## Breadcrumb - [Home](/) - [Advancing Research](/advancing-research) ## Breadcrumb - [Home](/) - [Advancing Research](/advancing-research) # Drug Discovery and Development Driving research through funding the development of new therapies aimed to treat, and ultimately cure, Parkinson’s. Through Parkinson’s Research Ventures, we believe we’ll deliver the next new treatment for people with Parkinson’s in years, not decades. ![Group of four researchers in a lab](/sites/default/files/styles/1902x600/public/images/venture-philantropy_0.jpg?h=83615a45&itok=3Io67CIW) IN THIS SECTIONIN THIS SECTION - [Understanding Parkinson’s](/understanding-parkinsons) View Menu - [Understanding Parkinson’s](/understanding-parkinsons) - [What is Parkinson's?](/understanding-parkinsons/what-is-parkinsons) - [10 Early Signs](/understanding-parkinsons/10-early-signs) - [Causes](/understanding-parkinsons/causes) - [Movement Symptoms](/understanding-parkinsons/movement-symptoms) - [Non-Movement Symptoms](/understanding-parkinsons/non-movement-symptoms) - [Getting Diagnosed](/understanding-parkinsons/getting-diagnosed) - [Statistics](/understanding-parkinsons/statistics) - [Living with Parkinson’s](/living-with-parkinsons) View Menu - [Living With Parkinson’s](/living-with-parkinsons) - [New to Parkinson's](/living-with-parkinsons/new-to-parkinsons) - [Our Care Programs](/living-with-parkinsons/care-programs) - [Finding Care](/living-with-parkinsons/finding-care) - [Treatment](/living-with-parkinsons/treatment) - [Management & Lifestyle](/living-with-parkinsons/management) - [Emotional & Mental Health](/living-with-parkinsons/emotional-mental-health) - [Legal, Financial & Insurance](/living-with-parkinsons/legal-financial) - [My PD Story](/living-with-parkinsons/stories) - [Resources & Support](/resources-support) View Menu - [Resources & Support](/resources-support) - [Helpline](/resources-support/helpline) - [In Your Area](/your-area) - [PD Library](/resources-support/pd-library) - [Hospital Safety Guide](/resources-support/hospital-safety-guide) - [Online Education](/resources-support/online-education) - [Events](/resources-support/events) - [Community Grants](/resources-support/community-grants) - [For Care Partners](/resources-support/carepartners) - [For Professionals](/resources-support/professionals) - [Find Your Chapter](/resources-support/find-your-chapter) - [Blog](/resources-support/blog) - [Advancing Research](/advancing-research) View Menu - [Advancing Research](/advancing-research) - [Finding A Cure](/advancing-research/finding-cure) - [Our Research](/advancing-research/our-research) - [Understanding Research](/advancing-research/understanding-research) - [Join A Study](/advancing-research/join-study) - [Advocate for Research](/advancing-research/advocate-research) - [For Researchers](/advancing-research/for-researchers) - [Drug Discovery and Development](/advancing-research/parkinsons-drug-discovery) - [How You Can Help](/how-to-help) View Menu - [How You Can Help](/how-to-help) - [Donate Now](/how-to-help/donate) - [Why Support Us](/how-to-help/why-support-us) - [Make a Tribute Gift](/how-to-help/tribute) - [Fundraise](/how-to-help/fundraise) - [Volunteer](/how-to-help/volunteer) - [Corporate Sponsors](/how-to-help/corporate-sponsors) - [Planned Giving](/how-to-help/more-ways-to-give/planned-giving) - [More Ways to Give](/how-to-help/more-ways-to-give) - [Policy & Advocacy](/how-to-help/policy-advocacy-priorities) ## 💡 Quick Summary - The Parkinson’s Foundation partners with Parkinson’s UK to accelerate the development of transformative treatments through Parkinson’s Research Ventures. - This program funds high-potential early-stage Parkinson’s treatments helping them advance to [clinical trials](/living-with-parkinsons/treatment/clinical-trials "Clinical Trials"). - By prioritizing people over profit, we work to deliver new, life-changing therapies in years, not decades, filling critical gaps left by traditional pharmaceutical pipelines. To accelerate breakthroughs in treatments that people with Parkinson’s disease (PD) need today, the Parkinson’s Foundation formed a strategic partnership with Parkinson’s UK to jointly invest in drug discovery and development through the global initiative called [Parkinson’s Research Ventures](https://www.parkinsons.org.uk/research/researchers/parkinsons-research-ventures). The Parkinson’s Foundation works to solve Parkinson’s through taking a big-picture approach to research, closing the gaps in knowledge about this disease. While our annual [research grants](/advancing-research/for-researchers "For Researchers") and global [genetics study](/advancing-research/our-research/pdgeneration "PD GENEration: Powered by the Parkinson’s Foundation") make progress toward understanding the biology of Parkinson’s, there remains a shortage in the development of new drugs for Parkinson’s. It is an expensive and a high-risk endeavor for pharmaceutical companies to pursue this critical research. Without increased and sustained investment in drug