Presentation at September 12-13, 2016 FDA Hearing on HCT/Ps

Statement by Leigh Turner 

I’d like to put the draft guidances in context by drawing attention to the hundreds of U.S. clinics selling unapproved stem cell interventions. I’m concerned years of inadequate regulatory oversight by the FDA fuelled the nationwide spread of such businesses.

Over 350 U.S. businesses advertise purported stem cell treatments provided at nearly 600 clinics. Many of these clinics sell costly stem cell interventions for ALS, Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, muscular dystrophy, cerebral palsy, autism, COPD, spinal cord injuries, and dozens of other conditions. Children are among the individuals receiving unapproved stem cell products.

Advertised interventions include autologous stem cells obtained from adipose tissue, bone marrow, and blood; allogeneic products derived from amniotic material and placentas; xenogeneic stem cells; and human embryonic stem cells.

It’s understandable that individuals with serious health problems respond to the compelling marketing claims stem cell clinics make. Less comprehensible is how companies get away with making unsubstantiated claims about cellular therapies without prompting swift regulatory action.

Clinics advertise using the rhetoric of “stem cell treatments” and “IRB approved patient-funded studies.” Numerous companies use the NIH’s ClinicalTrials.gov registry as a marketing platform for “studies” that have serious scientific, ethical, and regulatory flaws. Some of them falsely claim their studies are “NIH” or “FDA approved.” When journalists have contacted the FDA and asked questions about such studies the FDA has responded by stating that it cannot comment on trials conducted under Investigational New Drug applications. Since these studies are not conducted under INDs such replies provide cover for clinics selling unapproved stem cell products.

For many years now, the FDA has failed to regulate the U.S. direct-to-consumer stem cell marketplace in a risk-based, timely, and consistent manner. This is a marketplace where regulatory action is rare even when businesses have spent the last five years selling unapproved stem cell products for 20 to 30 diseases.

Acknowledging this regulatory failure, I commend the FDA for issuing the draft guidances. The documents clarify when pre-marketing authorization is required. They provide insight into how the FDA defines, interprets, and applies concepts such as the same surgical procedure exception, homologous use, and minimal manipulation. Addressing these concepts is crucial because such criteria are abused by clinics. Perhaps the guidances are a sign the FDA now plans to take action against marketing claims and business practices that often are based on nothing more than the unsubstantiated assurances of clinic owners. However, I’m concerned meaningful regulatory action will not follow this hearing.

In 2012 and 2013 I contacted the FDA and urged you to investigate numerous businesses selling autologous adipose-derived stem cell interventions for ALS, Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, muscular dystrophy, COPD, stroke, spinal cord injuries, and dozens of other diseases and injuries. Over three years later these companies continue to profit from administering stem cell products the FDA states require pre-marketing authorization.

During their advocacy for the 21st Century Cures Act, Senator Lamar Alexander and former Senate Majority Leader Bill Frist have used a pay-to-participate study run by a Florida-based physician as an example of dramatic progress being made in stem cell therapies. They presumably do not know that at least two patients whose eyes were injected with autologous bone marrow derived stem cells suffered serious complications. These outcomes were reported not by the doctor charging $20,000 per “research subject” to inject stem cells, but by the physicians who treated the patients he injured. I urged you to investigate this business back in January 2013, before these patients were harmed.

Disciplinary actions by medical boards in California, Florida, and elsewhere and published case reports reveal that numerous patients have been harmed by clinics selling unapproved stem cell interventions. Recall, for example, Domenica Fitzgerald and Richard Poling, two patients who died after their autologous stem cell transplants at Regenocyte. Lawsuits filed by former patients of various U.S. stem cell clinics also contain troubling allegations of injuries and fraud.

Widespread clinical use of unapproved stem cell products combined with continued regulatory inaction will likely be followed by additional reports of harmed patients. This is to be expected when so-called stem cell treatments have not been subjected to preclinical studies and tested for safety and efficacy in properly designed, conducted, and regulated clinical trials.

The out-of-control marketplace for stem cell interventions needs effective regulatory oversight. I therefore hope the draft guidances are more than stage props and this hearing is more than public theater. When patient safety and public health are at stake, the FDA must do more than function as a paper tiger. It is time for action.

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