What if muscular dystrophy (MD) could be treated with an injection of cells that build new, healthy muscle tissue? This is what some researchers hope will be the future of stem cell therapy. While there are no approved stem cell treatments for MD yet, researchers are studying safe ways to make this possible.

In this article, we’ll explain stem cell therapy and how it might help treat MD. We’ll also cover the potential risks, benefits, and frequently asked questions (FAQ) about stem cells as a therapeutic approach for MD.

What Are Stem Cells?

Stem cells are special cells that are capable of making many specialized cells in your body. There are two important qualities of stem cells:

• They can make more stem cells in a process called self-renewal.
• They can turn into specialized cells, with specific jobs, in a process called differentiation.

Our bodies have different types of stem cells that help maintain and repair tissues. For example, mesenchymal stem cells (MSCs) help make new muscle, fat, and cartilage. Hematopoietic stem cells make new blood and immune cells.

Stem cells are also grouped by how many cell types they can make.

• Pluripotent or embryonic stem cells — These can become any cell type in the body. They come from donated umbilical cord blood or embryos from in vitro fertilization (IVF).

• Multipotent and unipotent stem cells — These are more specialized and can only make new cells based on the tissue they live in. For example, multipotent adult stem cells found in the bone marrow can only make certain types of blood cells.

Induced pluripotent stem cells (iPSCs) are made in a lab. With the right genetic changes, iPSCs can act like embryonic stem cells. Researchers use these cells to study new therapies and to learn how diseases affect the human body.

What Is Stem Cell Therapy?

Stem cell therapy — also known as regenerative medicine — uses stem cells to repair damaged tissues and treat diseases. Researchers are studying how to turn stem cells into specialized cells. The idea is to implant or inject these healthy cells to replace diseased ones.

One example of stem cell therapy available today is a bone marrow transplant. This treatment replaces unhealthy blood-forming hematopoietic stem cells in the bones with healthy adult stem cells from a donor. Bone marrow transplants are used to treat blood cancers like leukemia, lymphoma, and myeloma.

Potential Benefits of Stem Cell Therapy for Muscular Dystrophy

Stem cell therapy has the potential to treat other diseases too — including MD. MD is a neuromuscular disorder that causes progressive muscle weakness. Symptoms vary depending on the type of MD you have. Most muscular dystrophies involve muscle wasting that makes it hard to walk, talk, eat, and perform other daily activities.

Researchers hope stem cell therapies will repair damaged muscle tissue and improve the quality of life of those living with MD. Once muscle tissue is lost, it’s hard to regrow. The goal is to add healthy cells to weak muscles, making them stronger again.

Current treatment options for Duchenne muscular dystrophy (DMD) — the most common type of MD — focus on fixing problems with the dystrophin protein. Gene therapies repair issues in the genetic code of muscle cells. However, they don’t replace damaged muscle tissue or fix degeneration.

Unfortunately, the available gene therapies — called exon skipping therapies — only work on some people. For example, eteplirsen (Exondys 51) may work for only 13 percent of children with DMD. These treatments can’t cure DMD, and some people end up having milder symptoms similar to Becker muscular dystrophy (BMD).

Stem cell therapy for MD could work for many types of MD and other degenerative conditions. Researchers believe it has the potential to treat these diseases, and there are promising studies underway, especially for DMD.

Stem Cell Therapy for Duchenne Muscular Dystrophy

Researchers are studying how stem cell therapy might help treat Duchenne Muscular Dystrophy. So far, all studies using stem cells have been done in mice. In one study, scientists turned human skin cells into iPSCs, then into muscle cells, and transplanted them into mice. The mice’s bodies accepted the new muscle cells, which produced healthy dystrophin proteins.

Using this research, scientists are working to create new skeletal muscle cells from stem cells. In July 2024, the U.S. Food and Drug Administration (FDA) approved a new stem cell therapy for clinical trials, called MyoPAXon. These large, well-regulated studies will test how safe and effective this stem cell treatment is for people with DMD.

The goal of MyoPAXon is to use muscle stem cells created from iPSCs to build new skeletal muscle tissue. The new muscle fibers from the stem cells would make dystrophin proteins, which could improve muscle strength and reduce muscle wasting.

MyoPAXon trials haven’t started yet, but you can ask your healthcare provider for updates on the study and whether you or your child might qualify to join.

Potential Side Effects of Stem Cell Therapy

Stem cell therapies are still being studied for many diseases, and there are risks. Possible side effects and safety concerns of stem cell treatments include:

• Bacterial infections
• Blindness
• Immune system reactions causing inflammation
• Abnormal growths or tumors
• Reactions at the injection site
• Veno-occlusive disease (VOD)

Frequently Asked Questions (FAQ) About Stem Cell Therapy for Muscular Dystrophy

Stem cell therapy is an exciting scientific field that’s constantly evolving. Here, we’ll answer some questions you may have about stem cell therapy for MD.

Can Stem Cells Cure Muscular Dystrophy?

Right now, we don’t know if stem cells can cure MD. Researchers are studying whether they can help treat the condition.

How Long Do Stem Cell Injections Last?

We don’t know yet how long stem cell injections or transplants will last for MD. Early studies in mice show that stem cells can become part of muscle tissue. As clinical trials begin, researchers will learn more about how these cells interact with human skeletal muscles.

Where Can You Find Stem Cell Therapy in the United States?

The best way to receive stem cell therapy is under the care of a trained medical professional. Joining a stem cell therapy clinical trial is one of the best ways to try this experimental treatment. Talk to your healthcare provider if you’d like to learn more about trials you or your child may qualify for.

How Expensive Is Stem Cell Therapy?

The cost of stem cell therapy depends on the type of treatment. Right now, the FDA hasn’t approved any stem cell treatments for MD. If you join a clinical trial for a new therapy — like MyoPAXon — treatment costs are usually covered by the company running the trial. Your insurance plan may also cover doctor’s visits and other routine care. A clinical trial coordinator can help explain any costs involved.

Connect With Others Who Understand

On myMDteam — the social network for people living with muscular dystrophy and their loved ones — members share their experiences, ask questions, and find support from others who understand the challenges of living with MD.

What questions do you have about stem cell therapy for muscular dystrophy? Would you join a clinical trial to try this treatment? Share your thoughts in the comments below, or start a conversation by posting on your Activities page.