Lupus and Stem Cell Therapy
Stem cell therapy is the newest frontier in the quest for better treatments (and possibly a cure) for lupus. What can this therapy mean for you – now or in the future? Read on to find out!
Stem cell therapy, as a treatment for lupus, is relatively new and still experimental. However, stem cells have been used for decades in the form of bone marrow transplants in the treatment of some types of leukemias and lymphomas. Currently, stem cell therapy is being either researched or used clinically in fighting a variety of diseases. These include: Parkinson’s, COPD, ALS, and autoimmune conditions like rheumatoid arthritis, multiple sclerosis, Crohn’s … and increasingly lupus!
Simply stated, stem cell therapy requires taking stem cells from the lupus patient, or from matching donor, and transplanting them back into the patient. Thereby, replacing or repairing damaged cells or even reducing the autoimmune and inflammation response. This, of course, is much more complicated than it sounds and requires a bit more explanation!
Important Note: Though not yet approved for lupus by the US Food and Drug Administration, there have been dozens of studies and hundreds of patients who have undergone stem cell therapy clinical trials and the results are encouraging.
The potential is great, and here are some of the basics you should know!
What are stem cells?
Stem cells are not easy to define. They are the body’s biological expression of “potential.” Meaning, they are cells that have the potential to become something else. When each of us began as one cell, a fertilized egg, that one cell was the ultimate “stem cell.” It was undifferentiated, meaning it had the potential to divide and give rise to every other cell in the body. Through the embryonic and fetal stages, as our cells became more specialized into bone, muscle, nerve, and other tissues, we lost the number of stem cells that remain unspecialized (or undifferentiated). Yet, even as adults, we retain some cells that maintain the potential to change and the ability to create new kinds of cells.
The first identified adult stem cells, called hematopoietic stem cells, and were found in bone marrow. These cells produce all of the new blood cells we need throughout our lives – including the white blood cells that control the body’s autoimmune response. Later, stem cells were found in other places, such as umbilical cord tissue, fat tissue, tooth pulp, and amniotic fluid.
This means that there are several types of stem cells, though they are usually divided into two basic kinds: embryonic stem cells and adult stem cells.
- Embryonic stem cells are obtained, as you might guess, from embryos that are three to five days old. They are usually the unused embryos of in vitro fertilization clinics that are donated for medical research. Their use has been controversial and restricted by some state and federal regulations.
- Adult stem cells, the ones used in lupus research and therapies, come from several sources such as bone marrow (remember the hematopoietic stem cells), adipose (fat) tissue and human umbilical cord blood.
Mesenchymal stem cells (MSCs), sometimes called stromal stem cells, are the most widely used adult stem cells in clinical trials for treating lupus. They are adult stem cells taken from bone marrow, fat tissue or umbilical cord tissue and then grown in a laboratory and processed in a way that promotes their ability to fight autoimmunity and inflammation.
Stem Cell Therapy and Lupus
When might stem cell therapy be used?
Since stem cell therapy for lupus is complicated, expensive and as of yet, unapproved by the FDA, it is and would be suggested for either serious or refractory forms of SLE – after other types of treatments have been attempted.
This means that the first line of treatments, such as immunosuppressives, hydroxychloroquine and steroids like prednisone would be tried first. Then, if needed, biologics, like belimumab (Benlysta), anifrolumab (Saphnelo) and voclosporin (Lupkynis) for lupus nephritis would also be attempted. Stem cell therapy would probably, at least initially, be used after these first two stages of therapy were unsuccessful.
What is the stem cell therapy procedure?
There currently is no standard procedure for stem cell therapy specific to lupus. In most clinical trials, mesenchymal stem cells are either transplanted directly on damaged tissue or more commonly, they are introduced to the blood stream by IV infusion.
In autologous stem cell transplantation (ASCT), these MSCs come from the patient’s own bone marrow. An important benefit to ASCT is that there is no need to find a matching donor.
In allogeneic hematopoietic stem cell transplantation (AHSCT), the stem cells come from a donor’s bone marrow, adipose tissue or umbilical cord blood.
In either case, the stem cells are separated from the non-stem cells and treated in a lab to make them more potent.
After infusion, the MSCs move throughout the body and eventually end up in tissue where they find damaged cells or areas of inflammation. This process can take several weeks or months.
Once the MSCs touch the damaged cells, the healing begins. Amazingly, these stem cells can repair and even replace unhealthy tissue. This is often called regenerative therapy, and in lupus, this has the potential to heal damage in the kidneys, heart and other organs.
Another benefit to MSCs is that they seem to act as immunomodulators, meaning they can “reset” the immune response. Specifically, stem cells are able to produce chemicals, such as cytokines that can suppress the autoreactive T cells that cause so much damage with lupus. Some studies have found that they can also enhance the role of Regulatory T cells (Tregs), which are one of the body’s natural ways of slowing the immune response.
The Benefits of Stem Cell Therapy:
- MSC’s are relatively easy to get from patients themselves or from donors.
- Many techniques do not require the kind of damaging radiation or chemotherapies used for bone marrow transplants that commonly target blood cancers.
- Stem cells could dramatically reduce the need for standard SLE drug therapies, like steroids, and their side effect
The Challenges and Risks of Stem Cell Therapy:
- Donated (allogeneic) stem cells carry the risk of either being rejected by the recipient or by what is called, graft-versus-host disease (GvHD), where some transplanted cells, specifically white blood cells, from the donor sees the cells of the recipient patient as foreign and attacks them.
- There is the chance that infections can take place as with any type of tissue transplant or blood infusion.
- Stem cell therapy is very expensive, and since it is not yet approved by the FDA, most insurance plans are not going to cover the costs. Stem cell therapies currently can range from $5,000 to $10,000.
- It is still unclear what the very long-term consequences might be for introducing such powerfully active stem cells into a person’s body.
It is an exciting time for stem cell research as a therapy for lupus! It is no longer theory; there are active clinical trials taking place now! Unfortunately, the mechanism for how stem cells work is still unknown and the long-term effects have yet to be completely understood. However, the potential for stem cells to actively attack the autoimmune response and significantly heal the damage caused by inflammation is too great to ignore. The FDA has yet to approve these treatments for lupus, but in the future, stem cell therapy will likely be a gamechanger. Even if stem cell therapy does not prove to ultimately be a cure, it could dramatically improve the quality of life of those living with lupus – especially those who are not adequately helped by current therapies.
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United States Food and Drug Administration (2021, November 11). FDA Warns About Stem Cell Therapies.https://www.fda.gov/consumers/consumer-updates/fda-warns-about-stem-cell-therapies
Author: Greg Dardis, MS
Professor Dardis was formerly the Chair of the Science Department at Marylhurst University and is currently an Assistant Professor at Portland State University. His focus has been human biology and physiology with an interest in autoimmunity.
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