CRISPR cancer treatment clears 1st safety tests

Several months after the first U.S. cancer patients were treated with CRISPR gene-editing technology, scientists shared Nov. 6 that initial results demonstrate the safety and feasibility of the treatment, NPR reports.

In the study, led by researchers from the Philadelphia-based University of Pennsylvania's Abramson Cancer Center, three patients received infusions of about 100 million of their own immune system cells that had been modified with CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats. The treatments occurred in January, April and August; two of the patients have multiple myeloma and one has sarcoma.

"This treatment is not ready for prime time. … But it is definitely very promising," lead investigator Edward Stadtmauer, MD, a professor in oncology at the University of Pennsylvania, told NPR. "We hope this is the beginning of the next generation of engineering cells to help many different diseases and many different tumors."

The trial, which will be presented at a meeting of the American Society of Hematology in Orlando, Fla., in December, was not meant to prove the effectiveness of CRISPR-based treatments, but their safety and feasibility. The researchers plan to continue recruiting patients for the study, with a goal of modifying the genes of a total of 18 cancer patients and monitoring their progress for several years.

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