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Early Career Innovators: Ion Transporter Gene Therapy for Epilepsy, Cell and Gene Therapy TIN

By Alina Shrourou, on 7 March 2022

In this interview as part of the Early Career Innovators series, recognising the amazing translational work being done by postdocs and non-tenured researchers at University College London (UCL), Dr Amy Richardson highlights her Cell and Gene Therapy Therapeutic Innovation Network (TIN) Pilot Data Fund awarded project based on testing a novel gene therapy for epilepsy. 

What is the title of your project and what does it involve?

My project, ‘Ion transporter gene therapy for epilepsy’, is based on testing a novel gene therapy as a treatment for epilepsy and validating a viral construct in tissue taken from the brains of human epilepsy patients. Epileptic seizures often spread across the brain from a focal epileptogenic zone due to a breakdown in neuronal inhibition. This gene therapy aims to strengthen that inhibitory restraint to prevent runaway excitability.

The project is two-fold, testing the therapy in vivo using a well-characterised model of epilepsy and validating that we can target the correct neurons in humans with our therapy by putting the viral construct in brain tissue resected from epilepsy patients. The information gained from this project will accelerate the pathway for this treatment to clinical translation. 

What is the motivation behind your project/therapeutic?

Epilepsy is a debilitating neurological disorder that affects around 1% of the global population. Anti-seizure drugs are the first-line treatment for epilepsy and are often effective, however a huge 30% of patients do not achieve seizure freedom with these current medications.

In addition, those that are free of seizures often experience severe side effects as these drugs often work by inducing a global dampening down of neuronal activity meaning other, non-epileptic brain regions are also affected. This creates a huge need for novel treatment options for epilepsy. Gene therapies are fast emerging as a potential therapeutic, however those currently in the pipeline are based on dampening down brain activity in all neurons that are expressing the therapeutic transgene. This gene therapy however, should only be active during epileptic activity, reducing potential cognitive and psychological side effects.

Can you highlight any challenges have you experienced as an early career researcher in the cell and gene therapy/translational research space?

The pathway to a becoming an independent researcher is very daunting when you have just finished your PhD. The next step following a postdoc job is to apply for fellowships, but you require publications and evidence that you have started to acquire support and funding for your own research ideas. This is difficult as many of the large research grants require the applicant to hold a permanent position and you are expected to provide a lot of preliminary data to prove the idea is valid, yet getting this data often requires funding itself.

Why did you want to apply to the Cell & Gene Therapy TIN Pilot Data Scheme? How has it helped you?

I really believe in this project and wanted to generate my own funding to test the idea. As mentioned above, it is very difficult to get funding as an early career researcher, so the TIN pilot fund was the perfect opportunity. It allows researchers of my level to apply and will enable me to generate the preliminary data I require to take this project forward. This also gave me a good introduction to writing a grant proposal and creating a Worktribe budget without being overwhelming.

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 How did you find the process for the TIN Pilot Data Scheme? What did you learn?

The application process was straight-forward and easy. The limited word count forced you to really think about the best way to explain your scientific concept. I was lucky enough to attend the ACCELERATE TIN pitch coaching session and gained some great advice for my presentation which was incredibly useful. The whole application process gave me the confidence to apply for other grants and fellowships in the future!

Learn more about the translational training offered through ACCELERATE 

What do you hope to achieve in the 6 months duration of your project?

These 6 months will be used to generate that all important preliminary data. I hope to evaluate whether my gene therapy is effective in reducing seizure burden in vivo using an established mouse model of epilepsy. I will then create a new viral construct that can be used to investigate the transgene expression in human brain tissue resected from epilepsy patients.

Join the Cell and Gene Therapy  TIN on Teams (UCL-Only) to be part of UCL’s multidisciplinary CGT community with direct access to expertise 

About Dr Amy RichardsonAmy Richardson

Dr Amy Richardson is a postdoctoral researcher working in Professor Dimitri Kullmann’s lab at the Institute of Neurology. Before moving to the ION, she did a PhD in auditory neuroscience at the University of Leicester. Her research focus is mainly synaptic physiology, shifting from an emphasis on the single neuron level at PhD to network excitability during her Postdoc. Her current work involves creating novel gene therapies for epilepsy.

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