US sets rules on stem cell therapies

The U.S. Food and Drug Administration unveiled long-anticipated guidelines about stem-cell therapies Thursday that are meant to clear up confusion about which treatments are likely to be both appropriate and safe, and which pose the most risks and fewest rewards for patients.

The carefully worded guidelines underscore the narrow path regulators walk between allowing, and even hastening, approval of potentially life-changing cellular therapies and preventing providers from selling unproven and possibly dangerous cell products directly to patients.

“The promise of this technology is why the FDA is so committed to encouraging and supporting innovation in this field,” agency Commissioner Scott Gottlieb said in a statement released with the guidelines. “But the rapid growth and promise of this field has increasingly sowed the ground for the entry of some unscrupulous actors.”

In the same announcement, FDA officials said they would step up enforcement of stem-cell clinics that offer dubious therapies, some costing thousands of dollars, to patients with chronic or life-threatening conditions. These clinics have flourished in the Bay Area and much of the rest of the U.S. in recent years, but federal regulators largely have left them alone.

The FDA has been under pressure for years from scientists and consumer providers to clarify its guidelines around regenerative medicine, a field that involves the use of cell therapies to repair and replace parts of the body damaged by illness or injury.

Scientists have argued that traditional avenues of regulatory approval for drug therapies shouldn’t apply to cell-based treatments, and that the old-style process was hindering promising research by making it too expensive and time-consuming.

Meanwhile, stem cell experts had decried the expanding web of consumer clinics that promised to treat a host of conditions, from orthopedic injuries to brain or immune illnesses such as Alzheimer’s disease and multiple sclerosis.

The issue gained urgency in March when reports were made public of three women blinded by stem-cell treatments at a Florida clinic. In September, the FDA published a scathing letter the agency sent to the clinic, stating that providers there were marketing stem-cell products without proper federal approval and that some of its practices could put patients at risk. The FDA has not taken further steps to intervene at the clinic.

In the Thursday announcement, FDA officials said they will prioritize enforcement at clinics that are offering the most problematic stem-cell therapies. Specifically, they’re looking at clinics that sell therapies that are given by intravenous infusion or injected directly into the central nervous system or the eye. Clinics that inject therapies subcutaneously — just beneath the skin — or into a joint will likely be less closely monitored, in large part because such methods are considered relatively safe.

“We’re not going to turn a blind eye to a manufacturer of stem cells for injections into the knee who might be making contaminated cells that put people at risk,” said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, in a telephone briefing with reporters.

“I don’t think it’s fair to say we’ll give anyone a free pass to do anything they want,” Marks said. But, he added, there are “literally hundreds” of clinics marketing directly to consumers, “and we simply don’t have the bandwidth to go after them all at once.”

“In the scheme of things, we’ll have tiers to prioritize,” he said.

The guidelines strike a reasonable balance between easing burdensome regulations that were impeding science and giving the FDA clearer authority over problematic clinics, said Dr. Maria Millan, president of the California Institute for Regenerative Medicine, the state’s stem cell funding agency.

She said she hoped that the FDA’s action would help patients make better informed decisions about their own health care when it comes to cell therapies.

“One of the things we battle is trying to help people sort through what is valid and what is not,” Millan said. “This guidance will help.”

There were few surprises in the documents, said Dr. Deepak Srivastava, director of the Roddenberry Stem Cell Center at the Gladstone Institutes in San Francisco.

“But I’m glad to see them and bring clarity to some of this field,” he said.

In addition to documents addressing concerns about regulatory enforcement, Srivastava said, he was excited to see formal guidelines about how to speed up the movement of experimental stem-cell therapies into FDA-approved treatments.

“The FDA is beginning to recognize that regenerative medicine therapies are going to be unique,” he said.