Treating IMCD: From Precision Medicine to Clinical Trial

(calming music) – Here I was sitting in my lab at Penn. My head was bald from my most
recent round of chemotherapy. I had just nearly died for the fifth time battling Idiopathic
Multicentric Castleman Disease in the last three years. Idiopathic Multicentric
Castleman Disease, or iMCD, the immune system becomes hyperactivated, attacks and shuts down key organs like the liver, kidneys, heart and lungs. No one knows why and no one knew how to treat relapsing patients like me. I had dedicated what
little time I had left to taking on this disease. Then, three years of
research, data analysis, and experiments came
together for me that day. Connecting the dots
between multiple data sets indicated to me that the mTOR pathway, a key communication line
in the immune system, was turned on. Could targeting it and turning it off shut down and stop this
relentless and deadly disease? I drug already existed that targeted that precise communication line. Based on my research as
a Physician Scientist, I began taking the drug
as the first patient ever with my disease. I have been in remission for
the last three and a half years and I’ve repeated the same experiments that lead me to try this drug on myself in other patient samples. The results suggest
that this drug may work for other patients too. The support from the Penn
Center for Precision Medicine, we’re translating these
findings into a clinical trial, to investigate whether this
drug that saved my life could save other patients’ lives too. I’m Dr. David Fajgenbaum, and this is Precision Medicine with Penn. (calming music)

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