Spring 2006
NEWS
TOPICS
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Times
Up!
Around the World - Khalid
Zia, Pakistan
 
March Miracles is a fundraising campaign. We want to bring the MGFA Annual Meeting to your home. Last year we were able to broadcast the event live from Michigan allowing MGnet members all over the world to participate in this event through our chat room. We want to do it again. And, we want to be able to place the medical sessions on our website for you to view at your leisure. WE NEED YOUR HELP NOW. Send emails to companies in your area, to online companies, to doctors, to companies that make your life easier, to friends and relatives. Although we are asking for donations as high as $3000, we are also asking for any donation at any amount. Let your friends, families and local businesses know they will be listed as a donor for any donation. Larger donations also receive a link to their website. Send emails with our PDF attachment, or letters with a printout of the attachment. DO IT TODAY. IT IS NOT TOO LATE. Here are some examples of what you might want to include in your email or letter:
Sample emails and letters can be found at: www.mgfa-mgnet.org/html/march_miracle_samples.htm PLEASE HELP MGNET HELP YOU. Our goal is $15,000. Without these funds we may not be able to broadcast this year’s MGFA Annual Meeting. Please take the time today to send emails and letters. © Dale Wurtenberger
ATTENTION
Annual Meeting 2006
MGFA strives to address and educate you with all aspects of the disease, including but not limited to new and future research, chapter involvement, and the effects the disease has on patients and families, in the presentations. MGnet will be presenting the annual meeting via clip streaming on our chat site. Please check now to make sure you can get in to the chatroom. For more information
and to register for the Annual meeting go on line to MGFA’s website:
www.Myasthenia.org Registration Costs:
Registration deadline is April 10, 2006. MGFA Annual Meeting Travel Fund: Donations are welcome to help others attend the conference who cannot afford to pay their own way. A checkbox for your donation is available on the registration form. Suggested donations include $100, $50 or $25. Hotel Accommodations:The Buttes, A Marriott Resort (formerly Wyndham Buttes Resort) 2000 Westcourt Way Tempe, Arizona 85282 www.marriott.com/property/propertypage/PHXTM Special MGFA room rates are: • Standard Room, Single (1-person)........$134 Hotel reservations may be made now. To make reservations, please contact the hotel directly at 1-800-228-9290 or www.marriott.com/property/propertypage/PHXTM . When making reservations by phone; reference "Myasthenia Gravis Foundation" to get the special rate. When making reservations online; use MYAMYAA as the Group Code to get the special rate. Hotel reservation deadline is April 5, 2006. You can find all of the
up-to-date information regarding the 2006 Annual Meeting through the
website
www.myasthenia.org/AnnualMtg2006
The MGFA Annual Meeting will be held at The Buttes, A Marriott Resort, in Tempe, Ariz., this year. Formerly called Wyndham Buttes Resort, the hotel is built into a cliff. The main lobby and conference rooms are in one building with walkways connecting to the guest quarters. We have taken a look at the hotel from the vantage point of a person with MG and offer the following information. Hotel safety engineer Robert Marrs told MGNet the hotel has only two handicapped-accessible, wheel-in showers. Those two rooms are located on the lower level. However, for the wheelchair guest to get to the conference room, he or she will have to go to the elevator, go up to the fifth floor, go across the sky bridge over to another section of the hotel, get into the elevator again and go down to the first floor. All of the guest rooms are far away from the conference room. The main building with the conference rooms, check-in desk and lobby are separate from the guest quarters, with walkways leading to each building and the pool area. The walkways might be difficult to maneuver in a wheelchair. One pool is handicapped-accessible. All hotel employees are trained in CPR. Defibrillators and oxygen are available. In case of emergency, guests should call 911 from their rooms. The hotel 24-hour security team will automatically be dispatched to the guest’s room. The closet hospital is St. Luke’s, which is 3-½ miles from the hotel (about a $25 taxi charge). In addition, MDI Clinic is two miles from the hotel. When checking in, those with special needs are asked to inform the hotel. In case of a fire, those capable of evacuating should do so. Hotel staff will go to the rooms where they have been notified special assistance is needed. Once evacuated from the hotel, those requiring assistance should go to the helicopter pad. It would be wise to find out where the pad is located at check-in. Small refrigerators are available for $10. An advance order is required since the hotel has only a limited supply and might need to order more. There is no free shuttle from the airport to the hotel. However, there is a shuttle, “Your Ride,” available for $25 from the airport. A. wheelchair accessible van will have to be called ahead for those in wheelchairs. Taxis and limousines are available. SuperShuttle operates daily 24 hours; charge is approximately $9, offering a door-to-door van service to all destinations in the Valley daily 24 hours. Bus: Phoenix Transit (tel: 602 253 5000 or TDD: 602 261 8208) run Red Line and no.13 wheelchair-accessible buses to central Phoenix and surrounding areas. There are several intercity bus and coach companies operating from the airport. The Ground Transportation Office can provide further information (tel: 602 273 3383).
