Commentary 10.1172/JCI131332

Targeting the mTOR pathway in idiopathic multicentric Castleman disease

Robert M. Stern1,2 and Nancy Berliner1,2

1Hematology Division, Brigham and Women’s Hospital, Boston, Massachusetts, USA.

2Harvard Medical School, Boston, Massachusetts, USA.

Address correspondence to: Nancy Berliner, Brigham and Women’s Hospital, 75 Francis Street, Boston, Massachusetts 02115, USA. Phone: 617.732.5840; Email: nberliner@bwh.harvard.edu.

Find articles by Stern, R. in: JCI | PubMed | Google Scholar |

1Hematology Division, Brigham and Women’s Hospital, Boston, Massachusetts, USA.

2Harvard Medical School, Boston, Massachusetts, USA.

Address correspondence to: Nancy Berliner, Brigham and Women’s Hospital, 75 Francis Street, Boston, Massachusetts 02115, USA. Phone: 617.732.5840; Email: nberliner@bwh.harvard.edu.

Find articles by Berliner, N. in: JCI | PubMed | Google Scholar |

First published September 16, 2019 - More info

Published in Volume 129, Issue 10 on October 1, 2019
J Clin Invest. 2019;129(10):4086–4088. https://doi.org/10.1172/JCI131332.
© 2019 American Society for Clinical Investigation
First published September 16, 2019 - Version history

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.

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