A recent New York Times article reported that dozens of spine surgery patients at NewYork-Presbyterian/Weill Cornell Medical Center were treated with a product that was not approved for use in such procedures. Burst Biologics, Inc., the manufacturer of BioBurst, a stem cell treatment derived from umbilical cord blood, was issued a warning letter by the U.S. Food and Drug Administration (FDA) last year, citing possible contamination problems and insufficient screening of donors, making the product potentially unsafe.

Despite the warning letter and employee complaints dating back years, BioBurst still ended up on hospital shelves nationwide. As regenerative medicine grows in popularity, without regulation, this may become a more common occurrence.

Dangerously Unregulated

BioBurst is a product of the rapidly growing field of regenerative medicine, which aims to replace or restore damaged cells and organs through gene therapies, stem cell therapies, and tissue engineering. Umbilical cord blood contains blood-forming stem cells and has been approved by the FDA to treat disorders that affect blood production.

There are increasing concerns over the largely unregulated world of these therapies, which have exploded in popularity in the past decade. Inspections of facilities producing these products have found significant regulatory violations in sterile manufacturing and monitoring processes. Burst Biologics and another biotechnology company, GeneTech, also failed to adequately screen donors for Zika virus and other communicable pathogens.

To date, hundreds of patients have become seriously injured by or died from unapproved stem cell therapies. Between 2004 and 2020, there were 360 reported adverse events related to unapproved stem cell therapies, including twenty cases that caused death. Just five years ago, twelve people across three states were hospitalized with serious bacterial infections after receiving cord blood-derived stem cell injections manufactured by GeneTech.

FDA’s Role in the Regulation of Regenerative Medicine

According to the FDA, stem cell products such as cord blood-derived injections fall under the category of human cells, tissues, or cellular or tissue-based products (HCT/Ps). HCT/Ps are typically only permitted for homologous use, meaning the product must perform the same basic function or functions of the cells in the recipient as in the donor. Many products in regenerative medicine are used outside of these parameters, against FDA regulations. BioBurst, for instance, was marketed without approval for spine surgery, a non-homologous use.

But the fight to regulate unapproved treatments is complicated. In 2017, the FDA issued new guidelines and clarified the approval and licensing process for regenerative medicine, requiring more rigorous testing for efficacy and safety. Yet today, nearly 1,500 businesses with 3,000 clinics across the U.S. are offering unapproved stem cell treatments. The vast majority of these clinics are unaccredited. Clinics assert that the FDA should not interfere with patients’ the right to choose what treatments they receive. Companies like Burst Biologics argued that their cord blood fluids required only minimal processing and therefore did not fall under the class of product that required heavy regulatory oversight and licensing requirements.

A Wider Problem

Even in the absence of an adverse event, it is dangerous for companies to make unsubstantiated claims about the potential benefits of unproven therapies. This may lead doctors and patients to use these products with a false sense of security about their safety and effectiveness, resulting in underreporting of adverse reactions. Unregulated stem cell clinics can also divert patients away from participating in legitimate clinical trials for therapies vying for full FDA approval.

Despite the FDA’s concerns, FDA warning letters do not require that patients be notified of a product’s potential dangers. It is up to medical providers to communicate these potential dangers to patients. NewYork-Presbyterian would not say whether patients had been notified about receiving BioBurst or about its potential problems.

Frustratingly, it is still unclear how a problematic product like BioBurst ended up being used in hospitals. Neither NewYork-Presbyterian nor Weill Cornell Medicine would explain how or when products are approved at the hospital on the record. Typically, though, hospitals have a formal review process for products that will be used on patients, with a committee vetting requests from doctors before the products are bought by the procurement department, according to the Times. Without proper transparency, dangerous stem cell therapies may appear in hospitals with increasing frequency — and with potentially deadly results.