SANFORD, CALIF. - Three women with macular degeneration were blinded after an unproven stem cell treatment described as a "clinical trial."
A recent paper published in The New England Journal of Medicine documents the cases and is a "call to awareness for patients, physicians and regulatory agencies" on minimally regulated, patient-funded research according to Jeffrey Goldberg, MD, PhD, professor and chair of ophthalmology at the Stanford University School of Medicine and co-author of the paper.
Three patients -- all women ranging in age from 72 to 88 -- suffered from macular degeneration, a common disease of the retina that leads to loss of vision over time. Before the patients underwent surgery, their vision ranged from 20/30 to 20/200.
Now, all three patients are likely to remain blind due to complications from the stem cell treatments.
"Although I can't say it's impossible, it's extremely unlikely they would regain vision," said co-author Thomas Albini, MD, an associate professor of clinical ophthalmology at the University of Miami, where two of the patients went to treat complications.
The trial appealed to patients 'desperate for care'
Two of the patients learned of the so-called clinical trial on ClinicalTrials.gov, a registry and results database run by the U.S. National Library of Medicine. The patients believed they were participating in a trial, although the consent form and other written materials given to the them did not mention a trial, Albini said.
"There's a lot of hope for stem cells, and these types of clinics appeal to patients desperate for care who hope that stem cells are going to be the answer, but in this case these women participated in a clinical enterprise that was off-the-charts dangerous," Albini said.
Each patient paid $5,000 for the procedure, which authors said should have raised a red flag.
"I'm not aware of any legitimate research, at least in ophthalmology, that is patient-funded," Albini said.
At the Florida clinic where the procedure took place, patients have fat cells removed from their abdomens and a standard blood draw. The fat tissue was processed in a lab with the goal of obtaining stem cells and platelet-dense plasma was isolated from the blood. The cells were then mixed with the plasma and then injected into their eyes.
Patients reported that the entire process took less than an hour. The patients had both eyes treated at once -- another red flag, Albini and Goldberg said, because most doctors would opt to see how one eye responds to an experimental treatment before attempting the other eye.
No evidence the procedure would have restored vision
Shoddy stem cell preparation may have led to some of the complications, Albini said. However, even if executed correctly, there is no evidence that the procedure could have help to restore vision both authors report.
There is sparse evidence that fat-derived stem cells, the kind that the clinic claimed to use, are capable of differentiating, or maturing, into the kind of cells that researchers are attempting to target to develop therapies that could slow down macular degeneration.
"There is a lot of very well-founded evidence for the positive potential of stem therapy for many human diseases, but there's no excuse for not designing a trial properly and basing it on preclinical research," Goldberg said.
The "trial" lacked nearly all of the components of a properly designed clinical trial, including a hypothesis based on laboratory experiments, assignment of a control group and treatment group, collection of data, masking of clinical and patient groups, and plans for follow-up, Goldberg and Albini said.
Listings on ClinicalTrials.gov are not fully scrutinized for scientific soundness, Goldberg said. Although still visible on the website, the listing now states: "This study has been withdrawn prior to enrollment."
Albini says the clinic is also no longer performing these eye injections, but still seeing patients.
The procedures were not subject to Food and Drug Administration approval because the cells were not transferred between patients and were considered "minimally processed," according to Title 21, Part 1271.10, of the Code of Federal Regulations. The FDA released more specific guidelines in October 2015, after these procedures were performed, establishing the requirement for FDA oversight and approval for these types of procedures.
'Lack of oversight can lead to bad players and bad outcomes'
"We expect health care providers to take every precaution to ensure patient safety, but this definitely shows that the lack of oversight can lead to bad players and bad outcomes. It's alarming," Albini said.
The authors acknowledged that it is difficult for patients to know whether a clinical trial, or a stem cell therapy, is legitimate. Goldberg recommended that patients considering a stem cell treatment consult a website, A Closer Look at Stem Cells, run by the International Society for Stem Cell Research and to check if the trial is affiliated with an academic medical center.
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