Stem Cell Therapy for Muscular Dystrophy
What Causes Muscular Dystrophy?
- Genetic mutations that lead to the production of abnormal or missing proteins necessary for muscle fiber function
- Spontaneous (not inherited) gene mutations during fetal development
- Duchenne muscular dystrophy (DMD): A disorder caused by a deletion in the DMD gene on the X chromosome. The DMD gene provides instruction for making dystrophin, a connective protein necessary for muscle and heart muscle functions. Because females have two X chromosomes and males have one X chromosome, DMD affects mainly boys. Symptoms can begin between ages 2 – 16, but may also show up as late as 25.
- Becker muscular dystrophy: A muscle disorder caused by a milder change in the DMD gene than the deletion that caused Duchenne muscular dystrophy. As a result, Becker muscular dystrophy comes from reduced dystrophin, rather than complete elimination.
- Myotonic dystrophy: A muscle disorder resulting from abnormal production of myotonin protein kinase, an enzyme involved in communication across muscle cells. Myotonic dystrophy causes muscle wasting, cataracts, and balding.
- Limb-girdle muscular dystrophy affects both genders and involves a variety of different gene mutations
- Facioscapulohumeral muscular dystrophy affects the face, shoulder, and upper arm muscles
- Progressive muscle weakness beginning in the pelvic and shoulder muscles, and eventually affecting all other muscles in the body
- Cardiomyopathy, where the heart muscle becomes weak and enlarged, and can lead to death
- Difficulty walking
- Muscle wasting, where the muscles become weaker and smaller in size
- Delayed motor milestones such as sitting, crawling, or walking
- Difficulty breathing due to weakened muscles that are involved in breathing and postural issues
- Vision and hearing loss
- Cognitive dysfunction and delayed cognitive development, such as deficits in verbal, short term, and working memory
Stages of muscular dystrophy
Muscular dystrophy worsens over time. The rate of progression varies, but there are generally four stages of muscular dystrophy:
- Early-stage patients experience some muscle weakness and difficulty with certain movements, such as climbing stairs or standing up from sitting. They can still perform most activities of daily living without assistance.
- Ambulatory patients experience significant muscle weakness and may need to use mobility aids, such as braces, crutches, or a wheelchair, to get around. They can still walk and stand with assistance.
- Non-ambulatory patients cannot walk and require a wheelchair for mobility. They may also need assistance with activities like dressing and grooming.
End-stage patients need 24-hour care and assistance with all aspects of daily living. Respiratory and heart function is also severely compromised and leads to death in many cases.
Stem Cell Therapy for Muscular Dystrophy FAQ
The Scientific Rationale Behind Stem Cell Therapy for Muscular Dystrophy
Currently, there is no known cure for muscular dystrophy. The standard-of-care treatments may slow down disease progression and manage symptoms but do not reverse the damage done to the respiratory system. Most drugs for muscular dystrophy also have unpleasant side effects that tend to lower the quality of life. Some patients may not respond well to the available treatments or may not be eligible for certain treatments due to other health conditions or contraindications.
This is where stem cell therapy can uniquely deliver benefits.
How Much Does Stem Cell Therapy Cost for Muscular Dystrophy?
The cost depends on the specific treatment you will be undergoing and how many treatment sessions you need. Book a consultation with us today to get your personalized stem cell therapy treatment plan.
How Many Treatments Will I Need?
Typically, you will need 1-2 treatments, depending on the plan of care established between you and your doctor. All treatment appointments are in person, but the initial consultation and follow-up can be done by phone.
Some patients come back for additional treatments over the course of months or years. This largely depends on the stage of your disease, how well you respond to the treatment, and what your treatment goals are.
Your Patient Journey with Stem Cell Therapy for Muscular Dystrophy
Step 1: Application and initial consultation
Our questionnaire and initial consultation help us determine whether you’re a good candidate for stem cell therapy, along with the best treatment plans for you. Our patient advocate will walk you through the process, pricing, and payment plans.
Step 2: Physical exam
You’ll come in for a physical exam in preparation for the treatment and also to assess your current symptoms and health status.
Step 3: Stem cell collection
Your procedure will begin where we harvest cells from your bone marrow or fat tissues. We will then isolate the stem cells from these tissues in the clinic before administering your first treatment
Step 4: Stem cell culture
If applicable, we will send your stem cells to the lab to expand them in numbers.
