08/21/2019 A paper published in the Journal of Clinical Investigation last week describes the treatment of three iMCD patients who previously did not respond to IL-6 blockers like siltuximab, the only FDA-approved drug for iMCD. One of those patients is CDCN’s own David Fajgenbaum, who started treating himself with sirolimus in 2012 after research led him to believe it could help– he was right and has been in remission ever since! Sirolimus blocks mTOR, and these findings suggest that mTOR is another pathway for iMCD in addition to IL-6. The other two patients included in the study exhibited the same clinical subtype of iMCD as Dave (TAFRO syndrome) and have also been in remission since starting sirolimus treatment 19 months ago. You can find the full text of the paper here.
These findings are really exciting because until now, IL-6 was the only known pathway for iMCD that could be targeted with treatment. But only one half to one third of iMCD patients actually respond to IL-6 blockers like siltuximab, and patients who don’t respond have had limited options. Thanks to this research, a clinical trial for sirolimus at UPenn is set to begin enrollment shortly! You can find more information on the trial at NCT03933904.