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 CDCN and CSTL 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
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.