- Gradual muscle weakness and wasting
- Delay of reaching motor milestones
- Loss of previous ambulation (ex. Standing/walking)
- Recurrent respiratory complications (due to abnormal breathing muscles)
- Cardiac abnormalities (usually by the age of 20)
- Physiotherapy: Physiotherapy is still the main form of therapy for muscular dystrophy – along with steroids (2).
- Steroids: Steroids are strong anti-inflammatory drugs that can also modulate the body’s immune response. There is current evidence that early use of glucocorticoids/steroids delays the onset of wheelchair dependence. However, to this day and despite extensive studies on the topic, there are still uncertainties regarding which steroid is the best to use and the possible risk of under or over-treatment. This is also in addition to the potential side effects of prolonged steroid use such as weight gain and higher risk of infections (2).
- Gene Therapy: this is yet again another promising mode of therapy – similar to stem cell therapy – with multiple drugs being approved by the FDA for muscular dystrophies due to the limited amount of options available. These drugs target the gene abnormality responsible for muscular destruction and dystrophy; however given that these medications target a certain genetic mutation, they depend on a certain genetic mutation/aberration being present in the patient in order to be able to use it. For example, one drug approved known as Eteplirsen has been reported to be efficacious only in around 15% of patients with DMD who carry a certain mutation in Exon 15 of the DMD gene. This means that in children who have a gene abnormality elsewhere or even a different abnormality within the same gene – which is common – don’t benefit from this medication. Therefore, gene therapy requires a meticulous genetic assessment before its use (3, 4).
- Mesenchymal Stem Cells: These are stem cells obtained from adipose tissues, bone marrow, or umbilical cord tissues – which we actually use at Beike. These cells are the most widely used stem cells since they can be easily produced in larger numbers to accommodate a higher number of patients and allow better efficacy, have a better response in neurological and muscular diseases, have better differentiation capacity into numerous cell types, and have better anti-cell death effect in case of degenerative conditions like muscular dystrophies that cause progressive muscle wasting – compared to other stem cells. Mesenchymal stem cells can be obtained from different sources including the umbilical cord, bone marrow, and adipose tissue.
- Muscle/Satellite Stem Cells: These are another type that has particular benefits in differentiating into the defective muscle fiber cells, of the same origin; though their use is limited in patients due to their limited migration abilities to the site of defect following their transplantation. These stem cells also require long-term suppression of immunity using immunosuppressants to prevent transplant rejection.
- Adult Pluripotent Stem Cells: These are another source that can be produced in large numbers, yet their differentiation abilities are limited.
- Embryonic Stem Cells: Another type of stem cell includes embryonic stem cells. These cells can also differentiate into photoreceptors, yet they are difficult to obtain and have ethical concerns regarding their sources.
- Intravenous (Into the blood)
- Intramuscular (Locally into the muscles)
As previously mentioned, this concomitant administration of both types of stem cells, mesenchymal and hematopoietic stem cells, provides better results.
Stem cells are processed at our laboratories accredited by the American Association of Blood Banks (AABB) where we follow the highest international standards in order to provide the best cell samples.
mobile functioning before permanent damage and muscle atrophy/wasting take place.
We still need to report that clinical benefit is not 100% guaranteed as is the case with any intervention, and consulting our specialists prior to undergoing the procedure is of utmost importance in order to gain more insight into the procedure and the estimated possibility of treatment success for your individual case.
Additionally, in studies specifically studying stem cell therapy in patients with Muscular Dystrophy, no significant side effects were reported and none were life-threatening or had life-long consequences when mesenchymal stem cells were used (10). They were also easily managed medically at the time of their occurrence.