A recent Phase I/II clinical trial has demonstrated that a CRISPR-modified stem cell transplant can significantly improve outcomes for patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). Conducted by researchers at Washington University School of Medicine, the trial involved 30 adult patients who received stem cell transplants with donor cells engineered to remove CD33, a protein that is present on both cancerous and healthy myeloid cells, thereby reducing the risk of toxicity associated with traditional CAR T cell therapies.
The findings, published in Nature Medicine, indicate that engraftment rates and recovery times for blood cell production were comparable to standard stem cell transplants. Furthermore, the average survival rate exceeded 14 months, with patients maintaining stable blood cell counts while undergoing maintenance therapy with gemtuzumab ozogamicin, an FDA-approved treatment for CD33-positive AML.
These results suggest that the combination of CD33-deleted stem cell transplants and targeted immunotherapies could represent a promising new treatment paradigm for aggressive blood cancers, potentially improving patient outcomes while minimizing adverse effects. As Dr. John DiPersio noted, the study paves the way for future therapies that harness the benefits of gene editing and immunotherapy to combat these challenging malignancies.
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