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Physicians Breathe Life Into Cutting-edge Stem Cell Procedure

Patients living with Myasthenia Gravis (MG) may breathe easier thanks to a rare bone marrow transplant procedure performed at The Bone Marrow Transplant Program at University of California, San Diego Medical Center, the only program in the western United States that has attempted this procedure.

Myasthenia Gravis (MG) is a rare neuromuscular autoimmune disease where the body's immune system, which normally protects the body, mistakenly attacks itself. The transmission of nerve impulses to muscles is interrupted, which ultimately prevents the muscles from contracting. Without the proper nerve impulses, muscles that control breathing can't function.

"It's like dying in your own body," said Ewa Carrier, M.D., associate professor of medicine and pediatrics in the UCSD Blood and Marrow Transplant Division at UCSD's School of Medicine. "Eventually, MG patients can't walk, can't breathe, can't swallow. The signal just doesn't go to the muscles."

This new procedure reprograms the patient's stem cells, destroying them with chemotherapy, before re-introducing purified blood-forming stem cells. After the transplant, the modified stem cells build new bone marrow, renewing the immune system with correct signaling, renewing the immune system with cells that don't attack the body.

The Patient

The Myasthenia Gravis Foundation of America estimates only 20 out of 100,000 individuals in the country have been diagnosed with MG.

However, MG is considered underdiagnosed and many more are likely affected but do not know it.

Martin Glasser, M.D., is one of the confirmed cases. Every other day for the past three years, he has visited the plasmapheresis clinic at UCSD Medical Center. Plasmapheresis is a procedure much like dialysis which is used to help MG patients feel better for short periods of time.

Glasser's disease was progressing, causing weakness in the legs, arms and diaphragm. Plasmapheresis made breathing easier "but it's a very crude way of keeping you alive," said Glasser.

The Procedure

The procedure was conducted by a team consisting of Ewa Carrier, M.D., Arnold Gass, M.D., professor of medicine at Veterans Affairs San Diego Healthcare System, Geoffrey Sheehan, M.D., UCSD professor of neurosciences and myasthenia gravis specialist and David Ward, M.D., UCSD professor of medicine and Apheresis program founder. According to the International Bone Marrow Transplant Registry (IBMTR) this rare procedure has previously been performed only three times, all at Northwestern University Hospital in Chicago.

In preparing for the transplant, Glasser's native bone marrow was literally obliterated. Most of the T cells in the body were destroyed. "The theory is that if the T cells are destroyed before introducing new stem cells, the new stem cells will not receive the old message to attack. After the transplant, the modified stem cells build new bone marrow, renewing the immune system," said Carrier.

Glasser's transplant involved harvesting 16 million of his stem cells. These cells were cleaned with a special device resulting in 8 million pure stem cells. Stem cells at this early stage of development have the greatest chance of producing a healthy line of blood cells. After the transplant, patients must take antibiotics to protect them from infection. They cannot go to crowded places and must follow special diet requirements until the immune system is fully recovered, which takes about three months.

"There's a possibility that there's also some form of tissue repair going on," said Carrier. "For example, Dr. Glasser did not have feeling in his feet before the transplant and now he has feeling in his feet again, possibly indicating that his peripheral polyneuropathy is improving as well."

Adapted from materials provided by University of California - San Diego, viaEurekAlert!, a service of

AAAS. University of California -San Diego (2007, October 11).

Physicians Breathe Life Into Cutting- edge Stem Cell Procedure. ScienceDaily. Retrieved August 4, 2008, fromhttp://www.sciencedaily.com

The Role of Eduction in the Treatment of MG

The course of myasthenia gravis depends largely on patient compliance. Patient teaching, therefore, plays an important role in how a

patient and the family will do. Family members must be included in disease education, for their co-operation is essential to patient wellbeing.

Education must include the following information:

• Disease process

• Factors that could create symptoms or worsen the ones already present

• Medications—why they work and their side effects

• Emergency measures and support systems

• Myths and misconceptions about MG

Every MG patient and at least one family member should know something about each one of these points. Any who do not should visit the local library, local MG Chapter or doctor's office as soon as possible. Determine to know as much as you can about the disease affecting you or the one that you love.

In teaching the disease process in M.G., the effort has been wasted unless you begin at the patient's level of understanding. Initially a simple diagram showing how a message from the nerve to the muscle gets "short-circuited" does infinitely more than a detailed, precise medical explanation with $50, five-syllable words will ever do. "Keep it simple" is always good advice. Always bear in mind that myasthenia gravis is not a household word. It's usually an unknown to a patient that is, therefore frightening to them.

