Background
Adults over the age of 60 with symptoms of major depressive disorder are said to have late-life depression (LLD), a condition that usually decreases a person's quality of life and is associated with other risks like physical frailty and dementia. A common feature of more severe LLD is psychomotor slowing, where a person's ability to think and move are impaired. For example, they might not be able to walk or process information as quickly, and they might have problems with their working memory.
Psychomotor slowing in LLD might be the result of a problem with the way a person's body produces or responds to the neurotransmitter dopamine. The drug Levodopa (L-DOPA), which can replace missing dopamine in the brain, has been used to treat to treat Parkinson's disease for many decades, and it might also affect psychomotor slowing in LLD.
Methods
In this study, participants are adults aged 60 years or older with moderate to severe major depression. Participants undergo the "L-DOPA challenge"-a 2-week period where they receive a dose of L-DOPA once a day for the first week and a dose of L-DOPA twice a day for the second week. Before and after a participant completes the L-DOPA challenge, the study team assesses their depressive symptoms and psychomotor function. After the L-DOPA challenge, if a participant still shows signs of moderate or severe depression, they receive an antidepressant for 12 weeks.
Aims
The first aim of this study is to test the feasibility of the L-DOPA challenge-that is, whether most of the 50 participants recruited for this study will complete the L-DOPA challenge. For example, participants might have to withdraw if they can't make the daily visits to the research site to receive their L-DOPA medication, if they can't tolerate the medication's side effects, or if their depressive symptoms get significantly worse. Our hypothesis is that 80% of the participants will complete the L-DOPA challenge.
The second aim of the study is to see if L-DOPA affects participants' depressive symptoms, processing speed, and working memory. Our hypothesis is that L-DOPA response, measured as an improvement in gait speed, is associated with a decrease in depressive symptoms and an increase in processing speed and working memory.

Start Date15 Aug 2024

Sponsor / Collaborator

University of British Columbia

[+1]

NCT06365515

/ RecruitingNot ApplicableIIT

Dopamine and Reward Learning Across Hormonal Transition Phases

Hormonal transition periods during the menstrual cycle may predispose women to mental disorders. Hormonal fluctuations provide specific neuroendocrine conditions that modulate brain structure and function and these actions affect cognitive and emotional behaviors and affect energy and mood homeostasis. It is thought that these changes are driven by altered dopamine transmission. Here, the investigators aim to examine (1) how sex hormones and dopamine are linked and also (2) how hormonal changes affect motivation, mood, and energy homeostasis.
To this end, dopamine intervention will be tested on effort-based decision-making and motivational circuits in three hormonal stages (i.e., women in early-follicular phase (EF), women in mid-luteal phase (ML), and men). Additionally, the effects of hormonal status on metabolic indices will be tested, and its effects on mood fluctuations in a period of a month.
The investigator hypothesizes that women in EF cycle phase (1) have naturally less dopamine and show less effort, and (2) they show greater improvement in effort-based decision-making after Levodopa administration. The investigator has exploratory outcomes about (3) sex differences in reward-learning with and without Levodopa administration and explores if these differences correlate with elevated female sex hormone levels. Moreover, it is hypothesized that (4) hormonal fluctuations affect energy homeostasis, thus women in their EF cycle phase have higher energy expenditure and (5) they report more negative mood than in their mid-luteal (ML) cycle phase.

Start Date01 May 2024

Sponsor / Collaborator

Deutsche Forschungsgemeinschaft

[+1]

JPRN-UMIN000054201

/ RecruitingNot Applicable

Low-dose L-DOPA treatment for drug-induced dystonia - Low-dose L-DOPA treatment for drug-induced dystonia

Start Date18 Apr 2024

Sponsor / Collaborator

Osaka Neurological Institute

100 Clinical Results associated with Levodopa hydrate

100 Translational Medicine associated with Levodopa hydrate

100 Patents (Medical) associated with Levodopa hydrate

38,166

Literatures (Medical) associated with Levodopa hydrate

01 Nov 2025·Talanta

Dual enzyme-mimicking bimetallic MOF for selective SERS detection of L-DOPA in human serum based on cascade catalytic reaction

