Cancer ResearchCRISPR-Edited Stem Cells Shield Healthy Blood Cells While Attacking Leukemia
Researchers at Washington University used CRISPR to remove the protein CD33 from donor stem cells before transplanting them into patients with acute myeloid leukemia and myelodysplastic syndrome. The problem with treating these aggressive blood cancers is that CAR-T therapies targeting cancer cells also destroy healthy blood stem cells carrying the same protein. By editing donor cells to lack CD33, the healthy transplanted cells become invisible to CD33-targeted treatments. In a 30-patient clinical trial across 15 sites in the US and Canada, the edited cells successfully engrafted and appeared to protect healthy blood cells. The results, published in Nature Medicine, suggest this approach could be combined with CD33-targeted CAR-T therapy in future trials, potentially making treatment both safer and more effective for cancers that currently have very limited options after transplant failure.
