Stem Cell & Tissue Therapies for Parkinson's Disease

When around 70% of dopaminergic neurons have died off, people experience the first overt symptoms of Parkinson’s disease. These neurons produce dopamine, an important messenger molecule (“neurotransmitter”) which neurons in the brain use to communicate with each other, especially neurons involved in movement. So to properly treat people with Parkinson’s, and even reverse the disease, it’s very important to “bring back” these lost cells. One way to achieve this is by using stem cells or other cells (e.g. dopamine-producing cells) and to introduce them into the brain area where most cells are lost, namely the substantia nigra. Some companies are working on creating stem cells, which are then differentiated (changed) into cells that produce dopamine. These cells are then infused into the brain (the substantia nigra). Scientists discovered that actually using cells that are in between stem cells and differentiated dopamine-producing cells could actually work better than injecting stem cells or dopaminergic cells. These “hybrid” or “in-between” cells are called “dopaminergic precursor cells” or “dopaminergic precursor cells”. What also has become more clear in recent years, is that it’s more effective to protect the injected cells. Most cells, when injected into the brain, die off due to the sudden change in environment and stress. Also, very few cells graft properly. An important reason for this is that the cellular environment in the brain is pro-inflammatory, pro-oxidative and “aged" (most Parkinson patients are old, and inflammation also plays a role in Parkinson’s disease). This causes many injected cells to not properly graft and die. Therefore, embedding the cells into gels that protect them, or first (genetically) modifying the cells before they are injected, leads to better survival and grafting of the cells.
Lund University (Sweden)
STEM-PD is a cell/tissue therapy using dopamine cell replacement derived from human embryonic stem cells (hESCs). STEM-PD targets motor impairment by replacing damaged or lost dopamine-producing neurons to improve motor control in Parkinson's patients.

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Bayer AG and BlueRock Therapeutics
Bemdaneprocel (MSK-DA01/BRT-DA01) is a cell/tissue therapy using dopamine cell replacement derived from human embryonic stem cells (hESCs). The goal is to replace lost dopamine-producing neurons in patients with Parkinson's disease.

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S. Biomedics
TED-A9 is a cell/tissue therapy that uses A9 dopaminergic neuron precursor cells to address motor impairment. It aims to replace lost dopamine neurons and restore dopamine production.

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Algorae Pharmaceuticals
NTCell is a cell/tissue therapy utilizing pig choroid plexus cells. These cells are transplanted into the brain to produce neurotrophic factors and other supportive molecules, aiming to slow the progression of neurodegenerative diseases by providing cellular support and promoting repair mechanisms.

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University of Texas
Uses bone marrow-derived allogeneic (from other people, not from the patient) mesenchymal stem cells to treat neurodegeneration. The goal is to support brain repair mechanisms and slow the progression of diseases like Parkinson's.

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Hope Biosciences
Develops adipose-derived mesenchymal stem cells (HB-adMSCs) to slow disease progression by supporting cellular repair and regeneration.

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IMAC Holdings
This therapy uses umbilical cord-derived mesenchymal stem cells for cell/tissue therapy. Stem cells are introduced to support neuron health and repair, aiming to reduce bradykinesia and motor impairments in neurodegenerative diseases.

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Shanghai iCELL Biotechnology
A cell/tissue therapy using human amniotic epithelial stem cells to support neuron regeneration and slow progression. Stem cells are believed to offer potential by replacing or supporting damaged cells in neurodegenerative conditions.

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Aspen Neuroscience
This company is developing autologous neuron replacement therapy in sporadic Parkinson’s disease, and a gene-corrected autologous neuron replacement therapy for genetic variants that increase the risk of Parkinson’s disease. This means they would take skin cells from a patient, convert them into induced pluripotent stem cells (iPSCs) and then reprogram them into dopaminergic neurons, which are then transplanted into the brain.

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Mitochondrial Therapies for Parkinson's Disease

Mitochondrial dysfunction plays a central role in the pathogenesis of Parkinson's disease. Mitochondria are crucial for energy production and regulating cellular health. In Parkinson’s disease, impaired mitochondrial function leads to reduced energy (ATP) production, increased oxidative stress, and the release of harmful reactive oxygen species (ROS). This dysfunction contributes to the degeneration of dopaminergic neurons in the substantia nigra, a hallmark of Parkinson's disease. Additionally, mutations in genes like PINK1 and Parkin, involved in mitochondrial quality control, further compromise mitochondrial function. Overall, mitochondrial dysfunction is linked to neuronal death, contributing to Parkinson's disease progression and severity.
Mitokinin
Mitokinin is developing small molecules to increase the activity of PINK, a protein involved in the breakdown of (damaged) mitochondria. If PINK does not work well, damaged mitochondria are not properly broken down and start to accumulate in cells, including in dopamine-producing cells. These damaged mitochondria do not work well and produce free radicals, which hinders the proper functioning of cells, and also damaging them.