discovery and development, people with PD will likely have to continue to rely on a therapy that is more than 50 years old and has significant limitations. We are dedicated to changing this. ## How does our investment in drug discovery and development work? In collaboration with Parkinson’s UK, projects with the greatest potential to improve life for people with Parkinson’s are carefully reviewed and selected. We invest in rapidly testing and developing them. We understand the drug development business and are driven by results, but because our work is driven by people with Parkinson’s, not profits, we invest where others will not. Funding is provided to high-potential pre-clinical and clinical programs that aim to modify disease progression or significantly improve the management of specific symptoms. Our early support of certain projects has been instrumental in attracting follow-on funding, accelerating the journey of new treatments closer to people living with Parkinson’s. ### Drug Discovery and Development Goals Our goal is to provide targeted funding to biotech companies looking to develop new therapies to treat, and ultimately cure, Parkinson’s disease. While our goal is ambitious, we will also leverage our efforts to: - Develop promising new therapies or approaches, resulting in a greater understanding of the disease. - Increase resources for research and development in a woefully underfunded space. - Partner with a broad range of industry organizations allowing us to lead, leverage and direct resources. - Develop a people-first approach for drug discovery and development. ## Progress Toward Breakthrough ### Types of Research Projects ![Cure projects vs Life projects graphic](/sites/default/files/styles/no_sizing/public/images/Virtual-Biotech-Projects-Graphic.png?itok=AlwADzHD) ### Stages of Research - **Scientific discoveries** — Researchers attempt to find out what goes wrong in Parkinson’s and come up with ideas for how to fix it. - **Developing treatments** — Dedicated teams turn the most promising scientific discoveries into potential new treatments. - **Clinical trials** — New treatments that have been proven safe and effective by all other methods are carefully tested in study participants. ## Latest Drug Discovery and Development Projects ### Developing Treatments Each of these studies can lead to a new Parkinson’s treatment. - ### Project EndLyz: Finding ways to boost cell recycling in Parkinson’s **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. - ### Project Acurex: Fine tuning CU-13001 to progress it toward clinical trials for Parkinson’s **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:** We are supporting the final stages of testing and development of CU-13001 ahead of clinical trials. This promising molecule could slow or stop Parkinson’s. ### Clinical Trials Each of these studies are currently being tested. - ### Project Top Hat: A phase 2 clinical trial to explore the potential of ondansetron for treating hallucinations in people with Parkinson’s or Lewy body dementia **Type:** Life (Symptom) **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. - ### Project Cannabidiol (CAN-PDP): Clinical trial to investigate cannabidiol (CBD) for Parkinson’s-related psychosis **Type:** Life (Symptom) **Overview:** There are many symptoms of Parkinson’s and not everyone will experience the same ones. Evidence shows up to 75% of people with Parkinson’s go on to develop hallucinations or delusions 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:** In 2023, the second stage of the trial started and has now completed recruitment. For this trial, 103 people with Parkinson’s who experience problems with these symptoms take part in a 12-week, double-blind, placebo-controlled study — the gold standard for testing new treatments. Results are expected in mid-2026. - ### Project Syntara (previously known as Pharmaxis): A phase 2 clinical trial of a new treatment to relieve PD-like symptoms and target inflammation to slow onset **Type:** Cure/Life (Symptom) **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. The first participant was recruited in Australia in November 2023, and in the UK in April 2025. By January 2026, all 40 participants had been successfully recruited. Results are expected by mid-2026. - ### Project Ambroxol (ASPro-PD): A phase 3 clinical trial investigating the potential of ambroxol for slowing down the progression of 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:** A world-first phase 3 trial began recruiting in 2025. This is the first large phase 3 study in the portfolio, and it offers hope that a drug to slow the progression of Parkinson’s may be on the horizon. - ### Project Mission: First clinical trial of MTX325 in people with Parkinson’s **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. In 2024, we announced plans to fund the next stage of research through the Parkinson’s Virtual Biotech – investigating the safety and benefits of the potential drug in people with Parkinson’s. The trial