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Are stem cell therapies curative for patients?

Health tip
Are stem cell therapies curative for patients?
November 4, 2025

Stem cell therapies, also known as stem cell transplants, are used to treat a variety of diseases. These therapies come in different forms with the goal of decreasing the number of symptoms that a patient may have because of their disease. In some cases, they may be able to cure a patient, but they are not miracle cures for all ailments. Currently, stem cell therapy is only regularly used to cure blood diseases.  

What are stem cells?  

Stem cells are a special type of cell that can regenerate and create different types of cells. The stem cells we use live in the bone marrow.  Stem cells can replace cells that have been damaged or changed by a patient’s disease.  Stem cells can come from the patient, a family member, or someone who volunteers to donate their cells. 

What are some risks of stem cell therapies?  

Receiving stem cell therapy is not an easy process. It is more than obtaining stem cells and placing them inside of the body. A patient must have chemotherapy prior to stem cell therapy to prepare the body to accept the stem cells. Patients require extended periods of time in the hospital to prepare for stem cell therapy and to recover. In total, the process can take over 6 to 12 months. The patient’s body must get accustomed to a new set of cells. This means that children who get stem cell transplants cannot go to school, participate in sports or engage in typical social activities as their bodies adjust to the new stem cells.  

Do stem cell therapies cure diseases?  

We believe that stem cells can cure a limited number of diseases. These include leukemia, immunodeficiencies and sickle cell disease. For all other diseases, stem cell therapies are in experimental phases. Even for diseases that they can cure, they don’t always work: the disease can recur, or the transplant process can cause other medical problems.   

Some patients may no longer have any symptoms of their disease after their transplant. Other patients may begin to have some symptoms again. Patients may have severe complications such as rejection of the therapy, and may continue to have symptoms of their disease. Additionally, these complications do not include the many side effects related to receiving chemotherapy before the stem cell transplant. Because patients can respond so differently to stem cell therapies, we cannot say that stem cell therapies cure all diseases.  

What are we still learning?  

New research studies aim to understand how stem cell therapies can be used for other diseases. Additional studies want to understand how to prevent the complications and risks associated with receiving stem cell therapies. At the Children’s Hospital of Philadelphia (CHOP), researchers are analyzing stem cells as a possible targeted therapy for neurological syndromes that cause severe epilepsy in children, and how to use a patient's own stem cells to fix blood disorders. The field of stem cell research continues to grow with the hope of benefiting more patients in the future. There is promise here, and we hope that stem cell therapies may benefit more children as we learn more. 

References

https://www.research.chop.edu/chop-als20-lentiviral-gene-therapy-for-beta-thalassemia 

https://www.chop.edu/news/chop-researchers-use-human-stem-cells-model-severe-epilepsy-syndrome-and-identify-potential 

https://www.mdanderson.org/cancerwise/pediatric-stem-cell-transplants--what-to-know.h00-159382734.html 

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