Unregulated Stem-Cell Therapy Causes Severe Vision Loss for Three Florida Women
“…participation in a study for an emerging therapy that is not regulated by the FDA or another well-recognized regulatory agency like the European Medicines Agency in Europe, is fraught with dangers and can lead to unexpected serious consequences.”
A report today in The New England Journal of Medicine (NEJM) describes the cases of three women with age-related macular degeneration (AMD) who lost much of their eyesight after receiving ocular injections of stem cells derived from their own fat tissue. All of the women had good enough eyesight to drive before the procedures. Each paid $5,000 to receive the injections from a private clinic in Sunrise, Florida. The New York Times and other major media outlets have published news stories on the NEJM report.
The administration of the stem cells was performed without oversight from the U.S. Food and Drug Administration (FDA), because, the clinic claims, the cells were the patients' own — the tissue was extracted from the patients' bellies.
However, the patients said they believed they were participating in government-regulated trials, because the study was listed on www.clinicaltrials.gov, a website maintained by the National Institutes of Health.
What's important for the public to know is that the website listing does not mean that the procedure is approved by the NIH and has undergone FDA review and authorization. In an accompanying NEJM article, scientists from the FDA say the fat-derived stem cells, "are being used in practice on the basis of minimal clinical evidence of safety or efficacy sometimes with the claims that they constitute revolutionary treatments."
"The tragedy for these three women, and others who opt for these unproven, unregulated treatments, is they don't understand the risks and lack of evidence for efficacy," says Stephen Rose, PhD, chief research officer, Foundation Fighting Blindness, the world's leading private funder of research for inherited retinal diseases. "As seen in this case and others reported in the literature and press, participation in a study for an emerging therapy that is not regulated by the FDA or another well-recognized regulatory agency like the European Medicines Agency in Europe, is fraught with dangers and can lead to unexpected serious consequences."
Dr. Rose says that the Foundation regularly receives inquiries from people who are inquiring about unregulated stem-cell trials in the United States and around the world. Some of the alleged therapies cost tens of thousands of dollars.
"Understandably, people are anxious to do whatever they can to save their vision from retinal diseases like retinitis pigmentosa and AMD," says Dr. Rose. "Unfortunately, there are many unregulated 'trials' that are taking advantage of the desperation associated with loss of vision, and peoples' vision and lives are at risk. Patients participating in legitimate clinical trials of potential treatments do not typically incur any treatment fees. A fee associated with participation in a clinical study may be a red flag. Anyone contemplating participating in any clinical trial should consult their ophthalmologist or physician first."
FDA-regulated clinical trials for emerging stem-cell-derived therapies for AMD and RP are underway in the United States. Most of these studies are at an early stage and primarily evaluating safety. For more information on these studies, contact the Foundation at firstname.lastname@example.org or visit www.fightblindness.org.