Patients should be made aware of factors that can cause an increase of symptoms. Such things as extremes in temperatures (hot or

cold) or emotional stress can cause "flare-ups". Exposure to infectious diseases can be equally hazardous—especially for those patients on immunosuppressant therapy as Prednisone. Having MG does not preclude having other medical problems. These and other

medications that the patient. may be talking should be thoroughly discussed at the time of diagnosis.

Learning to cope with stress or to avoid stressful situations can be a real challenge. A lot of stress surrounds just accepting the diagnosis. Worry about new symptoms that might crop up can make the situation worse. Patients should be encouraged to vocalize their fears and to use their support systems when they feel anxious.

Myasthenic patients often feel remote and isolated because of changes in physical appearance, caused by the disease itself (droopy eyelids, snarl-like smile) or by medications they take (fluid retention, hump back, moon face). Again, explaining why these things are happing helps the patient to understand and cope. Ways to improve appearances should be carefully explored.

Most MG patients have been told that certain medications are contra-indicated. Few know why. A more detailed explanation of the neuromuscular junction is called for. As the patient learns more about myasthenia, he will understand why certain drugs are "bad" for him, as well as why MG medications work. The patient should always have a list of medications that could be harmful. And he should always check with his MG physician before taking any prescription or over-the-counter remedy.

Because the symptoms of too much or too little anticholinesterase can be very similar, patients must report any of the following

symptoms: abdominal cramps, nausea, vomiting, diarrhea, excessive sweating, excessive salivation, increasing muscular weakness, muscle twitching, irritability, anxiety, headache, increased tearing or bronchial secretions. Patients on immunosuppressant therapy

should know the potential side effects and be monitored closely by a doctor or by clinic.

Understanding and compliance has to be the goal of any patient education program. Taking the disease "out of the closet" and dispelling misconceptions about MG is absolutely essential. MG is not, for example, contagious. Neither is the disease thought to be hereditary. And thanks to recent advances, it should no longer be fatal. Through dedicated research and enormous strides in therapy, today's myasthenia gravis patient can not only dream, but live to see those dreams fulfilled.

AMPS:Greater South Texas and Nutmeg Chapters

Disclaimer

Any views expressed in this newsletter are those of the individual author and do not reflect any official position of the Georgia State Chapter, MGFA. Each person's situation is unique. If you have any medical questions, please discuss them with your doctor as he/she knows your situation best.

 A ”Pocket” Full of Nickels: Myasthenia Gravis Remission

By: Beverly J. Nason

On a good morning, we awake with a dollars worth of invisible nickels

in our pocket, or perhaps we find them after our

first dose of Mestinon.

We must decide how to spend this precious supply.

Shall we shower, dress, make the bed, have breakfast, go to a job,

and recklessly spend them all at once?

Perhaps, we should just use twenty-five cents,

and dress without the shower or shave, leave the bed for later,

and spend five to fifteen cents for breakfast.

We alone may judge from our recent experience.

Having decided, we act, and our precious deposits is made into an

invisible slot, filled with invisible batteries.

These batteries kick in slowly and we drain their reserve.

When it is close to gone, we force ourselves to rest.

As the day progresses, we decided to do a small chore.

The shave, shower, shampoo? Five more nickels perhaps?

The bed? One nickel perhaps. Cleaning house? Going to work?

How many nickels will we have to spend today?

For many of us, we can barely afford one nickel at a time, today.

If we have the luxury of time for a rest period or a nap,

we might awaken with a sudden new supply of nickels in our pocket.

Probably our next dose of Mestinon will provide us with a

fresh supply. For others, we may find we've chosen unwisely

and squandered our day's wealth,

Or borrowed from them tomorrow, to do what had to be done,

Or simply what we wanted to do to improve our quality of life.

The debt must be repaid, and time in bed will be the price.

We can gamble them all away, or spend them gratefully.

When we awake, morning after morning, with pockets full of nickels,

Such an abundance that we no longer have to count them

We have achieved Myasthenia Gravis remission.

May you all have a "pocket full of nickels"

REMEMBRANCES

In Memory of:                         By:

Harry E. Lawhon                                             Elizabeth Kendley

Dorothy Thomason Folds                               Vera R. Scalise

Malcolm W. Forte, Jr.                                     David Payne

Joseph Franco                                               Dr. & Mrs. Robert Spector

Howard Morris                                               Ellie Ruth Whelchel

Richard Johns                                                Beth & Bob Tarkington

Juanita Autry                                                 Clarence Autry

In Honor of:                           By:

Bill Bradley                                                    Ginnie Bradley

Dawn Warner                                                 Kareina Jackson

Karen Brooks

Kathy Russell