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Lucy Therapeutics
This company is developing small molecules to improve the function of mitochondria.

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Mthera Pharma
MT-101-5 is a small molecule that acts as both a mitochondrial enhancer and alpha-synuclein (aSyn) inhibitor. This multi-component therapeutic enhances mitochondrial function, which is crucial for maintaining cellular energy and health, thereby aiming to slow disease progression. It also works to inhibit the aggregation of alpha synuclein, which is involved in neurodegenerative processes such as Parkinson’s disease.

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Glaceum
Vutiglabridin (HSG-4112) is a small molecule that functions as a Paraoxonase 2 (PON2) modulator. PON2 is an enzyme involved in protecting cells from oxidative stress. Modulating PON2 activity may help in reducing oxidative damage, a key factor in the progression of neurodegenerative diseases like Parkinson’s.

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Sheffield Teaching Hospital
Ursodiol (UDCA) is a small molecule functioning as a mitochondrial enhancer. Mitochondria are essential for energy production in cells, and enhancing their function could help protect against neurodegenerative processes. By improving mitochondrial health, ursodiol aims to slow the progression of diseases like Parkinson’s.

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Autophagy & Protein-Aggregation Therapies

In many neurodegenerative diseases, including Parkinson’s disease, there are problems with autophagy. Autophagy is the process by which cells “digest” or break down damaged or old cell components, ranging from proteins to cell organelles like mitochondria. You can look at autophagy as the incinerators cells use to get rid of waste materials, and to recycle their components. Reduced autophagy leads to accumulation of proteins, like alpha-synuclein, which plays an important role in Parkinson’s disease, as explained here. Reduced autophagy also leads to accumulation of damaged or old cell components, like mitochondria. Various companies are developing methods to improve autophagy, or reduce protein accumulation. This can be done in various ways: - Using antibodies. Antibodies are proteins that can bind to the protein that accumulates, like alpha-synuclein. These antibodies attach to the proteins, and cause the breakdown of these proteins by the immune system. The immune system also uses antibodies to attach to and attack viruses and bacteria. - Methods to improve lysosomal function. The lysosomes are important cell components. These are little vesicles that break down waste or damaged molecules and components of the cell. - Tagging alpha-synuclein so they are broken down (transported to the lysosomes). Below you can find examples of therapies and companies that use this and other methods to reduce the accumulation of protein in the brain.
Nine Square Therapeutics
This company develops small molecules to upregulate autophagy to treat Parkinson’s disease.

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Verge Genomics
This company uses AI and tissue banks to find novel mechanisms and pathways involved in neurodegenerative diseases, including Parkinson’s disease.

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Caraway
This company is developing methods to activate the lysosomes, or improve lysosomal function, in order to treat Parkinson’s disease.

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University of Sydney
Doxycycline is a small molecule (originally an antibiotic) that is being investigated as a possible alpha-synuclein (aSyn) inhibitor. Alpha-synuclein aggregation is a hallmark of Parkinson’s disease, and by inhibiting this process, doxycycline could potentially slow the disease’s progression.

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1st Biotherapeutics
1st-102 (FB-101) is a small molecule that functions as a c-Abl kinase inhibitor. c-Abl is a kinase implicated in neurodegenerative processes such as Parkinson's disease. By inhibiting c-Abl kinase, 1st-102 aims to reduce neuronal damage and slow disease progression.

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Inhibikase
ikT-148009 is another small molecule that acts as a c-Abl kinase inhibitor. Inhibiting the c-Abl pathway is thought to protect neurons from oxidative stress and other forms of damage associated with Parkinson’s disease, potentially slowing the progression of the disease.

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Il-Yang Pharmaceutical
Radotinib is a small molecule c-Abl kinase inhibitor. By inhibiting c-Abl kinase, radotinib seeks to protect against neurodegeneration and slow Parkinson's disease progression.

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SPARC
Vodobatinib (K-0706) is a small molecule which inhibits c-Abl kinase. Its mechanism is focused on inhibiting c-Abl to mitigate neuronal damage and slow the progression of Parkinson’s disease by blocking the effects of harmful signaling in neurons.

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FAScinate Therapeutics / Kainos Medicine
KM-819 is a small molecule functioning as a FAF1 antagonist. FAF1 (Fas-Associated Factor 1) is a protein involved in apoptotic pathways, which can lead to cell death. By antagonizing FAF1, KM-819 aims to protect neurons from apoptosis, thereby slowing neurodegeneration and disease progression.