I would like to announce the Chapter Development Sessions (taking place on Friday, May 5th, from 2 to 5 pm – a full schedule of the Annual Meeting can quite conveniently be found at www.myasthenia.org/AnnualMtg2006. As many of you know that have attended Annual Meetings in the past, each year we hold a series of discussions specifically for chapter-based volunteers and staff. These discussions have been on a variety of topics, ranging from administration and financial management to fundraising and patient services. The discussions are generally facilitated by chapter volunteers themselves and provide chapter leaders with the opportunity for peer-to-peer discussions on specific topics, so that you can get ideas and learn from your cohorts. This year the Chapter Development Sessions will be on two topics: Support Groups and The National Foundation. The Support Groups discussion will be facilitated by Marika Bates, chair and support group facilitator for our Maryland/DC/Delaware Chapter, and Tamara Murphy, APN, MSN, CNS, a member of MGFA’s Nurses Advisory Board and a support group facilitator from the Pennsylvania Chapter. This discussion will cover starting, growing, and sustaining a group. By discussing several aspects to this topic, you should be able to strengthen your chapter’s own groups (or start new ones) using ideas gathered from one another. The National Foundation discussion will be facilitated by MGFA national staff members and will focus on (as the name implies) the National Foundation. With change afoot, we felt it was a good time for an encompassing discussion on how best to utilize our joined network of resources, presence, energy, and volunteers available throughout our chapters in order to fulfill our shared mission most effectively. Just as importantly, we want to make sure that all of our chapters are aware of the resources that are available through the National Foundation itself, and provide you with the opportunity for more feedback on what additional resources are needed. © Mat Spaan
Having myasthenia gravis sometimes causes concern for young women considering pregnancy. There is hope and help for those with these concerns. Kerri Sweeris has graciously agreed to tell her story of her pregnancy. The following article is the first in a series of articles describing Kerri’s experiences of bringing a new miracle into the world. My name is Kerri Sweeris. I have been married for just over five years to the most wonderful man on earth, Doug. Six weeks after we got married, I ended up in the hospital for tests. After an MRI under sedation during which I quit breathing, I was admitted to the hospital. Four days later, I was helicoptered, unconscious, to the University of Michigan in Ann Arbor. They had no idea what was wrong with me. After three months and every test known to man (which were either all negative or inconclusive), they decided I had MG, and started treating me for it. It was not until July of 2003, that I had a positive MuSK test, and finally confirmed my diagnosis. About three years ago, my husband and I started seriously talking about having a family. Because of my MG, I wanted to adopt. I did not think I could ever carry a child due to the myasthenia. My husband very much wanted his own child. In the spring of 2004, we found a solution: a surrogate. We would undergo fertility treatments, do in vitro, and have a friend of mine carry the baby for us. It seemed to be the perfect answer to a very complex situation. This however, was not what God had intended for us, because I did not respond well to the fertility treatments. We were not going to have a baby using a surrogate. In the winter of 2005, I started to seriously consider the possibility of trying to have a baby myself. I was terrified, to say the least. Images of bed rest, ventilators, and long hospital stays filled my mind. Yet, strangely, I had a sense of peace, after praying and realizing that this is what the Lord wanted to me do. I felt like I had to try, for my husband’s sake if nothing else. I did not think I would get pregnant because I did not respond using fertility drugs. How would I get pregnant without them? We spoke to the neurologist in March of 2005, and started making a plan. He said the first thing I had to do was change some medications, and stop others. I had to go off CellCept, my "miracle" drug, and be off of it for four months before we could even start trying. I was having plasmapheresis once a month, and would continue this throughout both trying to conceive and the pregnancy itself. Knowing there was a chance, I may