Step 5: Future treatments
Your future stem cell treatments will be done using your cultured stem cells.
Step 6: Follow-up appointments
Our team will keep in contact with you to monitor your treatment progress and support you through your recovery.
Advantages of Stem Cell Therapy vs Standard Treatments and Other Alternative Treatments for Muscular Dystrophy
Current standard-of-care treatments for Muscular Dystrophy and their shortfalls
The current standards of care for muscular dystrophy include a multidisciplinary approach to address issues of mobility, strength, and pain management. Some specific interventions include:
- Steroids, such as prednisone or deflazacort, slow the progression of muscular dystrophy by reducing inflammation and promoting muscle growth 16. Side effects can include:
- Weight gain
- Mood changes
- Osteoporosis (weakened bones)
- Delayed growth
- Increased risk of infection through suppression of the immune system
- Baclofen is a muscle relaxant that reduces stiffness and spasms. Baclofen increases the activity of the neurotransmitter GABA through the GABA-B receptor in the brain to reduce muscle activity 17. Side effects can include:
- Dantrolene reduces muscle stiffness, spasms, and creatine kinase (CK) – a marker of muscle inflammation. Dantrolene blocks the ryanodine receptor on muscle cells and prevents the release of calcium and muscle contraction 18,19 Side effects can include:
- Weakened breathing
Physical and occupational therapy
- Muscle strength
- Range of motion
- Communication devices
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- Biressi, S., Filareto, A. & Rando, T. A. Stem cell therapy for muscular dystrophies. J. Clin. Invest. 130, 5652–5664 (2020).
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- Wang, S. et al. Targeted Therapy for Inflammatory Diseases with Mesenchymal Stem Cells and Their Derived Exosomes: From Basic to Clinics. Int. J. Nanomedicine 17, 1757–1781 (2022).
- Gomzikova, M. O., James, V. & Rizvanov, A. A. Mitochondria Donation by Mesenchymal Stem Cells: Current Understanding and Mitochondria Transplantation Strategies. Front Cell Dev Biol 9, 653322 (2021).
- Barker, R. G., Wyckelsma, V. L., Xu, H. & Murphy, R. M. Mitochondrial content is preserved throughout disease progression in the mdx mouse model of Duchenne muscular dystrophy, regardless of taurine supplementation. Am. J. Physiol. Cell Physiol. 314, C483–C491 (2018).
- Moore, T. M. et al. Mitochondrial Dysfunction Is an Early Consequence of Partial or Complete Dystrophin Loss in mdx Mice. Front. Physiol. 11, 690 (2020).
- da Silva Meirelles, L., Caplan, A. I. & Nardi, N. B. In search of the in vivo identity of mesenchymal stem cells. Stem Cells 26, 2287–2299 (2008).
- Dai, A. et al. Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy – Phase I-II. Degener. Neurol. Neuromuscul. Dis. 8, 63–77 (2018).
- Rodriguez, A.-M. et al. Transplantation of a multipotent cell population from human adipose tissue induces dystrophin expression in the immunocompetent mdx mouse. J. Exp. Med. 201, 1397–1405 (2005).
- McDonald, C. M. et al. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet 399, 1049–1058 (2022).
- Sienkiewicz, D., Kulak, W., Okurowska-Zawada, B., Paszko-Patej, G. & Kawnik, K. Duchenne muscular dystrophy: current cell therapies. Ther. Adv. Neurol. Disord. 8, 166–177 (2015).
- Youngest child in world gets stem cell therapy for cerebral palsy at StemRx Hospital. http://www.pharmabiz.com/NewsDetails.aspx?aid=96256&sid=1.
- Topaloglu, H., Gloss, D., Moxley, R. T., 3rd, Ashwal, S. & Oskoui, M. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology 87, 238 (2016).
- Ghanavatian, S. & Derian, A. Baclofen. (StatPearls Publishing, 2022).
- Bertorini, T. E. et al. Effect of dantrolene in Duchenne muscular dystrophy. Muscle Nerve 14, 503–507 (1991).
- Ratto, D. & Joyner, R. W. Dantrolene. (StatPearls Publishing, 2022).
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- Elangkovan, N. & Dickson, G. Gene Therapy for Duchenne Muscular Dystrophy. J Neuromuscul Dis 8, S303–S316 (2021).