What is Muscular Dystrophy?
MD (muscular dystrophy) is a collection of diseases involving progressive weakness and loss of muscle mass. The underlying cause of MD is a genetic mutation that disrupts the production of proteins required to form muscle. Please continue on the treatment of muscular dystrophy with this and additional points below.
Key points about MD include:
- Muscular dystrophy: It all starts with genetics.MD is a genetic condition caused by mutations in genes coding for proteins necessary for muscle function. Genetic mutations are inherited, but they also predispose the body to not be able to maintain normal, healthy muscles.
- Types of muscular dystrophy: There are a multitude of types of MD, including Duchenne (DMD) and Becker. The most common and severe is DMD, which typically occurs in boys and presents itself early in childhood. Other types of MD are recognized as adult onset.
- How mesenchymal stem cells work: Mesenchymal stem cells are known to differentiate into many different tissue types, including muscle. There are also beneficial factors that MSCs secrete to stunt inflammation and enhance healing in the body, potentially facilitating regeneration of damaged muscle tissue.
- Rajput et al. study key findings: Rajput et al. study found evidence that umbilical cord-derived MSCs were beneficial as an adjunct to standard care of DMD for muscle power stabilization in patients without harmful side effects. I would interpret this as an indication that the umbilical cord-derived MMSCs were potentially safe for use.
- Limitations of stem cell therapy: The findings of the Rajput et al. study as an adjunct to standard care for DMD, although exciting, would still need to be supported by large, rigorous, double-blind studies to support claims of safety and improved efficacy, especially in muscular dystrophies.
Typical Treatment Approaches for Muscular Dystrophy
Medications
- Eteplirsen
- Corticosteroids (e.g., prednisone)
- Medications for the heart (ACE inhibitors, beta blockers)
Therapy
- Range-of-motion and stretching exercises
- Exercise programs
- Braces and mobility aids
- Help with breathing
Surgery
Correcting the curvature of the spine to help with breathing
Stem Cell Treatment: An Exciting New Frontier.
Mesenchymal stem cells (MSCs) are now being examined as a possible treatment for muscular dystrophy.
What are MSCs?
MSCs are multipotent stem cells that have the ability to differentiate into a multitude of different tissue types, including muscle. They have the capacity to secrete cytokines that can decrease inflammation and promote healing, aiding in muscle repair.
Where do MSCs come from?
- Bone marrow
- Trabecular bone
- Periosteum
- Adipose tissue
- Skeletal tissues
- Synovium
- Deciduous teeth
Clinical studies with MSCs and muscular dystrophy
Rajput et al. published an important study examining umbilical cord-derived mesenchymal stem cells (UC-MSCs) in DMD patients.
- This was a single-blinded study with 11 DMD patients (5-18 years of age);
- UC-MSCs were delivered by IV and IM injection;
- Follow-up averaged 1.5 years, with a maximum of 3 years;
- There was a control group of 5 non-treated DMD patients;
Results:
- Stabilization of muscle power in treated patients;
- No evidence of graft-versus-host disease (GVHD) or any other adverse effects;
- The treatment was felt to be safe compared to control patients.
These results suggest MSC therapy may be a safe treatment option for DMD. MSC therapy is seeing potential in muscle power stabilization without adverse effects.
Final Thoughts
Historically, interventions for muscular dystrophy have been limited to managing symptoms and slowing disease progression; however, stem cell therapy in India has the potential to affect clinical outcomes. The findings by Rajput et al. show us the safety and possible effectiveness of MSCs in DMD.
That being said, current stem cell therapy limited to muscular dystrophy has potential limitations to note. Current single-live trials leave much room for larger double-blinded studies to determine effectiveness and safety. So as research evolves, stem cell therapy may lead to new hope, but more work must be done to demonstrate if it can be considered a feasible treatment option.
FAQs
1. What is muscular dystrophy and what causes muscular dystrophy?
Muscular dystrophy (MD) refers to a group of genetic disorders that cause progressive muscle weakness and loss of muscle mass.
Muscular dystrophy is caused by a genetic defect that impedes the body’s ability to produce the proteins necessary to make and repair healthy muscle tissue.
The common and potentially life-threatening muscular dystrophy is Duchenne muscular dystrophy (DMD). DMD can occur in boys and is typically diagnosed in early childhood.
In the context of muscular dystrophy, MSCs will be delivered via intravenous (IV) and intramuscular injections and are intended to assist with repairing damaged muscle and restoring muscle function.
3. What do current studies suggest about stem-cell therapy for muscular dystrophy?
A large study conducted by Rajput et al. investigated umbilical cord-derived MSCs in 11 patients with DMD aged between 5 and 18 years and found that patients treated with MSCs appeared to stabilize their muscle power and did not appear to have any adverse effects or complications or other problems at an average follow-up of 1.5 years. This trial supports the hypothesis that MSC therapy may represent a safe form of treatment in DMD patients.
4. What are the current limitations of stem cell therapy for muscular dystrophy?
Although initial and preliminary results are promising, stem cell therapy for muscular dystrophy is currently not at a stage in development that it can be used in routine clinical practice, especially with regard to standard therapeutic treatment. At the present time there are significant limitations inherent in all of the studies reported following stem cell intervention. For instance, all studies to date have been non-randomized and single-blinded, with no multi-center or sham control studies.