Science is wonderous and powerful when applied after due diligence, but can be equally damaging and dangerous if it gets into the wrong hands. This was the case during a stem cell therapy by a Florida clinic that promised to cure three women’s retina disease but actually led to blinding them for life.
The New England Journal of Medicine reports that during a clinical trial, three women between the ages of 72 and 88 came hoping to cure their macular degeneration, which is a common progressive retinal disease that leads to loss of eyesight. None of the women were completely blind but were experiencing various degrees of vision loss when they came to seek help from the Florida clinic.
The clinic claimed to have a cure via a stem cell procedure that would treat macular degeneration. But just after a week of the procedure of injecting stem cells into their eyes in 2015, all three women became blind, and today after two years of efforts, doctors have concluded that there is no chance that their vision will ever be restored.
The report of the incident has been compiled by ophthalmologists Jeffrey Goldberg from Stanford University School of Medicine and Thomas Albini from the University of Miami, and they claim that the unfortunate incident is
“a call to awareness for patients, physicians and regulatory agencies of the risks of this kind of minimally regulated, patient-funded research.”
Stem cells are undifferentiated cells in our bodies that have the potential to turn into virtually any kind of cells under the right conditions, meaning they are incredibly useful in regenerative medicine. But despite their potential, the only truly effective clinical application of the stem cells is in bone marrow transplants where stem cells are extracted from a donor’s bone marrow and used to help a leukemia patient produce fresh blood systems.
The problem is that there are nearly 600 clinics in the United States are working on unproven and unapproved stem-cell procedures for a wide range of conditions, such as arthritis, autism, muscular dystrophy, cerebral palsy, stroke and even cancer. These clinics have no monitoring or regulation from any government authority. Thus their results and procedures are always dubious and risky.
Two of the three patients were also misled by the information related to the stem cell trial for macular degeneration on ClinicalTrials.gov, which although is a registry run by the US National Library of Medicine, but the data isn’t fully scrutinized for scientific efficacy. The patients fooled by the assumption that they were taking part in a bonafide clinical trial, and even the consent form and other materials did not make any mention of a trial. Each patient had to pay $5,000 for the one-hour procedure, which is highly unorthodox for a clinical trial and should have served as a red flag for the patients.
“I’m not aware of any legitimate research, at least in ophthalmology, that is patient-funded,” Albini said in a statement.
The NEJM study quotes the name of the trail as: “Study to assess the safety and effects of cells injected intravitreal in dry macular,” which when googled calls the trial up with the name of the company, Bioheart Inc. also known as US Stem Cell. According to Goldberg and Albini, the trail center is no longer offering the procedure, but it is still seeing patients.
The trial itself lacked all the components for a properly designed test, with no prior laboratory experiments, no control group, no data and no plans for follow-ups any follow ups. Standard blood withdrawal was taken from the patients, where their fat cells (i.e. adipose tissue) were then processed using an enzyme to separate the stem cells. Once isolated, the cells were injected into both eyes of each patient which again is a massive mistake as normally only one eye would be injected for such an experimental procedure.
A week later, all three women were blind, along with suffering from detached retinas and hemorrhaging.
The patients’ severe visual loss after the injection was associated with ocular hypertension, hemorrhagic retinopathy, vitreous hemorrhage, combined traction and rhegmatogenous retinal detachment, and lens dislocation. After one year, the patients’ visual acuity ranged from 20/200 to no light perception.
The Florida clinic exploited the desperation of their patients while taking advantage of loopholes in the regulatory system. The authors write in their report:
Adipose tissue–derived “stem cells” have been increasingly used by “stem-cell clinics” because of the relative ease of obtaining and preparing these cells. Many of the clinics that provide these stem-cell therapies have done so under the auspices of patient-funded, institutional review board–approved research, and the research is listed on ClinicalTrials.gov without an investigational new drug filing with the FDA.
At the time of the test, the procedure didn’t need FDA approval since the cells weren’t transferred between patients and they were considered “minimally processed.” Since then, the FDA has finally revised its requirements which mean these types of procedures will also require approval as the authorities look to clamp down on stem cell clinics.
But it’s too little too late for the three women involved.