Parkinson’s Virtual Biotech

Type: Cure

Overview: This project aims to find a way to stop harmful inflammation from damaging brain cells. Inflammation is a process that is vital for defending the body against harm from things like infection, injuries and toxins. It should only be activated when there is a threat. If inflammation is active when it shouldn’t be, it can cause harm to healthy cells. There is increasing evidence that this might be the case in Parkinson’s.

Key Takeaway: This project looks to uncover a way to reduce inflammation in the brain, in the hope to protect brain cells. This could pave the way for the design of a drug to help slow or stop the condition.

Type: Cure

Overview: We’re working with EndLyz Therapeutics, Inc. to help find therapeutic ways to clear cells of damaging or unwanted materials that might contribute to the causes of Parkinson’s. Recent research suggests that lysosomes, packets of digestive chemicals that help break down and recycle unwanted material inside cells, may be central to the development and progression of Parkinson’s. When lysosomes don’t work properly, brain cells can’t get rid of old and damaged proteins, so these build up and clump together, slowly choking cells.

Key Takeaway: This project will focus on developing new therapies to restore efficient lysosomal function, which may have the potential to slow or stop Parkinson’s.

Type: Cure

Overview: We’re partnering with researchers at the University of Sheffield to develop molecules that can boost the function of mitochondria (the power plants of brain cells). This research aims to take important steps toward creating a drug that can protect dopamine-producing brain cells and slow down the progression of Parkinson’s.

Key Takeaway: Over the next 12 months, the team will develop and test the drug-like molecules in cells from people with Parkinson’s. If this is successful, the molecules will then move forward into further testing in animal models, before moving into clinical trials in people with Parkinson’s.

Type: Cure

Overview: We’re partnering with NRG Therapeutics Ltd to find ways to boost the functioning of mitochondria in Parkinson’s. Mitochondria (the power plants of the cell) play an important role in both sporadic and inherited forms of Parkinson’s.

Key Takeaway: The aim of this project is to identify new molecules that can enter the brain and support the mitochondria. If successful, these protective molecules could provide a safe and effective new treatment that will protect brain cells, slow the progression of Parkinson’s and extend quality of life. In 2022, NRG secured additional funding to continue developing these molecules and progress toward clinical trial.

Type: Cure/Life

Overview: We’re collaborating with Eurofins, a leading contract research company in the UK, to create molecules that can increase activity of a selection of genes. Dialing up the activity of these genes has the potential both to increase dopamine production and boost the production of protective proteins to slow or halt the damage and loss of precious brain cells.

Key Takeaway: If successful, this could lay the foundation for research into new treatments that could not only improve Parkinson’s symptoms, but also slow, stop or even reverse the underlying condition.

Type: Cure

Overview: Keapstone was co-founded by researchers at the University of Sheffield and Parkinson’s UK, and this was the first project taken on by the Parkinson’s Virtual Biotech in 2017. The project looks at developing drugs that act on multiple pathways, which are believed to be important for the development of Parkinson’s.

Key Takeaway: Recent findings from this project have provided interesting avenues for further studies, so the Parkinson’s Virtual Biotech has put an additional investment into this project. The hope is that these drugs could slow or stop the progression of Parkinson’s.

Type: Cure

Overview: Research shows there is more inflammation in the areas of the brain affected by Parkinson’s. This is thought to play a role in damaging the dopamine-producing cells in the brain, causing Parkinson’s to progress faster. We’ve partnered with Neumora Therapeutics Inc., a US-based company, to accelerate the final lab-based research needed to advance a potential new drug that targets inflammation.

Key Takeaway: This drug aims to protect brain cells affected by Parkinson’s by stopping inflammation. If this two-year project is successful, the drug will be ready to move toward clinical trials involving people with Parkinson’s.

