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Stem Cell Treatment for Muscular Dystrophy

Thursday, December 22, 2022

Muscular dystrophy is a genetic disease that affects muscles throughout the body. It causes progressive weakness and wasting of the dystrophic muscle tissue. The condition usually begins in childhood and progresses rapidly.

There is currently no cure for muscular dystrophy. However, there are treatments that can slow down the progression of the disease. Your healthcare provider may recommend physical therapy, occupational therapy, speech therapy, E-stim therapy and various pharmaceutical drug therapies. 

Stem cell therapy is another treatment option for people with muscular dystrophy. In recent years, stem cell therapy has been used to treat patients with various dystrophic muscle diseases, including Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and Limb-Girdle muscular dystrophy (LGMD).

In this article, we will explore the basis of stem cell therapy for myogenic cells, its current uses for skeletal muscle function, and its prospects for skeletal muscle regeneration.

What Is Stem Cell Therapy?

Stem cells are biological cells that signal repair molecules in tissues. A stem cell may be derived from embryonic tissues or adult tissues. At Stemaid Institute, we work with pluripotent embryonic stem cells derived from inner cell mass of a pre-differentiated blastocyst. These cells are the youngest and the most potent stem cells and can signal repair in all 220+ tissue types of the body, including muscle tissue.

How do pluripotent stem cells repair skeletal muscles?

Stem cells secrete repair molecules (peptides and proteins) and transcriptions factors (mRNAs and microRNAs) that signal tissues to repair. These signaling factors usher in the immune system and DNA repair mechanisms, instigate the body to grow more blood vessels, regrow nerve connections and dissolve fibrosis, replacing damaged and injured tissue with flexible elastin-rich healthy tissue. The pluripotent signaling factors also communicate directly with skeletal muscle stem cells to resume long lasting skeletal muscle regeneration. 

How can pluripotent stem cells help with Muscular Dystrophy?

Muscular dystrophy is caused by the presence of a defective gene, the dystrophin gene. This abnormal gene codes for a protein called dystrophin that helps keep muscle cells intact.  Dystrophin-deficient satellite cells, the resident satellite skeletal muscle stem cells, cannot function properly, leading to chronic oxidative stress and muscle degeneration. 

Although stem cell therapy is not considered a gene therapy for muscular dystrophy, the epigenetic reprogramming that pluripotent stem cells instigate sets whole body physiology to continual repair and regeneration. The dystrophic muscle, in the presence of pluripotent signaling proteins and transcription factors, receives the go-ahead to grow, to repair, and to revive quiescent cells unaltered by the dystrophin gene. Muscle degeneration slows down and the physiological effects of the defective gene and the dystrophic satellite cells are offset.

What are the outcomes of Pluripotent Stem Cell Treatment in people with Muscular Dystrophy?

Muscles are the "organ of the will”, it is said, and no one shows more “will to move" than individuals diagnosed with muscular dystrophy (MD).  Muscle wasting conditions like MD can cause problems with walking, breathing, swallowing, respiratory infections, urinary incontinence, cardiac muscle, skeletal muscle strength and overall coordination. The effort MD adults and children put into moving freely and using the precious muscle organ is impressive and always inspirational. Every patient is different, outcomes vary depending on vitality and severity of the condition. Let's introduce you to our latest Stemaid patient with MD. 

Alex L., Stemaid Patient with Muscular Dystrophy

Alex came to Stemaid Institute with a diagnosis of Muscular Dystrophy (MD) as undefined by genetic testing. He began showing signs of muscle weakness as a child, first appearing as clumsiness and later on in his teens as more progressive weakness. Alex is now a successful 33 year old man, however the muscle degeneration and muscle spasms affect his ability to breathe, his physical stability and his capacity to move. He needs to sit on a high chair to be able to stand up, the strength to get up on his own is just not there. He also has difficulty going uphill. The difficulty breathing and the muscle weakness has led to weight gain, which affects his energy levels and health overall. The “will” to be healthy and fit is strong, it’s just the muscles are not cooperating, the strength is just no there.

Alex's Stemaid Journey

Alex came in for a one week Stemaid Pluripotent Stem Cells and Plurisomes program. After just 6 days, the two key blood markers of muscle wasting went down significantly. Elevated CPK and CPKMB levels are indicative of muscle disease and tells us a lot about severity of MD. His blood tests showed a significant decrease in both, in just one week.  A month post-treatment, Alex reached out to let the Stemaid Doctors know that he is feeling great. He has lost 10 lbs and his strength and exercise endurance are improving daily. This is a big win! in such a short period of time.

Alex's Future 

Alex will need intermittent treatment with Pluripotent Stem Cells and Plurisomes in the years ahead. Regular pulsing doses of Pluripotent factors gradually repair and reprogram muscle tissue, increasing muscle fiber number (hyperplasia) as well as muscle fiber size (hypertrophy). This will bring on more strength so that Alex can have a well working diaphragm, better core stability and power to move and exercise.

Pluripotent stem cells and their signaling factors act on the body for months after treatment. We are looking forward to seeing his improvement over the next few months ahead. Pluripotent stem cell therapy takes time, but so worth it.

"Where there is a will, there is a way!" 

Feel free to check with your healthcare provider or call Stemaid Institute to see whether Pluripotent Stem Cell Therapy is right for you. 

References

1. Targeting muscle stem cell intrinsic defects to treat Duchenne muscular dystrophy - npj Regenerative Medicine

2. Muscular Dystrophy Information Page | National Institute of Neurological Disorders and Stroke

3. Stem cell-based therapies for Duchenne muscular dystrophy - PubMed

4. Stem cell therapy for muscular dystrophy - PubMed

5. Ameliorating the hallmarks of cellular senescence in skeletal muscle myogenic progenitors in vitro and in vivo

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