Parkinson’s disease (PD) is a common neurodegenerative disorder characterised by symptoms such as tremors and slow movement – experimental treatments for genetic disorders have so far targeted mutated proteins/enzymes, but scientists from the Northwestern University Feinberg School of Medicine (Northwestern Medicine) in Chicago, US, have instead amplified healthy enzymes to alleviate symptoms of PD.

According to the study,as PD-associated mutations can produce misshapen glucocerebrosidase (GCase) enzymes, which are important for neuronal function, the scientists thus developed a series of small chemical activators that stabilised and amplified normal GCase. The activators were noted to improve PD-related cellular dysfunction in patient-derived neurons and also worked in several varieties of PD, meaning that treatment would be effective for a wide range of patients.

Dr. Dimitri Krainc, Director of the Center for Neurogenetics at Northwestern Medicine, said, “Wild-type GCase activation has the potential for modulating multiple forms of PD; the findings also highlight the importance of personalised therapies for drug development in genetic and idiopathic forms of PD.”

Meanwhile, another 2017study led by Krainc found that some of the key pathological features of PD were only seen in human neurons and not in mouse models. Krainc thinks it will be important to examine human brain cells, alongside animal models,in future testing for the disease.