Scott Gottlieb

The US Food and Drug Administration (FDA) has released new guidelines in a bid to quicken the implementation of treatments involving human cells and tissues, including gene therapy. 

However, such an update comes with a warning from the agency. According to The New York Times, they have announced that they would be cracking down on rogue clinics offering dangerous or unproven versions of such treatments. 

Scott Gottlieb, commissioner of the FDA, said, “These concepts are no longer the stuff of science fiction, 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.”

Although the FDA will still require clinical trials, gene and cell therapies that can show the potential to treat unmet medical needs and serious illnesses may now qualify for expedited review to get the products to market more quickly. 

Despite progress in the field, problematic clinics have continued to raise concerns among experts about hastening the approval process. The FDA have said that these clinics, many of which use products taken from patients’ own fat, are making treatments that must be approved before they can be marketed. 

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

For him, he is overjoyed that the FDA has stated clearly 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.

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

However, the prospect of faster approvals concerns Michael Carome, director of Public Citizen’s research group, an advocacy organisation. He believes that the industry is still very young, and needs careful FDA oversight. 

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

From the two new final guidelines proposed, one of them would be a boon to small clinics and independents researchers. It would permit them to apply as a group and to pool data. If approved, each would end upw ith 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 synthesised. 

In addition, 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. 

On the same morning that the FDA released the new guide lines, the FDA sent out a “consumer update” which insinuated the burden was on patients to check up on stem cell clinics before undergoing treatment.

The premise of the statement explained how consumers shouldn’t 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. 

At the news briefing itself, Dr Peter Marks, director of the FDA’s center for biologics evaluation and research, reinforced that practitioners would not get “a free pass to do anything they want.” He also 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.”

Despite this, it felt as though there was a mixed message. Dr Gottlieb and Dr Marks added that orthopedic procedures were of lower priority than treatments that involved injecting or infusing the cells into the central nervous system or bloodstream. 

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