In this issue:

	A message from our Foundation chair
	Together, we are stronger on the journey to a cure
	The Bermeos
	2007 Annual Meeting
	Clinical trials: update on CellCept® (mycophenolate) and myasthenia gravis
	Spreading the word in Spain
	Across the country, great things are happening!
	Pakistani plasmapheresis project recognized
	MGFA has a new Web site!
	MG Awareness Poster
	Did you know?
	Calendar
	Associations
	Make a gift to MGFA
	Subscription information

Do you remember when you first heard about myasthenia gravis? It is my guess that the majority of our Foundation Focus readers (excluding medical professionals) first learned about myasthenia gravis when they or someone they knew were diagnosed with the illness. At MGFA, we continually strive to promote greater awareness of MG by educating people about the illness before they or a loved one even experience myasthenia symptoms, and to alert them to what the symptoms are should they occur. Our Communications Committee has been diligently strategizing on various ways MGFA can enhance awareness nationally and in local areas—especially during MG Awareness Month, June 2007.

At the national level, we will target the top radio stations and newspaper markets. At the local level, we will provide media packets to chapter leaders. These packets will contain posters, sample press releases, public service announcements (PSAs), and “how-to” recommendations and resources to reach media personnel within their community. If you are not affiliated with an MG chapter, I encourage you to participate in this awareness campaign as outlined in the article “Together, We Are Stronger on the Journey to a Cure” inside this issue.

Future goals include production of new print ads and PSAs for television. We are searching for a public relations and/or advertising agency to help in these endeavors. If you know of a firm interested in pro bono assistance for our nonprofit organization, please contact our national office.

In 1952, Jane Dewey Ellsworth was the crusader who founded the Myasthenia Gravis Foundation of America. One of her driving concerns was the lack of information available about this all-too-often unknown illness. Her vision of MGFA was that it would lead the charge in broadcasting information about MG to as wide a spectrum as possible, including the media, the medical arena, patients, and the general public. In its long history, MGFA has never lost sight of that vision.

Over the years, various media tools and spokespersons have helped spread the word about MG. These tools have included posters, PSAs, and articles featuring Tony Randall, Ann-Margret, Suzanne Rogers, and Cherokee Chief Wilma Mankiller, to name just a few.

MGFA has also been active at both the local and national level through what might seem to be more routine—although equally important—outreach activities. For example, our national office sends out more than 1,500 informational packets annually to patients, physicians, nurses, and the general public. Our chapters combined offer more than 100 support groups and educational programs across the U.S. on a regular basis. Our members regularly speak with their local media to educate their community about the disease and their experiences with it.

To enhance these and many other “regular” outreach activities, in 1992 we began actively promoting an MG Awareness Week. Since that time, this “week” has grown into a full month of dedicated, focused educational and outreach activities by our entire community: the national Foundation, our chapters, and many of our members. In fact, through these shared efforts, June has been formally declared as MG Awareness Month in many cities, counties, and states across the country.

This year, we want to focus our community with the message that “Together, we are stronger on the journey to a cure.” Help us get this message out and educate everyone about MG!

You will find a link to a copy of our June Awareness poster—featuring the Bermeo twins from our East Central Florida Chapter—enclosed in this newsletter. We urge you to post it in a prominent location in your community. Get the conversation started!

We have also created a media kit that includes a sample press release, MG fact sheet, PSA script for radio announcers, resource links to local radio stations and daily and weekly newspapers, and recommendations on how to contact local media. These packets will be forwarded automatically to all of our local chapters.

This issue of Foundation Focus illuminates a family well known to our myasthenia community: Ashley and William Bermeo and their twin sons, Javier and Miguel. The boys were diagnosed with MG as infants and, since that time, the whole family has embarked on a journey of learning about and promoting awareness of myasthenia gravis.

The challenges of raising a child with a chronic, life-altering, neuromuscular disease are many. For the Bermeos, they added to the usual uncertainties that accompany all first-time parents. These challenges ranged from the mystifying time of recognizing that their children were ill to their diagnosis and learning the boys’ tolerances for their medications and activities.

Javier and Miguel’s first forays into school were not especially easy or happy; it was difficult for Ashley and William to convey the intricacies of MG to school and county officials involved in their education. Javier’s myasthenia has been more involved than his brother’s; he has had a thymectomy, and his treatment consists of CellCept® and Mestinon®. Miguel requires only Mestinon® at this time.

