FINDING ALTERNATIVES AT ALL STAGES —Talking with ALS Center Director Jeffrey Rosenfeld

Jeffrey Rosenfeld

by Margaret Wahl

Jeffrey Rosenfeld has directed the MDA/ALS Center at Carolinas Medical Center in Charlotte, N.C., since 1998. He's also chief of neurology at Carolinas and an associate professor of neurology at the University of North Carolina at Chapel Hill.

In 1983, Rosenfeld completed a doctoral degree in neuroanatomy at the University of Connecticut in Storrs, followed by postdoctoral studies at Johns Hopkins in Baltimore.

After deciding that he wanted to treat patients as well as conduct research, he completed his medical degree at the University of Maryland in 1990 and, after a general medical internship, completed residency training in neurology at the University of Pennsylvania in 1994.

From 1994 to 1998, Rosenfeld was on the neurology faculty at Emory University in Atlanta, where he also served as director of the MDA/ALS Center.

Q: How did you first become interested in amyotrophic lateral sclerosis?

A: ALS is perhaps the most devastating of the neurodegenerative diseases. But when you can make a difference in the life of an ALS patient, the magnitude of that difference is very obvious. That became very motivating to me.

Q: How would you describe the philosophy at your center in Charlotte?

A: Our center in Charlotte is unique in many ways. The way it came into being was quite unusual. About six or seven years ago, a group of prominent families in Charlotte got together. Each had been touched by ALS.

Because of their means, they had all been to different areas of the country for care. They came together to discuss what patients without those resources could do, and they then created an endowment of several million dollars within a year. [The center is now supported by the original endowment, with additional support from Carolinas Medical Center, from MDA and from the community.]

When they contacted me, I was directing the MDA/ALS Center at Emory University, and I wasn't looking for a job. I was under the impression the Charlotte families were asking me for ideas on how to start a center, and I was happy to come up there and give them my best ideas. In most instances, those were things that I couldn't do at Emory. I painted the sky — creating the most ideal situation I could imagine if resources weren't an issue.

After a time, it became obvious they were looking at me to be the director. What became of all this was that we were able to create the best-case scenario for care of patients with ALS.

Our philosophy is that we will offer the most aggressive, proactive, multidisciplinary care available, and that care will be delivered independently of patient resources. The stress of getting care should never be more than the stress of having the disease.

Q: Describe your center now.

A: The center now has a treatment team of 24 members who span at least 14 different disciplines, and most of them are dedicated just to our center. We have a physical therapy coordinator, a nutritionist, a speech therapist and a social worker, all of whom are working with us 100 percent of their time. We also have several research coordinators, a nurse manager and a variety of essential support staff members.

Q: What's the first visit like?

A: Patients who come to our ALS program come for an initial two-day evaluation. During those two days, they're seen by all of our team specialists, regardless of the problems they're having. At the end of that process, I sit down with them to tell them two things: what I think is going on — what I think the diagnosis is — and what we're going to do next.

The most gratifying part of this job is watching the transformation in patients and families

Solving problems for patients with ALS is Rosenfeld's priority at his center in Charlotte, N.C.

Q: What goes on in the clinic at a follow-up visit?

A: During our follow-up clinic sessions, up to 40 patients return to the clinic, with their families, and spend the day visiting with each other as well as with our entire team.

If a patient with an unstable gait needs a piece of equipment, he leaves here with the equipment, not just a prescription for equipment. We figure out how to get it paid for after we provide the care.

We have one of the largest, best-equipped communications labs for motor neuron disease patients in the country. When switches and mounts have not been available, we've had them engineered.

Q: What clinical trials are going on at the center?

A: We currently have seven trials going on. In one of our newest efforts, we're doing a pilot study of a multiple drug cocktail. It will be a small, open-label feasibility study, with 10 to 15 patients. There will be a lot of compounds with some potential interactions we will be evaluating. In the treatment of ALS, I think multidrug combinations, or drug "cocktails," will be one of the mainstays of treatment.