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Alpha Synuclein Threapies

Alpha-synuclein therapies, particularly antibodies, aim to treat Parkinson's disease by targeting the abnormal accumulation of alpha-synuclein protein in the brain. In Parkinson's, misfolded alpha-synuclein aggregates form toxic clumps (Lewy bodies), leading to neuronal damage and degeneration. Antibodies designed to recognize and bind to these toxic forms of alpha-synuclein can help neutralize and clear them from the brain, reducing their spread and protecting healthy neurons. By preventing further aggregation or aiding in the removal of existing clumps, these therapies may slow disease progression and alleviate symptoms related to motor and cognitive decline in Parkinson's disease.
Roche/Prothena
Pasinezumab is an antibody that binds to alpha-synuclein, so it will be broken down by the immune system. However, various clinical trials showed disappointing results. One reason is that such antibodies mainly work outside the cells, while alpha-synuclein mainly accumulates inside cells. Also, this approach does not address the root cause why alpha-synuclein accumulates in the first place.

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ABL Bio & Sanofi
ABL-301 is an antibody designed to inhibit alpha-synuclein pathology, which is a key mechanism involved in neurodegenerative diseases such as Parkinson's disease. By targeting alpha synuclein pathology, the therapeutic aims to reduce the spread of toxic alpha-synuclein aggregates in the brain.

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MODAG & Teva
Emrusolmin (also known as Anle-138b/TEV-56286) is a small molecule inhibitor of aSyn pathology, working to prevent or reduce the accumulation and misfolding of alpha-synuclein proteins.

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Bioarctic
Exidavneumab (BAN-0805) is an antibody that acts as an alpha synuclein pathology inhibitor, with the aim of neutralizing toxic alpha-synuclein aggregates and preventing their further spread.

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Vaxxinity
UB-312 is a vaccine that targets alpha synuclein pathology by generating an immune response against pathological forms of alpha-synuclein, promoting its clearance from the brain.

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UCB
UCB-7853 is an antibody that works as an alpha synuclein pathology inhibitor, designed to reduce the pathological spread of misfolded alpha-synuclein.

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Janssen Pharmaceuticals
YTX-7739 is a small molecule that acts as an alpha synuclein pathology inhibitor, potentially inhibiting the aggregation of alpha-synuclein and thus halting the progression of diseases like Parkinson's.

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Biogen & Ionis
This is an antisense therapy that aims for alpha synuclein reduction by targeting the production of alpha-synuclein at the genetic level, reducing the amount of the protein being produced.

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AC Immune
ACI-7104 is a vaccine that functions as an alpha synuclein pathology inhibitor, helping the immune system recognize and clear pathological forms of alpha-synuclein.

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Alterity Therapeutics
ATH-434 is a small molecule that inhibits alpha synuclein pathology, designed to bind to pathological alpha-synuclein and prevent its toxic effects.

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Lundbec
Lu-AF82422 is an antibody targeting alpha synuclein pathology, with the goal of neutralizing toxic alpha-synuclein aggregates.

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AstraZeneca & Takeda
This therapeutic is an antibody that works as an aSyn pathology inhibitor, aiming to block the propagation of toxic alpha-synuclein in neurodegenerative diseases.

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Novartis & UCB
Minzasolmin (UCB-0599/DLX-313) is a small molecule that acts as analpha synuclein pathology inhibitor, potentially reducing the accumulation of toxic alpha-synuclein.

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University of Tübingen
This is the same antibody as listed above, prasinezumab, used for inhibiting alpha synuclein pathology.

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Annovis Bio
Buntanetap (ANVS-401) is a small molecule aimed at alpha synuclein reduction, designed to lower the production or aggregation of alpha-synuclein, potentially mitigating neurodegeneration.

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Gene Therapy Companies for Parkinson's Disease

Various companies are developing gene therapies for Parkinson’s disease. However, these approaches will be less effective for people with “sporadic”, non-genetic Parkinson’s disease. Sporadic Parkinson’s disease is caused by many different mechanisms, risk factors and exposures. For example, mitochondrial dysfunction, oxidative stress, the microbiome and protein accumulation all seem to play a role in the origin of this disease. So changing just one gene with a gene therapy will likely have little impact. Also, gene therapy is very expensive, and it remains to be seen if it will be approved by regulatory bodies. Often, these companies use viral vectors or lipid nanoparticles (LNPs) to deliver a gene into cells. Viral vectors are modified viruses that carry a gene and infect cells, thereboy introducing the gene into the cells. Examples are Adeno-Associated Viruses (AAVs). LNPs are small vesicles that carry the gene and merge with cells, introducing the gene into the cells. The gene is then expressed in the cells, meaning it contains the instructions to build a specific protein. This protein then can for example produce missing substances for the cell, or take over the function of a malfunctioning protein. The gene in the viral vectors or LNPs can be encoded by DNA or RNA. For example, AAVs contain DNA encoding for a gene (protein). LNPs often contain RNA encoding for a gene (protein).
MeiraGTx
This company uses AAV-based viral vectors to deliver a gene. These vectors will be injected into the subthalamic nucleus in the brain, introducing their genes into brain cells and causing the expression of a protein (glutamic acid decarboxylase or GAD) that can increase the production of GABA.

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Oxford Biomedica
This company is developing lentiviral vectors (viruses) to deliver three genes that are involved in the production of dopamine.

