FDA Expedites Review of Gene Therapy, Targets Unscrupulous Stem-Cell Clinics

The U.S. Food and Drug Administration (FDA) issued new guidelines to speed the introduction of treatments involving human cells and tissues, including gene therapy.

“These concepts are no longer the stuff of science fiction,” said FDA Commissioner Scott Gottlieb, in a statement, “but rather, real-life science where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; where new genes can be introduced into the body to combat disease; and where adult stem cells can generate replacements for cells that are lost to injury or illness.”

Gene and cell therapies that demonstrate the potential to treat unmet medical needs and serious illnesses may be eligible for expedited review. The FDA has approved only two products that qualify as gene therapy.

The guidelines also detail steps to rein in the hundreds of stem-cell clinics that treat ailments by liposuctioning belly fat from patients and processing it to extract so-called stem cells, which are then injected back into the patients. Practitioners who perform these procedures have argued that they do not come under the FDA’s jurisdiction. But the guidelines state that if the fat tissue is processed specifically to isolate stem cells, then the procedures must meet FDA safety requirements.

At the press briefing announcing the guidelines, Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research, insisted that practitioners would not get “a free pass to do anything they want.” He added that the FDA “will not turn a blind eye to a manufacturer of stem cells for injection into the knee who might be making contaminated cells that also put people at risk.”

Dr. Marks said the FDA’s goal was to make clear to regenerative medicine developers that they will be held to the same standards as other drug and device makers. “Our goal here is not to flood the market with products. It’s to get products that are safe and effective,” he said.

Source: The New York Times, November 16, 2017.

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