In May one of the study participants, Edwin McClure, walked across the stage to receive his degree after completing a rigorous graduate program at Virginia Commonwealth University. The young man appeared strong, healthy, and confident.

The scene was in stark contrast to four years earlier when the high school star football player was battling a severe cold, fatigue, and inexplicable visual changes.

“It was like someone turned down a dimmer switch,” he recalls. “My mom thought the problems were due to sinus pressure and would eventually go away, but when I got over the cold and still had difficulty seeing, she took me to an optometrist.”

When nothing surfaced during visits to an optometrist and an ophthalmologist, McClure was referred to a neurologist for follow-up.

After a series of tests and an MRI scan, the doctor delivered the diagnosis — multiple sclerosis (MS). The visual changes the young man was experiencing were due to optic neuritis, an inflammation of the optic nerve that occurs in approximately 50 percent of patients with the disease.

McClure was placed on steroids and interferon injections — a regimen that successfully controlled symptoms for two years. But when the MS started to break through, his physician switched to another medication.

“Over the course of four months, I started to develop an allergic reaction to the drug,” McClure says. “Meanwhile, my disease was still progressing.”

McClure was at a crossroads: begin medications with significantly greater risk of side effects or, as his neurologist suggested, investigate a promising clinical trial underway at Northwestern University in Chicago.

He chose the latter, qualified, and enrolled in Dr. Burt’s study. McClure was one of the 21 patients in the trial, ages 20 to 53, who had relapsing-remitting MS for an average of five years and had not responded to at least six months of treatment with interferon beta. After an average follow-up of three years posttreatment, 17 patients (81 percent) improved and none got worse, according to Dr. Burt, whose findings were published in the March issue of The Lancet Neurology.

Resetting the Immune System

Dr. Richard Burt, M.D.
Courtesy Dr. Richard Burt, M.D.

“The concept is that your immune stem cells — your blood stem cells — could be used to regenerate a new immune system in virtually any autoimmune disease,” Dr. Burt tells the Post. “If we treated patients in the early relapsing-remitting phase of MS who were experiencing frequent acute attacks despite the use of interferon, patients got better. Six months after the procedure, they were even better. By two years, they seemed to have reached their peak improvement in neurological function. Most people tend to be early- to mid-range in their disability, and that’s when this therapy is really effective. But if you treat MS in a later stage, called secondary progressive MS, it doesn’t really help. In this stage, patients experience a steady worsening of irreversible neurological damage.”

In the procedure, Dr. Burt and colleagues first push immune stem cells from the bone marrow into the blood by using a growth factor and a drug called Cytoxan (cyclophosphamide). Ten days later, they harvest cells from the blood via catheter. The cells are then separated, frozen, and cultured to ensure that none are contaminated with bacteria during the process. Next, the patients are treated with drugs to inhibit the old immune system, and then the frozen stem cells are thawed and infused back into the patients to make a new immune system.

Reversing the Tide

“I started to feel improvement while I was in the hospital,” McClure says. “I realized that I didn’t need my glasses to see. At home my parents noticed that my balance was improving and that I didn’t seem as fatigued as before. Honestly, these changes started within the first month after coming home. My life continued to improve. By the third month, I was actually going to the YMCA to exercise.”

Three years after treatment, McClure remains off medication and now experiences no symptoms of MS.

Like McClure, the majority of trial participants experienced benefits.

“We’ve seen patients who have had marked improvement in symptoms,” notes Dr. Burt, principal investigator of the clinical trial. “Your nervous system controls everything, so the part of the brain attacked by MS determines where you have a problem. Some patients had trouble walking — falling down and having to hold on to things — but after the procedure, they had marked improvement. Others had issues with incontinence, and that’s gone away. If you’re worried about incontinence, that’s quite remarkable. Numbness, tingling, inability to feel things, visual problems — blurred and double vision — can all reverse. Basically, any type of deficit can reverse. In some patients, we actually had complete reversal — everything went away, and they were completely normal in all functional exams.
In others, symptoms never completely reversed, but improved dramatically.”

