Upcoming MG Awareness Screening 

Set for September 17, 2005 in El Paso, Texas 

The Northwest Texas Chapter provides services to a large portion of Texas.  In order to better serve all MG patients in our Chapter area, the Board voted to alternate the Annual MG Awareness Screening between the Lubbock location and an El Paso Location.  Mark your calendars and make plans to attend the first screening to be held in El Paso.  If you need additional information, you may call Lajuana Miller in the Abilene area at 325-554-7038, Myrna Rasmussen in the El Paso area at 915-581-4029, and in the Lubbock area call Coleen Shinn at 806-749-3126 or Marvin Burnett at 806-894-3171.

Congratulations to the El Paso Support Group!!!

The Junior Woman’s Club of El Paso has awarded the Northwest Texas Chapter $900.  El Paso Support Group Leaders, Jay and Myrna Rasmussen attended the awards luncheon in April to accept the donation from the Junior Woman’s Club.  This along with other donations received the last two years will be used to help fund the MG Awareness Screening this year in El Paso. 

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New Clinical Study for MG

A large clinical study of a new therapy for MG is about to start enrolling patients in several centers in the UK, Europe and North America. Sponsored by F. Hoffman-La Roche Ltd/Inc/AG, in partnership with Aspreva Pharmaceuticals, the study will assess the effectiveness and safety of the immunosuppressant mycophenolate mofetil (CellCept) for the treatment of MG.

MG is an autoimmune disease that occurs when the immune system, in the form of antibodies, attacks and damages the connection between nerve and muscle. These rogue antibodies target the acetylcholine receptor, a protein on muscle cells required for muscle contraction. The ensuing damage to these receptors reduces the muscle’s ability to respond to nerve signals, resulting in the apparent muscle weakness. Muscles that control the eyes, face, neck and limbs are commonly affected.

Treatments for MG target different stages of this process. Thymectomy, which is a surgical treatment option, may lead to improvement in some patients in whom the thymus is responsible for abnormal antibody production. Cholinesterase inhibitors, as discussed in previous newsletters, delay the breakdown of acetylcholine when it is released from the nerve endings. More acetylcholine is then available to compete against the antibodies to complete the nerve-muscle connection. Intravenous immunoglobulin (IVIg) and plasma exchange (PE) remove circulating antibodies and the cells that produce them, and are used primarily in crises, serious disease or prior to surgery. These treatments do not offer remission or sustained symptom control.

The mainstay of treatment is long-term immunosuppression. Suppressing the production of antibodies reduces the number of antibodies in the bloodstream and can lead to good function and prognosis. The damage to the acetylcholine receptors caused by the antibodies is reversible if the autoimmune process can be controlled.

Immunosuppressants such as corticosteroids, azathioprine and cyclosporine are commonly used in MG and they can be effective. However, these older immunosuppressants can have serious side effects and there can be a time delay before they become effective. Although these medicines represent important advances in the treatment of MG, there is still a critical need to improve therapy with safer, effective medications.

Mycophenolate mofetil (MMF) is a good candidate for further study. For the past nine years, MMF has been an important therapy for the prevention of rejection after organ transplant. It has been well studied in transplant patients and has a good safety record. It works by reducing the production of the immune cells responsible for producing antibodies that can lead to organ rejection—the same cells responsible for producing the problem-causing autoantibodies in MG.

This study is designed to fully evaluate MMF as a new treatment for MG. Once the study is complete the results will be assessed using clinical and statistical methods before a decision is made as to whether or not to seek regulatory approval. The information gathered will enable patients and their physicians to make treatment decisions based on solid clinical evidence.

To be eligible for the study, patients must have a diagnosis of generalized MG treated with oral corticosteroids at a certain dose level for at least four weeks prior to starting the study treatment and using no other immunosuppressant. Patients may also be taking cholinesterase inhibitors if this is their normal treatment.

Study treatment will be taken as tablets twice daily. Patients may be assigned either placebo or active treatment and neither the patient nor the doctor will know which treatment is being taken. The study will last for 36 weeks. Patients will need to attend the clinic on a regular basis for their MG to be assessed and for questionnaires to be completed.

Within Canada, this study is currently being conducted in the following cities:  London, Ontario -- Montreal, Quebec -- Edmonton, Alberta.   Within the US, this study is currently being conducted in the following cities:  Chapel Hill, NC -- Chicago, IL -- Columbus, OH -- Dallas, TX -- Davis, CA -- Durham, NC -- Kansas City, KS -- Miami, FL -- Rochester, NY

If you require any further details on the study, please contact Trinh Le at (760) 707-5032 or Lalaine Papa at (760) 707-5043. They will then be able to provide you with more detailed information.