Article

Author: Chen, Ying ; Cheng, Xingliang ; Li, Gongke ; Hu, Yuling ; Liu, Shuojiang

Surface-enhanced Raman scattering (SERS) in conjunction with nanozymes has emerged as a powerful technique for bioanalysis due to their signal conversion and amplification effect. However, clinical applications continue to encounter challenges such as inadequate selectivity. Herein, we proposed a novel approach for the selective SERS detection of L-DOPA in human serum with dual enzyme-mimicking MOF-919(Fe-Cu). This material exhibits both catechol oxidase-like and peroxidase-like activities, enabling it to catalyze catecholic compounds to o-quinones and subsequently oxidize 3,3',5,5'-tetramethylbenzidine (TMB) molecules into SERS-active ox-TMB. Ultrasonic-assisted synthesis ensures uniform and dense loading of AgNPs within MOF-919(Fe-Cu), thereby enhancing SERS activity. A sensitive and selective method for SERS detection of L-DOPA was developed. The catechol oxidase-like activity of MOF-919(Fe-Cu) provides ideal selectivity and anti-interference to L-DOPA, while the cascade catalytic system transforms weak SERS signals of L-DOPA into amplified SERS signals of ox-TMB. The mechanism of dual enzyme-mimicking activity was further discussed. The detection process was optimized to achieve high sensitivity towards L-DOPA with a linear range 0.5-100 μmol/L and low limit of detection (LOD) of 0.16 μmol/L. Furthermore, it has been applied in serum sample of Parkinson's patients, demonstrating excellent recovery and low relative error compared to HPLC-UV. This approach offers an economical, efficient and accurate strategy for the detection of L-DOPA and provides valuable insights for the integration of selective multifunctional nanozyme and sensitive SERS detection method.

01 Sep 2025·Journal of Environmental Sciences

Long-term cadmium stress influenced the immune response of Pardosa pseudoannulata by modulating its gut microbiota and hemolymph metabolome

Article

Author: Peng, Yuan-de ; She, Qingling ; Zhou, Rong ; Peng, Jie ; Yang, Xinyi ; Wang, Juan ; Li, Jinjin ; Song, Qisheng ; Wang, Zhi

Cadmium (Cd) is a common environmental heavy metal pollutant known for its toxic effects on various plants and animals. Cd contamination also poses a threat to Pardosa pseudoannulata, a predator in rice paddies. Results indicate that Cd pollution significantly reduced the levels of Metallothionein, phenoloxidase, and Ca²⁺. Furthermore, through metabolomics and 16S rDNA sequencing, alterations in the gut microbiota and immune-related metabolites of P. pseudoannulata were identified. The abundance of harmful bacteria such as Staphylococcus increased significantly, while levels of l-Tyrosine, glutathione disulfide, l-Dopa, among others, decreased markedly. Pearson correlation analysis revealed significant associations between various gut microbes and immune-related metabolites, suggesting that Cd may alter the immune system of P. pseudoannulata by modulating its gut microbiota.

01 Jul 2025·Phytomedicine

Integrated network pharmacology, metabolomics, and microbiome studies to reveal the therapeutic effects of Anacyclus pyrethrum in PD–MCI mice

Article

Author: Shataer, Samire ; Wu, Qin Fen ; Ma, Liang Ying ; Shan Li, Pei ; Chen, Yan ; Tuersong, Tayier ; Shataer, Munire ; Yong, Yu Xuan ; Yang, Xin Ling

