, which can be expressed outwardly in symptoms such as loss of coordination and cognitive function.

Scientists believe that manipulating a patient's own OPCs or transplanting OPCs could be a way to restore myelin.

And, they have long known that pluripotent stem cells have the potential to differentiate into OPCs. But, efforts to push stem cells in that direction have resulted in a mix of cell types, unsuitable for studying the developmental process that produces myelin, or to be used in therapies.

Tesar and colleagues are now able to direct mouse stem cells into oligodendrocyte progenitor cells in just 10 days. The team's success relied upon guiding the cells through specific stages that match those that occur during normal embryonic development.

First, stem cells in a petri dish are treated with molecules to direct them to become the most primitive cells in the nervous system. These cells then organize into structures called neural rosettes reminiscent of the developing brain and spinal cord.

To produce OPCs, the neural rosettes are then treated with a defined set of signaling proteins previously known to be important for generation of OPCs in the developing spinal cord.

After this 10 day protocol, the researchers were able to maintain the OPCs in the lab for more than a month by growing them on a specific protein surface called laminin and adding growth factors associated with OPC development.

The OPCs were nearly homogenous and could be multiplied to obtain more than a trillion cells.

The OPCs were treated with thyroid hormone, which is key to regulating the transition of the OPCs to oligodendrocytes. The result was the OPCs stopped proliferating and turned into oligodendrocytes within four days.

Testing on nerves lacking myelin, both on the lab bench and in diseased mouse models, showed the OPCs derived from the process flourished into oligodendrocytes and restored normal myelin within days, demonstrating their potential use in therapeutic transplants.

Because they are able to produce considerable numbers of OPCs – a capability that up until now has been lacking - the researchers have created a platform for discovering modulators of oligodendrocyte differentiation and myelination. This may be useful for developing drugs to turn a patient's own cells into myelinating cells to counter disease.

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The National Institutes of Health, CWRU School of Medicine, the New York Stem Cell Foundation, the Myelin Repair Foundation, the National Center for Regenerative Medicine, and the Case Comprehensive Cancer Center funded the research.

26-Sep-11 1:00 PM

Clinical Successes and New Technologies Revive Gene Therapy

Greg Benton - noemail@myelin.org — Fri, 09 Sep 2011 20:00:00 GMT

***Click the link below to read the article.***

http://www.genengnews.com/analysis-and-insight/clinical-successes-and-new-technologies-revive-gene-therapy/77899451/

9-Sep-11 1:00 PM

Current and Future Pharmacological Treatment Strategies in X-Linked Adrenoleukodystrophy

Greg Benton - noemail@myelin.org — Thu, 28 Jul 2011 17:00:00 GMT

Click here to view the article.

28-Jul-11 10:00 AM

Adrenolukodystrophy Advanced Nutrition Food Therapy

Kimberly Kushner - noemail@myelin.org — Sun, 12 Dec 2010 21:00:00 GMT

Click here to view the article.

Find Kimberly Kushner on Facebook! Click the link below.

http://www.facebook.com/kimberly.kushner.nutritionist

12-Dec-10 1:00 PM

New research for childhood genetic disorders

Patti Chapman - noemail@myelin.org — Tue, 02 Nov 2010 18:00:00 GMT

New research for childhood genetic disorders

November 2nd, 2010 11:13 pm PT

Do you like this story?

Genetic disorders are diseases caused by an abnormality in an individual's DNA, and can be passed from parents to children. Recently, gene therapy has made major inroads in stabilizing two boys with a rare inherited genetic disorder known as Adrenoleukodystrophy (ALD). This genetic disorder is passed to sons from symptom-free mothers (carriers), although in rare cases mothers develop very mild symptoms between the ages of 40 to 60.

The 1992 movie Lorenzo's Oil brought ALD to the attention of the world. ALD affects approximately one in every 18,000 boys worldwide, an incidence more frequent than ALS (Lou Gehrig's disease). It is caused by a mutation in the ALD gene and is a crippling and fatal brain disorder in young boys between the ages of 4 and 10. The good news is that the recent gene therapy study mentioned above suggests that the progressive symptoms from ALD can be halted by correcting the mutated gene.

