Experts Laud FDA’s Stepped-Up Stem Cell Efforts

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By Greg Langlois

The FDA’s new plans to crack down on “unscrupulous” stem cell clinics but also speed up new therapy development is welcome and necessary, experts said.

Food and Drug Administration Commissioner Scott Gottlieb announced Aug. 28 his agency will launch a working group to go after service providers hawking unapproved, possibly dangerous stem cell therapies. At the same time, the agency will issue new policies to more clearly define when emerging regenerative medicine products fall within the FDA’s purview and speed up approvals.

Both actions are badly needed, regenerative medicine law and policy veterans told Bloomberg BNA. Emerging cell therapies offer hope for effective treatment of a range of diseases and conditions, such as cancer, Parkinson’s disease, diabetes, and heart disease. Policing abuses and streamlining new product development will help achieve this potential, they said.

Stemming Abuses

The FDA trumpeted its aggressive enforcement efforts on the same day Gottlieb announced the agency would “pursue unscrupulous clinics through whatever legally enforceable means are necessary to protect the public health.” The agency said it issued a warning letter to a Florida clinic for using unapproved stem cell products and had a smallpox vaccine seized from California clinics.

Going after purveyors of unproven stem cell products “was a long time coming,” said Sean Morrison, former president of the International Society for Stem Cell Research and now its policy committee chairman. With lax enforcement, more and more clinics in the U.S. “started pushing the envelope,” making implausible claims about what conditions their products and therapies could cure, he said. And companies marketing such products benefit financially from avoiding the clinical trial process, he noted.

“Hopefully FDA will show an enduring commitment to enforcement because it will do good things for American patients to get this snake oil off the market,” said Morrison, who is director of the Children’s Medical Center Research Institute at the University of Texas Southwestern Medical Center.

Removing Ambiguity

Courts have upheld the FDA’s assertion of authority over modified stem cell products, even for autologous treatments (in which a patient’s own cells are used), Alta Charo, a professor of law and bioethics at the University of Wisconsin, said in an email. The FDA’s more assertive stance promises to “clarify just what kind and level of manipulation triggers that authority,” she said.

With clear lines drawn between stem cell products subject to FDA regulation and the practice of medicine, which is not, “so-called clinics” will be less able to take advantage of gray areas to skirt safety and efficacy regulation, Charo said.

"[C]larity lays the foundation for the next step, which is shutting down dangerous practices, followed (I hope) by shutting down totally unproven practices,” she said.

Speeding Development

Although protecting the public from unsafe stem cell products and therapies is necessary, it’s also important for the FDA not to over-regulate and to allow promising stem cell therapies to get approved, said Marc Scheineson, a partner with Alston & Bird LLP and head of its food, drug and device practice. In the past, the FDA has been very conservative in determining when stem cells are so modified they need to be treated as products subject to its oversight, said Scheineson, a former FDA associate commissioner for legislative affairs.

“It’s good to see an activist new commissioner with a lot of energy taking steps to expand investments and availability of cell therapy … if they draw the right line,” he said.

Although some clinics clearly have stepped over the line, he said, stem cell therapy thus far has largely been safe.

“There haven’t been a lot of reports of adverse events,” said Scheineson, who helped draft provisions for streamlined stem cell product approvals in the 21st Century Cures Act of 2016, which is designed to speed development and approval of innovative medical therapies. “While cell therapy has not been as effective as other treatments, there haven’t appeared to be a lot of safety issues,” he said.

Many advocates, he noted, say patients should have the right to use cells taken from their own bodies in treatment and that most of the danger stems from contamination.

Ultimately, the FDA’s Gottlieb is trying to strike a difficult balance between allowing innovation and protecting the public, Morrison said.

“FDA is damned if they do, damned if they don’t,” he said. “There’s pressure on them to reduce regulation … but when something comes out that proves to be unsafe, it gets criticized for not regulating it efficiently.”

To contact the reporter on this story: Greg Langlois in Washington at glanglois@bna.com

To contact the editor responsible for this story: Randy Kubetin at RKubetin@bna.com

For More Information

FDA Commissioner Gottlieb's statement is at http://src.bna.com/scO.

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