ambroxol oral / Generic mfg. - LARVOL DELTA

LC-MS/MS-Based Determination of Ambroxol in Human Plasma and Cerebrospinal Fluid: Validation and Applicability in a Phase II Study on GBA-Associated Parkinson's Disease Patients. (PubMed, Int J Mol Sci) - "We evaluated and confirmed the stability of the analyte in plasma and CSF across various storage conditions. The method was successfully validated according to European Medicine Agency (EMA) guidelines and its applicability was confirmed in the context of a multicenter, randomized, double-blind, placebo-controlled, phase II study, designed to monitor the ambroxol levels in the plasma and CSF of GBA1-PD."

Clinical • Journal • P2 data • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA1

Efficacy and safety of inhaled ambroxol hydrochloride solution in patients with lower respiratory tract infectious diseases: a randomized, double-blind, placebo-controlled, multicentre clinical trial. (PubMed, BMC Infect Dis) - "Inhaled ambroxol is better at ameliorating respiratory symptoms and has comparable safety to placebo in patients with LRTI diseases."

Clinical • Journal • Cough • Infectious Disease • Respiratory Diseases

Ambroxol as a Treatment for Parkinson Disease Dementia: A Randomized Clinical Trial. (PubMed, JAMA Neurol) - P2 | "However, the effect of ambroxol on cognition was not confirmed. ClinicalTrials.gov Identifier: NCT02914366."

Clinical • Journal • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia • Movement Disorders • Parkinson's Disease • Progressive Supranuclear Palsy

Ambroxol induces myeloma cell death by inhibiting autophagy. (PubMed, Blood Neoplasia) - "Existing anti-MM drugs demonstrate various effects on autophagy; panobinostat, a histone deacetylase inhibitor, induces autophagy, whereas bortezomib and lenalidomide do not. Gene set enrichment and pathway analyses also revealed that ambroxol increased the expression of genes related to autophagy inhibition and unfolded protein response. These results suggested that autophagy is a promising therapeutic target for MM."

Journal • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Oncology • MCL1

Modulation of NF-κB/NLRP3 inflammasome axis Nrf2/HO-1 signaling and attenuation of oxidative stress mediate the protective effect of ambroxol against cyclophosphamide cardiotoxicity. (PubMed, Biochem Biophys Res Commun) - "In conclusion, ABX confers significant protection against CP-induced cardiotoxicity through multifaceted mechanisms, including attenuation of oxidative stress, inhibition of NF-κB/NLRP3 inflamamsome axis, and upregulation of Nrf2/HO-1 signaling. These findings suggest that ABX could serve as an effective adjunct therapy to improve the safety profile of CP in clinical oncology."

Journal • Cardiovascular • Inflammation • Oncology • IL1B • NLRP3

Multisite Active Material for High-Performance Blue Perovskite Light-Emitting Diodes and Active-Matrix Displays. (PubMed, ACS Nano) - "Here, we investigate a biopharmaceutical agent, ambroxol hydrochloride (AMB), with multifunctional groups as a multifunctional perovskite crystallization regulator...High-quality active-matrix array displays are further demonstrated, achieving precise independent control of each pixel. These displays exhibit superior brightness and color consistency, making them highly promising for advanced optoelectronic applications."

Journal

Cost-effectiveness analysis of ambroxol for the treatment of Chinese patients with Gaucher disease. (PubMed, Front Med (Lausanne)) - "Sensitivity analysis showed that the cost of imiglucerase had a significant effect on ICER as well as demonstrating the stability of the results. Ambroxol combination therapy is a cost-effective regimen compared with imiglucerase monotherapy."

HEOR • Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Safety and efficacy of inhalable ambroxol hydrochloride aerosol for adult patients with respiratory diseases: an open-label, single-arm, multicentre study. (PubMed, BMJ Open Respir Res) - P4 | "Inhalable ambroxol hydrochloride aerosol is well tolerated and effective in easing expectoration and alleviating cough, reducing sputum and improving the QoL of adult patients with acute and chronic respiratory diseases."

Journal • Chronic Obstructive Pulmonary Disease • Cough • Infectious Disease • Respiratory Diseases

The safety, tolerability, pharmacokinetics, and pharmacodynamics of nebulized pegylated interferon α-2b in healthy adults: a randomized phase 1 trial. (PubMed, BMC Pharmacol Toxicol) - P1 | "Nebulized PegIFNα-2b at doses of 90 µg and 180 µg showed acceptable safety and tolerability. Minimal systemic absorption was observed following inhalation. Further studies are needed to explore its potential, especially in patients with lower respiratory tract infections."

