Emerging therapies
Gene therapy, stem-cell transplantation, alpha-synuclein immunotherapy (prasinezumab, cinpanemab), LRRK2/GBA-targeted therapy, GLP-1 agonists and other investigational treatments.
In this category
State of the art
No update yet for Emerging therapies. An update is a standalone state-of-the-art for the topic — what someone with Parkinson's needs to know about where this approach stands today.
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Exercise and smoking: health rivals revealing shared protective mechanisms in Parkinson’s? Exercise mimetics
This perspective explicitly frames the mechanistic overlap between exercise and tobacco as a roadmap for identifying novel drug targets — compounds that could mimic the neuroprotective biology of exercise without tobacco's harms, potentially informing the next generation of disease-modifying therapies for PD. -
Levodopa increases substantia nigra iron: implications for Parkinson's disease Iron chelation
If levodopa actively increases nigral iron, iron-chelation strategies (e.g. deferiprone) become more compelling as adjunct therapy — not only to counteract disease-driven iron accumulation but potentially also drug-driven accumulation. -
Observations on an Open‐Label Phase 1/2 Dopamine Gene Therapy Trial (OXB‐102/Axo‐Lenti‐PD) in People with Parkinson's Disease Gene therapy
This is the first complete peer-reviewed publication of the SUNRISE-PD open-label Phase 1/2 data for OXB-102/AXO-Lenti-PD, a lentiviral vector delivering TH, GCH1, and AADC genes to the putamen. Six participants tolerated the therapy without serious drug-related adverse events, and UPDRS-III off scores improved ~40% from baseline; the programme was terminated commercially in 2022 before a sham-controlled efficacy trial could be completed. -
Genome-wide association and population-tailored polygenic risk for Parkinson’s disease in Taiwan LRRK2 inhibitors
The gene-dosage analysis identifies double East-Asian LRRK2 variant carriers (p.G2385R + p.R1628P) as a high-risk subgroup with markedly elevated LRRK2 kinase activity (elevated pRab10 phosphorylation), making them a priority population for stratified trials of type II LRRK2 kinase inhibitors now under development. -
バイオジェンとデナリ、パーキンソン病治療薬の開発を中止 執筆 - Investing.com - FX | 株式市場 | ファイナンス | 金融ニュース LRRK2 inhibitors
The Phase 2b LUMA trial of BIIB122 (DNL151) — one of the most advanced LRRK2 inhibitor programmes — failed to slow disease progression versus placebo in early-stage idiopathic Parkinson's, leading Biogen and Denali to discontinue development for the general Parkinson's population. The drug did hit its biological targets (>90% peripheral LRRK2 inhibition, ~30% CSF biomarker reduction), suggesting the mechanism itself may be insufficient for broad Parkinson's, though the separate BEACON trial in people with a pathogenic LRRK2 gene variant continues, with data expected in the first half of 2027. -
Parkinson : une voie cérébrale protège les neurones féminins - Daily Beirut
This preclinical Journal of Neuroscience study (2026) identifies the β2 nicotinic acetylcholine receptor pathway as a potential neuroprotective target in female Parkinson's models; earlier work from the same lab showed the smoking-cessation drug cytisine (a partial nAChR agonist, not nicotine) could activate this protection, suggesting cytisine or similar non-addictive nAChR-boosting agents as repurposing candidates specifically for female patients. -
Safety and efficacy of faecal microbiota transplantation in Parkinson’s disease Faecal microbiota transplantation
This systematic review of 11 human studies (5 RCTs, 236 subjects) found FMT to be broadly safe in Parkinson's, with only mild transient gut side effects reported. Motor outcomes were inconsistent across RCTs — one showed benefit at 8–12 weeks, another only at one year, others none — and most changes hovered around the threshold of what patients would notice in daily life, leaving the procedure investigational rather than ready for clinical use. -
Cellular Intelligence acquires global rights to STEM-PD program Stem-cell therapy
Cellular Intelligence has acquired global rights to STEM-PD from Novo Nordisk and will use an AI platform to advance the program. The Phase 1/2 trial (NCT05635409) has dosed eight participants across UK and Swedish sites, with positive early safety data in the first four patients allowing progression to a higher-dose cohort; the FDA has cleared the program for Phase 2 and granted it Fast Track designation. -
Cell Replacement Approval in Japan and Other Cell Therapy Updates Stem-cell therapy
The video discusses the recent regulatory approval of stem cell therapy in Japan and outlines the current state of global clinical trials aimed at replacing dopamine-producing cells in the brain. -
Phase 3 trial of solengepras for Parkinson’s disease now fully enrolled GPR6 antagonists
Solengepras (CVN424) is a first-in-class oral GPR6 receptor antagonist targeting striatal neurons; with Phase 3 enrollment complete and readout expected late 2026, it represents a novel non-dopaminergic mechanism that could reach patients if Phase 3 data confirm Phase 2 results. -
Research to get excited about?
A Norwegian nationwide registry study (14,289 PD cases, 2004–2020, published in Neurology 2025) found tadalafil — a PDE5 inhibitor used for erectile dysfunction and prostate symptoms — associated with reduced 8-year mortality in people with Parkinson's. The authors caution that the finding is exploratory and may reflect selection bias (healthier patients being prescribed the drug) rather than a true neuroprotective effect; existing preclinical data for PDE5 inhibitors in Parkinson's is limited and mixed. -
Parkinson's Disease.
The review surveys a broad pipeline of disease-modifying trials, including LRRK2 kinase inhibitors, GBA1-targeting agents, anti-alpha-synuclein monoclonal antibodies (such as prasinezumab), antisense oligonucleotides, gene therapies, and GLP-1 receptor agonists (the same class as semaglutide/Ozempic, now in Phase 2/3 trials for Parkinson's). None are yet approved to slow disease progression, but the field is described as more active than at any prior point. -
Driving motor cortex oscillations restores plasticity and improves bradykinesia features in Parkinson’s disease
Combining gamma-frequency tACS with iTBS (two non-invasive scalp stimulation methods) is shown here for the first time to both restore motor cortex plasticity and produce functional motor improvement in Parkinson's patients, establishing this paired approach as a credible candidate for future therapeutic development. -
Preformed fibrils of α-synuclein rapidly activate LRRK2 on early endosomes, driving Rab5 phosphorylation and disrupting endolysosomal and synaptic function
Pharmacological LRRK2 inhibition with MLi-2 fully restored lysosomal function, chromatin accessibility, gene expression, and neuronal excitability in PFF-treated neurons, providing strong preclinical evidence that LRRK2 inhibitors could benefit a broad PD population beyond those with LRRK2 mutations.