The study participants are also off all conventional disease-modifying medications now used to slow the rate of disease progression.

While the small trial is only a first step, the results offer a completely new way to treat MS. “This is the first time in the history of any therapy used to treat MS where it actually reverses neurological deficit,” stresses Dr. Burt.

“All other therapies were studied or approved for their ability to slow the rate of progression — in terms of clinical deficits or MRI load of lesion burden — but nothing has, up to this time, reversed deficit. That’s what’s exciting. However, I want to stress that we cannot say it is a cure and current results with three years of follow-up are encouraging.”

Dr. Burt and colleagues are enrolling patients in a larger trial to test the procedure in a randomized setting. “If the results of the trial hold up, I believe it will help open the door for it to be accepted as standard therapy,” adds Dr. Burt.

At present, clinical trials are underway at the University of Calgary in Canada, the University of São Paulo in Brazil, and at Northwestern University. If interested in learning more about the trial, e-mail d-spahovic@northwester.edu.

A Different Approach

Sergeant Preston WalkerCourtesy Sergeant Preston Walker

After undergoing conventional therapy for MS for several years, Fort Worth police sergeant Preston Walker learned about a new treatment for autoimmune disorders. Researchers were utilizing adult stem cells derived from cord blood at The Institute of Cellular Medicine in Costa Rica. Walker inquired about the potential of the treatment for multiple sclerosis.

“We knew that if the treatment worked, the potential benefits for multiple sclerosis patients could be limitless,” says Walker.

Dr. Neil Riordan, CEO of the Institute, suggested a therapy under consideration — using stem cells derived from a patient’s fat tissue. In May 2008, Walker flew to the clinic where doctors removed samples of his abdominal fat through a mini-liposuction, drawing out stem cells, which were later re-injected. According to Dr. Riordan, Walker and a colleague were the first to undergo this treatment protocol. “My quality of life has improved significantly,” Walker told the Post. “The problems with depression, fatigue, and balance have been corrected. I feel really good.”

In June 2009, Walker, who continues to take Avonex as a maintenance drug, plans a return trip to Costa Rica for a “tune-up,” as he puts it. “I’m curious to see if they can further improve my cognitive abilities.”

Breakthroughs on the Brink: Turning the Tide on MS The Saturday Evening Post

“It’s a fascinating time in MS research,” says Patricia O’Looney, Ph.D., vice president of biomedical research at the National Multiple Sclerosis Society. “The knowledge is so great now.”

Multiple sclerosis is an unpredictable and often disabling disease of the central nervous system. It is believed to be an autoimmune disease, which means that the immune system reacts against itself. One of its quirks is that its progress, severity, and symptoms are different in everybody.

An estimated 400,000 people in the United States are affected by MS. Someone is diagnosed with MS every hour, every day of the year.

Symptoms that might send someone to a neurologist for tests include: loss of mobility; numbness, tingling, or pain; and sudden, often temporary, blindness. People may have mysterious, erratic symptoms for years before diagnosis. Most people — two to four times as many women as men — are diagnosed between ages 20 and 50.

Not surprisingly, genetics plays a major role in current research. “So far, researchers have found as many as 12 genes (out of about 20,000 in the human genome) that are linked to MS. Most of these are connected to the regulation of the immune system, which is inherited from our parents. In the end, we may find as many as 80 MS genes,” says Dr. O’Looney. “Identifying these genetic markers will help us understand why there’s such variability and unpredictability in the disease. There might even be some genes linked to particular types of MS. When you identify genes, it may lead to targets for new therapies.”

By eating away myelin - the protective nerve coating - MS shorts out the flow of information within the brain and between the brain and body, similar to how a stripped electrical wire can short out an appliance.
Image © 2009 RelayHealth and/or its affiliates

Even with genetic susceptibility, something has to trigger the disease, and researchers are looking for these catalysts. One hypothesis is that a lack of sunlight and vitamin D — which, in part, regulates the immune system — might play that role. One bit of evidence is a higher prevalence of MS the farther you live from the equator in your early years. What complicates this research is that there may be different triggers in different people. If a virus is the culprit, as many suspect, it likely has vanished from the body years before the onset of symptoms.