BACKGROUNDAnacyclus pyrethrum (l.) DC has potential value in treating Parkinson's disease (PD)-mild cognitive impairment (MCI), manifesting as impaired memory, attention, executive function, and language. However, the specific targets and modes of action of A. pyrethrum remain unclear.PURPOSEThe aim of this study was to identify the active components of A. pyrethrum and examine their effectiveness in treating a mouse model of PD-MCI.METHODSWe generated ethanol extracts of A. pyrethrum root (EEAP) and identified its active components and related targets using UHPLC-MS/MS and network pharmacology.The PD-MCI model was induced via intraperitoneal administration of 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine(MPTP). After following continuous administration of EEAP,Altered learning or memory, as well as anxiety, were tested using the morris water maze, eight-arm radial arm maze (RAM), and open-field test,elevated plus-maze. Brain histopathology and ultrastructural changes were examined using brightfield microscopy, and electron microscopy, respectively. Furthermore, protein expression was assessed using western blotting.Stool samples were used for metabolomics analysis by UHPLC-MS/MS and for 16S rDNA sequencing to determine the compositional changes of the gut microbiota.We conducted a short-chain fatty acid targeted metabolomics experiment to study their role in the gut-brain axis in PD-MCI.RESULTSUsing UPLC-MS-MS, 126 compounds were identified from A. pyrethrum samples.After searching the databases and literature reports, 31 active components and 544 drug-disease targets were screened. Biological processes and molecular functions, such as energy channels, cell signaling, and metabolism, were discovered through GO analysis. The water maze experiment showed that the average swimming distance and escape latency of mice in EEAP groups decreased. The eight-arm maze experiment showed that model had a much higher number of errors related to working memory than the control mice. In the open field experiment, compared with the control group, the mice in the EEAP group exhibited an increase in the average movement speed and total movement distance, along with a decrease in the residence time.In the elevated plus maze, the control had less anxiety than the Model. Donepezil/Levodopa(D/l) mitigated anxiety-like behavior, and EEAP (100-400 mg/kg) showed a dose-dependent increase in open-arm metrics, suggesting it may ease anxiety in mice.Hippocampal tissue of mice treated with different doses of EEAP showed intact cellular layers and the hematoxylin-eosin-stained cones were slightly better;cells were arranged neatly; their morphology was normal, and were distributed uniformly. Electron microscopy revealed that the nuclear membrane, chromatin, and nucleoli were clearly demarcated in the hippocampus of mice treated with different doses of EEAP, contrary to that in the model group. In brain extracts of the EEAP group, lighter thinner bands for amyloid precursor protein (APP) and Aβ were observed compared to those in the model group. In model mice, APP and Aβ protein expression was higher than in the blank group, as shown by stronger bands. In EEAP-treated mice, the bands were weaker, indicating reduced expression. In the model group had lower Bcl-2 and higher Bax levels. EEAP treatment increased Bcl-2 and decreased Bax expression.Compared to the control group, the model showed substantially low glutathione peroxidase (GSH-Px),superoxide dismutase(SOD),catalase (CAT)activity (p < 0.05),much higher (p < 0.05) in the EEAP-H group than that in the model. EEAP intervention significantly modulated the fecal metabolic profile of PD-MCI mice. The abundance of steroid and lipid metabolites, including linoleylethanolamine, was markedly altered in the model group compared to the control group, with EEAP treatment reversing several of these abnormalities. PLS-DA and OPLS-DA revealed significant separation between groups (Q2= 0.542, p < 0.01), confirming a dose-dependent effect. Random forest analysis identified 15 key metabolic markers, such as dose-dependent changes in d-glutamine and hydrocodone. Metabolic pathway analysis demonstrated significant enrichment in phenylalanine, tyrosine, tryptophan metabolism, and arginine biosynthesis pathways (p < 0.05). The Support Vector Machine (SVM) model achieved an AUC approaching 1, indicating substantial differences in metabolite profiles. EEAP intervention significantly influenced the composition and functional profile of the intestinal microbiota. The Venn diagram illustrates that each group shared 342 operational taxonomic units (OTUs), with the EEAP 400 group exhibiting a distinct Bacteroidetes proportion. LEfSe analysis identified g_Prevotella as the characteristic bacterium in the control group, c_Epsilonproteobacteria in the model group, and g_Adlercreutzia in the EEAP 100 group. The Faith's Phylogenetic Diversity (PD) index was highest in the EEAP 100 group, and Non-metric Multidimensional Scaling (NMDS)/Principal Coordinates Analysis (PCoA) revealed significant differences in microbial community structure. Short-chain fatty acids (SCFAs) analysis indicated that acetic acid was the predominant metabolite, while EEAP dose-dependently regulated propionic acid and isovaleric acid levels (VIP > 1, p < 0.001). These findings demonstrate that EEAP exerts its regulatory effects by reshaping the structure and metabolic functions of the gut microbiota.CONCLUSIONEEAP holds great promise as a potential therapeutic agent for PD-MCI, exerting its effects through multiple mechanisms, including regulating protein expression, modulating the fecal metabolic profile, and reshaping the gut microbiota and its metabolites.

468

News (Medical) associated with Levodopa hydrate

22 Apr 2025

·www.prnewswire.com

XADAGO/EQUFINA Set to Revolutionize Parkinson's Disease Treatment Landscape | DelveInsight

