MIT Technology Review – A breakthrough therapy that uses genetically altered immune cells to treat an often-fatal type of childhood blood cancer passed an important milestone in August when the U.S. Food and Drug Administration approved it. The highly personalized treatment, called CAR-T therapy, uses a patient’s own immune cells and takes about three weeks to manufacture—two factors that lend to its $475,000 price tag.
Similar therapies are in the pipeline, but the cost and time it takes to make each dose could put these life-saving treatments out of reach for patients who desperately need them. To address these issues, academic and commercial labs are already working on a newer approach—one that uses immune cells from a healthy donor instead of the patient. The idea is that these treatments could be manufactured in bulk and be readily available whenever a patient needs them. And one donor sample could hypothetically make a dozen—or even hundreds—of doses.
“It’s a very attractive concept,” says Bruce Levine, a professor of cancer gene therapy at the University of Pennsylvania’s Perelman School of Medicine who helped develop CAR-T cells. “Clearly, there are patients in need from whom we cannot generate adequate cells.”
Dubbed “off-the-shelf” immune cells, these therapies have problems of their own. The FDA this week halted two clinical trials of an off-the-shelf therapy developed by French biotech firm Cellectis after a 78-year-old patient died. The company is still investigating the death, and CEO André Choulika says he isn’t discouraged by the setback. But the tragedy underscores that these therapies aren’t yet ready for prime time, despite their potential benefits over personalized CAR-T therapies.
Read more at MIT Technology Review.