Treating Duchenne Muscular Dystrophy with Umbilical Cord Tissue-Derived Mesenchymal Stem Cells
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The genetic condition known as Duchenne muscular dystrophy (DMD) is characterized by increasing muscle degeneration and weakness. In patients who suffer from muscular dystrophy, the abnormal genes, also known as mutations, interfere with the protein production that is needed in order to form healthy muscles. As a result, patients have problems with their muscles and can experience muscle wasting. Muscle wasting is when a person’s muscles weaken and shrink due to either lack of use or in this case, disease.
Unfortunately, DMD is a condition that continues to worsen over time. The majority of patients who suffer from DMD are men. However, there are rare cases where females are also affected by this progressive disorder.
How Many People Have Duchenne Muscular Dystrophy?
According to statistics, approximately 250,000 people have been diagnosed with DMD. Every year, DMD affects approximately one out of every 5,000 live male births annually.
Global statistics show that approximately 20,000 children are diagnosed with Duchenne muscular dystrophy annually. Across the entire world, approximately one out of every 3500 male births is affected by DMD.
What are the Symptoms of Duchenne Muscular Dystrophy?
People who suffer from Duchenne muscular dystrophy can experience an array of symptoms associated with this disease. The most common symptom that patients experience as a progressive weakness of muscles. Some other symptoms associated with this condition include:
- Problems running and jumping
- Learning disabilities
- Delayed growth
- Walking on the toes
- Frequent falls
- Problems rising from a lying or sitting position
- Large calf muscles
- Waddling gait
- Muscle pain
- Muscle stiffness
As DMD progresses, patients can experience various types of complications. These include:
- Trouble using their arms
- Trouble walking
- Shortening of muscles
- Shortening of tendons around joints
- Breathing difficulties
- Curved spine
- Problems swallowing
- Heart problems
Patients who suffer from DMD may be able to undergo various types of treatment in order to help alleviate some of the symptoms that they experience.
What Treatment Options are Available for Duchenne Muscular Dystrophy?
Although there is currently no cure for muscular dystrophy, there are various forms of treatment and medication that can help prolong the time a patient can continue to be mobile. Treatments and medications may also aid in the development of heart and lung muscle strength.
In many cases, patients who suffer from DMD will have to visit various specialists. These include cardiologists, pulmonologists, endocrinologists, and even orthopedic surgeons. It is important that patients are able to focus on various aspects of their health in order to address any problems before they cause major complications.
In most cases, patients will utilize medications in order to help treat some of the symptoms that they have associated with Duchenne muscular dystrophy. Common medications provided for patients include:
- Corticosteroids: Patients are routinely prescribed corticosteroids, such as Emflaza and prednisone. These medications are designed to help muscle strength and inhibit the progression of various types of muscular dystrophy.
- Eteplirsen: This type of medication is designed to target specific mutations that are present in patients who suffer from DMD. This medication is designed to treat the symptoms of the condition by helping the body produce
- Golodirsen: This medication is prescribed to patients who suffer from DMD, and it works by inducing exons skipping in the dystrophin gene, thus increasing the amount of dystrophy and protein that is available for the muscle fibers.
In addition to medications that are designed to treat DMD specifically, patients may also receive heart medications. These medications are typically angiotensin-converting enzyme (ACE) inhibitors or even beta-blockers. These medications are generally described when DMD damages the heart in the patient.
In addition, patients may have to undergo various types of therapy. These include a range of motion therapy and stretching exercises. These therapies are designed to improve the flexibility and mobility of the joints. Exercise is also recommended. Most patients will engage in low-impact aerobic exercises, like walking or swimming. Braces may also be used to help keep the muscles and tendons flexible and stretched. In some cases, patients may utilize breathing assistance machines or mobility aids like walkers and canes, or even wheelchairs.
If a patient has curvature of the spine, they may have to undergo surgery in order to improve their ability to breathe. Pacemakers and other cardiac devices may also be implanted in cases where cardiac damage occurs as a result of the disease.
Unfortunately, there is no known cure for Duchenne muscular dystrophy. However, over the past decade, we have seen many medical professionals and scientists utilize umbilical cord tissue-derived mesenchymal stem cells in order to effectively treat DMD and patients. The use of UCT-MSCs has been effective thus far.
