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The Myelin Project's Annual Scientific Conference 2012 on 4-Oct-12 8:30 AM

Thu, 04 Oct 2012 08:30:00 GMT

The Myelin Project's Annual Scientific Conference 2012
Start Date: 4-Oct-12 8:30 AM
End Time: 5-Oct-12 5:30 PM
Location: The Mercure Hotel Frankfurt, FRANKFURT, KELSTERBACH 65451
Event Details:

The Myelin Project's 2012 Annual Scientific Conference is sponsored by:

The Myelin Project will be meeting in Frankfurt, Germany at the Mercure Hotel on October 4th and 5th for The Myelin Project's 2012 Annual Scientific Conference.

The conference aims to induce collaboration among scientists throughout the world that are in the field of neurology who specifcally deal with demyelinating diseases which affect more than two million people worldwide. More information on the event TBD.

The Mercure Hotel Frankfurt

Am Weiher 20

FRANKFURT, KELSTERBACH GERMANY 65451

[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

Your login information is listed below. To log into the site, visit http://www.myelin.org/en/ and enter your Username and Password.

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Password: [password]

If you have any questions please contact the Site Administrator.

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.

###

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

Sisters rally genetic researchers to tackle fatal brain disease

noemail@myelin.org — Fri, 06 Nov 2009 19:00:00 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 22:00:00 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 21:00:00 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 21:00:00 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 21:00:00 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 21:00:00 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 20:30:00 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 20:00:00 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 18:00:00 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 16:00:00 GMT

/attachments/wysiwyg/1/AmericanBrainCoalition.htm

Take Action!

Thu, 03 May 2012 00:25:50 GMT

1. DONATE to the CAUSE: The most effective way to make an immediate impact is to fund myelin research by making a charitable and tax-deductible contribution directly to our organization of any amount you can afford. All donations are fully tax-deductible. Just click the button below: 2. VOLUNTEER and FUNDRAISE: You can make a difference by hosting your own fundraiser in your community, e.g. a bake sale, walk or run-a-thon, fundraiser dinner, etc. in honor of the Myelin Project. We can provide you with whatever you may need to make it happen. Simply email info@myelin.org with your fundraising ideas. Below are links to our brochure and most recent newsletter to help you fundraise. Fundraising Tools : 2011 Year in Review Newsletter The Myelin Project's Official Brochure 3. SPREAD the...

RESEARCH PROJECTS

Fri, 13 Apr 2012 01:17:42 GMT

Research projects currently funded by The Myelin Project: 1. Peroxisomal proliferation and degeneration - A therapeutic approach in AMN mice ***Winner of The Augusto Odone New Investigator Award*** Dr. Celia Kassmann The Max-Planck Institute for Experimental Medicine, Goettingen, Germany Klaus-Armin Nave Max-Planck-Institute of Experimental Medicine, Gottingen, Germany 2. Roles of microgliamacrophages and their therapeutic use in combination with lentiviral gene transfer in Krabbe's Disease Ian Duncan University of Wisconsin, Madison, Wisconsin USA 3. Lorenzo's Oil Clinical Study at the Kennedy Kreiger Institute Dr. Gerald Raymond Kennedy Krieger Institute For more info click here...

2012 Letter from the President

Thu, 29 Mar 2012 23:56:55 GMT

March, 2012 To whom it may concern: Since our initial founding in 1989, we have not, despite our valiant efforts, attained our definitive goal of remyelinating the human central nervous system. This does not mean, however, that we have not made progress. Far from it. We have created a model that closes the gap between the lay person and scientist. Although we have not reached our ultimate objective, we have developed all the prerequisites to reach this goal in the near future. These results are possible through collaboration with our Scientific Advisory Board and our international affiliates: The Myelin Project USA, The British Trust for The Myelin Project, The Myelin Projekt Deutschland and Il Progetto Mielina. The Myelin Project's 10th Annual Scientific Conference in Paris on October 13-15, 2011 was extremely productive to say the least. Those attending included Dr. Annik Baron-Van Evercorren (France), Dr. Wolfgang Bruck...

March 2012 News

Thu, 29 Mar 2012 02:04:49 GMT

Myelin News March 28th, 2012 For Lorenzo's Oil boys: Race at Hammonasset this Sunday inspired by Branford man Published: Tuesday, March 27, 2012 They don't stand out, two pictures in the bright, airy living room of 23-year-old Brian Kelley's Branford home. But they each tell a story. One is a snapshot of 6-year-old grinning, blond-haired Brian on the beach. The other: a photograph of the Kelley house. Hanging from the garage and on the railing of the porch are Welcome Home signs. The photo was taken in 1995. The signs were put up by the Kelley's neighbors. They were welcoming Brian home after a stay of 85 days at St. Raphael's Hospital during which time he turned 7. And that's the story here. Not so much the adrenoleukodystrophy, or ALD, a debilitating genetic disorder, that out of nowhere stripped the insulation, or myelin, protecting Brian's nerves, and with it, his sight, speech and ability to walk. Read more... March 7th, 2012 Disease...