As a selective monoamine oxidase B (MAO-B) inhibitor, XADAGO offers a unique mechanism of action that complements traditional therapies, potentially improving motor function and reducing "off" time in patients. The increasing prevalence of Parkinson's disease and the growing demand for effective therapies create a substantial market opportunity for XADAGO, particularly in regions with aging populations. LAS VEGAS, April 22, 2025 /PRNewswire/ -- DelveInsight's " XADAGO/EQUFINA Market Size, Forecast, and Market Insight Report" highlights the details around XADAGO/EQUFINA, an inhibitor of monoamine oxidase B (MAO-B) indicated as an adjunctive treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of XADAGO/EQUFINA. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Newron Pharmaceuticals/Eisai/Meiji Seika Pharma's XADAGO/EQUFINA (safinamide) Overview XADAGO is a monoamine oxidase B (MAO-B) inhibitor used alongside levodopa/carbidopa to help manage "off" episodes in individuals with Parkinson's disease. While its exact mode of action in treating Parkinson's is not fully understood, it is thought to work by inhibiting MAO-B, thereby reducing dopamine breakdown. This leads to increased dopamine levels and enhanced dopaminergic activity in the brain. XADAGO contains the active ingredient safinamide in the form of a mesylate salt and is available in 50 mg and 100 mg film-coated oral tablets. Each tablet provides either 65.88 mg or 131.76 mg of safinamide mesylate, equivalent to 50 mg or 100 mg of the safinamide base. The medication is marketed as XADAGO in the United States and Europe and under the name EQUFINA in Japan. Learn more about XADAGO/EQUFINA projected market size for Parkinson's disease @ XADAGO/EQUFINA Market Potential Parkinson's disease is a progressive neurological disorder that mainly impairs movement control. It results from the slow degeneration of dopamine-producing neurons in the brain, especially in the substantia nigra, a region crucial for managing voluntary motor function. According to estimates by DelveInsight, there were 2.7 million diagnosed prevalent cases of Parkinson's disease in the 7MM in 2023, with the United States accounting for around 45% of these cases. While a cure for Parkinson's remains elusive, a combination of pharmacological and non-pharmacological therapies is used to manage symptoms. Physical, occupational, and speech therapies form key components of treatment, and surgical procedures may benefit select patients. Additionally, complementary therapies are sometimes employed to help address particular symptoms. Frequently prescribed medications for Parkinson's include levodopa, dopamine agonists, MAO-B inhibitors, COMT inhibitors, amantadine, anticholinergics, and adenosine A2A antagonists. These treatments primarily target motor symptoms, which are a major concern for those affected. DelveInsight reports that the Parkinson's disease market in the 7MM was valued at USD 3.2 billion in 2023. The market is expected to grow over the forecast period (2020–2034), fueled by the launch of new therapies, increasing prevalence, better access to healthcare, and rising demand for more advanced and effective treatment options. Discover more about the Parkinson's disease market in detail @ Parkinson's Disease Market Report Emerging Competitors of XADAGO/EQUFINA Some of the drugs in the pipeline include Solengepras (Cerevance), Minzasolmin (UCB Biopharma SRL/Novartis), Buntanetap (Annovis Bio), and others. Buntanetap has successfully completed Phase III trials for early-stage Parkinson's disease. Annovis Bio has scheduled a meeting with the FDA in Q1 2025 to determine the development path forward for Buntanetap in the treatment of Parkinson's disease. Buntanetap is also being developed for Alzheimer's disease, Lewy Body Dementia, and other neurodegenerative disorders. In November 2024, Cerevance announced the dosing of the first patient in its pivotal Phase III ARISE trial, evaluating solengepras as a potential adjunctive treatment for Parkinson's disease. Cerevance expects to report topline data in the first half of 2026. In December 2021, UCB partnered with Novartis to globally co-develop and co-commercialize UCB0599, a pioneering alpha-synuclein misfolding inhibitor currently in Phase II clinical trials for Parkinson's disease. To know more about the number of competing drugs in development, visit @ XADAGO/EQUFINA Market Positioning Compared to Other Drugs Key Milestones of XADAGO/EQUFINA In April 2020, Newron Pharmaceutical acknowledged that US WorldMeds, who held the US commercialization rights for the licensed drug, XADAGO, as a sub licensee of Zambon, had entered into a definitive agreement with Supernus Pharmaceuticals under which Supernus would acquire the CNS portfolio of US WorldMeds, including the US rights to XADAGO In November 2019, Eisai announced that it had launched the EQUFINA 50mg tablets for the indication of improvement of the wearing-off phenomenon in patients with Parkinson's disease under treatment with a drug containing levodopa in Japan. Manufacturing and marketing approval of EQUFINA was obtained in September 2019. In April 2017, Eisai Co., Ltd. and Meiji Seika Pharma announced entering into a license agreement for the commercialization of safinamide (development code: ME2125) to treat Parkinson's disease in Japan and Asia. In March 2017, Newron Pharmaceuticals announced the US FDA approval of XADAGO to treat Parkinson's disease as an add-on therapy to levodopa/carbidopa for patients experiencing "off" episodes. In March 2016, Zambon announced a strategic agreement with US WorldMeds to commercialize Newron's lead compound, XADAGO, to treat