For Immediate Release: Saturday, October 24, 2026, 9:45 a.m. U.S. Eastern Time
Media Contact: Evelyn Mantegani, +1 240-833-2014, [email protected]
Presented By: Nikita Marcou, BA, Johns Hopkins University School of Medicine
Rockville, Md. – Acute myeloid leukemia (AML), the most common type of leukemia, has the lowest survival rate. A donor stem cell transplant is currently the only potentially curative treatment, but nearly half of recipients relapse. Researchers have developed a logic-gated CAR T-cell therapy that could offer a more precise treatment for patients whose AML returns after transplant.
CAR T-cell therapies are designed to recognize and kill cells carrying a specific marker. Targeting CD33, a marker found on AML cells, can also destroy healthy blood-forming cells with the same marker, potentially causing serious side effects. The researchers sought to distinguish between a patient’s leukemia cells and healthy cells produced by the transplant donor.
Their experimental therapy combines two signals. The first directs the engineered T cells to attack CD33-positive cells. The second acts as an inhibitory switch when the T cells encounter an HLA marker specific to the donor. As a result, the therapy attacks leukemia cells that carry CD33 but lack the donor marker, while sparing healthy donor-derived cells that carry both.
In laboratory experiments, the logic-gated T cells completely eliminated AML cells lacking the donor-specific marker while preserving cells that represented healthy donor tissue. Researchers then tested the approach in mouse models of human AML, where the engineered cells again eradicated the targeted leukemia cells without attacking cells carrying the protective donor marker.
The findings suggest that genetic differences between transplant recipients and half-matched donors could be used to make CAR T-cell therapy more selective. The approach remains preclinical and has not been tested in humans, but it could eventually provide a new treatment option for AML patients who relapse after transplant.
About the American Society of Human Genetics (ASHG)
The American Society of Human Genetics (ASHG) is the leading global organization advancing the science of human genetics and genomics. Representing nearly 8,000 researchers, clinicians, genetic counselors, educators, and other professionals, ASHG drives progress in understanding the human genome and its role in health and disease. The Society fosters innovation and collaboration through its flagship events and publications—including the ASHG Annual Meeting and its peer-reviewed journals The American Journal of Human Genetics and Human Genetics and Genomics Advances. ASHG also champions science advocacy, supports the next generation of genetics professionals, and promotes responsible policies and public engagement in genetics. Learn more at www.ashg.org.
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