There are probably multiple spots along the cascade that leads to motor neuron death that can result in a treatment opportunity. Any single intervention may not have the same impact as adding interventions.

One compound we've just tested is oxandrolone, which is an anabolic steroid. The study showed that there may be a selected group of muscles — those that are weakest at the outset — that benefit the most from oxandrolone treatment.

Q: What do you think about the various theories of ALS causation that are now being discussed?

A: I think what we've learned in the recent past is that there are multiple mechanisms contributing to and interacting in motor neuron death. What's most exciting is that hypotheses that we used to think of as independent now appear to be interrelated.

A few years ago, I would have said maybe one hypothesis would pan out. Now we understand that they cross over.

Q: Some people feel that ALS should be treated as more of a chronic disability, like a spinal cord injury, rather than as a rapidly fatal disease. What do you think?

A: The short answer is that ALS is a progressive disease, while a spinal cord injury is often static. A spinal cord injury patient can accommodate to a problem and move forward from that point. The ALS patient does that, too, but down the road there may be another hurdle to jump.

But we don't tell patients that they're going to die with this disease. Instead, we tell them that they're going to live with this disease. That difference between living and dying with the same disease is very powerful.

That's the challenge for the health care community — to provide different and new opportunities for treatment alternatives. For some people, a new communication device is the difference between working and staying home, between communicating with grandchildren and not. For others, maybe it's the motorized wheelchair that makes the most difference. Or it might be participation in a research trial and contributing to the fund of knowledge.

Instilling hope and offering alternatives at all stages are critical in ALS.

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ALS Research Roundup

MDA Seeks Researchers to Conduct Cord Blood Trial

In response to recent developments, MDA is calling on researchers to begin a trial of cord blood transfusion for ALS.

Transfusions of human umbilical cord blood — which contains stem cells that can form blood and immune cells, and perhaps other cell types — have recently been tested in mice with ALS. At least one U.S. company is offering the procedure to people with the disease, at a price of nearly $25,000 for a one-time intravenous infusion.

After a thorough review, MDA's scientific advisers have concluded that this experimental

This would be the first trial of its kind. Over the past decade, cord blood transfusion has become an accepted treatment for diseases of the blood and immune system, but it hasn't yet been tested in people with neurological diseases.

In 2001, Robert Brown, director of the MDA/ALS Center at Massachusetts General Hospital in Boston, reported that transfusions of human cord blood produced a small but significant increase in the life span of mice with ALS.

But for people with the disease, the risks of the procedure might outweigh its potential benefits. Cord blood transfusion can trigger graft-versus-host disease, a potentially fatal condition wherein immune cells in the transplant attack the recipient's tissues. Also, preparation for transfusion usually involves total-body or partial irradiation and treatment with immunosuppressant drugs, leaving the body vulnerable to infections.

For these reasons, MDA discourages people with ALS from seeking cord blood transfusion until the procedure can be safely and adequately tested in a clinical trial. MDA has requested that researchers respond to its RFA by Feb. 1.

Topiramate Shows No Benefit in ALS

A 20-center, phase 2 study of the drug topiramate (see "MDA-Supported Topiramate Trial," April 2001),

Merit Cudkowicz

The disappointing results were announced at the 13th International Symposium on ALS/Motor Neuron Disease held in Melbourne in November.

The trial was conducted by the Northeast ALS Consortium, with support from MDA and the National Institutes of Health. Cudkowicz, a neurologist at Massachusetts General Hospital in Boston and an MDA research grantee, said there are no plans to do further studies of this drug.

Topiramate was considered a candidate for ALS treatment because it blocks molecular structures called AMPA receptors and decreases release of the chemical glutamate in the area around nerve cells, Cudkowicz noted. AMPA receptors are cell-surface docking sites for glutamate, an excess of which has been implicated in ALS causation or progression.

