CRISPR Stem Cell Transplant Shows Promise in Treating Acute Myeloid Leukemia
A recent Phase I/II clinical trial led by researchers at Washington University School of Medicine in St. Louis has demonstrated promising results in treating acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) using a CRISPR-modified stem cell transplant. The study involved 30 adult patients at high risk of relapse, who received a stem cell transplant with CD33 removed from donor cells using CRISPR technology. This approach aims to prevent cancer recurrence by avoiding the toxicity associated with targeting CD33, a protein present on both cancerous and healthy myeloid cells. The trial also included a maintenance therapy with gemtuzumab ozogamicin, an FDA-approved drug for CD33-positive AML, which helps prevent relapse but carries risks of liver toxicity and blood cell damage. 
