2015 · 2015 International Symposium - Orlando · biomedical · Scientific

Theme 13: In vivo Experimental Models

When you think of Orlando you think vacations, theme parks and sunshine. However, for one weekend in December, ALS/MND researchers from around the globe gathered in America’s sunshine state for the 26th International Symposium on ALS/MND. This was the first Symposium I have attended during my PhD and I was excited to finally present all the research that the MND Association’s grant has allowed me to achieve over the past couple of years. Of course the unseasonably warm weather we enjoyed was a plus in comparison to what we left behind in the UK!

I primarily attended the conference to present a poster of my work entitled ‘Zebrafish C9orf72 loss-of-function models of Amyotrophic Lateral Sclerosis (ALS)’ as part of theme 13: in vivo experimental models. I was pre-warned these sessions are busy but nothing could prepare me for the real deal. With the draw of up to date scientific research (and a free bar!) the conference hall was packed with people networking and discussing their research. There was a great buzz and I thoroughly enjoyed the opportunity to discuss my work with researchers from around the world.

There was a great variety of work presented as part of theme 13, including in vivo models of C9orf72, SOD-1, FUS, TDP-43, Profillin-1 and Matrin-3 – to name but a few! It really highlighted to me the genetic heterogeneity of ALS/MND and the importance of generating models to investigate potential molecular mechanisms underlying the disorder.

As I work on zebrafish myself, I found poster p252, ‘’Identification of a novel neuroprotective drug for ALS using ‘ZNSTRESS’, a zebrafish high throughput phenotypic screen and validation in the SOD1 G93A mouse model’, of particular interest. The group successfully exploited the high-throughput capabilities of zebrafish. They used a transgenic SOD-1 zebrafish model of ALS to screen for neuroprotective compounds by utilising a novel fluorescent reporter that labels stressed neurons in the mutant zebrafish. The screen resulted in the identification of several hit compounds which play a role in reducing neuronal stress in the model. A lead compound that came out of the study is now being tested in a mutant SOD-1 G93A mouse model of ALS and preliminary data suggests the drug is safe. This study highlights the advantages of using zebrafish in high-throughput drug screens and how they can be used to identify promising compounds to test in murine models of ALS.

To find out about all the research from this theme, you can find the abstracts on http://www.mndassociation.org/research/international-symposium/abstracts-online/ and all the posters will be uploaded onto the F1000Research open access platform.

Overall, my first experience at an international conference was excellent – sun and science are a great combination. I have returned to the UK with an up to date knowledge on current ALS/MND research and lots of inspiration for my final year of my PhD. I would especially like to thank the members of the South Yorkshire branch of the MND Association for kindly providing funds in order to attend the conference – it has been an invaluable experience!