Parkinson's disease in the US. In February 2015, XADAGO was approved in the European Union as an add-on therapy to levodopa, alone or in combination with other Parkinson's medications, in mid-late stage PD patients with motor fluctuations. XADAGO/EQUFINA Patent Details XADAGO tablets are currently protected by three XADAGO patents with US patent numbers 8,076,515, 8,278,485, and 8,283,380, which expire between June 2027 and December 2028 and are listed in the FDA's publication, Approved Drug Products with Therapeutic Equivalence Evaluations, commonly referred to as the Orange Book. The company has a license agreement with Zambon S.p.A., Newron's partner, related to the XADAGO Patents, and as a new chemical entity, XADAGO is under the 5-year FDA exclusivity period that expires on March 21, 2022. In May 2017, Zambon filed a worldwide patent under the patent number WO2017207587A1. The invention relates to pharmaceutical compositions comprising safinamide and, more particularly, to taste-masked particles comprising said active ingredient or pharmaceutically acceptable salts thereof, oral dosage forms that include said particles, and a process for preparing them. Discover how XADAGO/EQUFINA is shaping the Parkinson's disease treatment landscape @ XADAGO 50mg XADAGO/EQUFINA Market Dynamics XADAGO, marketed as EQUFINA in some regions (notably in South Korea and Japan), is a once-daily oral selective MAO-B inhibitor used as an add-on treatment for Parkinson's disease patients experiencing "off" episodes while on a stable dose of levodopa. The drug's unique dual mechanism of action—MAO-B inhibition and modulation of glutamate release—offers both dopaminergic and non-dopaminergic effects, positioning it as a differentiated product in the PD adjunctive therapy landscape. This has helped it carve a niche in markets where patients are inadequately controlled by levodopa alone, particularly those in mid-to-late-stage PD. The global market dynamics for XADAGO/EQUFINA are shaped by a mix of competitive intensity, regional adoption trends, and payer dynamics. In the U.S. and Europe, the drug has faced stiff competition from generic MAO-B inhibitors (like selegiline and rasagiline) and newer Parkinson's disease therapies, including COMT inhibitors, dopamine agonists, and extended-release levodopa formulations. Despite this, its favorable safety profile and once-daily dosing have supported its uptake in certain patient segments, especially in Europe. Market penetration has been slower in the U.S. due to both reimbursement hurdles and physician preference for more established adjunctive treatments. In Asia, particularly Japan and South Korea, EQUFINA has demonstrated stronger market performance thanks to focused commercial strategies and a high unmet need for effective "off time" management. Meiji Seika Pharma and Eisai, the regional marketing partners, have successfully leveraged the drug's differentiated profile in clinician education, which has led to growing adoption among neurologists. In Japan, where the aging population is rapidly expanding and Parkinson's prevalence is rising, EQUFINA has benefited from both demographic tailwinds and formulary access. Looking ahead, the market outlook for XADAGO/EQUFINA depends on several strategic levers, including lifecycle management, regional partnerships, and real-world evidence generation. The manufacturer's ability to expand into new geographies, secure favorable reimbursement, and differentiate against both generics and newer entrants will be critical. Moreover, as Parkinson's disease treatment moves toward more personalized approaches, XADAGO/EQUFINA may find sustained relevance in specific subpopulations, particularly those requiring a balance of efficacy and tolerability. Dive deeper to get more insight into XADAGO/EQUFINA's strengths & weaknesses relative to competitors @ XADAGO/EQUFINA Market Drug Report Table of Contents Related Reports Parkinson's Disease Market Parkinson's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Parkinson's disease companies, including UCB Biopharma SRL, Novartis, Annovis Bio, Supernus Pharmaceuticals, Inc., Britannia Pharmaceutical, Pharma Two B, Mitsubishi Tanabe Pharma (NeuroDerm), AbbVie, Cerevel Therapeutics, Cerevance, among others. Parkinson's Disease Pipeline Parkinson's Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Parkinson's disease companies, including Cerevel Therapeutics, Inhibikase Therapeutics, Neuraly, Peptron, Biogen, Roche, Brain Neurotherapy Bio, Inc., Modag, Annovis Bio Inc., BioVie Inc., United Neuroscience Ltd., Luye Pharma Group, AbbVie, UCB Biopharma SRL, InnoMedica Schweiz AG, Integrative Research Laboratories AB, H. Lundbeck A/S, Shanghai WD Pharmaceutical Co., Ltd., Cerevance Beta, Inc., Nobilis Therapeutics Inc., BlueRock Therapeutics, Taiwan Mitochondrion Applied Technology Co., Ltd., among others. Cell and Gene Therapy in Parkinson's Disease Market Cell and Gene Therapy in Parkinson's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key cell and gene therapy in Parkinson's disease companies including MeiraGTx, Hope Biosciences, Sumitomo Pharma, Prevail Therapeutics, BlueRock Therapeutics, Voyager Therapeutics, among others. Parkinson's Disease Psychosis Market Parkinson's Disease Psychosis Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Parkinson's disease psychosis companies, including Sumitomo Pharma America Inc., Vanda Pharmaceuticals, Acadia Pharmaceuticals Inc., Otsuka America Pharmaceutical, Lundbeck LLC, Jazz Pharmaceuticals, Alkahest Inc., Sandoz, Sio Gene Therapies, Axovant Sciences Ltd, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Drug ApprovalLicense out/inPhase 2

