ambroxol oral / Generic mfg. - LARVOL DELTA

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

Antiprotein Corona-Fouling Effect of the Double-Stranded DNA Coating Layer on the Gold Nanoparticles-Small Molecule Adsorbent Probe. (PubMed, Anal Chem) - "To alleviate this problem, in this study, double-stranded DNA (dsDNA) was introduced to modify the AuNP probes to mitigate the negative effect of PCs on the detection of small molecules by taking the AuNPs-dichlorofluorescein (DCF) probe-based detection of ambroxol hydrochloride (AMB) as a study model...Moreover, based on dsDNA10bp90@AuNPs-DCF probes, the recovery rates of AMB analyzed in commercial milk samples greatly improved compared with that with the AuNPs-DCF probe, particularly when the samples contained AMB with much lower concentrations. This study demonstrates a dsDNA-based antiprotein corona-fouling strategy for the AuNPs-small molecule adsorbent probe, which provides beneficial ideas for dealing with the interference resulting from PCs to the studying of biological samples."

Journal

Septic cardiomyopathy or myocardial infarction?: A case report of septic shock with ST-segment elevation on ECG. (PubMed, Medicine (Baltimore)) - "In cases of acute heart failure resulting from trauma-induced sepsis, it is crucial to consider myocardial ischemia as a potential factor. Early surgical removal of infected foci may prove beneficial in improving the patient's prognosis. However, differentiating between septic cardiomyopathy and sepsis-combined myocardial infarction can be challenging, and the appropriateness of the diagnostic criteria for sepsis at this stage is debatable."

Journal • Anesthesia • Cardiomyopathy • Cardiovascular • CNS Disorders • Congestive Heart Failure • Heart Failure • Hematological Disorders • Infectious Disease • Myocardial Infarction • Myocardial Ischemia • Septic Shock

Novel methotrexate long-acting system using ambroxol coating and hydroxypropyl methylcellulose encapsulation for preferential and enhanced lung cancer efficiency. (PubMed, PLoS One) - "The apoptotic, anti-metastatic, and MTX preferential lung cancer uptake profiles were higher using the MTX-ABL solid dispersion than in the MTX or ABL. The MTX-ABL-HPMC gel could serve as an alternative to the MTX-oral tablets available in the markets with enhanced efficacy and safety profile."

Journal • Gastrointestinal Disorder • Lung Cancer • Oncology • Solid Tumor • BAX • BCL2 • TGFB1

Comparison of Jinzhen oral liquid and ambroxol hydrochloride and clenbuterol hydrochloride oral solution in the treatment of acute bronchitis in children: A multicenter, non-inferiority, prospective, randomized controlled trial. (PubMed, Acta Pharm Sin B) - "Combined with clinical trials, the mechanism of JZOL against AB was uncovered by network target analysis, it was found that the pathways in TRP channels like IL-1β/IL1R/TRPV1/TRPA1, NGF/TrkA/TRPV1/TRPA1, and PGE2/EP/PKA/TRPV1/TRPA1 might play important roles. Animal experiments further confirmed that inflammation and the immune regulatory effect of JZOL in the treatment of AB were of vital importance and TRP channels were the key mechanism of action."

Clinical • Head-to-Head • Journal • Cough • Inflammation • Pulmonary Disease • Respiratory Diseases • IL1B • IL1R1 • TRPA1 • TRPV1

Broaden properties of ambroxol hydrochloride as an antibiofilm compound. (PubMed, Rev Argent Microbiol) - "In summary, ambroxol kills planktonic cells, reduce biofilm biomass as it increases cell death, and affect the expression of efflux pumps. Furthermore, it presents a viable alternative for the treatment of biofilm infection alone or in combination with antibiotic therapy."

Journal • Infectious Disease • Respiratory Diseases

Efficacy of Ambroxol Combined with Loquat Syrup on Bacterial Pneumonia in Mice. (PubMed, J Inflamm Res) - "Furthermore, the combined therapy of ambroxol and loquat syrup achieved better therapeutic effects on P. aeruginosa pneumonia compared to ambroxol and/or loquat syrup alone (p<0.05), as evidenced by significantly reduced P. aeruginosa-induced lung injury, improved lung permeability, decreased inflammatory cell infiltration, and lower expression of inflammatory cytokines. These findings suggest that the combination therapy of ambroxol and loquat syrup presents a safe and feasible new treatment strategy for bacterial pneumonia, offering promising benefits for ameliorating lung tissue damage and inflammation."

Journal • Preclinical • Infectious Disease • Inflammation • Pneumonia • Respiratory Diseases

Unveiling the antibacterial action of ambroxol against Staphylococcus aureus bacteria: in vitro, in vivo, and in silico investigation. (PubMed, BMC Microbiol) - "Finally, the in silico investigations were performed to elucidate the potential of ambroxol on five possible targets of S. aureus. Ambroxol showed good affinities on the five investigated targets in S. aureus, with CrtM being the highest, proposing its probable role in the mechanisms for ambroxol's action on S. aureus."

Journal • Preclinical • Infectious Disease • Oncology • TNFA

Protective Effects of Ambroxol on Aβ and α-Synuclein-Induced Neurotoxicity Through Glucocerebrosidase Activation in HT-22 Hippocampal Neuronal Cells. (PubMed, Int J Mol Sci) - "These findings suggest that by activating GCase and enhancing autophagy, AMBX may help alleviate DLB-associated neurodegeneration. This study underscores the potential of AMBX as a therapeutic agent for DLB and supports further investigation in animal models and clinical trials to validate its efficacy in neurodegenerative disease contexts."

Journal • Alzheimer's Disease • CNS Disorders • Dementia • Lewy Body Disease