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Prevail
Prevail is developing AAV vectors carrying the gene that contains the instructions to produce the glucocerebrosidase enzyme.

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uniQure
This company is developing AAVs (viral vectors) which encode for antibodies and also contain miRNA to suppress the production of alpha-synuclein protein in brain cells.

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Asklepios Bio and Bayer
AAV2-GDNF is a gene therapy that delivers GDNF (glial cell line-derived neurotrophic factor) agonists to enhance neuron survival and regeneration, particularly for dopamine-producing neurons, aiming to slow disease progression.

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Microbiome Companies for Parkinson's Disease

The microbiome likely plays an important role in Parkinson’s disease, as explained here. Various companies are developing therapies to modify the microbiome to partially reverse or slow down Parkinson’s disease.
Axial Therapeutics
This company is developing compounds to reduce the production of harmful proteins that gut bacteria secrete, and which can contribute to protein accumulation.

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University of California San Francisco
Rifaximin is a small molecule antibiotic. It targets gut bacteria to reduce inflammation and potentially improve motor impairment. This approach is based on the idea that gut health may influence neurodegenerative processes, and managing harmful bacteria could slow or improve motor symptoms in Parkinson's disease.

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Medical University of Warsaw
Fecal Transfer is a biologic therapy designed to modulate the fecal microbiota (microbiome modulator). By transferring healthy gut microbiota from a donor to the patient, this therapy aims to improve motor impairment and possibly slow progression of neurodegenerative diseases like Parkinson’s. The goal is to restore a healthier microbiome balance, which could have positive effects on brain function and motor control.

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University of Texas
Uses bone marrow-derived allogeneic (from other people, not from the patient) mesenchymal stem cells to treat neurodegeneration. The goal is to support brain repair mechanisms and slow the progression of diseases like Parkinson's.

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Oxidative & Metabolic Stress

Treating oxidative and metabolic stress in Parkinson's disease can help slow disease progression and protect neurons. In Parkinson's disease, oxidative stress results from an imbalance between reactive oxygen species (ROS) and the body's ability to neutralize them, leading to mitochondrial dysfunction and neuronal damage, especially in dopaminergic neurons. Metabolic stress further exacerbates this by impairing energy production, critical for neuronal survival. Therapeutic strategies aimed at enhancing antioxidant defences, improving mitochondrial function, and optimizing cellular metabolism could reduce oxidative damage, protect neurons, and ultimately slow the degeneration process in Parkinson's disease.
Clene Nanomedicine
CNM-Au8 uses bioenergetics through gold nanocrystals to improve cellular energy production. By enhancing mitochondrial function and cellular metabolism, CNM-Au8 aims to slow the progression of neurodegenerative diseases like Parkinson's by boosting cellular health and resilience.

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HealthPartners Institute
Novolin R is an insulin receptor agonist, administered via an intranasal route to enhance insulin signaling in the brain. Insulin plays a role in brain metabolism and function, and enhancing its activity may help alleviate multiple symptoms of neurodegenerative diseases, including cognitive and motor deficits.

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Gateway Institute
ISN-GSH (insulin-glutathione) is a combination of insulin receptor agonist activity and a reducing agent (glutathione), aimed at improving both insulin signalling and reducing oxidative stress in the brain. The dual approach targets cognition/dementia by protecting neurons from damage caused by oxidative stress and enhancing cellular metabolism through insulin pathways.

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Zhejiang University School of Medicine
Idebenone is a small molecule reducing agent. It works by decreasing oxidative stress in the brain, which is a key contributor to neurodegenerative processes. By acting as an antioxidant, Idebenone aims to slow the progression of diseases like Parkinson's by protecting neurons from oxidative damage.

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Stockholm Health Care Services
Exenatide is a peptide that functions as a GLP-1 agonist (glucagon-like peptide-1 agonist). GLP-1 agonists help regulate glucose metabolism and are thought to have neuroprotective effects. This therapy is being investigated for its potential to slow the progression of Parkinson's disease by reducing inflammation and oxidative stress.

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Novo Nordisk and Cedars-Sinai Medical Center
Liraglutide is a GLP-1 agonist that acts as a peptide therapeutic. Like exenatide, liraglutide is believed to have neuroprotective properties, and it is being studied for its potential to slow the progression of Parkinson’s by reducing cellular damage.

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Novo Nordisk & Oslo University
Semaglutide is a peptide that acts as a GLP-1 agonist, similar to the other therapies in this list. It is being studied for its potential neuroprotective properties, aiming to reduce neuroinflammation and oxidative stress in Parkinson’s disease and thereby slow disease progression.

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Sanofi and University Hospital Toulouse
Lixisenatide is a peptide GLP-1 agonist, working in the same pathway as exenatide and liraglutide. It is being investigated for its ability to regulate cellular metabolism, reduce inflammation, and possibly slow the progression of neurodegenerative diseases.