have to have more treatments more often, if I did indeed get pregnant. We set the target date of going off the CellCept in June, which meant in October we could start "trying" for our baby. In August, I went to a high-risk OB. I am also diabetic due to the long-term steroid use. I have a trach due to vocal cord damage from being emergently intubated. I prayed they would not laugh me out of the office. They did not! In fact, they were quite optimistic. The first thing they did, however, was put me on insulin. They have a very tight window in which they require blood sugar to be, and insulin was easier to adjust to meet that goal. I had to do five shots a day for a while, but after about two months, I got the insulin pump. What an awesome device! September came and went uneventfully. I joined a website online to monitor my temperatures and bought ovulation predictor kits. If I was going to get pregnant, I thought I would need all the help I could get! My blood sugar was under fantastic control, my medications were all baby-safe, and October was just around the corner. We were ready. The first month we
tried to conceive was a nail-biter. I took a pregnancy test way too
November I did not even test, just waited to see what would happen. Well, I was not pregnant this month either, but it was a little bit easier not seeing that negative test. Doug was almost happy that I was not pregnant yet. He needed my help wrapping the deer he butchered from hunting season! In December, I just knew I was not pregnant. I had the worst case of PMS I had ever had in my life. I was probably the crankiest person on the planet. I was hyper-emotional, and ready to bite anyone’s head off who got in my way. On Saturday, December 10, Doug and I went to a Michigan MG get-together in Dundee, Michigan. We went to Cabela’s, and of course the Russell Stover outlet store, and then met our friends at the restaurant. They all knew we were trying to have a baby and were very encouraging. I kept telling them I was not yet, and was terribly crabby about it! I was supposed to get my period that day, and could not figure out why I had not. I was going to wait until morning to test, but thought, what the heck, I might as well finish this day upset and crying (because of a negative result) instead of starting the day off tomorrow that way. I took the test at about 9:30 at night. It was positive.
© Kerri Sweeris 3/06
Have you ever wondered why a new drug that shows such promise, in studies, to make the sick well, seems to take so long to get to the very people it was made to help? This article will show what requirements drugs have to pass through from chemist to the consumer. The Food and Drug Administration (FDA) is the governing body that has set the regulations on drug research and development. It was set up to protect us, the consumers, from drugs that may be quite harmful to us. Before the FDA, there was never any assurance of quality---nor efficacy of any drugs. Drugs have been around since the Native Americans here in the United States. However, there were no standards, we knew very little about them and many times the drugs caused illness instead of being of benefit. Let us take an imaginary drug from the chemist’s mind. We shall call it IR376 (later to be called Invigitron). This drug is purported to fight all the fatigue associated with Myasthenia Gravis. Step 1. Chemist comes up with IR376 that he feels will work against the fatigue of MG. Step 2. He begins testing on molecules and cells to prove his theory. Step 3. Animal testing begins with IR376. This is the step that will show any potentially toxic effects. Start to formulate the appropriate dosage, absorption rates, blood levels, ETC… And must be accomplished on at least two different species. This step lasts on avg of 3.5 years. Step 4. At this time the FDA must be petitioned to allow human trials. This is accomplished thru the NDA (new drug application). The studies on animals must be carefully reviewed by the FDA. A panel of scientists, ethicists and nonscientists must also approve the protocol for clinical trials as well as oversee the trial itself. This step can take the FDA up to one year to complete the approval process. And many times the process must be repeated due to insufficient information to meet criteria for approval for trials. (currently the FDA is revising their NDA to make the process smoother and allow more first application approvals) Step 5. Phase I
clinical trials begin. Safety is the primary issue at this stage.
IR376 has the go ahead to give it to a number of healthy volunteers.