will recruit people with Parkinson’s to take part in an early phase clinical trial in early 2026 across multiple sites in the UK and other European countries. - ### Project Herantis: First clinical trial of a pioneering new treatment, HER-096, 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. In October 2025, results suggested the treatment is safe, well-tolerated and effective at reaching the brain. Additional biomarker data in January 2026 provided further confidence that the drug is acting as expected. In early 2026, Herantis Pharma received funding from the European Union to run a phase 2 trial of HER-096, which is now preparing to start as soon as possible. ### Past Projects Our drug development program is dynamic and agile — new projects are added and other investments come to an end. Projects may be moved to “past” because they are seeking other funding to continue or a decision has been made to stop an investment because the project is not meeting its aims. This is a vital part of making sure the most promising research is moving forward as quickly as possible. - ### Project Neurolixis: A phase 2 trial of the drug NLX-112 for treating dyskinesia in people with Parkinson’s **Type:** Life (Symptom) **Overview:** This project investigated a drug called NLX-112 for the treatment of involuntary movements (dyskinesia). This is a common side effect experienced by people with Parkinson’s who have been taking levodopa-based medications for several years. Studies investigated NLX-112 in the lab and secured approval from regulatory authorities to take the drug into clinical trials in people with Parkinson’s. In November 2020, a new phase 2a trial in 22 people with Parkinson’s was announced. Results showed the drug was safe and was beneficial for those who experience dyskinesia. The trial also showed that the drug had a positive effect on other motor symptoms of Parkinson’s. This was an early-stage trial, and its main aim was to prove the safety and understand how the body reacts to the drug, so that it can be tested on a larger scale. **Key Takeaway:** Neurolixis is currently planning how to best take this project forward to understand the effectiveness of the drug in more people and over a longer period of time. - ### Project Eurofins: Creating new drugs to improve symptoms and slow Parkinson’s **Type:** Life (Symptom)/Cure **Overview:** Eurofins aims to create new molecules to increase the activity of a selection of genes to both increase dopamine production, and boost the production of protective proteins to slow or halt the damage and loss of brain cells in Parkinson’s. **Key Takeaway:** There is still potential in this work, but after an independent review, we decided not to invest further at this time. - ### Project Sheffield: Discovering molecules that restore brain cell batteries **Type:** Cure **Overview:** Through our partnership with Sheffield University, drug-like molecules were tested to see if they can help protect brain cells and boost the function of mitochondria, the batteries of the cell. **Key Takeaway:** As the project progressed, we made new discoveries that provided valuable insights. We have no current active investment in this work. - ### Project Galaxy: Finding ways to dial down inflammation in Parkinson’s **Type:** Cure **Overview:** Inflammation is a vital process for defending the body against harm from things like infections, injuries and toxins. It should only be activated when there’s a threat. If inflammation is active when it shouldn’t be, it can cause harm to healthy cells. There’s increasing evidence that this might be the case in Parkinson’s. Announced in December 2021, this project looked to uncover ways to dial down inflammation in the brain to protect brain cells. **Key Takeaway:** The project was stopped because it looked increasingly unlikely to produce a new treatment. - ### Project Vivifi GDNF: Planning a new clinical trial of device-delivered GDNF **Type:** Cure **Overview:** Glial cell-derived neurotrophic factor (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. This project aimed to move forward a clinical trial of device delivered GDNF that has potential to help slow, stop or reverse the progression of Parkinson’s. **Key Takeaway:** We decided not to continue investing in device delivered GDNF as the costs and difficulties became too high. There are other trials in progress that continue to investigate the potential of GDNF. We’re also now funding a clinical trial of an injectable form of a growth factor that has similar potential to GDNF. - ### Project Enterin: Investigating a new treatment for Parkinson’s-related dementia **Type:** Life (Symptom) **Overview:** A drug called ENT-01 has the potential to reduce the effects of alpha-synuclein, a protein thought to play an important role in the development of Parkinson’s, in the gut. Early-stage clinical trials in people with Parkinson’s suggested that the drug improved constipation and revealed it may hold promise for treating other important aspects