Forbes July 11, 2024
Juergen Eckhardt

In April 2012, a seven-year-old with acute lymphoblastic leukemia named Emily Whitehead became the first pediatric patient ever to receive an experimental new blood cancer treatment.

For many years, the mainstream approach for treating blood cancers has been to use very high doses of chemotherapy to wipe out the patient’s entire blood cell production systems along with the cancerous cells. Then, a bone marrow transplant from a matched donor is required to restore the patient’s ability to produce blood cells. All in all, the process is grueling and carries many side effects.

In Whitehead’s case, her disease had become resistant to chemotherapy, so she was not eligible for a bone marrow transplant. Given just weeks to live, physicians decided to...

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