16 Apr 2025

·www.biopharmadive.com

New research kindles excitement around stem cell therapies for Parkinson’s

The most effective drug for Parkinsons disease hasnt changed in 50 years. But fresh research published in one of the countrys top scientific journals is helping build the case for a more cutting-edge approach that uses stem cells to restore important brain functions.The second most common neurodegenerative illness, Parkinsons, is caused by the loss of certain nerve cells. These cells produce a chemical messenger, dopamine, that plays a crucial role regulating movement. Since the 1970s, a drug called levodopa, which the body converts into dopamine, has been the mainstay treatment for combating the tremors, slowness, stiffness and balance issues that come with Parkinsons.Levodopa doesnt stop the disease, however, so scientists have spent decades trying to find a more permanent fix. The latest development in that search comes from two clinical trials testing stem cell-derived therapies transplanted into the brains of Parkinsons patients.The first of those trials, conducted at the Kyoto University Hospital in Japan, evaluated seven people who received what are essentially blank slate cells that can turn into dopamine-producing neurons. These cells were created using a technology pioneered at Kyoto University, where roughly 20 years ago, scientists discovered how to reset some adult human cells to act like stem cells.The studys main focus was safety. Results, published Wednesday in Nature, show no serious adverse events were reported, though researchers did identify 73 mild to moderate events. Importantly, there was no evidence the introduced cells started growing in an unchecked, tumor-like fashion a major concern with stem cell implants.The study also explored whether this therapy had any effect on patients disease. Two years post-treatment, the six participants who were evaluable had gotten better on a scale clinicians use to measure the severity of Parkinsons motor symptoms. Researchers looked at whats known as on time, when medications are adequately controlling these symptoms, as well as the opposite off time, and found scores improved an average of 36% and 20%, respectively.Another test indicated that dopamine-producing cell activity had increased substantially. This was determined through a technique in which researchers strap a radioactive element to a molecule of levodopa and then use a PET scan to follow its path through the nervous system, somewhat like how a submarine tracks objects on a sonar display.The second trial took place at sites throughout the U.S. and Canada, and employed a different kind of stem cell that comes from human embryos. It enrolled a total of 12 participants and followed them for a year post-transplant.Similar to the Japan study, there were no deaths, serious adverse events or tumor-like tissue growth related to the introduced cells. Trial runners did report two serious events overall. One participant was hospitalized with COVID-19, while another had a seizure that was attributed to the surgical procedure.The trial also saw improvements in off scores on that disease severity scale and positive results from the radioactive levodopa test.The cell therapy tested, called bemdaneprocel, is being developed by Bayer subsidiary BlueRock Therapeutics, which back in August 2023 announced some of the data published Tuesday. The company expects to start a Phase 3 trial before the end of June.Hideyuki Okano, a stem-cell scientist at Keio University in Tokyo, argues more research is needed to confirm these types of cell therapies are effective against Parkinsons.Still, in an accompanying Nature editorial, he called the new trial results encouraging because they suggest treating Parkinsons patients with donor cell transplants is likely safe. That both trials proved to be safe, and hinted at possible efficacy, is an important step towards the establishment of this cell therapy for Parkinsons disease in wider society, he wrote.Cell therapy for Parkinsons isnt a new idea. In 1989, a team led by neuroscientist Olle Lindvall performed the first cell transplantations for Parkinsons patients. While the landmark study didnt show a major therapeutic benefit, a few interesting signals encouraged the research community to keep exploring.One ethical concern from that trial was that it used a relatively large amount of tissue derived from aborted fetuses. In the decades since, Parkinsons and cell biology experts have made significant progress identifying other, less controversial sources of stem cells that can be scaled more easily.A milestone of that work came in 2020, when the technique invented at Kyoto University was used to turn a Parkinsons patients own skin cells into the early stages of dopamine-producing neurons, which were then implanted into his brain.According to Okano, as of December, there were 115 clinical trials testing 83 products derived from pluripotent stem cells. And among those targeting central nervous system disorders, the ones for Parkinsons disease are at a more advanced stage of development.Further behind Bayer and BlueRock, Aspen Neuroscience, a San Diego-based biotechnology company, has a Parkinsons therapy derived from patients own cells currently in early-stage human testing. '

Cell TherapyClinical ResultPhase 3Clinical Study

08 Apr 2025

·www.globenewswire.com

Cerevance Presents Positive Results from Phase 1 Study of CVN293 at American Academy of Neurology 2025 Annual Meeting

