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.