A High Content Screen for Mucin-1-Reducing Compounds Identifies Fostamatinib as a Candidate for Rapid Repurposing for Acute Lung Injury during the COVID-19 pandemic

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Abstract

Drug repurposing is the only method capable of delivering treatments on the shortened time-scale required for patients afflicted with lung disease arising from SARS-CoV-2 infection. Mucin-1 (MUC1), a membrane-bound molecule expressed on the apical surfaces of most mucosal epithelial cells, is a biochemical marker whose elevated levels predict the development of acute lung injury (ALI) and respiratory distress syndrome (ARDS), and correlate with poor clinical outcomes. In response to the pandemic spread of SARS-CoV-2, we took advantage of a high content screen of 3,713 compounds at different stages of clinical development to identify FDA-approved compounds that reduce MUC1 protein abundance. Our screen identified Fostamatinib (R788), an inhibitor of spleen tyrosine kinase (SYK) approved for the treatment of chronic immune thrombocytopenia, as a repurposing candidate for the treatment of ALI. In vivo , Fostamatinib reduced MUC1 abundance in lung epithelial cells in a mouse model of ALI. In vitro , SYK inhibition by Fostamatinib promoted MUC1 removal from the cell surface. Our work reveals Fostamatinib as a repurposing drug candidate for ALI and provides the rationale for rapidly standing up clinical trials to test Fostamatinib efficacy in patients with COVID-19 lung injury.

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  1. Excerpt

    Two recent preprints indicate that Fostamatinib might help with severe COVID-19 symptoms and point to the importance of detailed examination of immune response to COVID-19 vaccines.

  2. SciScore for 10.1101/2020.06.30.180380: (What is this?)

    Please note, not all rigor criteria are appropriate for all manuscripts.

    Table 1: Rigor

    Institutional Review Board StatementIACUC: Mesenteric Ischemia-Reperfusion (I/R): All animal procedures were performed in accordance with the guidelines and approval of the Institutional Animal Care and Use Committee (IACUC) of the BIDMC.
    RandomizationMice were randomly assigned to sham or I/R groups and were anesthetized by intraperitoneal injection of 72 mg/kg pentobarbital.
    Blindingnot detected.
    Power Analysisnot detected.
    Sex as a biological variableMice: Adult, 7-week-old male C57BL/6J mice were purchased from Jackson Laboratory (Bar Harbor, ME) and maintained in specific pathogen-free conditions at the Beth Israel Deaconess Medical Center (BIDMC) and allowed to acclimate for 1 week before use in experiments.

    Table 2: Resources

    Antibodies
    SentencesResources
    5g/L Blocking Reagent [Roche]), then incubated 90 min at RT with 1:2000, monoclonal mouse anti-MUC1 (214D4) antibody (Millipore) in Roche Blocking solution, followed by four PBS wash cycles.
    anti-MUC1 ( 214D4 )
    suggested: None
    Then the secondary antibody Alexa Fluor® 546 Goat anti-mouse IgG, Thermo Fisher Scientific and Hoechst 33342 stain, Thermo Fisher Scientific, were applied at a 1:1000 dilution in Roche blocking solution and incubated at RT for 45 min, followed by four PBS wash cycles.
    the secondary antibody Alexa Fluor® 546 Goat anti-mouse IgG
    suggested: None
    anti-mouse IgG
    suggested: None
    Experimental Models: Organisms/Strains
    SentencesResources
    Mice: Adult, 7-week-old male C57BL/6J mice were purchased from Jackson Laboratory (Bar Harbor, ME) and maintained in specific pathogen-free conditions at the Beth Israel Deaconess Medical Center (BIDMC) and allowed to acclimate for 1 week before use in experiments.
    C57BL/6J
    suggested: RRID:IMSR_JAX:000664)
    Software and Algorithms
    SentencesResources
    All graphs were visualized using ggplot2.
    ggplot2
    suggested: (ggplot2, RRID:SCR_014601)
    For the MUC1 membrane prevalence, the images acquired during 10-dose screening were analyzed using the Harmony software STAR morphology feature, which calculates the signal distribution across different cell compartments.
    STAR
    suggested: (STAR, RRID:SCR_015899)
    Statistical analysis: Statistical analysis was performed and presented using Graphpad Prism version 7.0 software.
    Graphpad Prism
    suggested: (GraphPad Prism, RRID:SCR_002798)

    Results from OddPub: We did not detect open data. We also did not detect open code. Researchers are encouraged to share open data when possible (see Nature blog).


    Results from LimitationRecognizer: An explicit section about the limitations of the techniques employed in this study was not found. We encourage authors to address study limitations.

    Results from TrialIdentifier: We found the following clinical trial numbers in your paper:

    IdentifierStatusTitle
    NCT02112838CompletedSafety and Efficacy Study of Fostamatinib to Treat Immunoglo…


    Results from Barzooka: We did not find any issues relating to the usage of bar graphs.


    Results from JetFighter: We did not find any issues relating to colormaps.


    Results from rtransparent:
    • Thank you for including a conflict of interest statement. Authors are encouraged to include this statement when submitting to a journal.
    • Thank you for including a funding statement. Authors are encouraged to include this statement when submitting to a journal.
    • No protocol registration statement was detected.

    About SciScore

    SciScore is an automated tool that is designed to assist expert reviewers by finding and presenting formulaic information scattered throughout a paper in a standard, easy to digest format. SciScore checks for the presence and correctness of RRIDs (research resource identifiers), and for rigor criteria such as sex and investigator blinding. For details on the theoretical underpinning of rigor criteria and the tools shown here, including references cited, please follow this link.