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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.”


This is a problem for the companies developing cell- and CRISPR-based treatments for blood or immune-related diseases. While these cutting-edge therapies promise one-time cures, many eligible patients could turn them down because of the toxic side effects and small risk of death associated with the decades-old cancer medicine that is used to “condition” or prepare bone marrow for treatment.

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  • Very interesting piece. One challenge may be getting buy-in to use new preconditioning tech in early stage programs, where generating proof of concept on the treatment platforms themselves is the priority. I’ve seen some reluctance to play around too much with preconditioning before there is proof of efficacy. The problem is then that approval is generated on the basis of the existing protocol, so undoubtedly changing the status quo will take some time. Good to see bluebird experimenting with it though.

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