Stem cells have been shown to do everything from regrowing skull bones to healing damaged lung tissue to repairing burned skin to fighting brain tumors and much more. They've even been used to restore vision in rabbits. But when three adult women tried an unproven stem-cell treatment at a clinic in Florida to combat vision loss from macular degeneration, they all went blind. A paper detailing the procedure and its tragic results was published today in The New England Journal of Medicine.

The paper was written by Jeffrey Goldberg, a professor of ophthalmology at Stanford University School of Medicine and Thomas Albini, an associate professor of clinical ophthalmology at the University of Miami where two of the three women were treated after complications from the surgery emerged. Goldberg says that the paper is a "call to awareness for patients, physicians and regulatory agencies of the risks of this kind of minimally regulated, patient-funded research."

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Warning signs

The women who underwent the procedures – aged 72-88 – found the opportunity on the website ClinicalTrials.gov, a site where listings are not screened for scientific rigor according to Goldberg. Signing up through such a site gave the women the logical impression that they were going to be participating in a trial, however, Albini says it was anything but.

One warning sign should have been that the women each were made to pay $5000 for the procedures, when in legitimate clinical trials there are rarely fees. Another issue was that there was no mention of a clinical trial in the paperwork the women were asked to complete. The webpage for the study now reflects that it has been withdrawn and, according to the New York Times, the clinic is no longer performing the procedure.

"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.

During the procedures, the women had fat removed from their abdomens. They also had blood taken. The fat was then processed with enzymes meant to isolate stem cells and the resulting material was mixed with plasma from the blood. That mixture was then injected into both eyes of all three patients – which is another issue, according to Albini, who says that a cautious researcher would have just used one eye each to see how the procedure went before proceeding.

Because the treatment used biological material that came from the participants themselves, they did not require FDA approval.

Jeffrey Goldberg, MD, PhD, professor and chair of ophthalmology at the Stanford University School of Medicine and co-author of the paper (Credit: Norbert von der Groeben)

Now, one of the women has gone completely blind while two others are considered effectively blind, says a report from NPR. Two of the women have sued the clinic and settled, according to the Times.

Prior to the procedure, all of them were suffering from macular degeneration, a condition that leads to blurry vision and eventual vision loss. While their vision would have no doubt continued to degrade without the procedure, two were still able to see well enough to drive before the procedure. Albini says the complications could have come from injecting a contaminant into the eye, or from the fact that the stem cells may have turned into myofibroblasts after the injections, which are cells associated with scarring.

Not all bad

Proving that stem cell treatments can also be perfectly sound, The New England Journal of Medicine also published a paper today that details a Japanese study that used a type of stem cells known as induced pluripotent stem cells (iPS) to treat a subject suffering from macular degeneration. A year later, the patient suffered none of the additional vision loss that would be common with the condition. iPS stem cells have a body of research behind their potential healing abilities, while the fat-based cells used in the procedures do not.

Adding to the flurry of activity regarding stem cells in today's New England Journal of Medicine, was an editorial by leading stem-cell research and dean of Harvard Medical School, George Q. Daley, as well as a piece by a team of researchers entitled "Clarifying Stem-Cell Therapy's Benefits and Risks." Both call for stricter regulation of the stem-cell industry and holding those who operate in that arena accountable to the same rigorous research standards applicable to all scientific advances.

Until then, Albini advises that individuals thinking about stem-cell treatments check out a website called A Closer Look at Stem Cells, while also investigating whether the person proposing to do the treatment is associated with an academic medical center.

"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.

Source: Stanford Medicine News Center

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