CVN293 was generally well-tolerated in healthy volunteers with evidence of robust brain penetration, supporting utility of KCNK13 inhibitors for neurodegenerative diseases characterized by neuroinflammationAdditional AAN 2025 poster presentations highlight the company’s NETSseq platform and solengepras, a novel non-dopaminergic investigational treatment in clinical development to treat Parkinson’s disease BOSTON, April 08, 2025 (GLOBE NEWSWIRE) -- Cerevance, a clinical-stage biopharmaceutical company advancing multiple cell type-specific therapies for the treatment of neurodegenerative, psychiatric, and central nervous system-controlled metabolic disorders, today announced results from its Phase 1 trial of CVN293, a novel KCNK13 inhibitor targeting neuroinflammation. The data showed that CVN293 was generally well-tolerated in healthy adults following single and 14-day multiple dosing and showed evidence of robust brain penetration. The data was presented today in a poster session at the American Academy of Neurology (AAN) 2025 Annual Meeting in San Diego. “The favorable tolerability and pharmacokinetic profile observed with CVN293 in this first-in-human study are promising indicators for its potential as a disease-modifying intervention for neurodegenerative diseases driven by neuroinflammation,” said Sagar Vaidya, M.D., Ph.D., chief medical officer of Cerevance. “We look forward to advancing our family of KCNK13 inhibitors for the treatment of neurodegenerative disorders characterized by neuroinflammation, where new therapeutic options are urgently needed.” The randomized, double-blind, placebo-controlled Phase 1 single/multiple ascending dose (SAD/MAD) study evaluated the safety, tolerability and pharmacokinetics of oral CVN293 in 72 healthy adult volunteers. Study participants in the SAD part of the study received a single dose of CVN293 up to 1,000 mg or placebo (CVN293 n=36; placebo n=12) and those in the MAD part received repeated doses of CVN293 up to 750 mg (375 mg twice daily) or placebo over 14 days (CVN293 n=18; placebo n=6). Results showed that CVN293 was generally well-tolerated following single and 14-day multiple dosing, with 100% of participants completing the trial. There were no severe or dose-limiting adverse events (AEs), treatment-related discontinuations or clinically meaningful changes in vital signs or laboratory parameters. All related AEs were considered mild. In the SAD and MAD parts of the study, CVN293 plasma exposure increased in a generally dose proportional manner. Cerebrospinal fluid (CSF) sampling demonstrated high levels of central nervous system (CNS) exposure with CVN293. Additional Cerevance Presentations at AAN 2025The company presented a poster demonstrating the potential of its proprietary NETSseq platform to identify and/or validate cell-specific gene targets and biological insight into human brain disorders, which can be developed into novel therapies for neurodegenerative diseases. The poster detailed two clinical-stage novel therapeutic targets with specific expression in the brain that resulted from NETSseq: solengepras and CVN293. Another poster presentation will detail the novel mechanism of action of solengepras, an oral, non-dopaminergic therapy in clinical development for the treatment of Parkinson’s disease. A brain-penetrant, specific inhibitor of GPR6, solengepras is designed to selectively target and modulate the brain circuits responsible for controlling motor and non-motor functions without directly affecting dopaminergic pathways. This novel mechanism of action is designed to reduce motor complications, such as dyskinesia, decrease periods of symptomatic worsening, known as “OFF” time, and improve functional non-motor symptoms. The poster will describe the primary results of the randomized, double-blind, placebo-controlled Phase 2 ASCEND study of solengepras as monotherapy in patients with early, untreated Parkinson’s disease. This data was previously presented at the International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD™ 2025). About CVN293CVN293 is an investigational, highly selective oral inhibitor of the potassium two pore domain channel subfamily K member 13 (KCNK13). By selectively inhibiting KCNK13, a potentially novel regulator of NLRP3 inflammasome activation, CVN293 aims to reduce neuroinflammation and slow disease progression in a variety of neurodegenerative disorders. This mechanism may provide a disease-modifying approach for challenging central nervous system (CNS) disorders and obesity. KCNK13 was identified by Cerevance’s proprietary NETSseq platform. About Solengepras (formerly CVN424)Solengepras is designed to provide a potentially novel approach to the treatment of Parkinson’s disease. Unlike dopaminergic therapies, which primarily act by replenishing, enhancing, or mimicking dopamine, solengepras is designed to selectively address the indirect pathway by modulating the GPR6 receptor. By inhibiting GPR6, solengepras aims to restore both motor and non-motor function without directly affecting dopamine levels or signaling, improving the relative balance between the direct and indirect pathways, and potentially reducing the risk of common side effects associated with dopaminergic therapies, such as dyskinesias and motor fluctuations. Solengepras is currently being evaluated as a once-daily, oral treatment for use as an adjunctive therapy to levodopa and other anti-Parkinsonian medication in the Phase 3 ARISE trial. About CerevanceCerevance is focused on advancing cell type-specific therapies for the treatment of neurodegenerative, psychiatric and central nervous system-controlled metabolic disorders. Our proprietary platform, Nuclear Enriched Transcript Sort sequencing (NETSseq), allows us to identify targets that are expressed at very low levels, that are present in rare cell types, or that change over time as a disease progresses. Our most advanced investigational treatment, solengepras, is currently in Phase 3 development and has the potential to be a first-in-class, oral non-dopaminergic therapy for both the motor and non-motor symptoms of Parkinson's disease. Our second investigational therapy, CVN766, is designed to be a highly selective oral antagonist of the orexin 1 receptor for the potential treatment of binge eating disorder and schizophrenia. Our third investigational treatment, CVN293, is a highly selective investigational oral inhibitor targeting potassium two pore domain channel subfamily K member 13 (KCNK13). CVN293 represents a potentially novel intervention point for neurodegenerative disorders and obesity. For more information, please visit www.cerevance.com and follow us on LinkedIn and X. Contacts Cerevance:Johnna Simoes, ir@cerevance.com MediaApril Dovorany, adovorany@realchemistry.com, +1-262-909-8739