'Corn Belt' Study Expands Across United States

Naomi Bienfang at the University of Northern Iowa in Cedar Falls has expanded her study of the possible role of environmental exposures in the development of ALS to include people beyond the U.S. corn-growing regions (see October 2002). She's studying people with ALS and people with a similar background without ALS.

Bienfang is now including everyone in the United States for the mail survey portion of the study, while people from the Corn Belt will contribute a survey and a blood sample.

For more information, contact Bienfang at (319) 273-3689 or nomeinroma@yahoo.com.

Gene Behind pmn May Hold Clues to ALS

Two new studies, published in November in Nature Genetics and in the Journal of Cell Biology, reveal the defective gene harbored by the progressive motor neuronopathy (pmn) mouse, used as an animal model of ALS in older studies.

Like humans with ALS, pmn mice lose their muscle-controlling nerve cells (motor neurons) and undergo progressive muscle wasting and paralysis. But because they don't have all the characteristic features of ALS, in new studies, they've been largely replaced by mice carrying mutations in the SOD1 gene — discovered in the early 1990s to cause some 25 percent of familial ALS (FALS).

Still, the defective gene found in pmn mice — which encodes a protein called the tubulin-specific chaperone e (Tbce) — should be considered a possible cause behind unexplained cases of human FALS, the authors of the new studies say.

Tbce normally helps maintain the stability of microtubules, long cablelike structures that provide support and help move essential nutrients within neurons.

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ALS — A Disability or a Fatal Disease?

by Cheryl Carter New

Health professionals both in and out of neurology have told me I will die of ALS. The

Cheryl Carter New

I have had the disease for more than seven years now and am losing the battle to walk. I lost my speech a few years ago.

What I want to know is why ALS isn't treated like any other disability. Why are we told we will die of it? ALS affects voluntary muscles, not the critical ones one needs to live. Eventually it affects one's diaphragm, which is necessary for breathing, but there's technology to restore that function.

Disease or Disability?

Quadriplegics with spinal cord damage aren't necessarily told they'll die of their injuries. Yet their functions are similar to those of us who have ALS.

Quadriplegics have rehabilitation centers. We have hospices.

Comparable diseases such as Parkinson's, multiple sclerosis and various muscular dystrophies are seen by many doctors as disabling conditions — not inevitably fatal diseases. If you have one of these diseases, it's assumed that health care professionals will help you to "overcome" the symptoms and get on with your life.

Too Expensive?

The first time I visited a prominent center for ALS, the coordinator told me it was "too expensive" to prolong my life. She told me to take that trip I'd always wanted to take before "it was too late."

When I asked about research, she said, "Well, last year we thought XYZ would be a cure but it did not work out so we don't get excited about anything now." I left there with no hope and no future.

I'm a researcher by profession. So when I got over the shock of visiting this center I did some in-depth research.

I learned what nerves and muscles are affected in ALS. I learned what devices are currently on the market to overcome the most severe of the problems affecting ALS patients.

I looked at Christopher Reeve, who is making a difference in the world in spite of being on a tracheostomy tube for breathing. I looked at Stephen Hawking, perhaps the world's premier theoretical physicist. He teaches classes, writes books, has fathered children and has a full and active life. When he received his ALS diagnosis in 1963, he was undoubtedly told he would die.

Quality of Life

Too often, health care professionals tell people with ALS: It's too expensive to prolong your life, it's too much of a burden on caregivers, and "you don't want to live that way, do you?"

Let me take these one by one and give you my perspective.

1. It's too expensive to prolong your life.

Who are these people who put a price on a life? Do they know what I will contribute to the world if I live? Do they know the value of my life to those who care for me?

I've talked to many people who choose to live and have willing caregivers in the home. They aren't all rich. In fact, some would be considered low-income by the standards of many health professionals, but they manage just fine.

2. It's too much of a burden on caregivers.

This is a personal matter and not one for judgment by outsiders. There are people to whom a loved one with ALS is so important that they gladly provide care.