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·        Prior to 1970 40% of MG patients died.  Thanks to modern therapy, MG deaths have been cut to less than 5%.  Research helped to develop these new life saving therapies!

·        However, even today many MG patients are misdiagnosed or go undiagnosed for many years prior to receiving treatment.  Education on MG made available to our communities and health care professionals is a way that we can change this fact.

·        The mission of the Northwest Texas Chapter is  “To facilitate the timely diagnosis of individuals with MG and improve their lives through programs of patient services, public information, medical research, professional education, advocacy, and patient care”.

Your participation in this Walk-a-thon will put you right on “Target” to help promote MG Awareness through education and research! 

SUPPORT GROUP MEETINGS

ABILENE               Support Group Leader - Lajuana Miller           325-554-7038

Bi-Monthly Meeting - Last Saturday - May, July, September – E-mail:  nwtc@direcway.com

AMARILLO        Support Group Leaders - Grady & Peggy Wilkinson        806-878-3007

Cancelled due to lack of participation.  If you have need of information or personal support, please contact Grady and Peggy at the number above.

EL PASO           Support Group Leaders - Jay & Myrna Rasmussen         915-581-4029

2nd Saturday of Each Month  

Potluck lunch at 1:00 p.m. at various locations - call or e-mail contacts for more information

and to be added to the mailing list for the meeting notices.

E-Mail:  jbirdrasmu@aol.com

LUBBOCK              Support Group Leader - Coleen Shinn           806-749-3126

Bi-Monthly Meetings

Contact: Coleen Shinn at 806-749-3126 or cshinn1@att.net

Marvin Burnett at 806-894-3171 or bkamar@crosswind.net

We had a great support group meeting.  Had 16 attend.  Pattie Perkins gave the program.  "Roll of a Care Giver".   It was a great program, and Pattie is a retired special ed teacher, and has gone back to school studying medical technology, and had to do a "medical paper".  Her topic MG.  Her dad has MG and her mom was primary care-giver, until she suffered a stroke!!!  I gave Pattie a "volume" of information, and she done a lot of research, and was amazed at the number of patients in this area.

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Myasthenia Gravis patients need to be educated on MG in order to better cope and manage their disease.  To provide this education to the patients of the Northwest Texas Chapter, we include a “lunch and learn” session each year.  You will want to make plans to attend Dr. Nations’ presentation at noon.

Our annual free annual MG Awareness Screening promises to be an educational as well as an enjoy-able experience for everyone.  We hope to see you there!  If you need additional information, you may call Lajuana Miller in the Abilene area at 325-554-7038, Myrna Rasmussen in the El Paso area at 915-581-4029, and in the Lubbock area call Coleen Shinn at 806-749-3126 or Marvin Burnett at 806-894-3171.   Our Annual Meeting will be held immediately following the awareness screening. There will be no charge for the screening; however, contributions will be graciously accepted.

University Medical Center is located at 602 Indiana Avenue and is easily accessible from both West Loop 289 and North Loop 289.  (From West Loop 289, exit 4th St. Go east to Indiana Ave.  From North Loop 289, exit Quaker Avenue; go south to 4th St., then east to Indiana Ave.  You can see UMC from the corner of 4th St. and Indiana Avenue).  From Indiana take 10^th street and then turn on Cancer Center Drive.  The Southwest Cancer Center is part of the UMC complex.  See UMC Overhead View below. 

Did You Know?

ST. PAUL, MN—(May 18, 2004)—“The Myasthenia Gravis Foundation was deeply saddened to learn of the death of Tony Randall.  Our deepest sympathies extend to his family, friends and the millions of lives he touched throughout his career.   Mr. Randall was actively involved in our foundation as Campaign Chairperson for over 32 years and used his celebrity to selflessly promote awareness for Myasthenia Gravis, an autoimmune neuromuscular disorder that affects 14 of every 100,000 people.  Tony will be greatly missed and his dedication and sense of civic duty will long be remembered.”—Esther Land, Chairperson, Myasthenia Gravis Foundation of America

Did you know….you can choose to receive your national newsletter Foundation Focus by e-mail rather than by hard copy.  This would save printing and mailing expenses for the national foundation and you could save all the info on a floppy disc and/or a CD for future reference. 

If you would like to receive your Foundation Focus by e-mail rather than hard copy, please e-mail chapteroffice@nwtcmg.com to make your request. 

Did you know?

• The Myasthenia Gravis Foundation of America currently has 34 chapters.

• The current epidemiology being used for Myasthenia Gravis in the United States is 20/100,000.

