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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.

###

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, 02 Feb 2012 00:11:25 GMT

DONATE to the CAUSE: The most effective way to make an immediate impact is to fund myelin research by making a charitable contribution directly to our organization of any amount you can afford. All donations are fully tax-deductible. Just click the button below: VOLUNTEER and help us 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. SPREAD the WORD: One of our goals is to raise awareness of demyelinating diseases which affect more than 2 million people worldwide.. Like us on Facebook and keep the conversation going. Tell your friends and family about us and what we do. Have them visit us online for more...

A Letter from the President

Tue, 31 Jan 2012 23:16:56 GMT

May 15, 2010 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...

January News

Tue, 31 Jan 2012 18:35:44 GMT

Myelin News January 27th, 2012 Family of ALD sufferers pledge to raise money for The Myelin Project 11:30am Friday 27th January 2012 By Andy Martin Two brothers are following in their father's footsteps and becoming trustees of a global charity. Charles and Harry Fynn of Bournemouth have pledged to throw themselves into fundraising for The Myelin Project and to help raise awareness of its work. It is a cause close to their heart for a very good reason as both young men carry the rogue gene for ALD (adrenoleukodystrophy). It has been described as a cerebral timebomb which was once considered fatal. That was until the parents of an American sufferer, Lorenzo Odone, refused to accept the prognosis that there was no hope for their son. The outcome of their search for a cure was a special therapy that became known as Lorenzo's Oil. READ MORE... January 23rd, 2012...

Ways to Donate

Wed, 25 Jan 2012 02:19:43 GMT

We appreciate your consideration of donating to our organization. The Myelin Project is a 501(c)(3) non-profit organization, tax ID# 52-1545992; all donations are tax-deductible to the full extent of the law. We sincerely thank you for your contribution. The Myelin Project has partnered with Network for Good to securely process your online credit card donation via the Network for Good Donor Advised Fund. This transaction will appear on your credit card statement as Network for Good.All online donations are made through a secure server hosted by the Network for Good, a BBB Accredited CharityFor more information on the Network For Good, please click the NFG link. SSL certificates from GeoTrust are the ultimate online security and trust solution delivering both 256-bit encryption and the True Site™trust mark providing third-party website identity validation. By Mail: The...

SCIENTIFIC ADVISORY PANEL

Sat, 21 Jan 2012 01:31:41 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...

Videos

Fri, 20 Jan 2012 02:29:06 GMT

Luigi Naldini, San Rafaele Telethon Institute for Gene Therapy, and Member of The Myelin Project Scientific Advisory Board.

The 14th Annual Hammerfest Triathlon

Lorenzo's Legacy

Public Service Announcement - The Giving Back Series

Research Progress Reports

Tue, 10 Jan 2012 20:33:14 GMT

2011 News from the Laboratory Gianvito Martino Martino's lab has contributed to the field of stem cell biology by demonstrating that adult neural stem/precursor cells (NPCs) posses a constitutive and inducible 'immunotrophic' signature which makes these cells - once in vivo transplanted - capable to protect the central nervous system (CNS) from inflammatory and degenerative insults. The concept of therapeutic plasticity emerged as the capacity of in vivo transplanted NPCs to act as therapeutic weapons not only by replacing damaged cells but also by promoting neuroprotection via the release of immunomodulatory and neurotrophic molecules. Gianvito Martino, MD Institute of Experimental Neurology Division of Neuroscience DIBIT - II San Raffaele Scientific Institute Milan Annik Baron Our team investigates the cellular and molecular mechanisms of...

December News

Fri, 06 Jan 2012 00:03:33 GMT

Myelin News December 26th, 2011 New Drugs Raise Hope for Patients with MS By LAURIE TARKAN Published: December 26, 2011 Three years ago, Kristie Salerno Kent, a singer-songwriter, was standing in a security line at the airport on her way home from a gig when her legs went numb. From the waist down, it felt as though I was trying to walk through a bowl of oatmeal, said the 38-year-old musician, who has multiple sclerosis. Read more... December 23rd, 2011 Doctor Reverses MS in 9 Months by Eating These Foods Through her research into MS, Dr. Wahls discovered that, for some unknown reason, in addition to the commonly known symptoms, MS patients' brains also tend to shrink. This roused her curiosity, and led her to research other diseases that have similar brain shrinkage, namely Huntington's, Parkinson's...

2011 News from the Laboratory

Thu, 05 Jan 2012 23:39:17 GMT

Gianvito Martino Martino's lab has contributed to the field of stem cell biology by demonstrating that adult neural stem/precursor cells (NPCs) posses a constitutive and inducible 'immunotrophic' signature which makes these cells - once in vivo transplanted - capable to protect the central nervous system (CNS) from inflammatory and degenerative insults. The concept of therapeutic plasticity emerged as the capacity of in vivo transplanted NPCs to act as therapeutic weapons not only by replacing damaged cells but also by promoting neuroprotection via the release of immunomodulatory and neurotrophic molecules. Annik Baron- Everkoren Our team investigates the cellular and molecular mechanisms of myelin repair in the central nervous system (CNS), with a strong emphasis on the role of stem cells from both the CNS and PNS in the remyelination of demyelinated lesions. Over the past years, we demonstrated a contribution of the endogenous adult neural...

The Myelin Project's 2011 Annual Conference

Thu, 22 Dec 2011 05:08:12 GMT

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, the U.S. Myelin Project, The British Trust for The Myelin Project, The Myelin Projekt Deutschland and Il Progetto Mielina. We hosted a very productive meeting in Paris on October 13-15, 2011. Those attending included Dr. Annik Baron-Van Evercorren (France), Dr. Wolfgang Bruck (Germany), Dr. Ian Duncan (USA), Dr. Robin Franklin (UK), Dr. Charles ffrench-Constant (UK), Dr. Jeff Kocsis (USA), Dr. Gianvito Martino (Italy), members of our...

Lorem ipsum survey

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

Objectives:

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