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Heart failure happens when the heart muscle is too weak or stiff to pump blood effectively. As a result, less oxygen-rich blood reaches the rest of the body. This can lead to symptoms like shortness of breath, fatigue, and swelling in the legs, ankles, or feet.
Around 6.7 million Americans ages 20 and up are living with heart failure. Current treatments focus on managing symptoms and slowing disease progression, but they don’t repair damaged heart cells. A promising area of research is stem cell therapy (SCT) — sometimes also called a stem cell transplant — which aims to help regenerate heart tissue.
“It's been almost three weeks since my stem cell treatment and my energy level is still strong,” a MyHeartDiseaseTeam member shared. “I can walk more than half an hour without stopping and take half-hour bike rides too.”
Stem cells are nonspecialized cells found throughout your body. Think of them as seeds that can grow into different types of cells, depending on where they are and the signals they receive.
Stem cell therapy involves taking stem cells from your own body (autologous) or from a donor (allogeneic) and delivering them to areas of damage in the body to help replace or repair old or damaged cells.
SCT is a developing treatment for heart failure. As a form of regenerative medicine, it aims to repair or replace damaged heart muscle cells.
Several types of stem cells have shown potential to develop into heart cells and support cardiac repair, including:
Bone marrow-derived cells come from the bone marrow — the spongy tissue inside bones that produces blood cells. The first study using these cells for heart repair was published in 2001. In early animal studies, transplanting these cells into hearts with low oxygen levels led to the growth of new, functioning heart cells.
Mesenchymal stem cells are stored in fat tissue and bone marrow. These cells develop into bone, fat, or muscle cells through a process called differentiation. They also help form blood vessels and support healthy blood flow throughout the body.
Induced pluripotent stem cells are created by treating adult cells with special chemicals that reprogram them to behave like embryonic stem cells (ESCs). ESCs naturally develop in early stages of pregnancy and can become nearly any type of cell in the body. Both types are considered progenitor cells — cells that can turn into many different specialized cells.
Standard treatments for heart failure help the heart pump blood more effectively, but most don’t repair the underlying cell damage. Stem cell therapy is a promising option that may offer additional benefits. It often uses a person’s own cells and is based on the body’s natural ability to repair itself. Compared to major heart surgeries, stem cell therapy can also be less physically demanding.
However, even with its promise, stem cell therapy still faces challenges. While animal studies have shown benefits, results in humans have been more mixed.
One study looked at bone marrow-derived stem cells delivered to people 24 hours after a heart attack that damaged heart function. It showed some short-term improvements in heart function after one year, but those benefits were not seen at the five-year follow-up.
Another clinical trial tested the same type of therapy in people with ischemic cardiomyopathy. Researchers found no meaningful improvements in quality of life or how much blood the heart pumped, suggesting limited benefits in some cases.
In animal models of heart injury, mesenchymal stem cell therapy helped increase the release of repair signals that support healing. A review of several animal studies showed that MSCs helped hearts pump more blood with each beat.
Human studies have shown more promising results in certain groups. A 2024 phase 3 clinical trial found that mesenchymal cell therapy may reduce hospitalizations and deaths in people with heart failure, especially in those with inflammation.
While stem cell therapy has potential benefits for people with heart failure, it also comes with important challenges. For the therapy to work, the body must accept the cells, allow them to develop into the right type, and avoid attacking them.
Cell engraftment is when stem cells attach and survive in the body — but most transplanted cells are lost after treatment. Poor cell engraftment may result from factors like the type or number of cells used, how they’re delivered, or how well the body’s environment supports them. When cells can’t take hold, the therapy is less likely to succeed.
Stem cells must not only survive — they also need to turn into cardiomyocytes (heart muscle cells) in a process called differentiation. A 2023 report found that bone marrow stem cells and iPSCs can become heart cells. However, getting them to stay that way and function properly within heart tissue is still a major challenge.
Immune rejection happens when your body sees new stem cells as invaders and attacks them — and it’s especially common when the stem cells come from a donor. Younger donor cells may be more robust, but immune matching remains a key challenge. If the match isn’t close enough, the body may reject the therapy.
Stem cell therapy is associated with other adverse events, such as arrhythmia (irregular heart rhythms) and cancer.
Arrhythmias are heartbeats that are too fast, too slow, or uneven. Animal studies have shown a possible link between arrhythmia and embryonic stem cells used in therapy. However, researchers believe these effects may be temporary and related to how the cells settle into the heart.
Another concern is cancer. Human pluripotent stem cells can sometimes form tumors if they don’t turn into heart cells after therapy. This risk is linked to certain proteins inside PSCs that may support cancer growth.
Scientists are using artificial intelligence (AI) — computer programs that mimic human thinking — to improve how stem cell therapy works. When AI tools analyze large amounts of scientific data, they can find patterns and make predictions. This is called machine learning (ML).
By studying results from both animal and human research, AI and ML help researchers:
AI and ML are part of the growing field of personalized medicine, which focuses on tailoring care to each individual. For people with heart failure, this could mean more effective stem cell therapies with fewer side effects.
Although stem cell therapy shows promise, it still has limitations. Using AI and ML to analyze health records and research data may help scientists solve challenges like low cell survival, immune rejection, and inconsistent results across studies.
Stem cell therapy is an innovative approach to repairing damage in the heart, and it may offer benefits for people living with heart failure. But even as research grows, results from animal studies don’t always match what happens in humans.
Stem cell treatments are tightly regulated. In 2023, the U.S. Food and Drug Administration (FDA) approved a major clinical trial to study bone marrow-based therapy for heart disease. Looking ahead, advances in AI and ML could help improve how these therapies are developed, delivered, and tracked over time.
If you're interested in stem cell therapy, talk to your doctor or cardiologist (heart specialist). Ask about clinical trials and whether this type of treatment may be an option for you. Stay informed together by asking about new research and checking trusted sources for updates.
MyHeartDiseaseTeam is an online community for people with heart disease and their loved ones. On MyHeartDiseaseTeam, more than 62,000 members have built a community to ask questions, give advice, and share their stories.
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