Major study published uncovering mechanisms involved in Castleman disease

A study funded by the CDCN and published in a top journal, JCI Insight, identifies a potential cause for the increased mTOR activation previously discovered and reveals a new drug that may be effective in iMCD

07/13/2020- A study supported by the CDCN and published in a top journal, JCI Insight, reveals potential new insights into the pathogenesis of the TAFRO-subtype of idiopathic multicentric Castleman disease (iMCD-TAFRO). iMCD-TAFRO is the most severe subtype of iMCD and involves episodes of low platelets, fluid accumulation, fever, kidney dysfunction, and enlarged liver and kidneys. This work was led by first author Ruth-Anne Langan Pai, an Immunology PhD student who has dedicated the last four years to studying iMCD, and senior author Dr. David Fajgenbaum, co-Founder of the CDCN and an iMCD patient.

The study involved in-depth profiling of blood samples contributed by 15 iMCD-TAFRO patients in flare and remission. It builds on previous research identifying mTOR activation as a possible novel therapeutic target for iMCD. This study identifies a potential mechanism underlying the mTOR activation in iMCD patients and is another positive sign that blocking mTOR with sirolimus could help iMCD patients that don’t respond to siltuximab! This data gives us a better understanding of mTOR activation in iMCD and we look forward to the results of the clinical trial of sirolimus on iMCD patients at UPenn.

PENN Press Release

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