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Neuraly
NLY-01 is a peptide and a GLP-1 agonist, specifically a pegylated exenatide. Pegylation increases the stability and half-life of the drug in the body, allowing for sustained activity.

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Invex & Peptron
PT-320 is a sustained-release form of exenatide (exenatide SR), a GLP-1 agonist in peptide form. The sustained-release formulation ensures longer-lasting effects, and it is being tested for its neuroprotective potential in slowing the progression of Parkinson’s disease.

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University of Sydney
Doxycycline is a small molecule (originally an antibiotic) that is being investigated as a possible alpha-synuclein (aSyn) inhibitor. Alpha-synuclein aggregation is a hallmark of Parkinson’s disease, and by inhibiting this process, doxycycline could potentially slow the disease’s progression.

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Inflammation

Slowing down or inhibiting inflammation can be a potentially promising strategy in treating Parkinson's disease by reducing neurodegeneration. In Parkinson's disease, chronic inflammation driven by activated brain cells (microglia and astrocytes) leads to the release of pro-inflammatory cytokines that exacerbate neuronal damage, particularly in dopaminergic neurons. This ongoing inflammatory response contributes to oxidative stress, mitochondrial dysfunction, and the accumulation of misfolded proteins, all of which accelerate neuron loss. By targeting inflammatory pathways, such as blocking cytokines or modulating immune cell activity, therapies can potentially protect neurons, slow disease progression, and improve motor and non-motor symptoms, enhancing overall patient outcomes.
Stockholm Health Care Services
Exenatide is a peptide that functions as a GLP-1 agonist (glucagon-like peptide-1 agonist). GLP-1 agonists help regulate glucose metabolism and are thought to have neuroprotective effects. This therapy is being investigated for its potential to slow the progression of Parkinson's disease by reducing inflammation and oxidative stress.

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Partner Therapeutics
Sargramostim is a biologic that works by stimulating the granulocyte macrophage colony, aiming to activate the immune system and potentially slow disease progression.

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Biohaven
BHV-8000 is a small molecule that inhibits both Janus kinase 1 (JAK1) and tyrosine kinase 2 (TYK2). These kinases (proteins) are involved in inflammatory pathways, and inhibiting them may reduce inflammation and slow the progression of neurodegenerative diseases.

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IntelGenx
Montelukast (INT-0043) is a small molecule leukotriene antagonist, typically used to treat asthma by reducing inflammation. In the context of neurodegeneration, it is being tested for its potential to reduce brain inflammation and slow disease progression.

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Olatec Therapeutics & University of Cambridge
Dapansutrile (OLT-1177) is a small molecule that inhibits NLRP3, a component of the inflammasome complex that triggers inflammation. By inhibiting NLRP3, dapansutrile aims to reduce neuroinflammation and slow the progression of diseases like Parkinson’s.

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Roche
Selnoflast (RO-7486967) is a small molecule NLRP3 inhibitor. It works similarly to dapansutrile by targeting the inflammasome to reduce inflammation and potentially slow the progression of neurodegenerative diseases.

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Ventus Therapeutics
VENT-02 is also a small molecule NLRP3 inhibitor, aiming to decrease neuroinflammation by inhibiting the inflammasome and slowing progression.

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Ventyx Biosciences
VTX-3232 is a small molecule that inhibits NLRP3, targeting inflammation in neurodegenerative conditions and aiming to slow the progression of diseases like Parkinson's.

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BioVie
NE-3107 is a small molecule that inhibits ERK1/2, which are kinases involved in inflammation and cellular stress responses. By inhibiting ERK1/2, this drug aims to reduce motor impairment and slow disease progression.

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University of Cambridge
Azathioprine is an immunosuppressant used as a small molecule therapy. It suppresses the immune system’s activity, which may help to reduce neuroinflammation and slow the progression of neurodegenerative diseases.

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NodThera
NT-0796 is a small molecule that acts as an inflammasome inhibitor and NLRP3 inhibitor. It aims to block neuroinflammation, thus potentially slowing the progression of Parkinson’s disease by targeting the inflammasome pathway.

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LLRK2 Therapies

Targeting leucine-rich repeat kinase 2 (LRRK2) in Parkinson's disease could mitigate its progression by inhibiting the overactive LRRK2 protein, which chemically modifies other proteins by potting phosphate groups on proteins. Abnormal, overactive LLRK2 activity is implicated in abnormal cell signalling, inflammation, and neuron degeneration. Reducing LRRK2 activity may protect dopaminergic neurons, slow neurodegeneration, and alleviate motor and non-motor symptoms of the disease.
Neuron23
NEU-411 is a small molecule that functions as a LRRK2 kinase inhibitor. It targets the LRRK2 (leucine-rich repeat kinase 2) protein, which is involved in Parkinson's disease progression. By inhibiting LRRK2 kinase activity, NEU-411 aims to slow disease progression.

