Contribute Try STAT+ Today

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.”

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!


What is it?

STAT+ is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

What's included?

  • Daily reporting and analysis
  • The most comprehensive industry coverage from a powerhouse team of reporters
  • Subscriber-only newsletters
  • Daily newsletters to brief you on the most important industry news of the day
  • STAT+ Conversations
  • Weekly opportunities to engage with our reporters and leading industry experts in live video conversations
  • Exclusive industry events
  • Premium access to subscriber-only networking events around the country
  • The best reporters in the industry
  • The most trusted and well-connected newsroom in the health care industry
  • And much more
  • Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.
  • 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.

Comments are closed.