Advances in Stem Cell Research and Treatments

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artwork showing stem cell changing into bone, heart, nerve cell

Stem cell therapy is an exciting and challenging area of medical research. Stem cells are the basic building blocks of all other types of cells. You have them in your body right now. They are responsible for the repair and regeneration of your body’s tissues. For example, they make wound healing and new blood cell production possible. Stem cell research focuses on harnessing these capabilities to treat human disease.

The usefulness of stem cell treatment is their ability to turn into other types of cells and self-renew. Currently, the only FDA-approved (Food and Drug Administration) use is blood-forming stem cells for bone marrow and blood-related disorders. This includes stem cell transplant to replace diseased or damaged bone marrow. It can treat people with leukemia, lymphoma, multiple myeloma, and other blood disorders. It can also help people when cancer chemotherapy severely damages their bone marrow. All other stem cell therapy uses are investigational.

Stem cells to understand cell differentiation

Stem cell research for medical treatment has three basic arms. The first is to gain a better understanding of how stem cells go from being basic building blocks to being highly specialized organ and tissue cells.

Scientists are studying the genetic and molecular controls that allow the process to happen. The goal is to apply this knowledge to conditions that result from problems with genes or cell division. Cancer and birth defects are examples. Studying stem cells may give scientists insight into how such conditions develop. This, in turn, may help them develop new strategies for treating these conditions.

Stem cells for drug development

Another arm of stem cell research is using stem cell lines to test drugs. Scientists can grow stem cell lines in a laboratory. Once they establish the line, it can essentially renew itself forever. Then, they can take cells from the line and test potential new drugs. This lets them see the effects—either good or bad—in human cells grown in the lab, without testing the drug on an actual person.

An exciting example of this research is in Alzheimer’s disease. Scientists can’t exactly mimic the disease in animals. So, animal models for testing drugs have limited usefulness. Drugs that work in animals don’t necessarily always work in humans. Researchers are currently using stem cells from Alzheimer’s patients to create nerve cell lines. They can test new drug molecules on these human nerve cells to find ones that will treat the disease.

Stem cell therapy for tissue regeneration

The third arm of stem cell research is tissue regeneration. It’s probably what most people think of when they hear about stem cell treatment. It’s an area of regenerative medicine. The NIH (National Institutes of Health) defines it as the process of creating living, functional tissues to repair or replace tissue or organ function lost due to age, disease, damage, or congenital defects.

Today, most tissue regeneration or cell-based therapies start with a person’s own stem cells. The stem cells in your body are tissue specific. This means they can only give rise to the different cells in a specific tissue or organ. Scientists can use these tissue-specific stem cells to regenerate the tissue. For example, they can use stem cells in the skin to grow skin replacement for burns.

Scientists are actively studying ways to engineer stem cells so they can turn into whatever kind of tissue you need. In the future, scientists may be able to transplant these cells using stem cell injection into the circulation or directly into the diseased organ. The goal would be to repair or replace damaged or diseased tissue.

Current areas of stem cell research include:

Some of these areas are in preclinical phases—meaning not in people yet. However, researchers have carried out small human studies in a few areas, such as heart disease and orthopedics. There are significant hurdles to clear before this area of regenerative medicine can go mainstream. Scientists must make sure stem cells can reliably do the following:

  • Make the kind of cells they need

  • Survive in the recipient

  • Integrate into (become part of) the tissue such as the brain

  • Function the way they should for the recipient’s lifetime

  • Not do any harm

Scientists also have to work around the body’s immune system. They are studying ways to make cells and tissues the body won’t reject.
Visit to learn about current stem cell research.

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Medical Reviewer: William C. Lloyd III, MD, FACS
Last Review Date: 2021 Feb 18
THIS TOOL DOES NOT PROVIDE MEDICAL ADVICE. It is intended for informational purposes only. It is not a substitute for professional medical advice, diagnosis or treatment. Never ignore professional medical advice in seeking treatment because of something you have read on the site. If you think you may have a medical emergency, immediately call your doctor or dial 911.
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