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Arvinas
ARV-102 is a small molecule that functions as a LRRK2 protein degrader. This means it works to degrade the LRRK2 protein rather than just inhibiting its activity. By reducing LRRK2 levels, ARV-102 aims to prevent or slow the progression of Parkinson’s disease.

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Biogen
BIIB-094 is an antisense therapeutic designed to reduce LRRK2 levels through LRRK2 reduction. Antisense therapies work by interfering with the production of specific proteins at the genetic level, thus lowering the amount of LRRK2 protein, which is believed to contribute to Parkinson’s disease progression.

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Denali & Biogen
This is a small molecule that functions as a LRRK2 kinase inhibitor. Like NEU-411 and NEU-723, it inhibits the kinase activity of LRRK2, with the goal of reducing the progression of Parkinson’s disease by preventing harmful effects from LRRK2 overactivity.

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GBA1 Therapies

GBA1 therapies target the enzyme glucocerebrosidase (GCase), encoded by the GBA1 gene, which is implicated in Parkinson's disease (PD). Mutations in GBA1 lead to reduced GCase activity, resulting in toxic accumulation of specific lipids which are normally processed by glucocerebrosidase (GCase). This accumulation of toxic lipids can lead to lysosomal dysfunction and the accumulation of α-synuclein, a protein which is normally broken down in the lysosomes, and which accumulates in Parkinson’s disease. GCase enhancers aim to restore the enzyme's activity, improving the breakdown of harmful substances, reducing α-synuclein buildup, and promoting cellular health. By targeting this metabolic pathway, these therapies hold promise in slowing disease progression and alleviating symptoms in patients with PD, particularly those with GBA1 mutations.
Gain Therapeutics
GT-02287 is a small molecule that acts as a GCase enhancer. GCase (glucocerebrosidase) is an enzyme involved in breaking down waste materials in cells. Enhancing GCase activity can help reduce the accumulation of toxic substances, which is believed to slow the progression of neurodegenerative diseases such as Parkinson's.

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Agyany Pharmaceuticals
Ambroxol is a small molecule functioning as a GCase enhancer, aimed at increasing the activity of glucocerebrosidase to reduce the buildup of harmful substances and potentially slow disease progression.

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IRCCS National Neurological Institute
This formulation of Ambroxol acts as a GCase enhancer and is designed to improve glucocerebrosidase activity to treat Parkinson's disease by slowing its progression through the same enzymatic pathway.

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Lawson Health Research Institute
A formulation of Ambroxol as a GCase enhancer, aimed at supporting GCase activity and reducing toxic buildup in cells to slow disease progression.

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University College London
Ambroxol as a GCase enhancer is used to enhance glucocerebrosidase activity, targeting the progression of Parkinson's disease by addressing cellular waste management.

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Bial Biotech
This is a small molecule functioning as a GCase enhancer, with the aim of boosting the activity of glucocerebrosidase to reduce the pathological accumulation of cellular waste in Parkinson’s disease.

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University Medical Center Groningen
This formulation of Ambroxol acts as a GCase enhancer and is being explored for its effects on both cognition/dementia and disease progression in Parkinson's. Enhancing GCase activity may address multiple symptoms by improving cellular health.

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Targeting Dopamine

Improving dopamine metabolism in the brain is crucial for treating Parkinson’s disease, as the condition is characterised by the loss of dopamine-producing neurons in the substantia nigra. However, most of these treatments do not address the root causes of Parkinson’s disease (such as protein accumulation, mitochondrial disease and others). They aim to increase dopamine levels or dopamine signalling (for example by stimulating dopamine receptors). Such approaches mainly lead to symptomatic improvement, as dopamine plays a key role in regulating motor control, and its depletion leads to the hallmark symptoms of Parkinson’s disease, such as tremors, stiffness, and bradykinesia. Enhancing dopamine metabolism can be achieved by increasing dopamine production, reducing its breakdown, or improving receptor sensitivity. Therapies such as levodopa, dopamine agonists, and inhibitors of enzymes like monoamine oxidase B (MAO-B) help maintain dopamine levels, alleviate symptoms, and improve patients' quality of life.
Dizlin Medical Design
Infudopa (levodopa/carbidopa, LD/CD) is a small molecule that provides a dopamine precursor to treat motor impairment in Parkinson's disease by replenishing dopamine levels in the brain.

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Lundbeck
Lu-AF28996 is a small molecule dopamine receptor agonist that targets motor impairment and "OFF" episodes (periods when medication isn't working). It stimulates dopamine receptors to improve motor function.

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Serina Therapeutics
SER-214 is a small molecule dopamine receptor agonist. It aims to alleviate motor impairment by activating dopamine receptors and enhancing dopamine signalling in Parkinson’s patients.

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Adhera Therapeutics
Armesocarb (MLR-1019) is a dopamine reuptake inhibitor designed to reduce dyskinesia by increasing dopamine availability in the brain, thereby modulating dopamine activity.

