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News
A clinical trial from Washington University shows that CRISPR-edited donor stem cells — stripped of the protein CD33 — can survive cancer-targeting therapies that would normally wipe out healthy blood cells too. All 30 patients with aggressive blood cancers like AML and MDS achieved successful engraftment, and one patient even hit complete remission after follow-up CAR-T therapy. It's a promising step toward making blood cancer treatment both safer and more effective.
Researchers at Washington University School of Medicine have found a clever workaround to one of blood cancer treatment's biggest challenges. In a 30-patient clinical trial published in Nature Medicine, they used CRISPR to remove the protein CD33 from donor stem cells before transplantation. The result? Healthy transplanted cells became invisible to CD33-targeting therapies — meaning doctors could go after the cancer without accidentally destroying the blood cells patients need to survive.
The strategy is especially relevant for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), two aggressive cancers where CAR-T therapy has historically underperformed. The problem has always been that CD33 appears on both cancer cells and healthy myeloid cells, making precision targeting nearly impossible — until now. In one standout case, a high-risk AML patient received CD33-deleted stem cells, relapsed, was treated with CD33-targeted CAR-T therapy, and entered complete remission for over a year.
By the Numbers
Why it matters: This approach could unlock the full potential of CD33-targeted immunotherapies — like CAR-T and antibody-drug conjugates — for some of the hardest-to-treat blood cancers, without the collateral damage that has long limited their use.
