The Cure Within:
Unlocking the Secrets of Our Stem Cells

The elusive fountain of youth isn’t in Florida. But it may be in your body. At the earliest stage of life, the cluster of cells in a mother’s womb are stem cells. These embryonic stem cells carry all the instructions needed to make a whole person.

Within a few days the cells begin to specialize, eventually creating more than 200 different kinds of cells. Some will head off to make heart muscle, some form skin, and others account for bone. When the body is fully grown most of the stem cells will have fulfilled their purpose and they die off.

Some rare stems cells, referred to as adult stem cells, continue to make new cells, to replenish the body with specific things like blood cells or skin tissue, for example.

Naturally, this—why some stem cells continue to function and others do not—has made scientists curious. Salamanders and starfish have the natural ability to regenerate a limb if it is severed. But in the human body—if a nerve is severed, if knee cartilage wears out, or heart muscle is damaged—there is no biological way to fix the problem. This may change in the not-too-distant future, however.

Petri dishes incubating adult stem cells.
What the excitement is about Scientists at the University of Minnesota and around the world are so excited about breakthroughs that have come in stem cell research during the past few years, that it’s difficult not to talk about potential cell therapies. The list is breathtaking.

In the next decade we have good reason to believe there will be cures for hemophilia, diabetes, multiple sclerosis, and Parkinson’s and Alzheimer’s diseases and regenerative therapies for worn out joints, damaged heart muscle, bones that won’theal, and stroke damage.

That’s just the short list. In theory, every organ of the body can benefit from the knowledge that is now being collected in preparation for clinical tests.

The University’s Stem Cell Institute is in the thick of discovery. Under the leadership of Catharine Verfaillie, the institute—the first of its kind in the country—is held in high regard among those pursuing answers to stem cell behavior.

This year Verfaillie was named one of the nation’s 10 leading innovators in science and technology by U.S. News & World Report. Most recently the Michael J. Fox Foundation for Parkinson’s Research honored the institute with a one-year $100,000 grant.

A key discovery made at the University A little more than three years ago Verfaillie and the University began to rewrite a few pages from the textbook on stem cell biology.

In a project designed to grow stem cells in a petri dish to regenerate bone and cartilage for a rare childhood genetic disease, something was changed in the regular way of doing things. Verfaillie instructed her lab researcher Morayma Reyes to leave an ingredient that might have been harmful to the child out of the medium normally used for growing stem cells.

The result was surprising. What Verfaillie saw when she peered into the microscope was completely unexpected. Instead of seeing only stem cells for bone and cartilage, the culture dish also included endothelial stem cells, cells that line blood vessels.

This was a huge discovery Until then it had been believed that an adult stem cell could only develop into its own rigid cell type—blood, skin, smooth muscles, and so forth. But evidently a stem cell has chameleon-like qualities, able to change its function to match the surrounding environment.

Verfaillie’s team saw an opening and immediately began studying how modified growing conditions could trick an adult stem cell into being as flexible as an embryonic cell. Soon the group was able to grow solid bone that actually stuck to the bottom of a culture dish. They’ve been able to grow little balls of cartilage—imagine what those might do for a bad knee joint?

With each investigation, the team made slight changes to a stem cell’s growing habitat to see what they could get. They tinkered with the recipe to mimic conditions and timing that take place during a stem cell’s natural evolution.

One day what they saw astounded them. Heart muscle stem cells were found in a culture dish, beating. In addition, they found functioning brain and liver cells.

From bone marrow to stem cells Bone marrow has been the primary source of adult stem cells since the 1960s when University of Minnesota immunologist Robert Good successfully transplanted bone marrow for the first time. The University has been at the forefront of bone marrow use ever since. In fact, Verfaillie came from Belgium in 1988 as a hematologist to learn how to perform bone marrow transplants under Philip McGlave, another pioneer in the field.

An adult stem cell project begins by extracting a small amount of bone marrow, usually from the hip-bone area. Bone marrow is used because it contains more adult stem cells than can be found elsewhere in the body. The bone marrow is then puried in a four- to six-month process that allows stem cells, but not other cells, to thrive. The new stem cells can be frozen and later used one cell at a time to answer investigators’ questions.

Catherine Verfaillie, director of the Stem Cell Institute, holds three endowed chairs: the McKnight Presidential Endowed Chair, the Andersen Chair in Stem Cell Biology, and the Edmund Wallace Tulloch and Anna Marie Tulloch Endowed Chair in Stem Cell Biology, Genetics, and Genomics.
The value of adult stem cells At about the same time Verfaillie revealed the potential of adult stem cells, a storm was brewing over the ethical use of unneeded embryos—previously frozen for in vitro fertilization—as a source of stem cells.

So the timing of Verfaillie’s discovery couldn’t have been better. While research using embryos could continue abroad, the University and other American research centers pursued research on adult stem cells in order not to lose important federal funding.

Adult stem cells may one day eliminate the need for organ transplants. In 5 to 10 years it is predicted that doctors could remove the stem cells from the body of someone with a damaged organ, purify the cells, genetically alter them, and place them back in the patient’s body to grow a new, healthy organ. And because the DNA would be a perfect match, there would
be little or no need for antirejection drugs.

An interdisciplinary effort More than 50 different stem cell research projects currently are under way at the University—from the College of Veterinary Medicine to the Institute of Technology.

When a stem cell needs to be genetically corrected, the work moves from the Stem Cell Institute to the new Center for Molecular and Cellular Therapy in St. Paul. To generate the quantity of cells that will eventually be needed for a prospective transplant, the institute has opened a dialogue with chemical and biological engineering to build a bioreactor—technology that employs fluid vats and micropatterning to grow massive amounts of stem cells efficiently.

It is also an international effort. The institute is collaborating with research units in Belgium, France, Denmark, and Germany, as well as the United States, to speed the work.

On the horizon The institute has three diseases in its immediate sites: diabetes, hemophilia, and mucopolysaccharidosis (Hunter’s syndrome). These are diseases known to be controlled by a single factor—a missing islet, protein, or enzyme. The singular focus for these diseases makes the work of the institute a little easier and, in the eyes of the Food and Drug Administration (FDA), less risky. The FDA has the final say on when clinical studies may begin.

Until the FDA gives the high sign for these types of studies, stem cells can help in drug discovery. Until now, no fool-proof method existed for pretesting a new drug for unwanted side effects. But because stem cells are like the human body in miniature, they can be used, rather than an actual patient, to test drug toxicity.

In the short run, drugs may also provide a quicker, less costly solution than stem cell therapy. To develop solutions for chronic or degenerative diseases, investigators are treating defective stem cells with an assortment of drugs to see which, if any, can correct the cells.

There’s a lot bubbling at the Stem Cell Institute. After some important findings, researchers are practically on the edge of creating a whole new medical industry. And for patients and doctors alike, that could be a big fountain of joy.

—by Carl Franzen

get more info For more inofrmation on stem cells, call the Stem Cell Institute at 612-626-4916.


Contact the editor
Last modified Feb 28, 2001
© 2001 by the Regents of the University of Minnesota
The University of Minnesota is an equal opportunity educator and employer.