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Targeting the mTOR pathway in idiopathic multicentric Castleman disease
Robert M. Stern, Nancy Berliner
Robert M. Stern, Nancy Berliner
Published September 16, 2019
Citation Information: J Clin Invest. 2019;129(10):4086-4088. https://doi.org/10.1172/JCI131332.
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Commentary

Targeting the mTOR pathway in idiopathic multicentric Castleman disease

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Abstract

Idiopathic multicentric Castleman disease (iMCD) is a rare hematologic illness of systemic inflammation and organ dysfunction, with unknown etiology. Although therapies targeting IL-6 have been proven effective, a subset of patients with iMCD are resistant to this approach. In this issue of the JCI, Fajgenbaum et al. performed an in-depth analysis of serum inflammatory markers in three iMCD patients refractory to IL-6 blockade, and identified activation of the mTOR pathway associated with symptom flares. Treatment with sirolimus, an mTOR inhibitor, induced remission in all three patients. This study models a precision medicine approach to discovering therapies for rare diseases.

Authors

Robert M. Stern, Nancy Berliner

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