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Could gene editing be used to treat cancer? Well, researchers say they could be a step closer to realizing that goal. NPR health correspondent Rob Stein has the details.
ROB STEIN, BYLINE: The new approach combines two of the hottest discoveries in biomedical science, the powerful gene-editing technique called CRISPR and immunotherapy - using patients' own immune system cells to fight their cancer.
EDWARD STADTMAUER: The last decade has been really a revolution in the immunotherapy for cancer.
STEIN: Edward Stadtmauer is at the University of Pennsylvania.
STADTMAUER: We've really learned how to harness the immune system to treat cancer to create these miraculous cells that are infused into patients as living drugs to treat cancer.
STEIN: But those genetically engineered living drugs don't work for lots of cancer patients or only work for a little while. That's where CRISPR gene editing comes in.
STADTMAUER: We're using this CRISPR technology to make these cells work even better and potentially attack the cancer better.
STEIN: Here's how it works. Doctors take key immune system cells out of patients' bodies. Next, scientists use CRISPR to edit genes in the cells. Then doctors infuse the modified cells back into patients' bodies.
STADTMAUER: We want to see if we can allow the cells to have sort of super powers to either proliferate even better, to stay in the patients better as living drugs and to potentially attack even more directly the tumor.
STEIN: Stadtmauer's team infused about 100 million of these edited cells into three patients, two fighting multiple myeloma and one with sarcoma. And so far, it looks like the cells survive, function and are safe.
STADTMAUER: We demonstrated safety. We demonstrated feasibility. And that's really the most important finding of this study, given that this is the first of its kind in patients with cancer.
STEIN: Now, Stadtmauer cautions that it's far too soon to know whether the approach will actually help patients live longer. This study was designed only to see if it's feasible and safe. But he's optimistic.
STADTMAUER: This treatment is not ready for prime time. But in many ways, this is the beginning of the next generation of engineering of cells to help many different diseases and many different tumors.
STEIN: Other researchers agree.
JENNIFER DOUDNA: I'm just so excited about this.
STEIN: Jennifer Doudna helped develop CRISPR at the University of California, Berkeley.
DOUDNA: It's an important step on the path towards using CRISPR genome editing in patients and shows the potential of this technology to be a safe and effective therapy.
STEIN: But others are more cautious. Michel Sadelain at the Memorial Sloan Kettering Cancer Center is doing similar research.
MICHEL SADELAIN: I don't think there's much to conclude yet - probably want more patients and a longer follow-up to really make the call that the use of CRISPR is safe. But the data are certainly encouraging.
STEIN: Several studies are already underway, or starting soon, testing CRISPR for genetic blood disorders, an inherited form of blindness and other forms of cancer. And Stadtmauer's group plans to treat more cancer patients soon.
Rob Stein, NPR News.
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