The boys, who are now six and a half years old, are experiencing some separation issues over their father’s second deployment to Iraq. This stress can wreak havoc with their MG as they miss their dad, who has been away for several months. They are also susceptible to the usual illnesses that affect children in school, but there is nothing “normal” about dealing with illness when myasthenia is involved. Their MG worsens, producing weakness to the point of not being able to go to class or enjoy their activities. This year, they have “shared” the flu twice. It is for this reason that their parents have decided to home school next year, hoping to minimize the boys’ exposure to illness and subsequent absences from classes while being more able to accommodate their fatigue and medication requirements in a more personalized and controlled environment.

During their strong times, the boys enjoy swimming, soccer and—new last year—snorkeling! Javier and Miguel are Cub Scouts, too, so there is no shortage of opportunities for them to experience activities with friends.

In many cases, this would be the end of the tale of a young family striving to integrate a difficult situation into the fabric of their lives. However, the Bermeos are not just any family. Ashley and William coach the boys’ soccer team and Ashley assists with the Cub Scout den (while she is finishing her bachelor’s degree in education). Javier, Miguel, and Ashley all participate in seminars for students of the St. Augustine University of Physical and Occupational Therapy and nursing students of Daytona Beach Community College. According to their mom, the boys have become quite adept at describing their condition, as well as how to treat it. The family takes a scrapbook with MGFA chapter members’ stories and photographs to share with the students so that they can get a larger sense of how myasthenia gravis affects people of all ages.

It was three years before the Bermeos met anyone else with myasthenia. William and Ashley remember the isolation and uncertainty of the boys’ early years, and made a conscious decision to reach out to other families when they decided to become involved in MGFA. Since January 2006, the parents co-chair the East Central Florida Chapter of MGFA and take turns leading the Palm Court Support Group. In William’s absence, his colleagues from work and the military step in to assist Ashley with newsletters, support meetings, and anything else that is required. A group of six families from around the country who have children with myasthenia are in close contact with the Bermeos, and they all share information and support. About every four months, the family travels from Florida to Duke University in North Carolina for the highly specialized follow-up care that the boys require—a drive of nine hours one way!

2007 Annual Meeting
of the
Myasthenia Gravis Foundation of America, Inc.

April 19-21, 2007

Join more than 150 chapter leaders, patients and their families, medical professionals, and caregivers who will attend the 2007 MGFA Annual Meeting in our nation’s capital.

The selection of the meeting venue, hotel, meals, and program have incorporated many of the comments and suggestions made after last year’s meeting. This year’s program is designed to address specific areas of interest, including advancements in medical diagnosis and treatment, research, and emotional and financial impacts on both patient and families. In addition, there will be chapter-focused breakout sessions as well as sessions for the newly diagnosed and their families. We will also take advantage of being in our nation’s capital to hear directly from those involved in advocacy efforts.

This will be a great meeting and we look forward to seeing you there!

Registration
The registration deadline is April 2, 2007. No refunds will be considered after this date. Visit www.myasthenia.org/mgprogram_annualmeeting.cfm and use either the online or the mail-in registration form.

Location
Doubletree Hotel Crystal City
300 Army Navy Dr
Arlington, VA 22202
(703) 416-4100
www.doubletreecrystalcity.com

Wednesday, April 18, 2007

• Board of Directors Meeting
• Registration open
• Town Hall Meeting

Thursday, April 19, 2007

• General Assembly & Business Meeting
• Session I: Research Update by Robert Pascuzzi, MD, MSAB Chair
• Breakouts: Chapter Development Sessions
  • Financial Planning by Mike Elder, MSW, Director, Financial Education Partnerships
  • Chapter Oversight and Fiscal Management by Beth Tabak, CPA, Partner, Ullbrich & Co.
  • Board Development, Recruitment and Retention by Robyn Spearot, Marilyn Buckner, and Sam Schulhof, members of the Board Development/Nominating Committee
• Breakouts: MG – The Basics
  (moderated by NAB Chair Wilma Koopman and other members of the Nurses Advisory Board)
  • Introduction to MG: Questions and Answers
  • Lifestyle Management

Friday, April 20, 2007

• Session II: National Health Council by Myrl Weinberg, President, National Health Council
• Session III: IVIG Treatments by Marinos Dalakas, MD, Chief, Neuromuscular Diseases Section, NINDS, MSAB member
• Session IV: Psychosocial Aspects of Living with MG by Lindsay Knudsen, MS, 2006 Student Fellowship Award winner
• Session V: A Pragmatic Approach to the Treatment of the MG Patient by Allan Weiss, MD, 2006 MGFA Doctor of the Year, MSAB member
• Lunch: Advocacy and Volunteer Health Organizations by Marc Boutin, Executive Vice President, National Health Council (invited speaker)
• Awards Banquet