(anywhere from 20-100) This phase tells the chemist how long IR376
stays in the body, what organs it effects, and how the body gets rid
of the drug. This phase lasts several months to a year. After phase
I is completed, trial reports must be submitted to the FDA for
review. If the review is positive (again this phase review can also
take a year or more and need many revisions to the application) and
without major problems, then IR376 has permission to proceed to
phase 2 of the trials. (failure rate at this point is about 30% of
drugs tested) Step 6. Phase 2
trials. During this time IR376 is given to patients who have MG. The
person in charge of the study must find MG patients who are similar
in age, disease progression and several other factors. Out of the
volunteers selected they form two groups. One group is given IR367
and the other group is given a placebo or “sugar pill“. This
information is kept from the observers/patients to allow for true
evaluation of effectiveness. This phase is for more safety study,
but mainly evaluating effectiveness and can take up to 2 years to
complete. (failure rate at this point is about 33% of those drugs
that made if thru phase I trial) After phase 2 trial, reports must
be submitted to the FDA for review. If the review is positive then
IR376 has permission to proceed to the phase 3 of the trials. (again
this phase review can also take a year or more and need many
revisions to the application) Step 7. Phase 3 trials. During this time IR376 is given to MG patients on a broader spectrum. From several hundred to several thousand. This phase is concerned with safety, effectiveness and dosage and takes 1-4 years to complete. (failure rate at this point is about 25% of those drugs that made it thru phase 2 trials) After Phase 3 trial, reports must be submitted to the FDA for review. If the review is positive then Invigitron has permission to be marketed to the public. However additional post marketing reviews must be submitted to the FDA. At this time if the
benefits clearly out weigh the risks, Invigitron is now available at
your pharmacy. It seems like a very long journey, but Invigitron
being safe and effective is the goal. Without these steps, we would
be back in the 19th century and taking serious and often unknown
risks each time we took any pill. Now, a word about orphan status of drugs. If a drug has a target population of 200,000 or less in the US, the FDA can designate it orphan status. The most recent drug labeled for MG has been CellCept. Many questions came up about the use prior to being labeled. Often times a drug will have uses other than those for which the drug was developed/researched/approved. Physicians may prescribe it for other uses. Orphan status is granted if the disease is “rare” and new drugs are seldom available to treat it. The FDA approves approximately 40 new drugs a year. This is double the number of approvals given just given just 5 years ago. The FDA is striving to stream line the entire process to allow much needed drugs to come to the consumer sooner, while maintaining the same level of safety. Bibliog.:*US FDA www.fda.gov/fac/special/newdrug *US Department of Health and Human Services *Phases of product Development By Dale E. Wierenga, PH.D and C. Robert Eaton Office of Pharmaceutical Research and Development Pharmaceutical Manufactures Association By DJ Butler
I followed up with my Doctor today.
FCC Consumer Fact:
For more information go
to the following website:
Diagnosed with generalized MG when he was 24, Khalid then gave up his career as an engineer and devoted his life to helping other myasthenics in Pakistan and eventually the world. Khalid, now 41, soon
established the Pakistan Myasthenic Welfare Organization The PMWO has a myasthenic drug bank for free provision of medicines to poor patients. It also provides free plasmapheresis services (3,800 to date) to hospitals in the region around Islamabad, the capital of Pakistan where Khalid lives. About 7,000 MG patients have been diagnosed and treated. In addition, the PMWO has done more than 8,000 EMGs and SFEMGs as well as nerve conduction studies at the Myasthenic Treatment and Rehabilitation Center in Islamabad. Neurologists all over Pakistan refer MG patients to the PMWO, a non-government organization that is renowned for the diagnosis and treatment of MG. Due to his outstanding work in the field of diagnosis and treatment of myasthenia gravis, Khalid received the Commonwealth Youth Asia Award for Excellence in Youth Work and Development in 1998. The Commonwealth is an association of 53 countries. Its 1.8 billion citizens, about 30 percent of the world's population, are drawn from the broadest range of faiths, races, cultures and traditions. (For more information, visit www.thecommonwealth.org ) “I had generalized MG condition with difficulty in chewing and swallowing,” Khalid says. “Easy fatigability in walking and could not take steps upstairs.” He used Mestinon and prednisolone in small doses, then had a thymectomy in May 1989. “After the thymectomy my MG condition much improved and I spent a few years without medicines and in complete remission,” Khalid says. MG symptoms returned in 1994 and were controlled with steroids and Mestinon.
