MDA's PhD Student Scholarships
Without a science degree of your own, understanding the intricacies of the research conducted within a research centre like the National Muscular Dystrophy Research Centre can be difficult to say the least. Predicting how close the scientists are to achieving a major breakthrough and how long it will be before the research will yield a solution to neuromuscular disorders is even more difficult. But that question is possibly the one most frequently asked of the staff at the NMDRC and at the MDA.
There is no definitive answer to the question. There are many small steps and countless hours involved in conducting just one experiment that may yield results that can then be applied to the next step in the process. And similarly, while there may be one ultimate goal in sight – to find a cure – there are many different methods that could be used to reach that same goal. Around the world there are several research racilities and scientists working specifically towards understanding and overcoming neuromuscular disorders. Each facility and each scientist has their own particular area of focus – for example in some cases this is to explore the possibility of correcting the gene that causes the disorder, in other cases this is to investigate the potential to remodel muscle cells that are already affected by the disorder, and so on.
Each of these research streams has the potential to provide a cure, or at the very least, a treatment for MD. Diversifying the research focus in this way means that both research streams can be explored at the same time, which in turn means that many research advancements can be made in a more timely way.
Sharon Wong and Marion Todaro are two students who have chosen to complete their PhD studies in the area of Muscular Dystrophy research. Both Sharon and Marion have been undertaking research at the NMDRC for the past 3 and a half years and are nearing completion of their PhD scholarships.
Sharon’s research has centred on remodeling dystrophic muscle and essentially replacing the affected dystrophic cells with stem cells that are able to function correctly when transplanted into the muscle. Marion has chosen to focus her studies on attempting to enhance gene correction of the mutated gene linked to Duchenne Muscular Dystrophy (DMD).
Marion describes the advancement of the research process as a “tag-team” setup. The results from one person’s research are often used as the basis to expand that research out into the next step. The initial results may form the basis for the next research stream and the next round of experiments to prove or disprove the idea.
Marion’s research focus builds upon findings from research already conducted by NMDRC scientists and uses the principles applied from previous studies as the basis for this next step in the process.
The use of alternative renewable cell sources for the purpose of autologous stem cell transplantation therefore seems a logical approach to the treatment of DMD.
Previous studies have shown that bone marrow cells were able to convert to muscle, albeit at a very low efficiency. Therefore, the first aim of my project was to optimise the delivery of these bone marrow cells and induce their conversion into muscle cells within the dystrophic muscles of the mdx mouse. This involved investigating the use of various methods as a means of enhancing uptake and expression of injected bone marrow cells and DNA. The second aim of my project focused on the delivery of naked DNA carrying the dystrophin gene into dystrophic mdx mouse muscle.”
Both Sharon and Marion are now in the final stages of completing their PhD studies, and over the next 6 months they will be busy “writing up” their findings. Once this is done, their thesis is then presented for review. In the review process, a committee of scientists who are established and respected in the particular field the PhD covers, assess the student’s research for validity of methods used, publication record, and research findings.
For PhD work to be considered successfully completed’ the research itself does not necessarily need to yield the desired outcome. The findings of Sharon and Marion’s research at this stage look promising, but as with all scientific experiments, a ‘successful’ experiment does not always mean a ‘positive’ result, it simply means that a result was successfully produced. An unsuccessful experiment is one in which the results are inconclusive. Similarly, with a PhD project, whilst a ‘positive’ result is ideal, provided the research conducted is valid and has produced conclusive results either way, the project is considered to be completed successfully.
Once the student’s PhD work has been reviewed and verified, the findings can then be published. And again, if the findings of the PhD research are favourable, they can then be incorporated into further research projects. If the results concluded that the avenue of research would not yield the desired result, then this information will help to shape new directions to be explored through future research projects.
For the NMDRC and for MDA, the primary benefit in offering scholarships to PhD students is the progress that can be made in a particular area of research and the fresh ideas that a new mind can bring to the research team. But another significant benefit is that by providing a PhD student with the opportunity to focus their studies on neuromuscular disorders, it encourages those students to build their knowledge of these disorders and to continue working in that area once they have completed their PhD. Thus it builds the worldwide pool of experts who are dedicating their research efforts to finding a solution to neuromuscular disorders.
Both Sharon and Marion are looking forward to the completion of their PhD projects and are planning a well-deserved break at the beginning of next year, once their project findings are submitted for review. Marion hopes to continue her research after a break, and ultimately plans to focus her research on understanding and combating Motor Neurone Disease (MND). Whilst this is a deviation from her current focus on DMD, the findings of research into MND can still have the potential for significant relevance to DMD.
Sharon plans to take a break from the rigours of research for a short while, having studied non-stop from the time she began school. She is considering venturing into the field of pharmaceuticals for a time, before eventually returning to research, and hopefully bringing with her a wealth of applicable knowledge gained in the meantime.
On behalf of the MD community we thank our PhD students, Marion and Sharon for their contributions to this pool of knowledge. MDA’s PhD Scholarship Program and PhD students - Making a Difference.
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