STAT Plus: Cutting-edge therapies promise one-time cures, but not without risk. Can biotech make them safer?
When Dr. Akshay Sharma sits down with his sickle cell disease patients to discuss potentially curative stem cell transplants or gene therapy, they’re shocked to learn that preparing for treatment requires injections of toxic and potentially lethal chemotherapy.
“They ask me, ‘I don’t have cancer, so why do I need to take chemotherapy?’” said Sharma, a bone marrow transplant expert at St. Jude Children’s Research Hospital. “These are mostly young people, teenagers, who can’t fathom even a small risk of dying during treatment, so it’s quite the reckoning for them. And it drives many of them away.”
