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Stem Cell Research: What Does It Mean for Scleroderma?

By Carolyn Weller, R.N. and Elaine Furst, R.N., B.S.N., M.A. (originally published in "Scleroderma Voice," 2001 Issue #3)

Carolyn Weller, R.N.

Carolyn Weller, R.N.

Editor's note: You can skip directly to the subheads that interest you, by clicking on these links:

President rules on embryonic stem cell
—New York Times

Parents freeze umbilical cord blood for stem cell use in the future
—Seattle Post-Intelligencer

Stem cell research might help cure Alzheimer's, Parkinson's and diabetes —Newsweek

Whew! What has been going on over the past few months? The headlines above come from leading newspapers and magazines. The Internet also has many articles and web pages devoted to the stem cell controversy.

The stem cell issue involves elements of ethical, religious, and scientific thought near and dear to each of us in some way. It can be very confusing to patients and families affected by scleroderma, as well as those of us who are involved with scleroderma support, awareness, and research funding.

The stem cell research controversy is a good example of how science, government, and ethical concerns can overlap.

Celebrities such as Michael J. Fox, who has Parkinson's, and Mary Tyler Moore, who has diabetes, both made news when they testified before Congress, adding to the number of stories on TV.

Because we've heard some confusion among our own members, this article will try to bring some light to the stem cell debate raging in Congress and the media, and show how it relates to people affected by scleroderma.

What exactly is a stem cell?

Let's start by explaining what an embryonic stem cell is. Immediately upon fertilization a human egg begins to divide. One cell becomes two, two cells become four, four cells become eight, and so on.

After several cycles of cell division, these cells begin to form a hollow sphere. This sphere is called a blastocyst. This blastocyst consists of two layers. The outermost layer of the blastocyst will eventually develop into the placenta and other supporting tissues that will support the fetus.

The inside layer is a hollow sphere that contains a cluster of unique cells. These cells will go on to form many different types of tissues. It is these cells, which contain the potential to develop in many different directions, that are referred to as the stem cells.

Is there more than one kind of stem cell?

Yes, there are two main types of stem cells: the embryonic stem cells described above, and stem cells that can be isolated from adult tissues. Both types can be used for research, but many researchers prefer to work with embryonic stem cells, because they are easier to isolate and reproduce faster.

1. Embryonic stem cells

The embryonic stem cells that have been used for research were isolated from frozen embryos, donated by infertile couples that no longer require them for pregnancy purposes.

Embryonic stem cells have the potential to help researchers in many ways. For instance, they could help unlock the mystery of human development at the cellular level-including what occurs during cell division that causes birth defects and cancer.

Many researchers believe they can be helpful in developing new drug therapies for a variety of diseases. For example, it could be useful to study the efficacy and safety of a drug at the cellular level before beginning studies with animals and humans.

One particularly exciting aspect of stem cell research is that scientists have been able to stimulate stem cells to specialize and develop into a variety of specific tissue types. This opens the possibility of a renewable source of replacement cells and tissue to treat many different diseases and conditions. Specialized heart cells encouraged to grow in a lab setting could be injected into a diseased heart, and ideally these new cells would work to heal the heart by continuing to grow new, healthy cells. Other diseases, conditions, and disabilities that could potentially be treated with stem cells include Parkinson's and Alzheimer's diseases, spinal cord injury, stroke, bums, diabetes, osteoarthritis, and rheumatoid arthritis. According to the National Institutes of Health (NIH), "There is almost no realm of medicine that might not be touched by this innovation" (Stem Cells: A Primer, National Institutes of Health, May 2000).

2. Adult stem cells

Adult stem cells also show promise. For example, stem cells have been isolated in the hair follicles of adult mammals, and in theory could become the specialized cells that would generate new hair follicles, epidermis, and sebaceous glands. This development could improve the results of skin grafts for burn patients, help us understand the origins of some skin cancers, and be used to treat hair loss and excess body hair.