“I need to do a lot of work to manage the earthquake victims with crush injuries with mobile plasmapheresis to save them from the amputations,” Khalid says. “This difficult task completed with zeal and courage, but my MG condition again worst.” Now his MG symptoms of drooping eyelids, double vision, limb weakness, difficulty walking and easy fatigability are well controlled with 20 mg prednisolone (a steroid in the same class as prednisone) on alternate days and 60 mg pyridostigmine (generic Mestinon) four times a day. “I am using all my energies to develop this charity work for the myasthenics,” Khalid says of the PMWO. His organization, of which he is secretary general, has done a tremendous job of creating awareness about MG in Pakistan through publications, TV interviews and visits to medical colleges. In big cities where neurologists are based there is no difficulty in getting a quick diagnosis, Khalid says, but in remote areas speedy identification of the problem is not assured. Once referred to the PMWO, a patient can receive comprehensive treatment including medicines, plasmapheresis and thymectomy. IVIG also is considered in clinical management of some critical MG cases. PMWO helps poor patients with the cooperation of the Pakistan Institute of Medical Sciences, a prominent 650-bed hospital in Islamabad. Khalid and his wife, Kalsoom, have two daughters, Javeria Khalid, 10, and Noor Fatima, 2. The proud father reports that Javeria is a “very intelligent” fifth-grader. Khalid suggests that positive thinking and doing some positive work for the care and welfare of others can help myasthenics improve. “We should join hands toward the cause anywhere in the world,” he says. MG Around the World is a regular feature of the MGnet Connect Newsletter. We feature a person with MG each issue, traveling all over the globe via e-mail to interview him or her. If you know someone who would make a good subject, please contact Kathleen Knorr, irishkathleen2@yahoo.com . © Debbie Marshall
If you are interested in helping with the MGnet Newsletter, please contact the editor, Kathleen Knorr at: newsletter@mgfa-mgnet.org Any Chapter of the MGFA may copy and reprint one of these articles if they comply with the following rules: All articles must be reprinted in their entirety including any references or links; The author's name must be included; MGnet and the MGnet web site must be included ( http://www.mgfa-mgnet.org/ ) MGnet must be notified of when and where the article will appear ( newsletter@mgfa-mgnet.org ); no article may be permanently placed on another web site, but permission may be granted to link to the article. Anyone outside the MGFA must submit a request in writing to copy any article. ( mailto:(newsletter@mgfa-mgnet.org ) The decision to allow an article to be reprinted is the sole right of MGnet. Under no circumstances may the article be placed on another web site. Permission to link to this site will be determined by MGnet upon receiving a request. ©2006 by MGnet
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early. Then was devastated at the big minus sign on the stick. I
could not believe how upset I was! I was the one who did not expect
to get pregnant anyway! My husband was calm and reassuring. He was
convinced we were going to get pregnant and have a wonderful,
healthy pregnancy.
Shock was a mild
term for what I was feeling. I was pregnant. I was PREGNANT! I,
who usually have a motor mouth, was completely speechless. I had no
words. I took
the
stick into the living room and handed it to Doug, slack-jawed. He
looked at it, looked at me, and said, "What does this mean?" I said,
"What do you think it means . . . Daddy?" I do not think either of
us slept a wink that night. Doug was on cloud nine. I had NEVER seen
him happier or more proud in our entire lives together. I thought I
would be terrified, but I was so excited. I was absolutely thrilled
to be pregnant. I knew it would not be easy, but I knew it would be
worth it. 
Khalid
Zia has done so much for myasthenics in his home country of
Pakistan, as well as around the world, that it’s hard to know where
to start. From hurrying to the remote areas affected by Pakistan’s
recent earthquake to providing Mestinon to a desperate patient in
Africa, Khalid is always finding ways to aid those with MG. 
He
experienced another exacerbation last September, but quickly went
into remission. That didn’t last long, because he responded
immediately when a devastating earthquake hit Pakistan in October.