Clinical • Journal • P1 data • PK/PD data • Hematological Disorders • Infectious Disease • Leukopenia • Respiratory Diseases • IFNA1

An advanced inhalable dry powder, mucus-penetrating aerosol platform: Bridging Andrographolide delivery with clinical translation. (PubMed, Biomaterials) - "The platform encapsulates Andrographolide, a bioactive compound derived from traditional Chinese medicine, together with a chitosan-ambroxol coating to achieve mucus penetration, sequential drug release, and prolonged retention in the lungs...In murine models of acute lung injury (ALI), bacterial pneumonia (Klebsiella pneumoniae), and fungal pneumonia (Candida albicans), HCLplga-Ab demonstrated enhanced mucus penetration and biofilm destruction, uniform and prolonged drug retention in the lungs, and significant reduction in inflammation and pathogen burden. This versatile platform bridges traditional medicine with modern aerosol technology, offering a promising solution for respiratory disorders and clinical translation."

Journal • Acute Lung Injury • Infectious Disease • Inflammation • Pneumonia • Pulmonary Disease • Respiratory Diseases

ASPro-PD: Ambroxol to Slow Progression in Parkinson Disease (clinicaltrials.gov) - P3 | N=330 | Recruiting | Sponsor: University College, London | Not yet recruiting ➔ Recruiting | Trial completion date: Sep 2028 ➔ Sep 2029 | Trial primary completion date: Mar 2027 ➔ Feb 2029

Enrollment open • Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA1

Evaluation of Additive Neuroprotective Effect of Combination Therapy for Parkinson's Disease Using In Vitro Models. (PubMed, Antioxidants (Basel)) - "We demonstrated that some of the medications, used in combination, can exert an additive neuroprotective effect in preclinical models of PD that is superior to that of each of the compounds individually. This project can lead to the development of the first treatment for PD that can slow or prevent its progression."

Journal • Preclinical • CNS Disorders • Movement Disorders • Parkinson's Disease • NEFH

Product Development of High-Dose Ambroxol HCl Capsules for an n-of-1 Clinical Trial Involving Dutch Patients with Gaucher Disease Type 3. (PubMed, Pharmaceutics) - "No degradation products or microbiological impurities were detected after production. The optimized formulation of 75 mg AMB capsules formulated within the hospital pharmacy setting resulted in qualitative and uniform capsules which can be used in clinical trials."

Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Rare Diseases

Sidransky Syndrome-GBA1-Related Parkinson's Disease and Its Targeted Therapies. (PubMed, Int J Mol Sci) - "Despite failed trials of substrate reduction therapies, current clinical trials with Ambroxol and other PCs highlight promising avenues for disease modification. This commentary advocates for increased awareness of Sidransky syndrome to advance diagnostic strategies, promote genetic testing, and refine targeted treatments, with the potential to transform care for GBA1-related PD and prodromal stages of the disease."

Journal • Review • Alzheimer's Disease • CNS Disorders • Dementia • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Movement Disorders • Parkinson's Disease • GBA1

Observational Study on the Efficacy and Safety of Ambroxol and Ezetimibe Intervention in Mucopolysaccharidosis (ChiCTR) - P4 | N=60 | Not yet recruiting | Sponsor: Children's Hospital,Zhejiang University School of Medicine; Children's Hospital,Zhejiang University School of Medicine

New P4 trial

Clinical study of the effect of ambroxol irrigation of salivary glands on xerostomia in patients with primary Sjögren's (ChiCTR) - P=N/A | N=54 | Recruiting | Sponsor: Nanjing Medical University Fourth Affiliated Hospital; Nanjing Medical University Fourth Affiliated Hospital

New trial • Dental Disorders • Xerostomia

PATIENT -DERIVED MIDBRAIN ORGANOIDS OF GBA1 -PARKINSON'S DISEASE RECAPITULATE LEWY PATHOLOGY AND GLUCOCEREBROSIDASE DYSFUNCTION (ADPD 2025) - "MOs display post -natal mature features, thus holding the potential for modelling age - related physiology and disease. MOs derived from GBA1 -mutated patients recapitulate biochemical dysfunction and neuropathological hallmarks of PD neuropathology observed in post-mortem brains. Our results support the role of ambroxol as a promising disease -modifying compound in PD."

Clinical • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA1

EFFICACY OF AMBROXOL IN A MOUSE MODEL OF PARKINSON'S DISEASE (ADPD 2025) - "Ambroxol mildly reduced brain SNCA and GlcCer levels, the latter only in the high -dose cohort, suggesting that very high doses were required. Despite moderate effects on lipids, ambroxol showed limited impact on sensory or motor functions. IntelliCage beha vior suggested a potential improvement in PD-associated encephalopathy that manifests in mice as slowness."