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 My Fortunate Disease by V’Lesha Wilcox

I bet you have never thought about being able to smile. You're probably thinking, "C'mon, everyone can smile, you don't have to think about it." I would've said the same thing before my sophomore year in high school.  In 2001 I was diagnosed with a neuromuscular autoimmune disease called Myasthenia Gravis. MG affects the transmission of nerve impulses at the neuromuscular junction, causing the characteristic fluctuating weakness in voluntary muscle groups that include ocular, oropharyngeal, shoulder, hip, and limbs. Although there are effective treatments, there is no cure for this disease. I take a total of 7 pills daily, and a couple more when needed. I also went through major surgery. I had a thymectomy, which uses the same procedures as open-heart surgery to remove thymus tissue in the chest cavity. I had to undergo a six-week long recovering period.

You're probably wondering how all of this started. My sophomore year started out great. School was going well, and my athletic endeavors were going even better.  I qualified for regionals in cross-country my freshman year, so the outlook was even brighter for the next year.  I was sick on the day of the meet though.  Basketball was going great.  I was a starter and the only sophomore on varsity. As games began we were winning, but I was having some problems. My eyes wouldn't stay open, and when they did, I had no depth perception at all. This of course threw off my game tremendously.  I went to an ophthalmologist, and he said my eyes were healthy and had no idea what the problem was. This was good news, but also not so good at all. I kept playing though.

I then began to experience problems with my facial muscles. It became very difficult and tiring to smile, chew, and talk. The muscles in my face just wouldn't work. I couldn't explain it to anyone. As time went on, I had trouble writing in school and doing other simple tasks like putting on my makeup and fixing my hair. In games, my legs would feel very tired even before tip-off. I spent 45 minutes before the game
stretching and rubbing them down with Icy Hot trying to loosen them up. I couldn't figure out why it was that way. We played in a Christmas tournament over the break, and during the first game, it was horrible. I couldn't get up and down the court or move my feet well on defense; I was one of the quickest people on the team. I told coach that my legs just wouldn't work, but naturally that is a little hard to understand, especially because we were out of shape due to the break. During the next game, I could not run. When I tried I would just fall. My legs would not move. This scared me. 

My parents took me to the doctor and that is when a long, grueling process began. By doctor's orders I couldn't play basketball anymore. She went down a long list of things it could be, and the testing
began. I visited the doctor almost twice a week to run more tests. After three months of being pricked, shocked, and drained the doctor referred me to a neurologist. He then began his tests. I missed almost 25 days of school that year, not to mention the rest of basketball and track. Finally, after six months of tests, I was diagnosed with Myasthenia Gravis, and told I would probably never be able to play or run again. This was not an option.

I started treatment which included a high dose of a steroid called Prednisone. This helped with the symptoms, but the side effects were awful. I had an increased appetite, my face swelled up, and I was extremely moody. But I was beginning to be able to do things that I couldn't do before. My eyes and facial muscles improved a lot, but my legs still weren't up to speed. Not playing was completely out of the question though.

That's when I decided to have the thymectomy. There was only a fifty percent chance it would work, but fifty was better than zero. The procedure took place in June 2002. Because of the surgery, I no longer have to take the steroids. I'm not cured but I'm not on steroids. On top of that, I played basketball my junior year, started, and we were area champs. I received 1st team all-district honors. I also ran track my junior year and qualified for regionals in the 800m relay and the 1600m relay. Now it’s my senior year. Although I am unable to run cross-country, I am playing basketball. We have a good team that is state ranked. I have endured a lot to get here, and I have a scar to prove it!

Throughout the whole experience I wished I wasn't this way, but I'm appreciative of the lessons I've
learned. Without this experience I wouldn't be grateful for the things people take for granted. I wouldn't have thought twice about being able to fix my hair or hold silverware or swallow food. Now I thank God for every opportunity I have to do these things. My spiritual life has also benefited from this. I consider myself lucky because although I will be taking medicine for the rest of my life, I have everyday reminders to be thankful and not take anything for granted.

This misfortune has pushed me to become an orthopedic surgeon, in order to help others that encounter a career ending mishap. Some people might call my condition sad or unfortunate, but I think I am very fortunate to be the way I am physically, mentally, and emotionally.

IN THE NEWS: Israeli drug eases suffering of Myasthenia Gravis patients
By ISRAEL21c staff December 14, 2003

An Israeli company is in the advanced stages of developing an effective treatment for Myasthenia Gravis (MG).

MG is a chronic and debilitating disease, which affects about 100,000 people worldwide, characterized by muscle weakness especially inability to open one's eyes, and hand and leg muscle problems. The body's immune system attacks acetylcholine receptors at the neuromuscular junction, interfering with normal muscular function. In severe cases the disease can involve the respiratory muscles, causing potentially life-threatening respiratory failure.