I know a woman with the disease whose husband provides such good care, the local hospice program said the family didn't need assistance. Her daughters-in-law have offered to spell her husband so he can get out and pursue his own interests.

My husband is a wonderful caregiver and we have become even closer through the challenges we face.

3. You don't want to live that way, do you?

Let's see — I don't lose my sight, hearing, sense of feeling or the ability to taste. I can still see the beauty of nature, hear the music of the wind in the trees, feel the rain on my cheeks, and experience the love of my husband and family. I can watch the boats go by on my little lake and see Big Bird (our great blue heron) as he walks in his dignified way along the shore.

With technology, I can continue to write and correspond with my family and friends. I can even talk. And there's new technology every day. With a power chair, even if I have to go on a trach I can be mobile and can travel. I may not be able to move, but I can experience the world.

How many people, in their rush to do the next thing, don't experience their world to a tenth of the degree that I may?

Focus on Living

It took many visits with various doctors of neurology and lots of miles on the car, but I finally found a clinic where every person is focused on helping you live with the disease — the MDA/ALS Center at Carolinas Medical Center in Charlotte, N.C. I am fortunate; I have talked with dozens of people who are not so blessed.

Living with ALS or dying of it is a personal choice. It's a choice, not for the health care professional to make, but for each person with the disease.

New, of Inman, S.C., is president of Polaris Corp., a grants consulting firm. She has co-authored four books in the field of grants acquisition published by John Wiley and Sons; a book on negotiation; and numerous articles on various business topics.

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Nodaros Helps Others While Helping Himself

by Tara Wood

Andy Nodaros fully understands why someone who has received an ALS diagnosis might want to slink away to hide from the world and hope the disease will go away.

But you won't catch Nodaros, who learned he had the disease in November 2001, doing any such thing.

Instead, the 56-year-old Brentwood, Pa., man has focused on making a difference by capitalizing on his most notable traits: motivating others and promoting ALS awareness with his keen "talkability."

Specifically, Nodaros has extensively involved himself with MDA and made a big effort to connect with others with ALS.

"I'll do what I can as long as I can for this organization and feel good about it, and that's my way of coping," Nodaros said.

Putting Himself Out There ... Way Out

A key step has been getting the story of how ALS has changed his life into the news media.

Andy Nodaros is joined by his wife, Carol, granddaughters Jordan (left) and Chandler, and friend Drew Gilbert.

Nodaros has given a Pittsburgh-area newspaper up close access to his life for an ongoing series titled "Life Without a Cure." The articles, complete with unflinching details, show the progress of the disease and the effect it's had on his life and family.

Nodaros has been featured in other area newspapers, and was seen in a taped profile and live interview on the local broadcast of the 2002 Jerry Lewis MDA Telethon. He has even traveled to Washington to encourage legislators to increase spending on ALS research.

Nodaros admits that the publicity at times makes him feel like a movie star. But part of his motivation for thrusting himself and his family (he's often accompanied by any of his five grandchildren to help "affect people") into the public eye is that it"s also a way to reach others with ALS, he said.

"I do it because I wanted people to know that, like I have MDA in my corner, they have Andy Nodaros in their corner," he said.

On a smaller scale, Nodaros has taken advantage of a free service of his local cable television company's information station to post an advertisement for anyone diagnosed with ALS to contact him.

The result has been a small but growing network of area friends with ALS with whom Nodaros regularly keeps in touch and works to encourage, either by phone, e-mail or visits.

"I want to let other ALS patients know that Andy Nodaros, who is in every newspaper in this city, on television, does MDA lunches, etc., is not giving up," Nodaros said. "I do have my down time, but I'm not giving up."

Now, with the help of MDA staff, he's expanding his efforts to start an ALS support group for the area, which is south of Pittsburgh. His goal is to include lots of member input and to schedule guest speakers who will give people useful information, such as vendors of mobility products.

A People Person to the Core

Nodaros' upbeat attitude, gift for gab and tendency to pepper his conversations with witty one-liners has helped him enjoy success in both his former and current occupations.

