FDA crackdown on unproven stem cell therapies

The US Food and Drug Administration (FDA) intends to investigate the use of unproven stem cell therapies being offered in the country's clinics.

Tighter enforcement from the FDA comes as an inspection at StemImmune Inc based in San Diego, California, revealed the use of potentially dangerous treatments administered to vulnerable cancer patients.

Only a small number of stem cell treatments are currently FDA approved, including use of bone marrow transplants in cancer patients and cord blood for specific blood-related disorders. However stem cell treatments using only the patient's own cells are not subject to the same level of regulation as drugs if the cells are only 'minimally manipulated'.

FDA commissioner Dr Scott Gottlieb said in a statement: 'The FDA will not allow deceitful actors to take advantage of vulnerable patients by purporting to have treatments or cures for serious diseases without any proof that they actually work. I especially won’t allow cases such as this one to go unchallenged, where we have good medical reasons to believe these purported treatments can actually harm patients and make their conditions worse.'

Five vials, each containing 100 doses of the live Vaccinia Virus Vaccine, were seized from StemImmune Inc by US marshals on 25 August 2017. 

The vaccine, which is used against smallpox, and is not commercially available – was combined with stem cells derived from body fat to create an unapproved therapy. The concoction was injected directly into tumours of cancer patients at California Stem Cell Treatment Centres in Rancho Mirage and Beverly Hills.

The effects of the vaccine in immunocompromised cancer patients have the possibility to cause severe complications such as inflammation and swelling of the heart and surrounding tissues.

In a separate case, a warning letter was also sent to chief scientific officer Kristin Comella at US Stem Cell Clinic in Sunrise, Florida, after three patients with macular degeneration were blinded following the use of unapproved stem cell injections into their eyes, in a sponsored study (see BioNews 893). The letter lists a number of non-compliance to procedures and 'significant deviations' to current good manufacturing practice and good tissue practice.

'Our actions today should also be a warning to others who may be doing similar harm, we will take action to ensure Americans are not put at unnecessary risk,' Dr Gottlieb commented. 'I also urge health care providers, patients and consumers to report these kinds of activities or any adverse events associated with these unproven treatments to the agency through MedWatch – a safety reporting programme.'

Professionals in the field blame the past lack of FDA attention for the widespread problem and are calling for stringent regulation. Professor Leigh Turner, from the Centre for Bioethics at the University of Minnesota, told CNN: 'This is a space where the FDA could have taken action four or five years ago as far as making this a policy priority.'