A drug which has been used to treat liver disease for over 30 years is being trialled in patients with Parkinson’s Disease to determine its safety and tolerability in Parkinson’s, but also to assess its potential to slow down disease progression for the first time.

The ground-breaking study, led by researchers at the University of Sheffield in partnership with Sheffield Teaching Hospitals NHS Foundation Trust, will assess the safety and tolerability of the liver drug ursodeoxycholic acid (UDCA) in Parkinson’s patients – a drug which is hoped could potentially be repositioned to help slow down the progression of the disease.

Parkinson’s Disease currently affects around 145,000 people in the UK. The progressive neurological condition severely affects a patient’s quality of life and symptoms include problems with mobility such as walking, coordination or tremor, but can also result in memory loss, low mood or abnormal bowel function.

The symptoms of Parkinson’s are mainly due to the loss of dopamine containing nerve cells in the area of the brain which controls movement. An important reason why these cells die in the brain of patients with Parkinsons is due to a malfunction of the cell’s batteries – known as mitochondria.

The trial, led by Oliver Bandmann, Professor of Movement Disorders Neurology at the Sheffield Insitute for Translational Neuroscience (SITraN) and also an Honorary Consultant Neurologist at Sheffield Teaching Hospitals NHS Foundation Trust, was made possible due to the NIHR Sheffield Biomedical Research Centre (BRC) for Neurological Disorders.

Professor Bandmann and his team previously identified UDCA as the most promising drug to rescue mitochondrial function in Parkinson’s in a drug screen where 2,000 drugs were assessed for their rescue effect on mitochondrial function directly in the tissue of patients with Parkinson’s.

“After nearly a decade of research we are extremely pleased to launch the first clinical trial of UDCA in Parkinson’s patients to see if the drug is safe and tolerated,” said Professor Bandmann.

“This is a pilot trial, which if successful, will lead to a bigger study to firmly establish the effectiveness of the treatment to slow down progression of Parkinson’s. Currently, Parkinson’s is relentlessly progressive but patients tend to respond very well to symptomatic medication in the early stages of the disease.

“A drug which will slow down the progression of the disease – even after the first few years of diagnosis – would help people to have an improved quality of life for longer.”

The trial, known as the UP Study (UDCA in Parkinson’s) will be conducted at two centres in the UK, Sheffield and London, in collaboration with Professor Tom Foltynie at University College London Hospitals. Working in collaboration with researchers from the University of Sheffield’s Institute for Insilico Medicine (Insigneo), the NIHR Sheffield Clinical Research Facility at Sheffield Teaching Hospitals and the NIHR Sheffield Biomedical Research Centre, the effectiveness of the drug will be assessed with two novel approaches:

The clinical trial is supported by the JP Moulton Charitable Foundation, The Cure Parkinson’s Trust and the pharmaceutical company PRO.MED.CS.

Helen Matthews, Deputy Chief Executive Officer of The Cure Parkinson’s Trust said: “Through its International Linked Clinical Trials Programme, The Cure Parkinson’s Trust has been working towards bringing UDCA into clinical trials - and this trial is the first step in understanding the drug’s potential to slow Parkinson’s progression. We are delighted to be supporting Professor Bandmann’s important work.”

A new link between diminished input from dopamine-firing cells deep inside the brain and the ability to form new memories could be crucial in detecting the earliest signs of Alzheimer’s disease.

Scientists from the National Institute for Health Research (NIHR) Sheffield Biomedical Research Centre at the University of Sheffield have discovered a loss of cells that use dopamine – a neurotransmitter that has a number of functions including regulating movement and emotional responses – may cause the part of the brain responsible for forming new memories to function less effectively.

The findings could revolutionise screening for the early signs of Alzheimer’s disease – which affects more than 520,000 people in the UK – changing the way brain scans are acquired and interpreted as well as using different memory tests.

Lead author of the study, Professor Annalena Venneri, from the Sheffield Institute for Translational Neuroscience (SITraN) at the University of Sheffield, said: “Our findings suggest that if a small area of brain cells, called the ventral tegmental area, does not produce the right amount of dopamine for the hippocampus, a small organ located within the brain’s temporal lobe, it will not work efficiently.

“The hippocampus is associated with forming new memories, therefore these findings are crucial to the early detection of Alzheimer’s disease. The results point at a change which happens very early on, which might trigger Alzheimer’s disease.

“This is the first study to demonstrate such a link in humans.”

Professor Venneri and fellow lead author Dr Matteo De Marco acquired 3Tesla Magnetic Resonance Imaging (MRI) scans on 51 healthy adults, 30 patients with a diagnosis of mild cognitive impairment, and 29 patients with a diagnosis of Alzheimer’s disease. 3Tesla MRIs are twice the normal strength of normal MRI scans generating the highest quality images.

The results showed a key link between the size and function of the ventral tegmental area, the size of the hippocampus and the ability to learn new material.

“More studies are necessary, but these findings could potentially lead to a new way of screening the elderly population for early signs of Alzheimer’s disease, changing the way brain scans are acquired and interpreted and using different memory tests,” said Professor Venneri who is also an Honorary Consultant at Sheffield Teaching Hospital NHS Foundation Trust.

“Another possible benefit is that it might lead to a different treatment option with the potential to change or halt the course of the disease very early, before major symptoms manifest.

“We now want to establish how early alterations in the ventral tegmental area can be seen and also test whether these alterations can be counteracted with treatments already available.”

The results of the study are published today (27 March 2018) in the Journal of Alzheimer’s Disease.

This study has been completed at the NIHR Biomedical Research Centre (BRC) in Sheffield which is dedicated to translational neuroscience for chronic neurological disorders. The Centre was launched in April 2017.

The NIHR Sheffield Biomedical Research Centre is a research partnership between the University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust, dedicated to improving the treatment and care of people living with chronic neurological disorders.

There are many problems that plague medicine and healthcare. It is a wonder why these problems still exist, given how crucial health is to everyone here on Earth.

What better way to accelerate the solving of these problems by integrating technology into our radical solutions, while providing a safe, creative environment for people to do so?

HackMed aims to bring together hackers, dreamers, and doers to solve problems related to humanity’s elementary need, health.

HackMed is a medical hackathon aimed at developing creative solutions to solve problems within medicine and healthcare. It is a student-run event for individuals from various backgrounds interested in the crossroads of life sciences and technology.

Throughout the weekend, hackers at HackMed will have a unique opportunity to learn from each other, build awesome projects together, and share them with other hackers. This will allow a multi-disciplinary mesh of people working on developing solutions to pressing problems within healthcare and medicine.

For further information and to apply to join, please click here.