Wednesday, August 15, 2012


As part of our support of the Gillingwater-Parson research work we were delighted when a scholarship was set up in Estella's name. We were even more delighted to receive this report from Katie Hoban detailing her work within this innovative environment. This is Katie's report 

Estella Star Scholarship Report

Having just completed my BSc degree in Neuroscience and looking forward to beginning my training to become a medical doctor at the University of Edinburgh, I was keen to spend time in a scientific research laboratory. I am particularly interested in the way in which basic research will inform my future clinical practice and when I saw the advert for the Estella Star Scholarship I immediately applied. I was delighted upon hearing I had been awarded the scholarship and the chance to work in a leading SMA research laboratory. My project was to be supervised by Dr Simon Parson and entitled “The role of the vascular system in Spinal Muscular Atrophy’.
You may be surprised that I was not to work on motor neurons, but instead the vascular system.  This is because it is becoming increasingly apparent there are other key targets, such as the heart and blood vessels, in the disease as well as the characteristic muscle atrophy. Previous research in the laboratory shown the amount of blood vessels in muscle is dramatically reduced in mouse models of severe SMA. My project was to investigate the blood supply to the spinal cord (where motor neurones are found) and nerve (which connect the motor neurons to muscle). First we carried out a detailed analysis of normal tissue upon which to base future SMA studies. I learnt new techniques to isolate specific tissues, prepare them for microscopy, stain them to highlight blood vessels and then quantify the amount of blood vessels present. This was challenging at first, but with help from the other students in the laboratory I soon became proficient and was able to collect data. We found that the newborn spinal cord was sparse of blood vessels, but steadily increased over the first few weeks of life. We specifically noted changes in the part of the spinal cord where motor neurons are found. Peripheral nerves were much better supplied with blood vessels at birth. This is important as defects in blood supply to either of these regions could have catastrophic consequences for motor neurons. I presented this data at a recent symposium on Motor Neurones and Diseases of Motor Neurones, and hope to submit it for publication later this year. Finally we began to assess tissue from mice which model severe SMA and found some potentially very interesting results, but these are too preliminary for us to publicize yet, so watch this space!
Working in a hospital environment in recent months has highlighted the fact there are patients in urgent need of therapies for currently incurable diseases. To be given the opportunity by your incredible Estella Star fundraising efforts, allowed me, in a small way, to add to current research. When previously speaking to you both during your visit to the lab, I was struck by your profound dedication and personal commitment to heighten the profile of SMA and I vowed that by the end of my placement, I would be able to report new developments which would provide a platform of information to help further understand the disease pathology of SMA.
I will make it my utmost effort to follow the progress of the Gillingwater-Parson lab because I sincerely believe that they are at the cutting edge of scientific research and with the advances to date, we are en-route to answering many of the unresolved questions in SMA and together with your fantastic fundraising efforts, are another step closer to SMASH SMA.
I can only thank you for this unique opportunity and have nothing but respect for your dedication to help us find treatments to the fatal disease that is SMA.
Katie Hoban

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