A week ago
the FDA released its 483 inspection report relating to Celltex Therapeutics
Corporation’s practices banking and facilitating the administration of adult
stem cells for therapeutic purposes. News
sources (here and here) and the Center for Genetics and Society published portions of its
startling findings. Celltex processes, multiplies,
distributes, and facilitates the injections of mesenchymal cells derived from
adipose tissue using technology licensed from RNL Bio based out of Seoul, South
Korea. Prominent physician Carl Elliot, MD, PhD and bioethicist Leigh Turner, PhD have both expressed public concern about Celltex’s seemingly
flagrant circumvention of FDA regulatory standards, licensing partnership with
a controversial corporation, and conflict of interest in the oversight of its operations.
Since its opening
in December 2011, Celltex is the largest stem cell bank in the US and
facilitates the process and supplies the product for individuals seeking to
receive stem cell injections, charging a hefty fee ranging from an estimated $20,000-$30,000
for its services. It has garnered significant
publicity arising in part from Texas Governor Rick Perry’s support, his personal
use of stem cell injections for a back injury, and Perry’s push for state
legislation to create a state adult stem cell banking initiative and revise
Texas Medical Board regulations relating to physicians’ ability to administer non-FDA
approved stem cell “treatments” to their patients.
Back in
February, Turner compiled a letter of listed concerns relating to Celltex’s
practices, requesting that the FDA investigate the company’s practices. Turner and other media sources have pointed
out the following, along with other issues:
Celltex has
not offered substantial data or clinical trials to show the stem cells it
offers are safe and efficacious. In January 2012 the FDA issued a Consumer Health Information guide cautioning consumers to make sure that any stem cell
treatment they consider has been approved by the FDA or is subject to a current
protocol submitted to the FDA to ensure that the stem cells are safe, effective,
and have undergone adequate and well controlled clinical trials. Furthermore, the FDA must oversee the manufacturing
process to assure the products’ safety, purity, and potency.
If individuals are receiving experimental injections, Celltex and the physician should thoroughly explain that
the procedure is experimental medical research rather proven clinical medicine and
the risks associated with the procedure.
Both the FDA and physician commentators have noted that patients seeking
to have cells injected face safety risks arising from the procedure, even if
they are the patient’s own stem cells and the corporation follows good manufacturing
practices. Jamshid Lofti, MD, a
neurologist who has administered injections to more than 20 patients has
admitted the need for controlled trials, but has problematically downplayed such risks, asserting that “the worst that can happen is it won’t work.” Not according to the FDA and numerous other physicians, who list potential risks to include tumors, cancer, and even death. Such risks are at the forefront of investigation (see here and here) into partner company RNL Bio’s practices in South Korea, where former patients and
their families have come forth with allegations of cancer and death linked to
receipt of RNL Bio’s stem cells.
Celltex contends
that it merely processes and expands individuals own stem cells, only
minimally manipulates the cells, and is currently coordinating prospective
clinical studies. According to Nature, Celltex
coordinates with physicians and pays physicians $500 per injection, and each
patient receives at least three injections.
Celltex argues that the process of culturing and preparing the stem
cells does not constitute the manufacture of a biological drug, so the process stands
outside the scope of FDA regulations normally required for biological drugs. A Colorado court is currently examining this
issue arising from the practices of another company Regenerative Sciences, who
proffered a similar argument to avoid applicability of FDA requirements.
However,
Rita Chappelle, a spokesperson for the FDA’s Center for Biological Evaluation
and Research asserts that any “expanded” cells cannot be considered minimally manipulated. Importantly, the FDA’s 483
inspection form classifies Celltex as a biological drug manufacturer in the
business of manufacturing mesenchymal stem cells rather than minimally
manipulating stem cells. Independently of
how Celltex classifies its processes, the 483 report made news headlines based
on the number of alarming deficiencies in the processing and manufacturing procedures. It found Celltex failed to validate processes
to prevent contamination; to distinguish between components being quarantined
or approved; to routinely calibrate and check the equipment; to review quality
processing systems; and could not guarantee the sterility, uniformity or
integrity of the cells. Celltex’s press
release in response is here.
Had there
been any uncertainty about the scope of FDA regulation, the Consumer Health
Information guide back in January clarified that the manufacture of stem cells must undergo FDA review.
Yet Celltex continued its operations charging clients tens of thousands
of dollars for unproven and unregulated “treatments,” evaded FDA’s clarification,
and exposed consumers to additional risks based on its manufacturing
deficiencies detailed in the 483 inspection.
More problematically, associated figures such as Gov. Perry and
physicians who administer the injections provide the imprimatur of Celltex’s practices- creatively flouting
regulatory requirements, downplaying risks of the procedure, and reaping hefty
profits. Even in the name of therapeutic
progress and statewide economic growth, it is time for the FDA to set an
example that such disregard for its jurisdiction and review process will not be
tolerated.
--Katherine Drabiak-Syed
