US FDA stem cell enforcement challenged by state legislation

In response to increased industry activity in the field of personalised medicines, the US Food and Drug Administration (FDA) has produced a series of guidance documents​, a dedicated expedited Regenerative Medicine Advanced Therapy (RMAT) pathway, and recently began clamping down on unapproved stem cell treatments and unregistered clinics.

Last month​, for example, it sent a warning letter to a Sunrise, Florida-based clinic for marketing autologous stem cell products without FDA approval, and – with the help of the US Marshals Service – seized material for an unapproved stem cell product intended to be directly injected into patients in two clinics in California.

But local state legislation may be holding back these efforts and undermining the FDA’s attempts at regulating the industry.

American Stem Cell Centers of Excellence​

Earlier this month, American Stem Cell Centers of Excellence opened a clinic in Dallas, Texas offering regenerative stem cell treatments for autoimmune, orthopaedic, degenerative and neurological conditions.

The clinic takes bone marrow or adipose tissue from a patient and reintroduces them into a specific location of injury or disease.

Currently the handful of stem cell products approved for limited use in the US (see list here​) all consist of HPC (hematopoietic progenitor cells), cord blood isolated from the placental, or umbilical cord blood. No FDA approved treatments are made from bone marrow of adipose tissue.

According to FDA spokeswoman Erin Baker, the Agency has “no record of a firm called American Stem Cell Centers of Excellence being registered with FDA,”​ though under House Bill 810, which passed in the state of Texas on September 1, the firm has every right to offer such treatments.

State laws​

HB810​ allows for the use of unapproved stem cell treatments to patients with certain severe chronic diseases or terminal illnesses, defined as “a condition, injury, or illness that: (A) may be treated; (B) is never cured or eliminated; and (C) entails significant functional impairment or severe pain.”​

American Stem Cell Centers of Excellence did not respond to requests for comment from Biopharma-Reporter.

But biopharma legal expert Kevin Noonan – a partner at McDonnell Boehnen Hulbert & Berghoff – told us the Bill could be at odds with the FDA’s efforts to regulate the sector.

“The Texas law is significant because the size of the state reduces the need to engage in interstate commerce, which has long been the justification for Federal government action/regulation of many activities. However, the FDA does have the ability to regulate stem cells if there are allegations that they are not safe or effective, and the FTC can also go after commercial activities that are fraudulent or deceptive,”​ he said.

“The Texas law will enable stem cell clinics to provide services to Texas residents until such time as there are consumer complaints that rise to the level of patient injury, ineffectiveness, or death, and then I expect the Texas authorities will step in.”​

And Texas is not alone as about 30 states have such legislation enacted or in the works, Noonan added.

So where does this leave the FDA?​

Tissues and cellular therapies – including stem cells – are regulated by FDA’s Center for Biologics Evaluation and Research (CBER) as human cells, tissues, and cellular and tissue-based products (HCT/Ps). 

According to Baker, any establishment that performs any of the manufacturing steps for regenerative medicine products must register with the Agency, listing their products and each of the manufacturing steps they perform, and are subject to FDA inspections.

“It is important to note that FDA expects anyone involved with recovering, processing, or otherwise manufacturing HCT/Ps to familiarize themselves with the regulations as they are responsible for complying with the regulations that apply to their products,”​ she told Biopharma-Reporter.

While the Agency recognises there are a number of clinics operating which are not registered with the FDA, for now Baker encouraged consumers to contact the Agency and the appropriate state authorities to report any potentially illegal or harmful activity related to stem cell based products.

“We also encourage patients and health care providers to report adverse events associated with cellular therapies to FDA.”​