Most Regenerative Medicine Therapies on the Market Are Not FDA Approved

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, Los New York Times reportado. The manufacturer of BioBurst Fluid and BioBurst Rejuv, stem cell treatments derived from human umbilical cord blood, was issued a warning letter by the FDA after unlawfully marketing their products for orthopedic use. The letter also reported possible contamination risks and insufficient donor screening, making the company’s products potentially unsafe.

These hazards are not unique to the rapidly growing field of regenerative medicine, which aims to replace or restore damaged cells and organs to normal function through gene therapies, stem cell therapies, and tissue engineering. Nearly 1,500 businesses with 3,000 clinics across the U.S. were found selling purported stem cell treatments in 2021, the vast majority of which are not approved by the FDA. From 2004 to 2020, 360 adverse events from these therapies were reported, including twenty deaths–and due to underreporting, the true number may be even higher.

FDA Rules on Regenerative Medicine

The FDA typically only permits human cells, tissues, or cellular or tissue-based products (HCT/Ps) like BioBurst for uses in which they will perform the same basic function or functions of the cells in the recipient as in the donor. Umbilical cord blood is approved by the FDA to treat disorders that affect blood production; use in spine surgery is therefore against FDA regulations. Many other products in regenerative medicine are unlawfully used outside of these parameters.

But clinics offering these therapies are popping up faster than the FDA can issue warnings. Clinics assert that the FDA should not interfere with patients’ right to choose their medical treatments. There is also disagreement between HCT/P manufacturers and the FDA over which of their products warrant heavy regulatory oversight and licensing requirements.

Long-Term Consequences

Clinics continuing to perform unapproved cellular and gene therapies can hinder the progress of the field of regenerative medicine. These clinics, most of which are unaccredited and do not take insurance, divert patients away from participating in legitimate clinical trials for therapies vying for full FDA approval (currently, only thirty-two are approved). When companies make unsubstantiated claims about the potential benefits of unproven therapies, healthcare providers and patients may overestimate their safety and efficacy, resulting in underreporting of adverse reactions.

It unfortunately is still unclear how BioBurst ended up at NewYork-Presbyterian/Weill Cornell. But until manufacturers see real consequences for marketing unapproved therapies for unapproved use, this may happen again–with possibly deadly consequences.