Saturday, April 21, 2007

• Session VI: Update on MG Treatments moderated by Audrey Penn, MD, Deputy Director, NINDS; MSAB member panel to include Marinos Dalakas, MD, and Daniel Drachman, MD
• Session VII: Update on the Thymectomy Trial for MG by Henry Kaminski, MD, Chairman, Department of Neurology, St. Louis University Medical Center, MSAB member
• Session VIII: Ask the Doctor moderated by Henry Kaminski, MD, with Drs. Penn, Pulley, Dalakas, and Weiss
• Chapter Networking Roundtables
  • Fundraising Strategies in Federated Organizations by Nancy Law, Executive Vice President, Programs & Services, National Multiple Sclerosis Society
  • Chapter and Support Group Management by Marie Ronnlof, Chapter Liaison, MGFA Board of Directors
  • How to Be an Advocate by Janet A. Myder, MPA, Board of Directors, Maryland/DC/Delaware Chapter

The neurological literature contains a series of reports authored by clinical experts from around the world who have attempted to document the effect of mycophenolate on MG. Up until 2006, these reports have been what we call anecdotal and have been collections of patients treated with mycophenolate who have been observed by their clinicians and had those observations noted and reported.

These various reports have suggested that mycophenolate is a useful drug in MG patients, with a general observation that up to 75% of patients benefit and the improvement begins within the first few months. While such reports are encouraging and have an impact on how the drug is used in MG patients, it is always important to recognize that there are limits to what we can truly know based on anecdotal reports, especially those involving treatment of a disease as complex and variable as myasthenia gravis.

Many of our patients are being treated with multiple medications simultaneously, and the disease has a tendency to come and go spontaneously. These make it challenging to know if a patient’s improvement is really due to a specific treatment or not. Furthermore, clinicians recognize that treating physicians and their patients are optimistic and enthusiastic about the approach to treatment and wish for treatments to work well. When we want a treatment to work and have confidence in it, then we tend to conclude that there may be more improvement than is, in fact, occurring. For such reasons, we always look to be more scientific and objective whenever possible.

The scientific and objective approach to studying the effect of treatment involves the testing of a therapy in a controlled and blinded trial. To put it simply, we identify patients and, if they consent to participation in the trial, we randomize them to one of two treatment options: one group gets the treatment and the other does not (the second group typically receives a placebo). The patients are then observed over a defined period of time and their condition is evaluated. In diseases like MG, there is concern that patients may not do well if they are randomized to a treatment that consists of a placebo, so we try to structure a study with sufficient safeguards that we think will allow patients to remain healthy.

Another key component to a good clinical trial is that of blinding. Blinding is a process of having the clinician not know in advance whether the patient is on the real drug or the placebo. Whenever possible, it is best to have the patient blinded as well. In the case of the thymectomy trial, in which some patients get thymectomy and some do not, it is obviously not possible to give the patients a placebo surgery, and so the patients are not blinded; those who have surgery certainly know it. But the clinicians are blinded in that study and those evaluating the patients must do so without knowing who has and has not had a thymectomy. In the end, the observations should be more objective, less biased, and more meaningful.

Controlled clinical trials are complicated to design and can be difficult to perform. If one looks back over the past 60 years, it is the case that most treatment options for myasthenia gravis have not been tested with truly optimal prospective controlled clinical trials. Prednisone, for example, has never been optimally studied in such a fashion, in part because it is in common usage and the overwhelming belief is that it is a very effective treatment.

The good news is that we have entered a new dawn of thinking about treatment of MG, and the medical community and our patients are looking to be as scientific and objective as possible. Accordingly, we are looking to perform controlled and blinded clinical trials whenever possible to try to fully understand the relative usefulness and safety of new treatment options for MG. One old treatment option is finally being tested in a large controlled clinical trial (thymectomy).

We have also been fortunate to have had clinicians perform two controlled and blinded clinical trials to evaluate the role of mycophenolate in the treatment of MG. Members of the MGFA Medical/Scientific Advisory Board (M/SAB) are reviewing the results of these two recently-completed clinical trials. While there is currently no consensus regarding the optimal role of CellCept® in treating MG patients, the results of these studies will help guide our usage of the drug. Additionally, the results of these studies will likely have an impact on the design of future clinical trials of CellCept® and other drugs therapies in MG. I have summarized in general terms the overall results of these two trials below. The full study results will be fully presented to the scientific and medical community and published in 2007.

