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Parkinson's Disease: The Role Of Genomics In Drug Discovery

Updated: Oct 19, 2023

✍🏻 Madeline Park | MBiol Biology student at the Uni of Leeds & NHS Lab Assistant in Haemotology


What is Parkinson's disease? 🧠

Parkinson's disease (PD) is one of the most common neurological diseases.

Neurological diseases are a group of syndromes that alter the function of the brain and nervous system.

It is thought that PD is caused by the loss of specific neurons in the brain which pass messages into the limbs to promote movement. Symptoms of PD include trembling, poor balance, and difficulty talking.

Although around 1% of the population over the age of 60 suffer from PD there is unfortunately no cure. Some drugs like levodopa can treat symptoms however, the disease will continue to progress and the patient will gradually become more ill over time. This highlights the importance of recent work to discover new drugs for PD treatment using genomics.


What is genomics? 🧬

DNA can be thought of as the bodies’ blueprint, comprising of a set of instructions that make up all the proteins and structures in the body.

The entire set of these DNA instructions is known as the genome and the branch of biology which is concerned with studying an organism’s genome is called genomics.

In recent years, the field of genomics has rapidly exploded, due to improved technology enabling the study of DNA which is known as sequencing. Many healthcare professionals believe that studying our DNA will help to vastly improve future diagnostics and treatment of a range of diseases.

One review discussed the possibility of using genomics to find new and better treatments for Parkinson's disease.


What are genome-wide association studies? 📑

A genome-wide association study (or GWAS) is when a group of individuals with the same disorder are studied in order to identify similar sections of DNA across patients.

These similar sections of DNA are then said to be associated with the particular disease studied. It has been shown that drugs targeting these disease-associated sections of DNA are more likely to be successful in clinical trials and be approved by drug administration boards.


How can GWAS impact the discovery of drugs to treat Parkinson's disease? 🧪

GWAS of patients with Parkinson's disease identified 31 genes associated with the disorder.

In general, genes are used by the body to produce proteins. Drugs can then bind to these proteins to slow or prevent disease progression.

Of the 31 Parkinson-associated genes, 23 protein products have been proposed as potential drug targets. Some of these targets, such as the NDUFAF2 gene, can already be targeted by clinically-approved drugs, such as metformin, which is currently used to treat type II diabetes.

In theory, then, metformin could be repurposed as a drug for Parkinson's disease. However, future work in patient cohorts will be required to determine if treating Parkinson's patients with metformin could actually act as a potential way to slow or stop Parkinson's disease progression.

Some of the 23 targets identified do not have pre-existing clinically approved drugs although this work has still shone a light on the potential of new drug discovery pathways.


What does the future of drug discovery and genomics look like? 🔮

The use of genomics in genome-wide association studies has provided a way to identify potential drug targets in both Parkinson's disease and other disorders.

This work could lead to the discovery of new treatment options both through identifying drugs which can be repurposed or through the design of new drugs to target specific proteins in the body.

Grace Pountney, Founder and Director at Write in the Loop

Edited by: Grace Pountney (Founder and CEO)


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