Considerations for Developing an Effective Stem Cell Therapy for Muscular Dystrophy   - Medical Science Assignment Help

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Considerations for developing an effective stem cell therapy for Muscular Dystrophy  
Alicia Sandoval sand0559@flinders.edu.au  
 

• Muscular Dystrophy’s (MD) are a group of inherited diseases which are distinguished through continuous degeneration and fragility of the skeletal muscles (1). Duchenne Muscular Dystrophy (DMD) differs from other MD’s due to the absence of dystrophin, a protein local to the surface of plasma membranes of muscle fibers, within the cardiac and skeletal muscles (1). Dystrophin provides physiological and anatomical stability to the sarcolemma, therefore, in the absence of dystrophin, the sarcolemma is unable to hold standard muscle contractions (1). Stem cell therapy utilizes stem cells which are harvested from the blood or bone marrow and injected to aid in preventing some diseases (1). The harvested stem cells can be administered through injection to the direct site, into the spinal canal or intravenously (1). This essay will investigate and research a possibility for an effective stem cell therapy for (DMD), by encapsulating the following: What cells would be used for this therapy, how will the cells be administered to the target tissue and how will the pre-clinical be run?  
• Within this study the use of allogenic embryonic stem cells (ESC) was used for the transfer, these contained cells which were harvested from a universal donor who has functioning dystrophin. Allogenic ESC’s were beneficial to use for this study as they are produced in larger batches and one universal donor provided ESC’s for multiple patients (2). In comparison to allogenic therapy's, autologous therapy’s required multiple donors per patient and therefore required more costs to pay donor participants (2). Running an allogenic therapy meant that the use of one universal donor was needed and ensured a more cost-effective form of treatment therefore more participants were included (2). Another benefit of using ESC’s for this therapy, was their unique preservation and growth in vitro, yet still being able to retain the capacity in developing and generating all cell varieties within the embryo (3). This was beneficial to this study, as cell death can be extremely detrimental to these kinds of therapies, by prolonging the results and increasing costs.   
• A con which followed with the process of allogenic therapy was the effect of an immune response from the patients. When allogenic therapy is used, it almost always needs to have immunosuppressive therapies which follow, to ensure that the immune system does not try and eliminate the administered cell therapy (2). If the therapy is not followed up with immunosuppressive therapy and the body creates an immune response to the new cells, this can become an issue with wasting grant funds, cells and time. If the therapy has these issues, it can become unethical for the participants who will need to be re-injected with the ESC's, and for the donors to have their cells re-harvested.  
• The cells were administered to the patients through an intramuscular injection directly into the affected sites. This form of administration was decided on, due to the possibility of the cells migrating to other areas of the body if done intravenously, therefore diluting the full effect of the cells. The direct intramuscular injection can allow for a higher amount of stem cells to reach the targeted dystrophic muscles and potentially have a stronger effect on the participants (4). The arms, thighs, hips and buttocks were the sites for the injection, and a total of 8 injections were given to each participant. Using ESC’s may be a potential long term and ethical stem cell therapy for DMD, based off the path of administration which was used within this study.    
• The pre-clinical was conducted within the Flinders Medical Centre laboratory where in vitro and in vivo studies were carried out to test the ESC’s. Male, dystrophin deficient rats where then used as models to perform these tests on. Rats were chosen for this study as they are small, easy to handle and cost effective. However, a more ideal animal to use for this study would be a dog or a pig, as they are larger dystrophin deficient animals. Therefore, it will be easier to assess the disease, however, they are more costly and time consuming (5). The pre-clinical consisted of administering the rats once weekly, intramuscularly in all four of their limbs with the ESC’s and observe their distance in movement on an exercise wheel, this was repeated for 3 months. After euthanasia of the rats, assays and eosin staining of the muscular tissues were conducted to observe how the ESC’s worked on the muscle fibers.  
• A study conducted by (PLOS One) named, “Characterization of Dystrophin Deficient Rats: A New Model for Duchenne Muscular Dystrophy”, stated that they performed a “grip” test on their mice (5). Within this current study conducted at Flinders Medical Centre laboratory, a grip test was not believed to be the most ideal way to observe the rats. It is believed that more limitations may arise through the grip test, such as human error, is the tail of the rat restricted with the same amount of force per rat? It is hard to have each individual rat restrained with the same amount of force when it is done manually by a human who may have more grip on the tail compared to their partner who has another set of rats.  
• It can be concluded from all the information provided above, that ESC’s are effective and beneficial stem cells to use for the study and possible treatment of DMD. Although there are pros and cons for each different stem cell available for stem cell therapy and research, it was observed that having allogenic ESC’s provided more cells per one donor, therefore providing a more cost-effective and ethical study. Using ESC’s also provided the researchers with cells which could live in vitro for larger amounts of time which allowed for error within the study. This study is believed to be an ethical and long-term possible stem cell therapy for DMD, strongly based off the route of administration of these ESC’s and the cells all rounded nature.       

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