A new study shows that CRISPR gene editing may help protect patients with Acute Myeloid Leukemia from the harmful side effects of intensive cancer treatment.
In the VBP101 trial, researchers used CRISPR-Cas9 to remove the CD33 protein from donor stem cells before transplant. This created a new blood and immune system that could resist damage from the leukemia drug Gemtuzumab Ozogamicin, which normally attacks both cancer cells and healthy donor cells.
All 30 patients in the study achieved successful blood cell recovery after transplant. Nineteen patients later received maintenance therapy with the drug without developing the severe blood count problems commonly seen after treatment.
Researchers reported high gene-editing accuracy, with edited cells remaining nearly 100% CD33-negative during therapy. The findings suggest CRISPR-edited stem cells could make future treatments, including CD33-targeted CAR T-cell therapy, safer and more effective for high-risk AML patients.