Stem-Cell Therapy Takes Off in Texas
With Texas pouring millions of dollars into developing adult stem-cell treatments, doctors there are already injecting paying customers with unproven preparations, supplied by an ambitious new company.
The US Food and Drug Administration (FDA) has not approved any such stem-cell treatment for routine clinical use, although it does sanction them for patients enrolled in registered clinical trials. Some advocates of the treatments argue, however, that preparations based on a patient’s own cells should not be classed as drugs, and should not therefore fall under the FDA’s jurisdiction.
There are certainly plenty of people eager to have the treatments. Texas governor Rick Perry, for instance, has had stem-cell injections to treat a back complaint1, and has supported legislation to help create banks to store patients’ harvested stem cells.
One company that has benefited from this buoyant climate is Celltex Therapeutics, which “multiplies and banks” stem cells derived from people’s abdominal fat, according to chairman and chief executive David Eller. Its facility in Sugar Land, just outside Houston, opened in December 2011 and houses the largest stem-cell bank in the United States.
Celltex was founded by Eller and Stanley Jones, the orthopaedic surgeon who performed Perry’s procedure, and it uses technology licensed from RNL Bio in Seoul. Because clinical use of adult-stem-cell treatments are illegal in South Korea, RNL has since 2006 sent more than 10,000 patients to clinics in Japan and China to receive injections.
Celltex says that although it processes and banks cells, it does not carry out stem-cell injections. It declined to answer Nature’s questions about whether its cells have been used in patients. But there is evidence that the company is involved in the clinical use of the cells on US soil, which the FDA has viewed as illegal in other cases.
In addition to the publicity surrounding Perry’s treatment, a woman named Debbie Bertrand has been blogging about her experiences during a five-injection treatment with cells prepared at Celltex. Her blog (debbiebertrand.blogspot.com) hosts photographs of herself alongside Jones; Jennifer Novak, a Celltex nurse; Jeong Chan Ra, chief executive of RNL Bio; and her doctor, Jamshid Lotfi, a neurologist who works for the United Neurology clinic in Houston. Another photo is captioned: “My cells are being processed in here for my next infusion!!!” A third shows Bertrand, Lotfi and a physician called Matthew Daneshmand, who is, according to the caption, injecting Bertrand’s stem cells into an intravenous drip, ready for the infusion. Nature has been unable to contact Bertrand.
Lotfi says that he has administered cells processed by Celltex to more than 20 people. “Five or six” — including Bertrand — have multiple sclerosis and “four or five” have Parkinson’s disease, he says. Lotfi explains that patients sign up for treatment by contacting Novak, and that cells are prepared by removing about five grams of fat — containing roughly 100,000 mesenchymal stem cells — from the patient’s abdomen. Over a three-week period, the cells are cultured until they reach about 800 million cells. Lotfi says that patients get at least three injections of 200 million cells each, and that the cells do not take effect for a few months. According to Lotfi, Celltex charges US$7,000 per 200 million cells, and pays Lotfi $500 per injection.
Lotfi says that most of his patients claim to get better after the treatment, but he admits that there is no scientific evidence that the cells are effective. “The scientific mind is not convinced by anecdotal evidence,” he acknowledges. “You need a controlled, double-blind study. But for many treatments, that’s not possible. It would take years, and some patients don’t have years.”
“The worst-case scenario is that it won’t work,” he adds. “But it could be a panacea, from cosmetics to cancer.” He says that Celltex is conducting a trial in which patients “will be their own control”. “If you can compare before and after and show improvement, there’s no need for a placebo,” he explains. “How can you charge people, and then give them a placebo?”
The answer, according to most biomedical researchers, is to follow the normal strategy for an unproven medicine: conduct a placebo-controlled clinical trial in which patients are not charged. “This is crucial for clinical trials to yield meaningful data and to avoid the placebo effect,” says Joshua Hare, director of the Interdisciplinary Stem Cell Institute at the University of Miami in Florida, who worked with the biotechnology company Osiris Therapeutics in Columbia, Maryland, to conduct a trial of adult mesenchymal stem cells in patients who have had a heart attack2.
The FDA does exempt some “compassionate use” procedures from these requirements, but only for very small numbers of patients with life-threatening disorders that demand immediate intervention.