The cures of the future still rely on a toxic drug from the '50s
The advent of gene therapy brings the promise of a one-time treatment for serious diseases, but clearing the way for curative treatment requires a decades-old chemotherapy whose side effects can ward off potential patients.
As STAT’s Adam Feuerstein reports, scientists use busulfan, a chemotherapy first developed in the 1950s, to eliminate disease-causing cells before administering gene therapy. It’s a necessary step, but one that comes with risks, including organ damage, severe infections, and, in rare cases, death.
That’s why a host of researchers are investigating ways to replicate busulfan’s much-needed benefits while leaving out the toxicity. And there’s some promising data, albeit from animal studies, suggesting they might be on the right track.
Read more.
As STAT’s Adam Feuerstein reports, scientists use busulfan, a chemotherapy first developed in the 1950s, to eliminate disease-causing cells before administering gene therapy. It’s a necessary step, but one that comes with risks, including organ damage, severe infections, and, in rare cases, death.
That’s why a host of researchers are investigating ways to replicate busulfan’s much-needed benefits while leaving out the toxicity. And there’s some promising data, albeit from animal studies, suggesting they might be on the right track.
Read more.
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