05/7/2020 A study funded by the CDCN and published in the top hematology journal, Blood, builds on previous research identifying mTOR activation as a possible novel therapeutic target for iMCD. The previous research describes David Fajgenbaum and two other iMCD patients’ response to sirolimus, a drug that blocks mTOR. This study further examines mTOR activation in iMCD patients.
The findings show that iMCD samples have increased mTOR not just compared to normal, but also compared to Hodgkins lymphoma and lupus, which suggests that mTOR activation is not just a result or symptom. In fact, the levels of mTOR activation in iMCD were comparable to those in ALPS, a disease that is driven by mTOR hyperactivation and responds to sirolimus treatment.
These are all positive signs 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.