First U.S. Cancer Patients Treated with CRISPR Gene-Editing Therapy

Patients have been treated in the U.S. using CRISPR gene-editing for the first time, with a University of Pennsylvania study utilizing the technique in treating cancer. Scientists have been hopeful that CRISPR could be used to treat many diseases, and these Penn researchers are optimistic about the trial.

Two patients were treated with the powerful gene-editing tool, one with sarcoma and one with multiple myeloma. Both of these patients had underwent standard treatment but were unable to eradicate their disease, and are instead partaking in the first case of CRISPR use in humans in the U.S. With recent news of a Chinese scientist using CRISPR to make genetically modified babies there is much controversy around the technology, however the science community is eager to see how the technique can help those with disease.

“2019 is the year when the training wheels come off and the world gets to see what CRISPR can really do for the world in the most positive sense,” said Fyodor Urnov, a gene-editing scientist at the Altius Institute for Biomedical Sciences in Seattle and the University of California, Berkeley.

To this point very few studies have attempted to use CRISPR to treat disease, with a majority of them taking place in China. Like this recent Penn trial, these studies aimed to use the technique in treating cancer.

This specific trial taking place in Philadelphia involves the removal of immune system cells from patients, genetic modification of them, then re-implantation. The goal is that these CRISPR-edited cells will be able to target and destroy the cancer cells.

Though only two patients have received this treatment, the study was approved to eventually treat 18 patients. No information has been released regarding how well the treatment is working.

“Findings from this research study will be shared at an appropriate time via medical meeting presentation or peer-reviewed publication,” wrote a Penn spokesperson in an email to NPR.

READ MORE: Gene Therapy Technology Advancing Rapidly

Outside of this trial, scientists elsewhere in the U.S., Europe, and Canada are planning a handful of studies analyze CRISPR as a treatment for various diseases. Two of these trials are designed to treat genetic blood disorders, one being sickle cell disease and the other a similar condition called beta thalassemia. These trials are sponsored by CRISPR Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston.

The beta thalassemia patient was actually treated already in Germany using a CRISPR gene-editing technique known as CTX001. Other patients may soon receive similar CRISPR therapy at that hospital and another clinic in Germany, followed by other patients at centers in Toronto and London.

Additionally, the first sickle-cell patients in the U.S. may soon receive CRISPR gene therapy in Nashville, San Antonio, and New York. Another study, sponsored by Editas Medicine in Cambridge, Massachusetts, plans to use the technique to treat a heritable form of blindness known as Leber congenital amaurosis.

Sources: NPR, CRISPR Therapeutics