How Stem Cells Work

Using Stem Cells to Treat Disease

stem cell applications
HowStuffWorks Lee Dempsey

The first step in using stem cells for disease treatment is to establish stem cell lines, which researchers have accomplished. Next, scientists must be able to turn on specific genes within the stem cells so that the stem cells will differentiate into any cell they wish. But scientists have not learned how to do this yet; so, studying stem cell differentiation is an active area of research. Once scientists can create differentiated cells from stem cells, then there are many possibilities for their use, such as drug testing and cell-based therapies. For example, let's say you want to test new drugs to treat heart diseases. Currently, new drugs must be tested on animals. The data from animal research must be interpreted and then extrapolated to humans prior to human clinical trials. But suppose you could test them directly on human heart cells. To do this, human stem cell lines could be treated to differentiate into human heart cells in a dish. The potential drugs could be tested on those cells and the data would be directly applicable to humans. This use could save vast amounts of time and money in bringing new drugs to market.

Stem-cell-based therapies are not new. The first stem-cell-based therapy was a bone marrow transplant used to treat leukemia. In this procedure, the patient's existing bone marrow is destroyed by radiation and/or chemotherapy. Donor bone marrow is injected into the patient and the bone marrow stem cells establish themselves in the patient's bones. The donor bone marrow cells differentiate into blood cells that the patient needs. Often, the patient must take drugs to prevent his or her immune system from rejecting the new bone marrow. But this procedure uses existing hemopoietic stem cells. How would you use stem cell lines? Let's look at how stem cells might be used to treat heart failure.


Ideally, to treat a failing heart, scientists could stimulate stem cells to differentiate into heart cells and inject them into the patient's damaged heart. There, the new heart cells could grow and repair the damaged tissue. Although scientists cannot yet direct stem cells to differentiate into heart cells, they have tested this idea in mice. They have injected stem cells (adult, embryonic) into mice with damaged hearts. The cells grew in the damaged heart cells and the mice showed improved heart function and blood flow.

In these experiments, exactly how the stem cells improved heart function remains controversial. They may have directly regenerated new muscle cells. Alternatively, they may have stimulated the formation of new blood vessels into the damaged areas. And the new blood flow may have stimulated existing heart stem cells to differentiate into new heart muscle cells. These experiments are currently being evaluated.

One major obstacle in stem cell use is the problem of rejection. If a patient is injected with stem cells taken from a donated embryo, his or her immune system may see the cells as foreign invaders and launch an attack against them. Using adult stem cells or IPSCs could overcome this problem somewhat, since stem cells taken from the patient would not be rejected by his or her immune system. But adult stem cells are less flexible than embryonic stem cells and are harder to manipulate in the lab. And IPSC technology is too new for transplantation work.

Finally, by studying how stem cells differentiate into specialized cells, the information gained can be used to understand how birth defects occur and possibly, how to treat them.

So, if there's so much potential in stem cell research, why all the controversy? Let's investigate the current ethical and political issues.