Michael Benton, who grew up in Pacific Palisades, California, tested positive for the mutated ALD gene at 8 years old. Although he showed no symptoms of the illness, the presence of the mutated gene meant that he had a 50% chance of developing symptoms as a child and a 50% chance of developing symptoms as an adult. Upon being diagnosed with ALD, he participated in a five-year trial for Lorenzo's oil, and remained symptom free until college. Because the ALD gene became active in adulthood, he is now considered to have Adrenomyelonueropathy (AMN), the late-onset form of the disease.

Patti Chapman, Michael's mother, lost her brother Bobby to ALD when he was 5 years old and her brother Richard to AMN when he was 44 years old. She is now facing her son's progressive development of the same life-threatening genetic disorder. She is currently the president of The Myelin Project whose goal is to accelerate research on preventing nerve damage and repairing myelin. There will be a fundraiser on February 26th, 2011 at Corpus Christi Church in Pacific Palisades, California to raise money for a similar gene therapy study for AMN. The gene therapy treatment is thought to be suitable for adults with the late-onset form of the condition.

This type of research is promising because rather than trying to find a genetic match from unrelated donors, which involves serious rejection risks and side effects, each patient will be using his own stem cells, thus eliminating the aforementioned risks. In addition, this is a powerful new approach to altering defective DNA. Such gene therapy could be used for other genetic conditions such as sickle cell anemia and cystic fibrosis, as well as a number of crippling myelin-damaging diseases including multiple sclerosis, canavan disease and krabbe disease.

2-Nov-10 11:00 AM

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

Candace Root - noemail@myelin.org — Fri, 18 Dec 2009 19:50:47 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

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18-Dec-09 11:50 AM

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

Candace Root - noemail@myelin.org — Thu, 05 Nov 2009 22:38:36 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

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5-Nov-09 2:38 PM

Help Someone with a Demyelinating Disease

Kim Hodgson - noemail@myelin.org — Wed, 09 Sep 2009 19:00:00 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 - noemail@myelin.org — Wed, 01 Jul 2009 16:00:00 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 - noemail@myelin.org — Tue, 19 May 2009 17:56:40 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

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19-May-09 10:56 AM

Treating The Other Face of X-ALD -- Adrenomyeloneuropathy

Margaret Weis - noemail@myelin.org — Tue, 19 May 2009 17:30:00 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 - noemail@myelin.org — Thu, 16 Apr 2009 18:00:00 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 - noemail@myelin.org — Mon, 29 Dec 2008 20:00:00 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 - noemail@myelin.org — Wed, 10 Dec 2008 22:00:00 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 - noemail@myelin.org — Wed, 10 Dec 2008 22:00:00 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

Advances In Hematopoietic Stem Cell Based Gene Therapy For The Treatment of Metachromatic Leukodystrophy

Alesssandra Biffi, MD - noemail@myelin.org — Wed, 10 Dec 2008 22:00:00 GMT

Dr. Alessandra Biffi, of the San Raffaele Scientific Institute, Milan, Italy, reported on her group’s advances in hematopoietic stem cell based gene therapy for the treatment of metachromatic leukodystrophy.

Metachromatic Leukodystrophy (MLD) is a demyelinating disease due to inherited deficiency of arylsulfatase A (ARSA). In the absence of effective therapies, MLD is a disease with an urgent medical need. This is particularly important, since donor hematopoetic stem cell (HSC) transplant in MLD has met with mixed results. In a mouse model, ARSA can be transplanted to the central nervous system. Specifically, the ARSA gene was transplanted to HSC of MLD mice. Then the modified HSCs were transplanted back to the MLD mice. After this treatment, the manifestations of MLD were corrected. Bboth the feasibility and safety of this therapeutic strategy were tested in a pre-clinical model. Using HSCs from human MLD patients, a similar strategy has successfully corrected the ARSA deficiency. These data have provided the basis for the next step, giving the treated HSCs back to an MLD patient. This clinical trial of HSC gene therapy for the treatment of MLD patients is expected to start by the second quarter of 2009. As in the mouse model, the protocol is based on isolating HSCs from the MLD patients, transplanting the normal ARSA gene into the HSCs, then giving the cells back to the patient. This strategy is expected to avoid the potential complications of graft vs. host disease and to achieve sustained long term ARSA expression in MLD patients.