Two clinical trials and results

In both studies, patients were randomly assigned to receive either CellCept® or a placebo, and were evaluated by blinded investigators. Patients in these studies were also treated with prednisone and pyridostigmine (Mestinon®), and some had previously undergone thymectomy. One study lasted three months and the other lasted nine months.

The two studies showed similar results in that patients treated with CellCept® (along with prednisone and Mestinon®) had an outcome similar to those patients who did not receive CellCept®. While the two studies did not demonstrate a benefit from CellCept®, it is important to look carefully at all of the data and consider the type of patients studied, the design of the studies, the impact of prednisone and Mestinon®, the duration of treatment, and many other factors that could influence the results.

What does this information mean to practicing physicians and their patients?

In current clinical practice, there are differing views on the use of CellCept® and other immunosuppressive drug options for treatment of MG. Patients with questions about their own use of CellCept® are advised to discuss their disease management with their individual physicians. Many clinicians feel strongly that CellCept® is an excellent drug for the treatment of myasthenia gravis even though these two studies did not demonstrate a benefit. Patients should also keep in mind that, since these studies have just recently been completed, their physicians may have limited information regarding the specifics of the results. Most physicians wish to have all available information prior to making significant decisions about treatment options for MG patients.

The M/SAB of MGFA has formed an ad hoc committee charged with further exploration of the results of these studies as well as the development of new trials that might better clarify the role of CellCept® in the treatment of MG. This M/SAB “CellCept® Study Group” will provide periodic updates to the MGFA membership. As of this writing, the CellCept® Study Group is exploring several new ways of studying mycophenolate in order to better understand its optimal role in the treatment of MG. I will provide MGFA updates on those activities in the ensuing months.

During the past fourteen months, we have been privileged to communicate with Esther Chiquero Lozano, chairperson of our international affiliate, Asociación Española de Miastenia (AEM) in Córdoba. AEM is the only organization in Spain dedicated solely to myasthenia gravis and, as Ms. Chiquero states, they have been diligently working to “facilitate and spread knowledge of this disease affecting more than 6,000 people in our country, and to improve the quality of life of the patients.” During their approximately three-year existence, they have distributed posters and brochures containing the main symptoms of MG to all the hospitals and Care Attention centers in the country, as well as to neurologists, radio and TV stations, and newspapers. Additionally, they have begun support groups in Córdoba, Jaén, and Murcia, are planning further ones in Madrid and/or Barcelona, and have established a Web site (www.aemiastenia.org).

One of AEM’s larger projects was the production of a manual for patients and their families and neurologists: Aprende a Convivr con la Miastenia. The manual text was written in Spain and reviewed for translation accuracy in the U.S. by Dr. Gaston Da Costa at Indiana University, under the direction of Dr. Robert Pascuzzi. Upon completion of the manual, AEM hosted a presentation ceremony at the Hospital Reina Sofía in Córdoba that included representatives from the Córdoba health department, Spanish Federation of Neuromuscular Diseases, Federation of Rare Diseases, and members of the neuropediatrics and neuromuscular pathology sectors.

“Together we are stronger….”

When the Foundation says that we are stronger when we work together, what exactly are we talking about? In honor of this year’s June is MG Awareness Month campaign, I want to briefly explore the above statement to see just how true it really is and what it really means.

MGFA was founded on the idea that through a shared purpose we will find ourselves in a future free of this disease. Our mission goes on to name several ways in which we try to fulfill this vision: programs of support, public information, research, and education.

Each of these areas—supporting one another, informing the public, funding meaningful research, and educating doctors, nurses, neighbors… everyone about this disease—requires us to all work together. What we do to defeat this disease cannot be accomplished without everyone’s help and everyone working together.

Only through the efforts of our chapters, our members, our volunteers, and you, working together, do we bring full meaning to the statement “Together we are stronger on the journey to a cure.”

What you will find below are just a few examples of the many activities that we use to collectively strengthen one another.

Last summer, our Nurses Advisory Board and our Alabama Chapter organized the 12th Annual “All Eyes on Myasthenia” nursing workshop. Through the joint efforts of many members involved with the Advisory Board or the chapter, this event educated almost 50 nursing professionals on the symptoms, diagnosis, and treatment of myasthenia gravis. Imagine the effect this education will have on the lives of MG patients these nurses see in the future.