After a successful decade in the bar business, Nodaros decided that he wanted a 9-to-5 job in which he could help people.

A friend suggested what turned out to be a perfect match for his outgoing personality: the Turtle Creek Valley Mental Health/Mental Retardation Center in Homestead, Pa., where Nodaros helps people with cognitive disabilities find and keep jobs.

"It's so much fun. It's challenging to convince managers that giving these people a chance is the right thing to do," Nodaros told a local newspaper.

Nodaros plans to work as much as possible for as long as he can. In his spare time, he strives to maintain his "Mr. Healthy" lifestyle, which has always included plenty of weightlifting and exercise.

A former champion body builder, Nodaros now combines a modified exercise program with a diet filled with herbs, vitamin supplements and "crazy shakes" like a carrot-wheat grass-parsley drink he makes.

Some might find the regimen extreme, but to Nodaros it's all about fighting ALS in any way he can — and that includes not being afraid to look good, too.

Nodaros credits his glowing complexion to a "bronzer" lotion that gives him an artificial, but fresh-from-the-beach-looking tan. Just don't let him get emotional when he's wearing it. "My tan will run!" he jokes. "Recently, somebody said to me, "Boy, you look good, Andy. You'd never know you have an illness," and I said, "As long as there's hair dye, makeup and shoulder pads, I'll be all right.'"

Nodaros knows that his new life with ALS is full of irony: He's a body builder who has a muscle-wasting disease; he works with people with disabilities and is now coping with his own progression toward disability.

Don't expect to hear him moan about it, but do expect an energizing pep talk.

"I would like people to know and understand, that this isn't the end of your life ... to not let this disease dictate what you feel like and want to do,' he said. "Don't give up!".

DROP HIM A LINE! Anyone who wishes to correspond with Andy Nodaros can e-mail him at a.nodaros@att.net.

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Caregiver Support Programs Innovative — But Going Nowhere

by Christina Medvescek

This is the best and worst of times for family caregivers, at least in terms of legislation designed to address their needs.

"There are some positive things — states are recognizing the need to increase funding for home and community-based care," says Sandra Newman, policy specialist for the Family Caregiver Alliance (FCA). But the uncertain economy and shifting national priorities mean that "legislators are really challenged right now by how to do that."

The FCA, a national caregiver advocacy group, sponsored a conference for legislators, policymakers and advocates in October 2001. "Who Will Provide Care? Emerging Issues for State Policymakers" focused on ways to strengthen and support informal family caregiving as part of long-term care (LTC) service delivery.

A summary of the conference proceedings and text of seven policy briefs written expressly for the conference are available at the FCA Web site, www.caregiver.org. The full proceedings, published in 2002, also may be ordered for $30 from FCA, Suite 600, 690 Market St., San Francisco, CA 94104; (800) 445-8106; or through the Web site.

Approximately 12 million Americans, almost half under age 65, require LTC. (LTC refers

"Who Will Provide Care?" focuses on ways to strengthen family caregiving.

FCA says that, to continue providing this level of care, family caregivers need help, such as respite services, in-home assistance, education and training, easy access to information about services, financial help, affordable long-term care insurance and greater control over how service dollars are spent. Working caregivers also need support from employers.

At present, these concerns are being addressed in a piecemeal fashion by individual states and some federal proposals, Newman says.

At the federal level, a proposal to double funding for the National Caregivers Family Support Program (NCFSP), which funds state caregiver support programs, is stalled along with other 2003 appropriations bills. Other promising but immobile proposals include the Lifespan Respite Care Act, which would provide states with grants to expand respite care services, and a proposal to extend Medicare benefits to caregivers.

On the state level, Newman pointed to several successes: California's enactment of a paid family leave program; Hawaii's creation of a statewide long-term care safety net; and Florida's extension of a project that gives cash allotments directly to LTC consumers and allows them to decide how to spend the funds. (For more on these programs, visit the FCA Web site.)