10-Dec-08 2:00 PM

Long-term Outcome of Bone Marrow Transplantation in a Mouse Model of Krabbe’s Disease

Yiochi Kondo, PhD - noemail@myelin.org — Wed, 10 Dec 2008 22:00:00 GMT

Dr. Yoichi Kondo, University of Wisconsin, discussed the long-term outcome of bone marrow transplantation in a mouse model of Krabbe’s disease

Bone marrow transplantation (BMT) or umbilical cord blood transplantation are the only therapies available to date for globoid cell Leukodystrophy (GLD, Krabbe disease). However, they do not cure the disease. To discover why, Dr. Kondo investigated twitcher (twi) mice, a model of GLD. If BMT was performed on these mice 10 days after birth, the twi mice lived for an average of 168 days. Those animals that did not receive BMT lived for only about 51 days. When compared to control twi mice, animals that received BMT had better myelin formation at 45 days of age. However, at 200 days, the mice receiving BMT had extensive loss of myelin and displayed evidence of progressive neuronal damage. This study demonstrates that enzyme replacement by simple BMT is not sufficient for the long-term treatment of GLD.

10-Dec-08 2:00 PM

Novel Strategy for Gene Therapy of Globoid Cell Leukodystrophy.

Alessandra Biffi, MD - noemail@myelin.org — Wed, 10 Dec 2008 22:00:00 GMT

Dr. Alessandra Biffi also reported on a novel strategy for gene therapy of globoid cell leukodystrophy.

Globoid leukodystrophy (GLD), also known as Krabbe Disease, is a genetic disease whose victims are unable to make galactocerebrosidase (GALC). The GALC enzyme helps to metabolize some of the lipid components of myelin. A gene therapy strategy for GLD based on hematopoietic stem cells (HSC) is under development. The gene transfer can be made by using a virus, called the lentivirus (LV). This has proved to be an efficient method to transfer the GALC gene to HSCs The treated cells are able to express GALC at levels that are very high. However, this high level of GALC expression may also cause functional impairment, or even death, of the HSCs. It could be that the unnaturally high levels of GALC expressed by the treated HSCs are damaging to them. However, GALC doesn’t damage the descendents of HSCs. This suggests a new therapeutic strategy that is now being tested. The LV vector has been modified, so that when the GALC gene is transplanted to the HSCs, the GALC gene lies dormant until the HCS divides and differentiates. That is, the GALC gene is not expressed until it is safe to do so. This novel strategy avoids GALC damage in HSCs, while allowing sustained GALC expression in the progeny of the HSCs. Evaluation of this strategy is in progress.

10-Dec-08 2:00 PM

Inflammatory Neurodegeneration Caused by Inactivation of Peroxisome Function in Oligodendrocytes

Celia Kassman, PhD - noemail@myelin.org — Wed, 10 Dec 2008 22:00:00 GMT

Dr. Celia Kassmann of the Department of Neurogenetics, Max-Planck-Institute of Experimental Medicine discussed her recent studies on inflammatory neurodegeneration caused by inactivation of peroxisome function in oligodendrocytes.

X-linked adrenoleukodystrophy (X-ALD) is the most frequent juvenile leukodystrophy. It is caused by mutations of the adrenoleukodystrophy protein (ALDP), a peroxisomal membrane protein of unknown function. Attempts to create a mouse model for X-ALD by inactivating the ALDP gene have failed. Although ALDP deficient mice accumulate very long chain fatty acids (VLCFA) inflammatory brain demyelination does not occur. Dr. Kassmann investigated how peroxisomes in oligodendrocytes (the cells that form myelin in the brain) help maintain CNS myelin. Her laboratory generated mutant mouse that lacked functional peroxisomes only in oligodendrocytes. She found that peroxisomes in these cells are essetial for maintaining white matter (myelin) tracts. The mutant mice developed normally, but within several months exhibited ataxia, tremor, and premature death. They also showed widespread axonal degeneration, progressive subcortical demyelination, and a strong brain inflammation. The exact function of oligodendroglial peroxisomes is still unknown. But, Dr. Kassmann's studies suggest that functional peroxisomes are required for axonal support.

10-Dec-08 2:00 PM