Lorenzo Odone

Fri, 23 Mar 2012 01:32:19 GMT

- Lorenzo - Lorenzo Michael Murphy Odone was born on May 29 1978 in Washington DC. During his first years at Kindergarten his teachers reported problems with his behaviour and a deterioration in his attention span. By 1984, when he was six, Lorenzo's behavioural problems had worsened and he began to suffer from blackouts and memory lapses, symptoms akin to those of stroke victims. A brain scan finally confirmed that Lorenzo was suffering from ALD. The prevailing medical wisdom decreed that children with ALD typically lived for only a few years beyond diagnosis. But Augusto and Michaela Odone refused to accept this, and began an exhaustive quest for an effective treatment. Despite having no medical training, they contacted specialists and combed the scientific journals. In 1987, having taken early retirement, Augusto Odone invented a treatment, in the form of a vegetable oil, that appeared to arrest the progress of ALD. To help with the oil's...

Michaela Odone

Fri, 23 Mar 2012 01:28:30 GMT

- Michaela - 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 lycee. In 1966 she made the list of Outstanding Young Women of America. Michaela had an important role both in developing Lorenzo's 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 Lorenzo's bedside, day in and day out. Her passionate caring for Lorenzo, however, did not prevent her from answering the constant flow of...

Augusto Odone

Fri, 23 Mar 2012 01:27:24 GMT

- Augusto - Augusto Odone was born in Rome in 1933. After a long career in the USA as an economist with the World Bank, he now lives in Italy. He has an honorary degree in medicine. Augusto Odone and his wife Michaela (died in 2000) founded The Myelin Project, an international scientific body which seeks to promote research into those diseases which destroy myelin, the white matter of the central nervous system without which the brain cannot transmit messages to other parts of the body. The goal is to bring hope to those suffering from conditions such as multiple sclerosis and the leukodystrophies (of which ALD is one). The cover of Augusto's Odone's much anticipated book, due to release in Europe in Italian on May 17th, 2011. The translation to English is currently in progress. Lorenzo's Oil, the film starring Nick Nolte as Augusto Odone and Susan Sarandon as his wife Michaela, moved the whole...

The Family, the Film, and the Oil

Fri, 23 Mar 2012 00:35:19 GMT

- The Family - Augusto Daniel Odone (Rome, May 4, 1933) and Michaela Teresa Murphy Odone (January 10, 1939 - June 10, 2000) are the parents of Lorenzo Odone (May 29, 1978 - May 30, 2008), a child afflicted with the illness adrenoleukodystrophy (ALD). They became famous for discovering a treatment using Lorenzo's oil, for their son's incurable illness, and this quest was recounted in the film Lorenzo's Oil (1992).[1] One of Augusto's other children is the journalist Cristina Odone.In recognition of the parents' work, Augusto Odone has received an honorary doctorate from the University of Stirling. He continues to raise funds and drive the scientific task force known as The Myelin Project. Michaela Odone battled cancer for some time, and finally died of lung cancer at 61.[2] Lorenzo died the day after his 30th birthday. He was almost totally paralyzed but was, according to his father Augusto, holding his own. He couldn't see, speak, or...

About the President

Mon, 12 Mar 2012 02:19:20 GMT

Dear Friends and Family of the Leukodystrophy Community, I have been involved with the Myelin Project and served on its Board of Directors since its inception in 1989, after having met Augusto Odone in 1987. Serving on the Board with a very devoted group of people has been inspirational and I look forward to continuing the work of The Myelin Project. Dr. Ian Duncan, will also continue to be involved in the day-to-day operation of The Myelin Project. He will remain as Chairman of the Scientific Advisory Panel, which is a very specialized and expert group of scientists devoted to our Mission. My journey with these illnesses began when I lost my little brother to adrenoleukodystrophy in 1960, and my other younger brother to adrenomyeloneuropathy (AMN) in 2001. I have three sons, Michael, Christopher, and Gregory, my eldest Michael unfortunately inherited the genetic defect. He is now 31 years old and currently...

SCIENTIFIC ADVISORY PANEL

Fri, 09 Mar 2012 20:25:07 GMT

Ian Duncan, BVMS, FRSE, PhD Chair of The Myelin Project Scientific Advisory Panel Professor of 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 Wolfgang Bruck , M.D. Professor of Neuropathology Institute for Neuropathology | University of Gottingen Robert-Koch-Strabe 40 | 37075 Gottingen | GERMANY Email: wbrueck@med.uni-goettingen.de Annik Baron-Van Evercooren, PhD Director, Unit on Disorders of Myelin and Muscle Ion Channels Faculte Pitie-Salpêtrière | Laboratoire des Pathologies de la Myeline INSERM CJF 97-11 | 105 boulevard de l'Hôpital 75634 Paris Cedex 13 | FRANCE | Email: anne.baron@upmc.fr Robin J.M. Franklin, BVetMed, BSc, PhD Professorof Neuroscience, MRC Cambridge Centre for Stem Cell Biology and Regenerative Medicine, University of Cambridge ...

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noemail@myelin.org — Thu, 09 Aug 2007 22:40:38 GMT

Objectives:

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