What is Stem Cell Therapy?

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What are Stem Cells and How Does Therapy Work?

Stem cells are the building blocks of all cells in the human body. Every cell in our bodies begins a stem cell and, through a series of genetic signaling, transform into the various cells that make up the human body. In Duchenne, a signaling error prevents some of these stem cells from transforming into the muscle fibers needed to generate and repair muscle, leading to muscle wasting. There are numerous forms of stem cell therapies. Our library contains a compilation of all therapies. Below is information on stem cell transplantation.  


There are two types of stem cell transplants; allogeneic and autologous. Allogeneic stem cell transplants are stem cells derived from a donor, via bone marrow, muscle fibers, fat tissue, and some times umbilical cord blood. The donor's blood must be matched to the recipient to ensure there is no rejection of the stem cells post-transplant. Autologous stem cell transplants take the patient's own stem cells and reintroduce them into the body. This type of transplant does not pose a risk of rejection since the cells are derived from the patient themself. Both of these procedures have been performed in Duchenne patients and both types of stem cells (from donors and from patient) have shown promise in the treatment of Duchenne.


Stem cell transplants are gaining recognition as a possible therapy for Duchenne around the world. In the US, stem cell therapy is currently used for certain cancers, cardiac infarction, Alzheimer's, Parkinson's disease, and stroke. There are currently several US stem cell trials for Duchenne in the clinical stages, and one application for Compassionate Use of stem cells for Duchenne has been approved by the FDA. Canadian researchers have also just reclassified Duchenne as a muscle stem cell disease, which will certainly lead to more clinical trials in the very near future.

Current Stem Cell Therapy for Duchenne

There are now over 100 in-vitro (non-human) and clinical (human) trials in progress around the world, with 7 current clinical trials in the US. Some of the most extensive  stem cell research is happening in India at the NeuroGen Brain and Spine Institute. Dr. Alok Sharma, executive director of NeuroGen and a global leader in stem cell therapy and Duchenne, has successfully treated over 400 Duchenne patients with stem cell therapy and over 800 patients with other dystrophies or other similar illnesses. In Ukraine, approximately 50 DMD patients were also treated with stem cell transplant therapy in clinical trials. France researchers conducted a clinical trial on stem cell transplant therapy for 10 throat muscular dystrophy patients. All clinical trials showed positive results. 


Along with stem cell transplantation, granulocyte-colony stimulating factor (G-CSF) is another therapeutic option for DMD. Commonly used prior to stem cell extraction, G-CSF has been shown to be therapeutic on its own as well. G-CSF therapy involves the mobilization of a patient's own stem cells without the need for a stem cell transplant. G-CSF stimulates the production of stem cells, allowing for more stem cells to reach maturation, resulting in increases in skeletal muscle mass, strength, respiratory function and satellite cells. Studies in Poland, Brazil and Japan have demonstrated the efficacy and safety of G-CSF therapy. Studies in the United States have long-explored the safety of G-CSF for a variety of diseases and conclude that G-CSF is a safe and effective therapy option. We invite you to explore the other pages here to learn more about research and clinical trials for Duchenne stem cell therapy.


A Brief History

In the mid 1800s it was discovered that cells were essentially the building blocks of the body and that some cells had the ability to produce other cells. Early experiments included attempts to fertilize mammalian eggs outside of the body. In the 1950's scientists attempted to combine the blood of healthy mice with those of diseased mice in an effort to cure the disease. This was among the early research which sought to use elements of our own bodies to cure ailments.

In 1968, the first bone marrow transplant was performed to successfully treat two siblings with severe combined immunodeficiency. Other key events in stem cell research include:


  • 1978: Stem cells were discovered in human cord blood
  • 1981: First in vitro stem cell line developed from mice
  • 1988: Embryonic stem cell lines created from a hamster
  • 1995: First embryonic stem cell line derived from a primate
  • 1997: Cloned lamb from stem cells
  • 1997: Leukemia origin found in hematopoietic stem cells, indicating possible proof of cancer stem cells
In 1998, Thompson, from the University of Wisconsin, isolated cells from the inner cell mass of early embryos and developed the first embryonic stem cell lines. During that same year researchers at Johns Hopkins University derived stem cells from fetal tissue. Then, in 1999 and 2000, scientists discovered that manipulating adult mouse tissues could produce different cell types. This meant that cells from bone marrow could produce nerve or liver cells and cells in the brain could also yield other cell types. These milestones have given rise to the extensive amount of research and therapies we see today. Today stem cell therapy is being studied or is already employed for many diseases including cancer, heart disease, Alzheimer's, Parkinson's,  Multiple Sclerosis, and traumatic injury, with scientists applying the therapy to new diseases every year- including Duchenne.