Stem cells have also been isolated in the bone marrow of adult rats. Scientists then altered the cells' environment, transforming them into nerve cells. These cells were injected into the brains of rats, where they took up residence and formed connections with other nerve cells. They are reported to have survived within the brain for months without any adverse effects. Scientists are hopeful that similar procedures could be used to repair damaged areas of the brain.

Stem cells have also been isolated in the blood, and in a lab setting have been used to produce liver cells. This line of research could significantly impact our ability to produce organs and tissues for transplants. If the necessary tissues could be produced from cells taken from the patient, it might be possible to avoid rejection complications.

As noted above, there are some drawbacks to using adult stem cells, compared to embryonic stem cells. They are more difficult to isolate, exist in smaller numbers, and researchers have not been able to isolate stem cells from all of the adults' tissues. It has been suggested that adult stem cells may have more genetic errors than embryonic stem cells, and these errors could be perpetuated in the cultured stem cells.

Dr. Daniel Furst has been in the news for his stem-cell rescue involving scleroderma patients. What does his procedure consist of?

The procedure used by Dr. Furst in Seattle and by other centers in Detroit and California consists of isolating stem cells from the blood of the patient who will be receiving the transplant. These stem cells are preserved for later reintroduction to the body of that same patient.

Next, the patient is given chemotherapy and total body radiation. This destroys all the body's immune system cells. The reasoning is that the patient's existing immune system is not functioning properly, and needs to be replaced.

The stored stem cells are then reinfused into the patient's blood stream. The therapeutic goal is for the stem cells to take up residence in the patient's bone marrow and begin to function as a normal immune system. This is called an autologous transplant.

This stem cell procedure does not use embryonic stem cells and therefore is different from the type of stem cell research that has recently been in the news.

What kind of stem cell research was President Bush talking about when he announced the federal government's guidelines?

President Bush has made a decision to allow federal funding of research through NIH using existing "stem cell lines." These are stem cells whose original sources were human embryos. The President stated the 60 existing stem cell lines have "great promise that could lead to breakthrough therapies and cures. This allows us to explore the promise and potential of stem cell research without crossing a fundamental moral line by providing taxpayer funding that would sanction or encourage further destruction of human embryos that have at least the potential for life" (cnn.com, August 10, 2001).

President Bush has also endorsed increased funding for research on stem cells obtained from adults and from umbilical cords, placentas, and animals. About $250 million will be spent on this type of research this year.

The President has also announced the creation of a council on bioethics, responsible for monitoring scientific and ethical concerns as the research proceeds. This council will be chaired by Leon Kass, M.D., Ph.D., of the University of Chicago.

What is the Scleroderma Foundation's position on stem cell research?

The Scleroderma Foundation has consulted its Medical Advisory Board, chaired by Maureen Mayes, M.D., for a position on this matter. The Medical Advisory Board has stated: "The Scleroderma Foundation supports excellence in medical research and will follow the guidelines of the NIH regarding the conduct of research."

Is stem cell research likely to help find a cure for scleroderma?

It is much too early for us to answer this question, but at the very least stem cell research may become important in the future by helping researchers understand the basics of how cells work.

We are here to help you

We hope this information helps you weave your way through all the information on the news about stem cell research. Please be sure to call us at the Scleroderma Foundation's National Office if you need further clarification (800-722-HOPE). We can also help you find medical centers, in addition to Dr. Furst's in Seattle, that are doing adult stem cell rescue for patients with severe systemic scleroderma.

Acknowledgement

We thank Daniel Furst, M.D., for reviewing this article for accuracy.

Additional Reading

Below we list some references if you want to leam more about stem cell research.

Publication

National Institutes of Health, Stem Cells: A Primer (May 2000)

Websites (search them for phrase "stem cell")

www.cnn.com
www.sciam.com
www.usatoday.com

Medical Research Center

Virginia Mason Medical Center, Seattle, Washington.

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