So far, a procedure that turbocharges the immune system to attack tumors seems safe, but it’s too soon to tell whether it helps patients.
Doctors have for the first time in the United States tested a powerful gene-editing technique in people with cancer.
The test, meant to assess only safety, was a step toward the ultimate goal of editing genes to help a patient’s own immune system to attack cancer. The editing was done by the DNA-snipping tool Crispr.
The procedure was feasible and safe, early results indicate, but whether it is fighting the disease is unclear. Only three patients have been treated so far, and the longest follow-up is nine months. All three patients are in their 60s, with very advanced cancers that had progressed despite standard treatments like surgery, radiation and chemotherapy.
“The good news is that all of them are alive,” said Dr. Edward A. Stadtmauer, the section chief of hematologic malignancies at the University of Pennsylvania Abramson Cancer Center. He added, “The best response we’ve seen so far is stabilization of their disease.”
The research was paid for by the Parker Institute for Cancer Immunotherapy and the company Tmunity. Some members of the research team, but not Dr. Stadtmauer, are equity holders in Tmunity, as is the University of Pennsylvania.
The findings have not yet been published in a medical journal, but will be presented next month at a meeting of the American Society of Hematology, and posted online on Wednesday.
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“This is really just the beginning of a whole next generation of treatments,” Dr. Stadtmauer said in an interview.
Researchers hope to use Crispr to treat a number of genetic diseases, and it has already been used experimentally to treat sickle-cell anemia.
Doctors in China say they have already started using Crispr to treat various types of cancer.
Two patients in Dr. Stadtmauer’s study have a blood cancer, multiple myeloma, and the third has a sarcoma, a rare cancer of connective tissue.