Gene therapy combats acute leukemia in small study

A tiny new study involving 5 patients with an acute and typically terminal case of relapsed leukemia has provided a few high-profile testimonials for a new and promising gene therapy strategy. 

Reporting in Science Translational Medicine, the investigators say they were able to take T cells from late-stage leukemia patients and modify them with genetic material, reengineering the immune cells to track down and kill B cells. A disabled viral vector was used to inject the genetic material to recognize the CD19 protein expressed on the surface of B cells.

Anywhere from 5 months to two years after the gene therapy, three of the 5 patients in the study are still alive. Two of the patients died--one from a blood clot--while the other relapsed. One of the sickest patients was free of the leukemia 8 days after he was treated, surprising everyone involved.

This isn't the first time this approach has worked in patients. But scientists still have only tested the technique in a handful of patients, leaving researchers to caution patients that plenty of development work remains to be done on the experimental strategy. But they're clearly excited by what they've seen so far.

"The results here are profound," says oncologist Renier J. Brentjens, according to The Wall Street Journal. "It's a very promising step forward."

"We're creating living drugs," Dr. Michel Sadelain of Memorial Sloan-Kettering Cancer Center tells The New York Times. "It's an exciting story that's just beginning." 

- here's the story from the Wall Street Journal
- get the report from the New York Times
read the Science Translational Medicine abstract