Cure SMA has awarded a $75,000 research grant to Jean Giacomotto, PhD, at the University of Queensland, for his project, “Zebrafish models of Spinal Muscular Atrophy optimized for chemical genetics and drug discovery. From proof-of-principle to new insights and treatments”.
In SMA, the lack of additional “druggable” targets, beyond SMN, creates a gap in the traditional drug discovery pipeline. In order to address this issue, Dr. Giacomotto and his team have created a zebrafish model of SMA designed to be suitable for large-scale drug screening.
In this proposal, the team will optimize their zebrafish model to make it compatible with drug discovery. Then they will run a pilot screening study using a pool of pharmacologically active compounds to potentially find beneficial drugs for treating SMA.
This research will generate the first animal model of SMA compatible with large-scale drug discovery experiments. It has the potential to identify existing drugs that might be beneficial in SMA as well as new compounds for further development. This study could help identify new ways to discover therapies that could potentially be used in combination with SMN-enhancing approaches, such as Spinraza, to achieve greater therapeutic benefit.
Meet Dr. Giacomotto
Who are you?
I am a French scientist working in Australia at the Queensland Brain Institute at the University of Queensland. I was initially trained in industry with a background in genetics and drug discovery. After realizing that many big pharmaceutical companies have not been interested in orphan diseases, I decided to make the transition to academic research. My main interest today is to apply my drug discovery skills to orphan diseases in hopes of finding treatments to alleviate the suffering of patients. I am also strongly interested in understanding the mechanisms that are involved in the progress and the severity of these orphan diseases.
How did you first become involved with SMA research?
After leaving industry, I initially focused my research on developing new drug discovery approaches to combat rare diseases such as spinal muscular atrophy and Duchenne muscular dystrophy. I started by recapitulating these disorders in small animal models, such as C. elegans (a tiny worm) or the zebrafish. By doing so, I was able to create a unique opportunity to search for drug treatments without a prior complete mechanistic understanding of the disease.
What is your current role in SMA research?
My work focuses on trying to develop new animal models of the disease that are compatible with large-scale drug discovery. My ultimate goal is to find new therapeutic targets and palliative treatments that would help to relieve the suffering of patients.
What do you hope to learn from this research project?
We hope to find pharmacologically active compounds suitable for SMA drug development. How will this project work?
We will optimize our recently developed zebrafish model of SMA in order to be able to conduct a large scale drug screening to potentially identify compounds beneficial in SMA.
What is the significance of your study?
This research will generate the first animal model of SMA compatible with large-scale drug discovery experiments. This model can then be used to screen drugs and identify those which may warrant further drug development for SMA.
Basic Research Funding
This grant to Dr. Giacomotto is part of $955,000 in new basic research funding that we’re currently announcing.
Basic research is the first step in our comprehensive research model. We fund basic research to investigate the biology and cause of SMA, in order to identify the most effective strategies for drug discovery. We also use this funding to develop tools that facilitate SMA research.