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Guangzhou Henovcom Bioscience
HNC-364 is a small molecule MAO-B inhibitor, targeting motor impairment by preventing dopamine breakdown, thus increasing dopamine levels in the brain.

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Intrance Medical Systems
Legicon (a LD/CD/entacapone pump) combines a COMT inhibitor with a dopamine precursor to treat motor impairment by improving the effectiveness of levodopa and prolonging its effects.

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SynAgile
DopaFuse (LD/CD) is a dopamine precursor delivered as a small molecule to manage motor impairment by restoring dopamine levels in the brain, similar to oral levodopa/carbidopa treatments.

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PureIMS
PIMS-703 is an inhaled levodopa formulation, delivering a dopamine precursor as a small molecule for treating "OFF" episodes in Parkinson’s patients.

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University Hospital Lille and InBrain Pharma
A-Dopamine is a small molecule dopamine receptor agonist that targets motor impairment by directly stimulating dopamine receptors to mimic dopamine’s effects.

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Alexza Pharmaceuticals
AZ-009 (apomorphine) is a small molecule dopamine receptor agonist, used to treat motor impairment and "OFF" episodes by stimulating dopamine receptors and improving motor control.

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Kissei Pharma and Affamed
KDT-3594 is a dopamine receptor agonist that addresses motor impairment by enhancing dopamine receptor activity, thereby improving motor function in Parkinson’s patients.

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Chase Therapeutics
Pramipexole ER (CTC-413) is a small molecule that acts as a dopamine receptor agonist and NK-1 receptor antagonist to treat motor impairment and progression in Parkinson’s disease by modulating multiple neurotransmitter pathways.

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IRLAB Therapeutics
Mesdopetam (IRL-790) is a small molecule dopamine receptor antagonist targeting dyskinesia in Parkinson’s patients. By blocking certain dopamine receptors, it aims to reduce involuntary movements (dyskinesia) that can result from long-term dopamine replacement therapy.

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Cerevance
CVN-424 is a small molecule dopamine signalling modulator aimed at improving motor impairment and managing "OFF" periods in Parkinson’s disease. It modulates dopamine signaling pathways to enhance motor control without directly stimulating dopamine receptors.

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Cerevel Therapeutics and Abbvie
Tavapadon (PF-06649751) is a dopamine receptor agonist targeting motor impairment in Parkinson’s disease. This small molecule aims to improve motor control by stimulating dopamine receptors in the brain.

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AbbVie
ABBV-951 (LDP/LCD infusion)is a dopamine precursor delivered via continuous infusion, targeting motor impairment. This treatment provides levodopa/carbidopa in a more controlled and continuous manner to manage motor symptoms more effectively.

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Pharma Two B
P2B-001 combines a dopamine agonist (pramipexole) and a MAO-B inhibitor (rasagiline) in a small molecule formulation. It targets motor impairment by both stimulating dopamine receptors and preventing dopamine breakdown, thus enhancing dopamine availability.

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Luye Pharma Group
LY-03003 (rotigotine ER) is a dopamine receptor agonist formulated as an extended-release small molecule to treat motor impairment. By activating dopamine receptors, it mimics dopamine’s effects in the brain to improve motor function.

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Amneal
IPX-203 (LD/CD) is a dopamine precursor in small molecule form, designed to treat motor impairment by providing extended-release levodopa/carbidopa, helping patients maintain more stable dopamine levels throughout the day.

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Neuroderm (MT Pharma)
ND-0612 (LD/CD pump) is a dopamine precursor delivered via a pump for continuous infusion, aimed at managing motor impairment by providing a steady supply of levodopa/carbidopa to reduce "OFF" periods and fluctuations in motor control.

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Supernus Pharmaceuticals
SPN-830 (apomorphine infusion) is a dopamine receptor agonist administered via infusion, intended to treat "OFF" periods in Parkinson’s disease by continuously stimulating dopamine receptors, improving motor function when oral medications are not effective.

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UCB
UCB-7853 is an antibody that works as an alpha synuclein pathology inhibitor, designed to reduce the pathological spread of misfolded alpha-synuclein.

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Novel Devices for Parkinson's Disease

1. Red and infrared light therapy (neurobiomodulation) Red and infrared light of specific wavelengths (namely 630-660 nanometer and 830-850 nm) can impact brain health, and can be helpful for Parkinson’s disease too. The red light (630-660 nanometer) can increase blood flow, and infrared light (830-850 nm) can penetrate the skull and brain tissues. (Infra)red light is absorbed by specific proteins inside the mitochondria, which improves mitochondrial function. Cells also react to the light by secreting healthy and repair molecules. 2. Epidural electrical stimulation This device stimulates the spinal cord (R). It has been shown to improve freezing-of-gait in Parkinson’s patients, and other problems with gait and maintaining posture. It works by stimulating nerves in the spinal cord.

