UCL researchers have identified up to 23 genes which could be targeted by drugs to treat Parkinson’s disease (PD). There are currently no disease-modifying treatments for the debilitating neurodegenerative disorder.
In the study published in Nature Communications, which used a type of genetic analysis known as Mendelian randomization, researchers found that six of these 23 had particular potential as drug targets.
The team, led by Prof Nicholas Wood and Prof Aroon Hingorani, looked at over 3,000 genes that encode ‘druggable’ proteins and predicted their efficacy as drug targets for PD. The team analysed how proteins might respond to medication.
Genetic analysis of this kind sheds light on which treatments may successfully treat a disease. Drugs which have genetic evidence are much more likely to be successful. So these data can be used by pharmaceutical companies to prioritise molecules for drug development.
Four drugs used to treat other diseases already target the genes identified by the team and could be repurposed to treat PD, according to the research team. One is metformin, a drug used to treat diabetes. A clinical trial published in 2019 suggested metformin may reduce rates of PD.
Prof Wood said: “Part of the reason why we don’t yet have effective disease-modifying treatments is that there has been a lack of potential drug targets. The data we have published helps to fill this gap and can be used by industry to prioritise potential treatments to take forward to clinical trials.”
The six genes with the most potential as drug targets were CTSB, GPNMB, CD38, RHD, IRAK3 and LMAN1.