Developing novel treatments for motor neuron disease

**WELSH LANGUAGE STUDENTSHIP**

Project Outline

This scholarship, which is fully funded by the Coleg Cymraeg Cenedlaethol will focus on research into innovative methods to treat motor neuron disease, also known as Amyotrophic Lateral Sclerosis (ALS). The research will take place in the Medicines Discovery Institute (MDI), which has connections with the School of Biosciences and the School of Medicine at Cardiff University. The primary goal of our Institute is to deliver modern drug discoveries to improve treatments and the lives of those in need.
This is a unique opportunity for an individual from a Welsh language education background to complete a PhD in medicinal chemistry through the medium of Welsh at the Medicines Discovery Institute.

Project background

Amyotrophic Lateral Sclerosis (ALS), which is the most common form of motor neuron disease, is a severe and fatal neuromuscular disease which currently has no cure. There are no effective medicines, meaning there is an urgent need to find therapies to change the course of the disease and to treat symptoms.
Across neurodegenerative diseases understanding is emerging that altered RNA metabolism is key to disease initiation and development. Small sequences of DNA known as microsatellites occur naturally in the human genome and have important roles in genome evolution and function. However, the expansion of microsatellites is associated with over two dozen neurological diseases. Prominent examples include myotonic dystrophy, Huntington’s disease, spinocerebellar ataxias and some forms of motor neuron disease - all of which are characterised by accumulation of toxic RNA species.
The research will encompass several aspects of the drug discovery process. Synthetic chemistry will be a substantial part of the design and development of small molecules that will be used to test whether increasing levels of our new RNA target (Nuclear Enriched Abundant Transcript 1, NEAT1) will provide an innovative new way of treating ALS.
In addition, there will be an opportunity to use state-of-the-art computational chemistry to help guide the process along with the use of biological assays to assess the synthesised molecules. We are looking for enthusiastic applicants that have a strong passion for medicinal chemistry, ideally from a background of studying organic chemistry or biochemistry.
The studentship is full time over the course of three years. The final thesis will need to be written in Welsh, and suitable support is available within the University to accomplish this and to improve the essential skills in the sciences and Welsh.

Project aims

The aim of this project is to design and develop small molecules to test the hypothesis that NEAT1 can provide an innovative new way to tackle ALS.

Environment

The main objective of the Medicines Discovery Institute is to translate fundamental discoveries in disease processes in order to identify novel molecule targets for new drugs.
By bringing together world-leading scientists to identify and produce new potential drugs, we offer a unique way to collaborate with the pharmaceutical industry. Our research covers modern drug discovery across the spectrum of oncology, immunology, neuroscience, central nervous system and respiratory diseases.
We are looking to build essential links between our researchers and the pharmaceutical industry to allow us to translate our research into real-life products which have the potential to improve the lives of people across the world, and to put Wales on the forefront in terms of medicinal innovation.
The MDI, located in the School of Biosciences (BIOSI), with excellent links to the School of Medicine (MEDIC) offers a dynamic and motivational environment for research and learning, and combines the most modern equipment with world-leading researchers. The MDI’s laboratories were recently refurbished, and as a result of this sizeable investment, we have the best possible facilities at our disposal for Medicines Discovery Research. We cooperate with schools, institutions and different departments within the University that means the student will have access to the best facilities possible.