F.D.A. Speeds Review of Gene Therapies, Vowing to Target Rogue Clinics

November 17, 2017
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The Food and Drug Administration on Thursday issued new guidelines to speed the introduction of treatments involving human cells and tissues, including gene therapy. But the agency also said it would crack down on rogue clinics offering dangerous or unproven versions of those treatments.

The therapies aimed at diseases like leukemia are known as regenerative medicine and have quickly grown into a booming industry worldwide.

“These concepts are no longer the stuff of science fiction,” said Scott Gottlieb, the commissioner of the F.D.A., “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 now qualify for expedited review to get the products to market more quickly, the guidelines say. The F.D.A. will still require clinical trials, but it is promising a faster process, as required by Congress under the 2016 21st Century Cures Act.

Even as the field has advanced in recent years, questionable centers have sprung up all over the country, offering treatments for everything from sore knees to heart disease and raising concerns among some experts about hastening the approval process.

The F.D.A. said that those problematic clinics, many of which use products derived from patients’ own fat, are making treatments that must be approved before they can be marketed.

For legitimate developers of the treatments, the guidelines were welcome news.

“This is an incredibly significant development for the gene therapy, regenerative medicine sector,” said Michael J. Werner, a partner with Holland & Knight and co-founder of the Alliance for Regenerative Medicine.

To Mr. Werner, the takeaway is that the F.D.A. has firmly stated that gene therapies can qualify for fast approval on a special pathway, which also carries other benefits for manufacturers. Gene therapies will be eligible for this designation provided that they make long-lasting changes to cells or tissue and provide a sustained therapeutic benefit.

“This should really open the door to a lot more gene therapies coming on the market quickly,” Mr. Werner said.

So far, the drug agency has approved only two products that qualify as gene therapy — Kymriah, from Novartis, and Yescarta, made by Kite Pharma. Both treatments involve genetically altering a patient’s own immune cells to fight leukemia or lymphoma. An advisory panel to the F.D.A. recommended approval for a third product, made by Spark Therapeutics, to correct a gene defect that causes a blinding hereditary eye disease. All three agency actions occurred this year.

Such treatments are extremely expensive, costing hundreds of thousands of dollars.

The prospect of faster approvals disturbs Michael Carome, director of Public Citizen’s health research group, an advocacy organization. Dr. Carome believes that the industry, still quite young, needs careful F.D.A. oversight.

“I think there is excessive hype,” Dr. Carome said. “We are talking about rushing to market very complex biologics products where we are still in the infancy of this field.”

The agency’s announcements included two final guidelines and two drafts that will be open for public comment. They are designed to help developers sort out whether they need to submit a licensing application to the F.D.A. to get approval for their treatments or fall into a lower risk category, which does not need premarket approval.

One of the proposals would be a boon to small clinics and independent researchers. It would permit them to apply as a group and to pool data. If approved, each would end up with a license for biologics, a category that refers to treatments like cell, tissue and gene therapies that come from natural sources rather than being chemically synthesized.

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. These largely unregulated procedures have been offered for arthritic knees, back pain, heart disease and other problems.

Several patients have been blinded after fat-derived cells were injected into their eyes.

Practitioners who perform these procedures have argued that they do not come under the agency’s jurisdiction. But the new guidance suggested that at least some of the fat-derived injections will be more tightly controlled by the F.D.A. The document stated that if the fat tissue is processed specifically to isolate stem cells — as the stem-cell clinics do — then the procedures must meet F.D.A. safety requirements.

But on the same morning it released the new guidance, the F.D.A. also sent out a “consumer update” that placed much of the burden on patients to check up on stem cell clinics before undergoing treatment.

Urging consumers not to believe the “hype,” the update said, “Some unscrupulous providers offer stem cell products that are both unapproved and unproven,” and added that some of those products may be harmful. The agency advised patients to determine whether the stem-cell treatment being offered had been reviewed by the F.D.A.

At the news briefing, Dr. Peter Marks, director of the F.D.A.’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 F.D.A. “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.”

But the message seemed mixed. Dr. Gottlieb and Dr. Marks also said that orthopedic procedures — injecting the fat-derived cells into joints — were of lower priority than treatments that involved injecting or infusing the cells into the central nervous system or bloodstream.

“There are hundreds and hundreds of these clinics,” Dr. Marks said. “We simply don’t have the bandwidth to go after all of them at once.”

Not everyone agrees that orthopedic clinics should be lower on the priority list. Leigh Turner, an associate professor at the University of Minnesota Center for Bioethics, School of Public Health and College of Pharmacy, who studies cellular therapies, said the use of stem cell therapy in athletic clinics must be scrutinized.

“Sport clinics that use stem cells are one of the largest components of the U.S. marketplace,” she said. “They’ve just been told that there is a three-year period in which they don’t have to be worried about the F.D.A.”

Dr. Marks said that the F.D.A.’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,’’ he said. “It’s to get products on that are safe and effective, and to clear up the field so that people know what they have to do.”