Clinical ResultPhase 2Phase 1Phase 3

100 Deals associated with Levodopa hydrate

External Link

KEGG	Wiki	ATC	Drug Bank
D00059	Levodopa hydrate	N04BA01	DB01235

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Parkinson Disease	Japan	Alfresa Pharma Corp.	07 Oct 1971

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Asthma	Phase 1	United States	Acorda Therapeutics, Inc.	01 Dec 2015
Smoking	Phase 1	United States	Acorda Therapeutics, Inc.	01 Dec 2015
Dyskinesias	Phase 1	United States	AbbVie, Inc.	01 Jun 2009
Dyskinesias	Phase 1	New Zealand	AbbVie, Inc.	01 Jun 2009
Dyskinesias	Phase 1	Germany	AbbVie, Inc.	01 Jun 2009
Parkinson Disease	Phase 1	United States	Shire Development LLC	04 Jun 1970
Parkinson Disease	Phase 1	United States	Hoffmann-La Roche, Inc.	04 Jun 1970

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EAN2024	Not Applicable	Parkinson Disease	-	Inhaled levodopa	tbdjzewplz(lruqnnpvfm) = reported by 12 (66.7%) patients lpirnhgbzc (cspwsofolc ) View more	-	28 Jun 2024
INVEST study (MDS2023)	Not Applicable	Parkinson Disease	-	Continuous intrajejunal levodopa infusion (CILI)	qftxqdbfuz(gmomtukgel) = tinqolcbft mrmtbbjnyc (onqvfruxwy, -2.9 to 11.0)	-	27 Aug 2023
Parkinson’s Progression Markers Initiative (PPMI) (MDS2023)	Not Applicable	Parkinson Disease	428	levodopa	gorikvjxta(ncaoldjoaf) = vvaswyuaja rologpfehu (ypykbuwsvw )	-	27 Aug 2023
				levodopa	gorikvjxta(ncaoldjoaf) = hgmitjgiwc rologpfehu (ypykbuwsvw )
PD MED early disease randomisation (MDS2023)	Not Applicable	Parkinson Disease	1,593	Levodopa-sparing therapy (dopamine agonist or monoamine oxidase B inhibitor)	xgcfjgzwkw(nuekttqeka): P-Value = 0.84 View more	Positive	27 Aug 2023
				Levodopa alone
MDS2023	Not Applicable	Dystonic Disorders	-	Pre-levodopa administration	rufkxkwfug(snemskaetx) = kqysnpvwzx shoxbclukc (jepndtunpo )	-	27 Aug 2023
				Post-levodopa administration	rufkxkwfug(snemskaetx) = pkzrjewitp shoxbclukc (jepndtunpo )
MDS2023	Not Applicable	Parkinson Disease	-	levodopa	zgfcvoatmj(nnstuooadw) = efywuztnkh taqtqotstz (apqcipfzxp )	Positive	27 Aug 2023
				levodopa	pupbluglie(jhzwzjabkz) = ghzsirbwlj pzomaqevpr (sywivrkkoz ) View more
EAN2023	Not Applicable	-	-	levodopa (PD patients with RBD)	ivvmymqdgi(oqqrobujkk) = bwqtctipjp qbzloltnlz (plvzuvqixt )	-	28 Jun 2023
				levodopa (PD patients without RBD)	ivvmymqdgi(oqqrobujkk) = hhtmypriac qbzloltnlz (plvzuvqixt )
WPC2023	Not Applicable	Parkinson Disease	-	levodopa (PwPD with ISA of LID (ISA+))	dwoouxgjgn(pfeqabdugj) = ywffimiegh kkddmxbbfq (meaubhzleb )	-	28 Jun 2023
WPC2023	Not Applicable	Parkinson Disease	-	levodopaLCIGdopa (LCIG home-titration with telemedicine-assisted technology)	yowgrfgcti(jncvreeppu) = tffhoeysno ccbmefjimk (vpfzrhfilj ) View more	-	28 Jun 2023

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