TY - JOUR AU - Stern, Robert M. AU - Berliner, Nancy T1 - Targeting the mTOR pathway in idiopathic multicentric Castleman disease PY - 2019/10/01/ AB - 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. JF - The Journal of Clinical Investigation JA - J Clin Invest SN - 0021-9738 DO - 10.1172/JCI131332 VL - 129 IS - 10 UR - https://doi.org/10.1172/JCI131332 SP - 4086 EP - 4088 PB - The American Society for Clinical Investigation ER -