En Español

Em Português

Myelin is produced by specialized cells:  oligodendrocytes in the central nervous system, and Schwann cells in the peripheral nervous system. Myelin sheaths wrap themselves around axons, the threadlike extensions of neurons that make up nerve fibers. Each oligodendrocyte can myelinate several axons.

The Myelin Project: An Overview

A multinational gathering of families joined the Odones in their efforts. Stricken by one or another of the demyelinating diseases, they refused to be passive victims.   By encouraging cooperation among researchers, and by providing scientists with funding for translational and clinical research.  While The Myelin Project has taken the conventional steps to advance remyelination research, we have taken the unconventional step of encouraging direct interaction between families and scientists, we continuously remind the scientists that there are people waiting for effective treatments.  Research has been focused on efforts to understand mechanisms of myelin repair, with a view to discovering therapies that will permit restoration of damaged myelin.  It is anticipated that these will permit at least partial restoration of function in patients suffering from myelin loss. 

The Myelin Project headquarters has recently been moved to Amarillo, Texas where it is housed on the Texas Tech University Health Sciences Center campus in the Laura W. Bush Womens Research Institute.   The Myelin Project has branches in Germany, Italy, Canada and the United Kingdom as well as an active partnership with the European Leukodystrophy Association, headquartered in France.  Project President, Margaret Weis, receives minimal compensation, members of the Board receive no compensation.  In Britain, Frances Germany, Italy and Canada Project Board members are also volunteers.

Using a motivated, time-conscious approach to attain specific goals, The Myelin Project has set up a Work Group from among the top international laboratories specializing in myelin repair. The Work Group includes researchers from Yale University and the University of Wisconsin at Madison in the U.S., the Istituto Superiore di Sanità and San Raffaele Scientific Institute in Italy, the Hôpital de la Salpêtrière and the Institute Pasteur in France, the Queen's University at Kingston in Canada, the University of Cambridge in the United Kingdom, and the Max-Planck-Institut in Germany.

The Myelin Project targets its funds toward clinically oriented experiments on the cutting edge of remyelination research. Basic research and studies directed toward the advancement of science for science's sake are excluded from Project financing.

We would be grateful if you would consider helping us. Supporting our work will not only be humanitarian, but will also make good business sense:

• We aim to keep our administrative costs to no more than 20% of total receipts, depending on the year.     

• Your donation will go toward financing practically oriented experiments conducted within the framework of a coherent overall plan.   All research proposals are reviewed in advance by our panel of leading experts in the field.

• We promise you that your donation will be well-spent, funding those proposals that are most likely to yield clinically relevant results.

Demyelinating Diseases In Brief

Demyelinating diseases are those in which myelin is the primary target. They fall into two main groups: acquired diseases (i.e., multiple sclerosis) and hereditary neurodegenerative disorders (i.e., the leukodystrophies). Although their causes and etiologies are different, they have the same outcome: CNS demyelination. Without myelin, nerve impulses are slowed or stopped, leading to a constellation of neurological symptoms.

Acquired Diseases

The most common of these is multiple sclerosis (MS), which usually manifests itself between the 20th and 50th years of life. Current estimates are that approximately 2.5 million people worldwide have MS, with between 250,000 and 350,000 cases in the United States, 50,000 cases in Canada, 130,000 cases in Germany, 85,000 cases in the United Kingdom, 75,000 cases in France, 50,000 cases in Italy, and 11,000 cases in Switzerland.

MS attacks the white matter of the central nervous system (CNS). In its classic manifestation (90% of all cases), it is characterized by alternating relapsing/remitting phases with periods of remission growing shorter over time. Its symptoms include any combination of spastic paraparesis, unsteady gait, diplopia, and incontinence.

Hereditary Neurodegenerative Disorders

This category includes the eight identified leukodystrophies metachromatic leukodystrophy, Refsum's disease, adrenoleukodystrophy, Krabbe's disease, phenylketonuria, Canavan disease, Pelizaeus-Merzbacher disease and Alexander's disease. The first six are storage disorders. The lack or the malfunctioning of an enzyme causes a toxic buildup of chemical substances. In Pelizaeus-Merzbacher disease myelin is never formed (dysmyelination) because of a mutation in the gene that produces a basic protein of CNS myelin. The etiology of Alexander's disease remains largely unknown.

The clinical course of hereditary demyelinating disorders, which usually tend to manifest themselves in infancy or early childhood, is tragic. Previously normal children are deprived, in rapid progression, of sight, hearing, speech, and ambulation. Equally tragic is their prognosis: death within a few years.

A number of government agencies and private foundations currently support research on various myelin diseases. Some efforts focus on identifying the cause of individual diseases; others are directed toward developing therapies for arresting disease progress or preventing onset.

In contrast, little attention is being given to the problems of repairing damage already done by the disease and of restoring lost function. Laboratories working on remyelination are relatively few in number and their programs are under-funded. In addition, rivalry among researchers is intense. Laboratories tend to work in isolation, learning of each other's progress through medical journal articles which are usually published a year or two after experiments are completed. This fragmented approach is clearly unsuitable to regenerating CNS myelin, a complex task which requires multi-disciplinary skills.

Scope and Strategies

Scope

Since myelin loss leads to the reduction or blockage of nerve impulse conduction, myelin regrowth would logically restore conduction in diseases for which therapies capable of halting demyelination have already been found (e.g., phenylketonuria, Refsum's disease, which are treatable, mainly through restricted diets). But regenerating myelin may also be beneficial in demyelinating diseases for which no effective treatment has been developed (e.g., multiple sclerosis). Indeed, the new myelin may well be able to withstand attack by the primary demyelinating agent, either permanently or for a long period of time.

Strategies

To attain its objectives, The Myelin Project relies on three major strategies: prompting researchers to work as a team and coordinating their research efforts, promoting interaction between researchers and laypeople, and rapid financing of practically oriented experiments.

The annual meetings of the Work Group as well as several teleconferences during the year provide a forum for exchanging information on member laboratories' respective work programs. They also serve to establish or strengthen personal ties among participating researchers, who now have come to see themselves as teammates rather than rivals. Today researchers visit each other's laboratories and join forces to conduct Project-financed experiments. They also exchange papers on the progress of their experiments continuously, even before publication.

Interaction between researchers and laypeople has been achieved at different levels. The presence of informed representatives of the families at scientific meetings (a quiet reminder that the objective of medical research is to save human lives) has served to provide researchers with increased motivation and focus. In some instances, we have helped researchers solve practical problems (e.g., assistance in locating and obtaining rare animal models).

The Myelin Project staff makes it a point to process requests for experiment funding rapidly—in a matter of weeks, rather than years. Two factors contribute to cutting down processing time:

• Proposals are reviewed within the Work Group, rather than by external committees, with the occasional addition of one outside reviewer. Reviewers are given a 3-week deadline for sending in their comments.

• The Myelin Project is managed in a businesslike, "no red tape" fashion. Being victims themselves, or relatives of victims of a demyelinating disease, the members of our boards of directors are naturally committed to moving research along at the fastest possible pace.

 

Last but not least, The Myelin Project is not a foundation in the traditional sense. We are not here to stay, but are working very hard to put ourselves out of business and demolish the project as soon as possible.