Drug Repurposing

Drug Repurposing

The Castleman Disease Collaborative Network (CDCN) and Center For Cytokine Storm Treatment & Laboratory (CSTL), under the leadership of Dr. David Fajgenbaum, are committed to the discovery of new uses for existing drugs to save patients’ lives. There are ~1,500 drugs currently FDA approved to treat ~2,500 of the ~10,000 human diseases. No treatments exist for over 7,000 diseases, many of which are rare and deadly.

The CSTL and CDCN have pioneered the following approach to drug repurposing for idiopathic multicentric Castleman disease (iMCD) and other rare diseases: 1) perform in-depth studies of high-quality biospecimens to identify cell types, signaling pathways, and genes/proteins that may be involved in a given disease, 2) validate the discovery using an orthogonal method, 3) utilize bioinformatic tools and drug databases to identify drugs already FDA-approved for another condition that modulate the target, 4) administer the drug off-label (giving a drug for a use other than what it is approved for) or through a clinical trial, and 5) systematically track the impact of the drug on the given disease.

This approach has led to the discovery and clinical development of two drugs for the treatment of iMCD. Dr. Fajgenbaum, also a patient with iMCD, began testing one of these drugs on himself in 2014 and is in his longest remission ever (more here). The CSTL opened up a clinical trial of this drug last fall. For more about this approach, check out this video.


Given the global crisis occurring due to COVID-19 and the urgent need to identify effective therapies, the heightened risk of COVID-19 to Castleman disease patients and other patients with immunological disorders and immunocompromise, and the CSTL’s expertise regarding drug repurposing, CSTL members and CDCN volunteers have begun directing efforts toward fighting COVID-19 (while maintaining focus on our other high-priority studies).

To begin, the CSTL is capturing data on all drugs being used off-label to treat COVID-19 patients, structuring the data on off-label drugs and reported response for future analyses, and making the data available online so that researchers and physicians can access these data quickly and easily.

During phase I, the team extracted data from published papers on PubMed and Biorxiv.

During phase II, we are partnering with CURE ID to encourage physicians to report de-identified information on their off-label drug uses in real time. In parallel, we are re-analyzing and identifying trends across research studies to create a COVID-19 Immune Map to dissect the immune response to COVID-19 that could guide therapeutic decisions.

You can visit the following link to access the CORONA database: COVID19 Drug Repurposing
You can visit the following link to access a resource for vaccines and drugs in development for COVID-19: Milken Institute Tracker

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