Three patients with advanced cancers, treated at the University of Pennsylvania in the first U.S. clinical study of gene editing, have had no serious side effects so far — a reassuring sign that the revolutionary technology can safely be used in humans, according to results released Wednesday.
The inaugural U.S. experiment using CRISPR-edited cells in humans is such a scientific and ethical watershed that Penn spent more than two years getting federal and other approvals. At least three other CRISPR trials — in sickle cell disease, non-Hodgkin’s lymphoma, and an inherited form of blindness — have since gotten go-aheads.
Experimental drugs must show safety in a pilot clinical trial. Then, more patients are treated to look for evidence of effectiveness.
Two of the patients treated at Penn have an incurable bone marrow cancer called multiple myeloma, while the third has a soft tissue cancer called sarcoma. Their cancers overproduce a protein called NY-ESO-1.
In previous research involving 25 patients, Penn researchers showed they could genetically engineer each patient’s own disease-fighting T cells to recognize multiple myeloma cells overloaded with that protein.