Another “event” that took place last summer was our Pacific Northwest Chapter’s “Dr. No” non-event. The chapter asked their membership to collectively skip a doctor’s appointment—an “appointment” with the imaginary “Dr. No.” Instead of going to the “appointment,” the membership was asked to use that time to send in a donation to help the chapter support myasthenics, their family members, and medical professionals in what is certainly the largest geographic area of any of our chapters: an area including Alaska, Hawaii, Idaho, Montana, Oregon, and Washington! Working together, the membership raised almost $4,000.

This past winter, our Ohio Chapter held its first “Bowl-a-Thon.” The successful event involved a bake sale full of goodies, prizes for participation, and tons of awareness, outreach, and fundraising for patient support and research. The chapter is now working hard on a “garage sale” event to take place this spring or summer to raise even more funds for patient support and research.

Towards these same goals of raising funds for patient support, research, education, and awareness, our Oklahoma Chapter will also be holding a “garage sale” event on Saturday, April 14th. Additionally, on June 3rd, the chapter will hold its annual “Picnic in the Park” in celebration of “June Is Myasthenia Gravis Awareness Month.” Taking place in Tulsa’s Lafortune Park, this event honors MG patients, their families and friends, and the chapter’s volunteers, and educates the public. To participate in either of these events, contact the chapter’s office at (918) 494-4951.

At the Pakistan Institute of Medical Sciences (PIMS), Islamabad, the plasmapheresis project of the Pakistan Myasthenic Welfare Organization (PMWO)—supported by Mobilink, Pakistan’s leading cellular service provider—was recognized with several special awards. The PMWO is the only health agency in Pakistan devoted solely to the conquest of myasthenia gravis, and is an international affiliate of the Myasthenia Gravis Foundation of America, Inc. for research and sharing of publications on myasthenia gravis.

In the December 7, 2006 Pakistani Times, the PIMS Executive Director Dr. Syed Fazal-e-Hadi said, “Pakistan Institute of Medical Sciences appreciates the task undertaken by the Pakistan Myasthenic Welfare Organization, as well as the compassion displayed by Mobilink in the success of this venture.”

The plasmapheresis project rendered free plasmapheresis treatments to 221 myasthenia gravis, Guillain-Barre Syndrome (GBS), and other patients for a total of 635 procedures in 2005-2006. The project works 24 hours a day with hospitals in the region using a mobile plasmapheresis service.

Further, the Pakistani Times quotes Sadia Khurran, Director Operations and CSR Mobilink: “Mobilink’s contribution is a mere token of our concern for those who need help; we… shall contribute monetarily to projects which can bring hope and happiness in the lives of our people. It is our way of changing lives.”

Mobilink has also donated a sizeable sum to the PMWO for the development of a special Mini Ward for the treatment of patients suffering from myasthenia gravis and GBS at the PIMS Hospital Islamabad.

Khalid Zia, Secretary-General of PMWO, said, “Today we are honoring the young men and women of our two organizations who have worked tirelessly in making this project a success. However, we have a long way to go, the results achieved so far are very encouraging, therefore, we would like to continue with this project and further improve the infrastructure so that more people from the far-flung areas of Pakistan can come and benefit from this facility.”

Under Khalid Zia’s leadership, the PMWO changes lives, bringing myasthenia gravis patients hope with appropriate treatments, including psychological counseling and needed medications. The PMWO participates with researchers in the United States, United Kingdom, and the Netherlands, providing all patients with the hope of a future without myasthenia gravis.

[Top]

Download and print this poster and use it to help promote MG awareness in your area. Make copies if you wish, or contact the MGFA national office or your local chapter for more posters and additional awareness materials.

www.myasthenia.org/docs/
MGFA_Poster_MGAwareness2007.pdf

Myasthenia Gravis Foundation of America Annual Meeting
April 19-21, 2007
Doubletree Crystal City
Arlington, Virginia
www.myasthenia.org/mgprogram_annualmeeting.cfm

International Conference on Myasthenia Gravis and Related Disorders
May 14–16, 2007
Renaissance Chicago Hotel
Chicago, Illinois
Hosted by the New York Academy of Science and MGFA
www.myasthenia.org/mgprogram_intlconference.cfm

Associations

MGFA is proud to be a member of the following associations: