Three Blinded by Unproven Stem Cell Treatment

12.04.2017

Three people with macular degeneration were blinded after undergoing an unproven stem cell treatment that was touted as a clinical trial in 2015 at a clinic in Florida. Within a week following the treatment, the patients experienced a variety of complications, including vision loss, detached retinas and hemorrhage. They are now blind.

A paper documenting the cases has been published in The New England Journal of Medicine.

The three patients—all women, ranging in age from 72 to 88—suffered from macular degeneration, a common, progressive disease of the retina that leads to loss of vision. Before the surgery, the vision in their eyes ranged from 20/30 to 20/200. Now, the patients are likely to remain blind, said Dr Thomas Albini, an associate professor of clinical ophthalmology at the University of Miami, where two of the patients were subsequently treated for complications from the stem cell treatments.

Some of the patients believed they were participating in a trial, although the consent form and other written materials given to the patients 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.”

Each patient paid $5,000 for the procedure. Any clinical trial that has a fee should raise a red flag. “I’m not aware of any legitimate research, at least in ophthalmology, that is patient-funded,” Albini said.

At the clinic, which is not named in the paper, the patients had fat cells removed from their abdomens and a standard blood draw. The fat tissue was processed with enzymes, with the goal of obtaining stem cells. Platelet-dense plasma was isolated from the blood. The cells were then mixed with the platelet-dense plasma and injected into their eyes. Patients reported that the entire process took less than an hour, Albini said. The patients had both eyes treated at once— another red flag, because most doctors would opt for a conservative approach to observe how one eye responds to an experimental treatment before attempting the other eye.

Shoddy stem cell preparation may have led to some of the patients’ complications, which could have been caused by injection of a contaminant or the cell wash solution into the eye, Albini said. When injected into the eye, the stem cells also could have changed into myofibroblasts, a type of cell associated with scarring.

“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,” said Dr Jeffrey Goldberg, professor and chair of ophthalmology at the Stanford University School of Medicine.

Medical News Today. March 17. New England Journal of Medicine. March 16.