Ester Neurosciences, based in Herzliya, has recently completed a successful Phase Ib trial for its drug Monarsen - an orally-administered anti-sense therapy for the neurological disease. As a result, The U.S. Food and Drug Administration has granted Orphan Drug Designation status for Monarsen, (formerly known as EN101).

"Obtaining orphan drug designation marks an important step in our regulatory strategy for Monarsen," said Dr. Eli Hazum, CEO of Ester Neurosciences. "Current MG treatments which include anti-cholinesterases, steroids and immunosuppressants, offer limited efficacy and often cause unpleasant and sometimes dangerous side effects. Monarsen offers the prospect of an efficacious and safe product that can address a very large market," added Hazum.

Orphan drug designation is granted by the FDA for treatments that might provide significant benefit to patients with serious, life-threatening diseases that affect less than 200,000 persons in the United States. The Orphan Drug Act was created by Congress to provide assistance and incentives for sponsors to develop drugs judged to be of potential benefit for a qualifying disease.

Orphan Drug Designation status gives Ester, upon marketing approval, the exclusive right to market a drug of this kind for MG in the US for seven years. In addition to marketing exclusivity, the advantages of the designation include eligibility for research grants to conduct clinical trials, certain tax benefits, and an exemption from certain user fees at the time of submission for marketing approval of a new drug application. A similar Orphan Drug application has been made to European regulatory authorities.

The prevalence of myasthenia gravis in the United States is estimated at 14/100,000 population, approximately 36,000 cases in the United States. However, myasthenia gravis is probably under diagnosed and the prevalence is probably higher. Previous studies showed that women are more often affected than men. The most common age at onset is the second and third decades in women and the seventh and eighth decades in men. As the population ages, the average age at onset has increased correspondingly, and now males are more often affected than females, and the onset of symptoms is usually after age 50.

Ester's Phase Ib results for Monarsen were presented at a special session of the National Academy of Neurology earlier this year. The breakthrough study was the first demonstration of the safe and effective use of an orally-administered anti-sense therapy for a neurological disease.

This study, where sixteen patients received oral liquid Monarsen, demonstrated significant improvement in MG symptom severity, with no cholinergic effects, nor significant adverse events. Fourteen out of sixteen patients had better scores on the Quantitative Myasthenia Gravis (QMG) scale on the last day of dosing as compared to the initial baseline.

Improvement of total QMG score for these days ranged from 27.8% to 53.4% (p less than 0.01). The Phase Ib trial results showed that Monarsen appears to have superior efficacy, longer duration of action and a more favorable side effects profile than currently used medications. Patient recruitment for extended clinical trials with Monarsen is underway.

Neurologist Jon Sussman, lead investigator at the Greater Manchester Neuroscience Centre, a UK trial site told Bio World, "We were very impressed with the striking improvement in the condition of our patients. Monarsen even enabled some patients with limited mobility to regain their ability to stand and to walk without aids."

The current means for treating MG is mainly a drug called Mestinon. While Mestinon is effective it deals only with symptoms of the disease and it has a short span of effectiveness. Mestinon works for only about two hours which means it must be administered up to six times a day by injection. It also has side affects such as diarrhea.

Monarsen on the other hand, is an antisense drug and works completely differently. Antisense technology was first developed about 10 years ago but first generation drugs started coming out only about four years ago. Monarsen is a third generation antisense drug and the first able to be administered orally instead of injected into the vein.

Antisense drugs have better penetration of the blood-brain barrier which many conventional drugs find difficult to cross. Thanks to its antisense technology, Monarsen need be administered only once a day, is more effective than Mestinon and has no known side affects.

According to Hazum, the same technology used in Monarsen can eventually be used to treat much more widespread diseases like Alzheimers and Multiple Sclerosis (MS). The company chose to target MG at first, because in US, drug markets of less than 100,000 potential patients, companies get exclusivity and the approval time is faster.

In addition, Alzheimers and MS are more complicated diseases which require testing on thousands of patients requiring tens of millions of dollars. In addition, approval takes longer - in this case up to three years more. Ester decided to initially target their concept on treating MG and then eventually partner with bigger companies for Alzheimers and other more widespread diseases.

"The next step is the Phase II study which we're preparing for, in which we'll compare head to head the efficacy of Monarsen versus Mestinon. The studies will likely be held in the U.S. and Europe beginning at the end of the first quarter of 2004," Hazum told ISRAEL21c. According to Hazum, if testing continues to go well in the second and third phases the drug could be ready for market by 2005.

Monarsen is based on pioneering research carried out by Prof. Hermona Soreq of the Hebrew University. Ester Neurosciences was established in 1997 by Medica Venture Partners, to commercialize discoveries pioneered by Soreq, who is the company's Chief Scientific Advisor.

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