Parkinson’s Virtual Biotech

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 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

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: The Parkinson’s Virtual Biotech has put an additional investment into this project to further investigate a molecule that has the potential to protect brain cells in lab experiments. The effectiveness and safety of the molecule will be assessed to inform whether clinical trials could take place in the future.

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: Cure

Overview: We are partnering with Lucy Therapeutics to drive forward research looking at new drugs to restore mitochondria. Mitochondria are responsible for producing the energy cells need to function properly. Lucy Therapeutics has developed molecules that early experiments show can target a key part of the mitochondria and improve its function. 

Key Takeaway: With investment from this partnership, they will study in more detail how the molecules might be able to protect brain cells. This will involve exploring whether the molecules can reduce clumps of a protein thought to damage brain cells. This testing should hopefully improve understanding of how the new drug could work and will bring it closer to clinical trials.

Type: Cure

Overview: CU-13001 is a molecule that has shown exciting potential for protecting the brain cells that are lost in Parkinson’s in laboratory-based tests. It targets an enzyme called 15-lipoxygenase, which emerging research suggests may play an important role in the death of dopamine-producing cells in PD.

Key Takeaway: An investment from the Parkinson’s Virtual Biotech will drive forward the development of a promising molecule which has potential to become a drug that slows or stops Parkinson’s. We are supporting the final stages of testing and development of CU-13001 in preparation for its progress into clinical trials.

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.

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: 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.

Type: Cure

Overview: Mitochondria are small structures responsible for producing the energy cells need to function properly, including brain cells. In Parkinson’s, mitochondria inside dopamine-producing brain cells stop working properly. MTX325 is a compound that has been designed to help get rid of faulty mitochondria. By removing ‘problem mitochondria’ the team hopes to protect dopamine-producing brain cells and ultimately slow or even stop the progression of the condition. 

Key Takeaway: After successful studies in the lab, MTX325 is currently being trialed in healthy individuals to understand its safety. The next stage of research involves investigating the safety and benefits of the potential drug in people with Parkinson’s.

Type: Cure

Overview: HER-096 is a new treatment that aims to protect and restore the dopamine-producing brain cells that are lost in Parkinson’s. It has been developed based on a growth factor the brain naturally produces called Cerebral Dopamine Neurotrophic Factor (CDNF). Research in the lab shows CDNF can help damaged dopamine cells survive and recover. However, CDNF is a large protein that can’t easily cross from the blood into the brain, so giving it to patients would require complex and invasive surgery. HER-096 has similar properties to CDNF but is smaller, can access the brain and be given as a simple injection. 

Key Takeaway: HER-096 has already been tested in a study with 60 healthy participants and was shown to be safe with no serious side effects. The Parkinson’s Virtual Biotech investment will fund a new study to test if regular injections of HER-096 are safe in people with Parkinson’s. The study is now underway in Finland.