In the current political and economic climate, the key to success is collaboration, Newman says. Caregivers and care recipients should work with local advocacy groups and lobby their state and national representatives. States can be encouraged to maintain and improve existing programs if resources aren't available for new programs.

"It's urgent that people speak up now," says Bonnie Lawrence, communication manager at FCA. "Legislatures all over the country are slashing programs. They need to hear that these programs are essential for public health."

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Scientist Traces His Journey

I Choose to Live: A Journey Through Life With ALS, by William Sinton, 2002, 155 pages. Banbury Publishing, (800) 247-6553, www.banburypublishing.com.

This autobiography chronicles Sinton's experience with ALS, from his first symptoms to his current life relying on a ventilator. It's also a memoir that spans his boyhood adventures, military service in World War II, the nurturing of his interest in science and the progress of a distinguished career.

An astrophysicist and professor of physics and astronomy, Sinton has a scientific nature which permeates the book, whether he's explaining the nature of the disease or a medical procedure he's undergone.

Readers also may find curious some of the factors in Sinton's past that coincide with some theories about what causes ALS. For example, he's experienced significant exposure to mercury, other chemicals and heavy metals in his work; been exposed to viruses and bacteria; and suffered a shrapnel injury in the war.

Sinton, who lives in Flagstaff, Ariz., seems to approach life's challenges with a scientist's analytical thinking, and his conclusion about having ALS is that he, as the title suggests, chooses not to dwell on what he has lost, but on what he still has.

"There are so many things that are interesting in life that I look forward to each day," Sinton writes, crediting his wife, Marge, family, friends and caregivers who enable him to do so.

As is the case with many other self-published memoirs, this book is best approached not as a profound piece of literature, but as a candid, first-person view of how one man with ALS and his family face the challenge of maintaining a good quality of life.

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ALS Chats Gear Up for New Year

As you open the brand new 2003 calendar that you received from Santa Claus last month, MDA's ALS Division would like you to take note of some upcoming events that you're invited to attend — without having to leave the comfort of your home.

Clinical-Research Online Chats

The 2003 MDA clinical-research online chat series is under way, offering you the opportunity to chat live with experts from the front lines of MDA's fight against more than 40 neuromuscular diseases, including ALS.

Two sessions should prove to be of special interest to those with ALS:

• Jan. 22 — Management of Pain with Neuromuscular Disease, hosted by Greg Carter, co-director of the MDA/ALS Center at the University of Washington Medical Center in Seattle. (Read an interview with Carter in The MDA/ALS Newsletter, October 2002).

• Feb. 19 — Respiration/ Ventilation with NMD, hosted by Jerold Reynolds, respiratory therapist in the Department of Neurology at Ohio State University in Columbus.

All clinical-research chats are scheduled on Wednesday nights from 5:30 to 6:30 p.m. (EST). Registration and participation are free. For more information, simply point your Web browser to http://www.mda.org/chat/calendar.html.

Chats Continue

MDA's regularly scheduled chat sessions will also continue in 2003:

Living With ALS, hosted by Jeff "ragingbear" every Monday at 2 p.m. (EST), is for people with ALS and caregivers to share information about life with ALS and ideas for making it better. Jeff received his ALS diagnosis in October 1994, and has used a ventilator since December 1997.

Issues That Matter, hosted by Julie "julie-s" Scurich and David "david-j" Jayne, a longtime advocate of issues important to people with ALS. (This monthly chat, held the last Tuesday of each month from 9 to 10 p.m. (EST), is on hiatus while Jayne continues to recover from surgery.)

ALS Chat Group from Connecticut is hosted by local MDA/ALS Task Force on Public Awareness members Dave "dgals" and Bill "friar-tuck" and MDA Health Care Service Coordinator Elissa "woof." This chat promotes a wide-ranging discussion about ALS and how MDA is helping those affected. It's held the third Thursday of each month from 2:30 to 4:30 p.m. (EST).

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