Other Therapies for Parkinson's Disease

Here we describe start-ups, companies and research groups that are developing other approaches to Parkinson’s disease from the ones already mentioned on this page.
Alkahest
Alkahest develops substances found in young blood (e.g. proteins) that could rejuvenate the body, including the brain. Also looks at methods to remove substances in the blood found that could accelerate aging (chronokines).

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Radboud University
Researches hypoxic preconditioning therapy, which involves exposing cells or tissues to low oxygen (hypoxic) conditions to stimulate protective responses. This can help cells build resilience against neurodegeneration, potentially slowing the progression of diseases like Parkinson's by enhancing the brain’s ability to cope with oxidative stress and energy deficiencies.

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Axoltis Pharma
NX-210c is a peptide that acts as a beta 1 integrin agonist, which helps promote cell adhesion and repair mechanisms. It is aimed at treating progression in neurodegenerative diseases by promoting cellular support and survival.

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Herantis
HER-096 is a peptide functioning as a CDNF (cerebral dopamine neurotrophic factor) agonist. It aims to support dopamine-producing neurons and slow the progression of diseases like Parkinson’s by enhancing neuroprotection and regeneration.

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University of Arizona
Allopregnanolone acts as a GABA receptor allosteric modulator, which means it enhances the effects of the neurotransmitter GABA. By modulating GABAergic activity, this therapy aims to reduce neurodegeneration and slow disease progression.

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InnoMedica
Talineuren is a therapy aimed at neural regeneration. By promoting the regeneration of neurons, it seeks to slow the progression of neurodegenerative diseases.

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Daewoong Pharmaceutical
ATH-399A is a small molecule that acts as a Nurr1 agonist. Nurr1 is important for maintaining dopamine-producing neurons, and this therapy aims to support their survival and slow disease progression.

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University at Buffalo
Lithium is a mineral found in nature that modulates signalling pathways related to neuroprotection. It is being tested to see if it can slow progression in neurodegenerative diseases by protecting neurons from degeneration.

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Neuronsanct
NNI-362 is a small molecule that stimulates p70S6 kinase phosphorylation, which is involved in cell growth and survival. This therapy aims to promote neural regeneration and reduce the impact of progression in neurodegenerative diseases.

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Io Therapeutics
IRX-4204 is a small molecule that acts as a RXR (retinoid X receptor) agonist. It modulates gene expression related to cell survival and inflammation, aiming to slow neurodegeneration and disease progression.

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Athira Pharma
Fosgonimeton (ATH-1017) is a small molecule that acts as a HGF (hepatocyte growth factor) agonist. HGF is involved in cellular growth and repair, particularly in the brain. By activating this pathway, Fosgonimeton aims to improve cognition and dementia by promoting neural repair and protecting against neurodegeneration.

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Technical University of Munich
Fasudil is a small molecule that functions as a ROCK (Rho kinase) inhibitor. ROCK inhibition is thought to reduce neuroinflammation and support neuron survival. Fasudil is being tested for its potential to slow the progression of neurodegenerative diseases by protecting neurons and reducing cellular stress.

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Cannabis-derived substances
Substances found in the cannabis plant, such as cannabidiol, cannabis oil and similar substances could mitigate Parkinson’s disease by stimulating cannabinoid receptors, reducing inflammation and protecting cells.

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Viage Therapeutics
DGX-001 is a peptide that functions as a vagal nerve stimulator. It aims to improve cognition and dementia by influencing the vagal nerve, which is involved in various physiological processes, including brain function.

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Kyowa Kirin
Istradefylline (KW-6002) is a small molecule acting as an adenosine receptor antagonist. It targets cognition and dementia by blocking adenosine receptors, which are involved in brain signalling pathways that can affect cognition and motor function in neurodegenerative diseases.

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Intra-Cellular Therapies
Lenrispodun (ITI-214) is a small molecule that inhibits phosphodiesterase 1. This mechanism is aimed at improving motor impairment by enhancing intracellular signalling in neurons, potentially improving motor symptoms in Parkinson’s disease.

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Eisai
Isrenonetine (E-2027) is a small molecule that inhibits phosphodiesterase 9A. It targets cognition and dementia by improving cyclic nucleotide signalling, which is crucial for memory and cognitive function in the brain.

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Celon Pharma
CPL-36 is a small molecule that inhibits phosphodiesterase 10A. It is being studied for its effects on dyskinesia, which are abnormal involuntary movements often associated with Parkinson's treatment. Inhibiting this enzyme could reduce these motor complications.

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University of Queensland
Levetiracetam is a small molecule that functions as an SV2A modulator, targeting synaptic vesicle proteins involved in neurotransmitter release. This therapy aims to improve cognition and dementia by modulating synaptic function, which could enhance communication between neurons.

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Jazz Pharma
JZP-385 is a small molecule that acts as a T-type calcium channel antagonist. This therapy aims to address motor impairment by inhibiting calcium channels that contribute to motor control and coordination, potentially reducing symptoms related to motor dysfunction in Parkinson's disease.

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