To date, there has not been a single study – to our knowledge – that has compared different types of stem cells, concerning safety and efficacy, particularly in patients with muscular dystrophies. However, we can summarize different stem cell sources that have been tested in these disorders. Each form of stem cells has its own benefits and drawbacks as will be mentioned. Different stem cell sources that have been tested in different muscle dystrophies – either on animals or in humans – include (5, 10):
- 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.
After carefully reviewing all of the benefits and risks of each type, we have decided to use mesenchymal umbilical cord-based stem cells as our main source since they have been the most extensively studied type in neuromuscular conditions, including muscular dystrophy, with the least reported side effects (6). This is in addition to also using umbilical cord blood cells (hematopoietic cells) concurrently to provide additional benefits from using two types of stem cells; with each contributing to muscle and nerve regeneration through different complementary mechanisms.
In addition to the source of stem cells, there are also multiple routes of stem cell administration. Most clinical trials testing stem cell therapy in Muscular Dystrophy use either one – or both – of preferably two routes; which are (10):
- Intravenous (Into the blood)
- Intramuscular (Locally into the muscles)
Studies have shown that in contrast to a single route of intravenous administration, administering stem cells through dual administration provides significant clinical results and improvement (10). At Beike, we use combined intravenous and intramuscular routes concomitantly to achieve the best results with the least possible side effects.