Type: Life

Overview: Involuntary movements (called dyskinesia) are a common side effect experienced by people with Parkinson’s who have been taking levodopa-based medications for several years. We’re partnering with The Michael J. Fox Foundation (MJFF) and biopharmaceutical company Neurolixis to investigate a drug called NLX-112 for the treatment of dyskinesia. The main medication currently available to manage dyskinesia is amantadine, which does not work for everyone and can have serious side effects. A new phase 2a trial was started in 2020. Results announced in July 2023 show the drug is safe to use for people with Parkinson’s and is beneficial for people who experience dyskinesia. The trial also showed the drug had a positive effect on slowness, stiffness and tremor.

Key Takeaway: This is an early-stage trial aimed to prove drug safety and understand how the body reacts to the drug, so that it can be tested on a larger scale. The next stage of the trial will aim to find out the best and safest doses and gather more information on the effectiveness with more participants over a longer period. Neurolixis is currently planning how to best advance to the next stage of clinical trials. If the next phases are successful, the drug could be approved and available by 2030.

Type: Life

Overview: It is estimated that around 75% of people with Parkinson’s experience hallucinations during the course of PD. However, current treatment options are limited. We’ve partnered with University College London to explore the potential of ondansetron as a treatment for visual hallucinations in people with Parkinson’s or Lewy body dementia. Ondansetron is currently used to treat sickness following operations or during chemotherapy.

Key Takeaway: This study is investigating whether ondansetron is beneficial and safe as a treatment for hallucinations in 306 people with Parkinson’s or Lewy body dementia. Following an interim analysis, Top Hat is no longer open to recruitment.

Type: Life

Overview: There are many symptoms of Parkinson’s and not everyone will experience the same ones. Evidence indicates up to 60% of people with Parkinson’s go on to develop symptoms of Parkinson’s psychosis as their condition progresses. We’re partnering with King’s College London to carry out a clinical trial to see whether CBD is safe and effective for treating symptoms of Parkinson’s psychosis. The first stage of the study, a six-week pilot to find the ideal dosage of oral CBD capsules, is complete.

Key Takeaway: Recruitment for the second stage of the trial started in 2023. In this stage, 120 people with PD-related psychosis will take part in a 12-week, double-blind, placebo-controlled study — the gold standard for testing if treatments work.

Type: Cure/Life

Overview: Inflammation is vital for defending the body against harm from things like infection and toxins. Researchers believe that inflammation may be linked to the causes and progression of Parkinson’s. We’re working with Syntara to investigate whether a drug called PXS-4728 can reduce inflammation in the early stages of Parkinson’s. This study will investigate PXS-4728 in 40 people who experience sleep disorders known as isolated rapid eye movement sleep behavior disorder (iRBD). Studies suggest that as many as 70% of people with iRBD go on to develop Parkinson’s.

Key Takeaway: The hope is this drug might be able to slow the onset of Parkinson’s symptoms in this group of people that are at a high risk of developing the condition. This could help find a way to the slow the progression of Parkinson’s in others with the condition. The first participant was recruited in November 2023.

Type: Cure

Overview: GDNF is a special protein that is naturally produced inside the brain. When GDNF is given to damaged dopamine brain cells in the lab, it helps them to regenerate. It may be able to do the same in people with Parkinson’s if it can be delivered to the right part of the brain in the right way. So far, clinical trials using different devices to deliver GDNF have given inconclusive results.

Key Takeaway: After reviewing scientific evidence from the first trial and listening to the views of people with Parkinson’s about whether it is right to move ahead, we have decided not to continue investing in device-delivered GDNF. There may be other opportunities for alternative ways to investigate the potential of GDNF for Parkinson’s.

Type: Cure

Overview: We partnered with research charity Cure Parkinson’s, Van Andel Institute and John Black Charitable Foundation to co-fund a trial looking at the potential of ambroxol, a drug found in a cough medicine which has been used for many years, to slow the progression of Parkinson's. Results from phase 2 of the clinical trial show that ambroxol increases a protein called GCase. A deficiency in GCase can contribute to Parkinson’s.

Key Takeaway: This trial offers hope that a drug to slow the progression of Parkinson’s may be on the horizon and is one of the closest in our portfolio to delivering a new treatment to patients.