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Drug trial brings hope to Parkinson’s patients

Cambridge researchers and Addenbrooke’s Hospital patients are joining forces to trial a new drug aimed at treating Parkinson’s disease.

Parkinson’s is the fastest growing neurological condition in the world, yet there are currently no therapeutics to alter its progression. Recent suggests that inflammation in the brain might be important in Parkinson’s and this is a target of interest for new therapies.

Now, Cure Parkinson’s, a UK charity working to end Parkinson’s, has granted an award to initiate a clinical trial to investigate the potential of an anti-inflammatory drug called dapansutrile.

Dr Caroline Williams-Gray
Dr Caroline Williams-Gray

It is being led by Dr Caroline Williams-Gray, who is a principal research associate at the University of Cambridge and an honorary consultant neurologist at Addenbrooke’s, specialising in Parkinson’s disease.

The work is supported by the Neuroscience Theme of the Cambridge Clinical Trials Unit and the National Institute for Health and Care Research (NIHR) Cambridge Biomedical Research Centre.

The trial will involve 36 people with Parkinson’s who will be treated with dapansutrile tablets for up to 12 months. In this early phase trial, the main aims will be to establish that the drug is safe in Parkinson’s, and to determine whether it reduces inflammation in the brain.

Parkinson’s symptoms include tremor, stiffness and slowness of movement, and arise after substantial loss of the dopamine-producing cells in an area of the brain known as the substantia nigra. This is associated with accumulation of a protein, known as α-synuclein, which is thought to disrupt the function of dopaminergic neurons. Once around half of the cells are affected, the clinical symptoms of Parkinson’s start to show. The movement problems can be accompanied by anxiety, sleep disturbance, gut symptoms, cognitive problems and dementia.

There is evidence from laboratory-based research studies that the abnormal α-synuclein protein leads to activation of a set of proteins called the NLRP3 inflammasome within immune cells, leading to inflammation and cell damage. It is thought dapansutrile has the potential to arrest this process - and in turn this may slow progression of the disease.

Dr Williams-Gray said:

There is a pressing need for a specific treatment, such as dapansutrile, which targets the most relevant aspects of the immune activation pathway in Parkinson’s without causing general immunosuppression and leading to unwanted side effects.

In this trial, we aim to determine dapansutrile’s safety and tolerability in people with Parkinson’s, and to establish whether the treatment can reduce inflammation in the brain. We will also investigate whether this results in a positive effect on clinical symptoms and disease progression.

Dr Caroline Williams-Gray

Dapansutrile is owned by Olatec Therapeutics Inc (opens in a new tab), which is based in America and Europe, and is developing novel oral inflammation therapeutics, known as specific NLRP3 inhibitors, that aim to improve the health and physical well-being of patients in a broad spectrum of inflammatory diseases.

Dr Simon Stott, Director of Research at Cure Parkinson’s said:

Professor Williams-Gray and her clinical team in Cambridge has a great deal of experience researching the role of inflammation in Parkinson’s. Cure Parkinson’s was delighted to facilitate this research collaboration between Olatec and Cambridge, and we are excited to be funding this important work.

Dr Simon Stott