With more than 130 clinical trials under way around the world, including more than a dozen phase 3 (late-stage) trials in process, nearing completion, or finished, MS research is making huge strides toward solving these unknowns. Many of these trials focus on treatment. The vast majority of people with MS have a type called relapsing-remitting and take one of the FDA-approved disease-modifying therapies. Fifteen years of data demonstrate their effectiveness. “What research has told us is that the disease is much more active than originally thought,” Dr. O’Looney explains. “Even if a person is aware of one or two flareups a year, the disease could still be active, although asymptomatic, particularly in the brain. There are times when a person feels OK, but damage is taking place. That’s why the National MS Society advocates so strongly that people diagnosed with MS should be on drug therapy.” Not everyone responds the same to these medications, so having several to choose among is important. One option we can expect to see within a year or so is an oral medication, which may be offered alongside or as a replacement for the injectable medications.

Another promising line of inquiry is understanding how sex hormones may regulate the immune system in people with MS. For example, levels of the hormone estriol typically rise significantly during late pregnancy. This is also when most women’s MS activity declines, leading some scientists to suspect that estriol may be responsible for the easing of symptoms during pregnancy. Preliminary results in a small pilot trial showed that oral estriol treatment decreased disease activity in women with relapsing-remitting MS. To further explore this, a larger trial has begun at the University of California at Los Angeles and several sites throughout the United States.
“Five or 10 years from now, it is certainly our hope that more and increasingly effective MS therapies will be available,” Dr. O’Looney concludes. “Our goal is to find a cure for everyone, and we will, eventually.”

To learn more, call 800-344-4867 or visit www.nationalmssociety.org.

Terri Garr

Courtesy: National Multiple Sclerosis Society

Garr takes one of the MS disease-modifying therapies, which has slowed the progression, she says. She also exercises for an hour a day on a recumbent bicycle. “It helps strengthen both my arms and legs and is aerobic as well. Plus, I can read or watch TV while I’m on it. In the summer, I swim, which I really love. It’s vital to keep moving.” She eats healthy, balanced meals and “was blessed with a dancer’s metabolism. I eat pretty much what I like, but in moderation.

“I decided to go public with my diagnosis partly because rumors had begun circulating about my health, and I wanted to be the one to set the record straight. Besides, what’s worse? Being disabled or being a woman over 50 in Hollywood? Once I went public about my MS, I quickly realized that I would be able to help people in the same condition, and there was nothing to be ashamed of. If people could relate to me through my work, why not in my real life? I became a spokesperson for MS Lifelines, a patient education program, and traveled the country speaking to people with MS, trying to encourage them to find out everything they could about the available therapies and how they could move forward with their lives. I still receive e-mails and fan letters that thank me for sharing my story and helping take the stigma and mystery away from MS.

“The most important thing I tell people with MS is to have a positive mental attitude and keep moving! I also advise anyone
recently diagnosed to relax. The status of MS research has never been more exciting. People talk about the ‘war’ against MS; this isn’t a war, but a movement by and for people with MS.

“Together we will create a world free of multiple sclerosis.”

Montel Williams

© Montel Media Group

Without a trace of self-pity, Williams admits he has 24-hour pain in his feet and side, and that it has recently migrated to his face. He’s tried many remedies, from over-the-counter painkillers to opiates, and from acupuncture to biofeedback. He deals with his condition by raising awareness and funds for MS research through the Montel Williams MS Foundation. “MS is a lifelong battle. There’s no wonder drug that arrests it with true efficacy,” he says. “We’re in a battle of time.” He also actively supports the Partnership for Prescription Assistance (pparx.org), which matches patients with free or nearly free prescriptions.

“The most important thing you can do as a patient, no matter the illness, is educate yourself,” he adds. “There is nothing more profoundly assuring and calming than knowledge.”

Unraveling Clues to Multiple Sclerosis The Saturday Evening Post