Bio's Bad Apples

Can unproven therapies spoil the sector’s sweet success?

By Karen Langhauser, Chief Content Director

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Bio's Bad ApplesFew will dispute the enormous potential of cellular therapy — and for patients suffering from vision loss, leukemia or lymphoma who have had life-changing experiences with these new treatments, cell-based therapies are nothing short of a miracle.

Although they lack a globally harmonized classification system, cell therapies fall under the broad umbrella of regenerative medicine. There are several types of cells that can be used for cell therapy and the type of cells administered depends on the treatment. Collectively, these therapies are heralded for their ability to promote the repair of diseased, dysfunctional or injured tissue. What this ultimately means is that cellular therapies offer the potential to treat conditions for which few, if any, treatments exist.

Around the world, there are clinics making dramatic claims about cure-all cell-based treatments that have not gone through regulated clinical trials — which means their safety and efficacy has not been proven to regulatory bodies. If this confusion surrounding cell therapies isn’t addressed, it could become a problem that ultimately discredits a whole industry on the brink of booming.

It is estimated that the unproven cell therapy market is currently worth $2.4 billion and treats approximately 60,000 patients annually. While it’s difficult to identify exactly how many clinics exist, a 2016 analysis of online direct-to-consumer marketing activity in the U.S. found 351 distinct businesses offering interventions at 570 physical locations — and this number has undoubtedly grown.

But when it comes to cell therapy, “potential” is still the key word in the discussion. Currently, the only stem cell-based products approved by the U.S. Food & Drug Administration consist of blood-forming stem cells (hematopoietic progenitor cells) derived from cord blood — and there are fewer than 10 of these products on the market. Kite Pharma, Novartis and Dendreon hold the only immune cell therapy approvals in the U.S. This is likely to change in the future, however. According to the FDA, there are nearly 800 active cell therapy Investigational New Drugs (INDs) on file with the agency.


The potential for miracle cures also brings with it the potential for large financial gain, which has sparked the growing private interest in the cell therapy space. While the pharmaceutical industry might be quick to dismiss these bogus cell therapy clinics, the unfortunate reality is that the general public is still confused. And in that confusion arises a genuine concern that the popularity of unproven cell-based therapies will have a negative impact on the future of legitimate, science-based cellular therapies — which is an issue the industry should not dismiss.

The FDA is not ignoring the rise of unproven cell therapy clinics and has acknowledged the role that regulatory gaps have played in the proliferation of direct-to-consumer cell therapy treatments.

In August 2017, the agency stepped up its efforts to enforce regulations and oversight of stem cell clinics. When this was announced, FDA Commissioner Scott Gottlieb acknowledged that new, complex therapies are challenging the traditional approach to regulations.

“The field of regenerative medicine, because of the very nature of the science and the rapidly evolving clinical developments, not infrequently lends itself to often close calls between what constitutes an individualized treatment being performed by a doctor within the scope of his medical practice on the one hand, and what constitutes a medical product that is currently subject to the authorities Congress has already charged the FDA with exercising,” said Gottlieb.

Part of the allure of unproven cell therapy clinics is that they promise to solve unmet medical needs. When a potential cure to an ailment is tied up in the FDA approval process, patients are more likely to seek help elsewhere. In order to find a balance between safety concerns and innovation, the FDA made a major move in November 2017 to modernize product licensing pathways by introducing an additional expedited program in which a product could be designated as a regenerative medicine advanced therapy (RMAT). The expedited RMAT program — like the fast-track designation, priority review, accelerated approval, and breakthrough therapy designation that were introduced a year prior with the 21st Century Cures Act — is now helping to speed the development of new regenerative therapies, particularly those aimed at life-threatening conditions.

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