of Parkinson’s, including dementia. **Key Takeaway:** Unfortunately, despite their best efforts, the company has not been able to raise enough investment to take the study forward at this time. However, Enterin remains fully committed to developing ENT-01 for Parkinson’s constipation. - ### Project Lucy: Development of potential treatments to protect brain cells using mitochondria **Type:** Cure **Overview:** In June 2024, we announced a partnership 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. With investment from this partnership, they looked to see in more detail how the molecules might be able to protect brain cells. This involved exploring whether the molecules can reduce clumps of a protein thought to damage brain cells. **Key Takeaway:** The data was not strong enough for further investment and the company has no funds to take this work forward. - ### Project Keapstone: Developing drugs to target oxidative stress **Type:** Cure **Overview:** Keapstone is a company co-founded by researchers at the University of Sheffield and Parkinson’s UK. This project looked at developing drugs that act on multiple pathways believed to be important in the development of Parkinson’s. **Key Takeaway:** The company reached the end of our funding. Recent findings have provided interesting avenues for further studies, and the company and University of Sheffield are currently evaluating strategic options to progress the program. - ### Project Neumora: Drug development to target inflammation in the brain **Type:** Cure **Overview:** Research shows that there’s more inflammation in the areas of the brain impacted by Parkinson’s. This is thought to play a potential role in damaging the dopamine-producing cells in the brain, causing Parkinson’s to progress faster. We partnered with Neumora Therapeutics Inc., a US-based company, to help fund and accelerate the final lab-based research needed to advance a potential new drug that targets inflammation. The drug aims to protect brain cells impacted by Parkinson’s by stopping inflammation being triggered in the brain. **Key Takeaway:** Our investment was to help safety test a new treatment that targets neuroinflammation in PD in animal models to progress it toward clinical trials. In 2025, the company shared it will be focusing on a lead indication of obesity. The company has not ruled out working on Parkinson’s again in the future. - ### Project NRG: Targeting brain cell batteries to slow the progression of Parkinson’s **Type:** Cure **Overview:** We partnered 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. With the help of the initial investment, NRG Therapeutics Ltd designed small molecules that are able to pass across the blood brain barrier and patch up a hole in the mitochondria wall to help prevent the loss of energy and cell death. In October 2024, more funding was secured and NRG selected NRG5051 as the molecule to focus on and do the final preparations before it can be tested in clinical trials. **Key Takeaway:** In September 2025, NRG successfully raised enough funding to take the drug into clinical trials in people with Parkinson’s and motor neuron disease. This offers hope as a potential way to protect mitochondria and help stop brain cells from dying. ### How You Can Help We need to ensure that promising Parkinson’s treatments are not lost in the development pipeline due to lack of funding. Our partnership with Parkinson’s UK keeps new drugs funded and moving through the development process. With your support, we can find the next life-changing treatment. [Make a difference and donate now](https://secure.parkinson.org/site/Donation2?11283.donation=form1&df_id=11283&mfc_pref=T) ## Related Materials Videos & Webinars ### [ Neuro Talk: Research You Fund ](/library/videos/research-you-fund) [Watch Now](/library/videos/research-you-fund) Videos & Webinars ### [ Neuro Talk: Top 3 Parkinson’s Disease Research Areas We Fund ](/library/videos/research-we-fund) [Watch Now](/library/videos/research-we-fund) Podcasts ### [ Episode 45: Patient Engagement in Research: The Parkinson’s Advocates in Research Program (PAIR) ](/library/podcast/45) [Listen Now](/library/podcast/45) ## Related Blog Posts ![2 women in a doctors office](/sites/default/files/styles/500x360/public/images/patientengagement-blog.jpg?h=8ecf0d06&itok=CWUH-kRd) Advancing Research ### [ Parkinson’s Foundation Drives Research with New Patient Engagement Framework ](/blog/awareness/patient-engagement-framework) READ NOW ![neurotalk-blog-jim](/sites/default/files/styles/500x360/public/images/neurotalk-blog-jim.jpg?h=8e7b635d&itok=ppJc-Tvr) Advancing Research ### [ Neuro Talk: How Does Basic Research Get Us Closer to a Cure? ](/blog/research/basic-research) READ NOW ![Microscope ](/sites/default/files/styles/500x360/public/images/researchvlog-blog.png?h=ed9ce7df&itok=ohguX2ml) Advancing Research ### [ Find Out What Our Researchers Are Working on Right Now ](/blog/research/research-now) READ NOW - Share on Facebook - Share on X - Share on LinkedIn