What are Umbilical Cord Tissue-Derived Mesenchymal Stem Cells?
Mesenchymal stem cells are a type of adult stem cell that can be found in various locations throughout the human body. Umbilical cord tissue-derived mesenchymal stem cells, in particular, are those that are taken from the umbilical cord. These stem cells are typically isolated from the Wharton’s jelly of the umbilical cord.
Umbilical cord tissue-derived mesenchymal stem cells are truly unique in that they have the ability to differentiate into various types of cells. These include cartilage cells, fat cells, and bone cells. In addition, the cells possess exceptional anti-inflammatory qualities, which means they are able to effectively reduce inflammation in various parts of the body. Furthermore, umbilical cord tissue-derived mesenchymal stem cells are considered to be immune privileged, which means that the body is less likely to reject the stem cells after they are injected into the patient.
More and more scientists are studying the use of UCT-MSCs due to the fact that they are considered to be more ethical and well-accepted. There is no loss of life required to remove these stem cells from the umbilical cord tissue.
Over the past decade, we have seen many scientists utilize these stem cells to treat various types of immune disorders and illnesses in the body. More recently, there have been several studies concerning the use of umbilical cord tissue-derived mesenchymal stem cells to treat Duchenne muscular dystrophy. Many of the studies performed have had extremely positive results.
Using UCT-MSCs to Treat Duchenne Muscular Dystrophy
Over the past two decades, we have seen numerous studies surrounding the use of mesenchymal stem cells to treat patients who suffer from Duchenne muscular dystrophy. Most of the studies have proven incredibly effective, with promising results for patients.
In 2009, a study was published by a group of scientists that focused on the use of mesenchymal stem cells to treat patients who suffer from DMD. Cell therapy proved effective when it comes to helping patients who suffer from this disorder. This study, which focused on mice, found that the use of mesenchymal stem cells was effective for rebuilding damaged muscle tissues. Other studies found that those who received the mesenchymal stem cells were able to regenerate new tissue in the dystrophy muscle cells and effectively replace the expression of dystrophin in the muscles of the dystrophin-deficient mice.
In 2010, a study was published in India on the use of umbilical cord tissue-derived mesenchymal stem cells to help patients who suffer from this debilitating condition. After one year, patients were able to achieve stability and increased strength in their muscles, whereas the control group presented decreases in strength within the proximal upper limb. This study further effectively achieved stability in the muscles of the hip extensors, hip flexors, abductors, and paraspinal muscles after a one-year follow-up from treatment. Conclusively, this study found that the use of mesenchymal stem cells was effective in stabilizing muscle power and did not show any adverse effects or deleterious effects on the patients. This further proved that this method of treatment is not only effective but safe as well.
Another study, published in 2020, found that the use of umbilical cord tissue-derived mesenchymal stem cells was effective when it comes to regenerating microfibers. In addition, mesenchymal stem cells effectively suppressed inflammation in patients and affected the immune response in dystrophy mice in order to protect muscles.
Another study published in 2015 found that patients who were injected with umbilical cord tissue-derived mesenchymal stem cells presented less tissue damage and less muscle injury at their 12-week follow-up. They also experienced no adverse reactions and showed promising results after receiving the treatment.
In 2016, human trials went underway in the United States to study the use of umbilical cord tissue-derived mesenchymal stem cells. These trials effectively helped patients stabilize their condition without any adverse effects.
In 2021, another study was conducted that specifically focused on the use of Wharton’s jelly-derived mesenchymal stem cells in patients who suffer from muscular dystrophies. The test concluded that the use of these stem cells was effective in improving the average muscle strength that was measured with a dynamometer and gait in various patients who underwent the study.
Umbilical cord tissue-derived mesenchymal stem cells have proven to be a promising new treatment for Duchenne muscular dystrophy. This revolutionary approach has been shown to significantly improve the lives of those with DMD and provides a great deal of hope for patients who suffer from this debilitating genetic disorder.
At Cellebration Wellness, we’re committed to your health. Contact us at 858-258-5090 to see if stem cell therapy is the right choice for you.