Preclinical • Anesthesia • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA1 • PINK1 • PTEN • SNCA

TREATMENT OF DLB AND PDD: STATUS AND NEW DEVELOPMENTS (ADPD 2025) - "There is however, Level 1 evidence for the use of cholinesterase inhibitors and mixed evidence for memantine for cognition in both conditions,, and for zonisamide for parkinsonism in DLB...Examples of drugs in ongoing trials based on ClinicalTrials.Gov are neflampimod, CT1812, and ambroxol, and memantine as add-on to cholinesterase inhibitor...There are currently no widely accepted DLB-specific outcome measures available, but development of such instruments is ongoing. These and other design elements will be discussed."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia • Lewy Body Disease • Movement Disorders • Parkinson's Disease • Psychiatry

Ambroxol attenuates detrimental effect of LPS-induced glia-mediated neuroinflammation, oxidative stress, and cognitive dysfunction in mice brain. (PubMed, Front Immunol) - "Ambroxol also improves synaptic integrity by upregulating synaptic biomarkers, including PSD-95 and SNAP-23. Overall, ambroxol demonstrated anti-inflammatory, antioxidant, and neuroprotective effects in LPS-treated mice, highlighting its potential benefits in neurological disorders."

IO biomarker • Journal • Preclinical • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Infectious Disease • Inflammation • Movement Disorders • Parkinson's Disease • FAP • GFAP • IL1B • MAPK8 • TLR4 • TNFA

Evaluation Of Additive Neuroprotective Effect of Combination Therapy For Parkinson's Disease Using in Vitro Models (AAN 2025) - "Therefore, combinations of medications targeting different disease mechanisms simultaneously may show additive or synergistic efficacy in slowing Parkinson's Disease progression.Design/We evaluated 44 combinations of the nine medications and supplements (sodium phenylbutyrate, terazosin, exenatide, ambroxol, deferiprone, coenzyme-Q10, creatine, dasatinib and tauroursodeoxycholic acid), chosen for their effect on different targets, that had shown neuroprotective effects in preclinical models of Parkinson's Disease. We demonstrated that some of the medications and food supplements, used in combination, can exert an additive neuroprotective effect in in-vitro models of Parkinson's Disease that is superior to each of the compounds individually. This preliminary data should be replicated and confirmed in other preclinical models of Parkinson's Disease and ultimately in clinical trials."

Combination therapy • Preclinical • CNS Disorders • Movement Disorders • Parkinson's Disease • NEFH

Identification of Causal Plasma Proteins in Hepatocellular Carcinoma via Two-sample Mendelian Randomization and Integrative Transcriptomic‒Proteomic Analysis. (PubMed, Cancer Res Commun) - "Potential drugs included ASS1 with arginine, aspartic acid, and citrulline; KRT8 with ambroxol, diltiazem, and amikacin; and STOML2 with chlortetracycline, chlorzoxazone, and dirithromycin. This study identified several novel causal plasma proteins, providing new insights into the early diagnosis and treatment of HCC."

Journal • Hepatocellular Cancer • Hepatology • Liver Cancer • Oncology • Solid Tumor • ASS1 • B2M • GABARAPL1 • STOML2

Investigating drug-liposome interactions using liposomal electrokinetic chromatography. (PubMed, Anal Bioanal Chem) - "Canagliflozin, which initially showed no electrophoretic mobility, migrated toward the anode in the presence of negatively charged liposomes. Mobility of positively charged substances, ambroxol and maraviroc, was suppressed by the interactions with liposomes...However, the effects were complex due to changes in API ionization and liposome surface charge, complicating the distinction between pH effects and liposome presence on API behavior. Our findings emphasize the significance of liposome composition, temperature, and pH in studying the interactions of liposomes with drugs, which is crucial for optimizing liposome-based drug delivery systems."

Journal

Drug Repositioning and Repurposing for Disease-Modifying Effects in Parkinson's Disease. (PubMed, J Mov Disord) - "Prominent candidates include glucagon-like peptide-1 receptor agonists, dipeptidyl peptidase-4 inhibitors, ambroxol, calcium channel blockers, statins, iron-chelating agents, c-Abl inhibitors, and memantine. Although preclinical and early clinical studies have demonstrated encouraging results, numerous phase III trials have yielded unfavorable outcomes, elucidating the complexity of PD pathophysiology and the need for innovative trial designs. This review evaluates the potential of prioritized repurposed drugs for PD, focusing on their mechanisms, preclinical evidence, and clinical trial outcomes, and highlights the ongoing challenges and opportunities in this field."

Journal • CNS Disorders • Inflammation • Metabolic Disorders • Movement Disorders • Parkinson's Disease • ABL1

Synergistic action of mucoactive drugs and phages against Pseudomonas aeruginosa and Klebsiella pneumoniae. (PubMed, Microbiol Spectr) - "N-acetylcysteine (NAC) and ambroxol hydrochloride (AMB) are commonly prescribed alongside antibiotics to alleviate sputum retention in lower respiratory tract infections, which are often caused by bacterial pathogens. Our results suggest that, under certain conditions, NAC or AMB provides an adjuvant effect by rendering the cells more susceptible to phage infection. These results contribute to advancing our understanding of the clinical combination of mucoactive agents and phage therapy, offering insights for optimizing treatment efficacy."

Journal • Chronic Obstructive Pulmonary Disease • Infectious Disease • Pneumonia • Pulmonary Disease • Respiratory Diseases