David Fajgenbaum: Untold story of a drug initially approved for Castleman Disease that saved a young girl’s life

David Fajgenbaum shared on X:

“Did you know that a repurposed drug saved the most promising treatment in all of cancer research? Here’s the untold story of a drug initially approved for Castleman Disease that saved CAR T-cell therapy and a young girl’s life.

A young girl named Emily Whitehead was the first pediatric patient to ever receive cancer cellular immunotherapy, a groundbreaking treatment where your own immune cells are reprogrammed to kill cancer.

Shortly after treatment, Emily ended up in the ICU with all of her organs shutting down. She was literally dying from the treatment meant to save her.

Desperate to save her, the doctors at Penn, including Dr. Carl June, tried a drug called tocilizumab, originally developed for Castleman disease decades earlier by Dr. Kazu Yoshizaki.

Miraculously, it worked. Tocilizumab saved Emily’s life, and in doing so, it saved the entire cancer immunotherapy program.

Had Emily tragically passed away from the treatment, the whole CAR T-cell therapy program would likely have been shelved. Instead, this repurposed drug has helped save tens of thousands of lives worldwide and counting.”

Source: David Fajgenbaum/X