Even the doctor can’t explain how the procedure really worked.
Following a controversial stem cell treatment, Vanna Belton from Baltimore regained some of her sight after being blind for over five years. This recovery is thrilling news for the blind community, but the treatment itself is perplexing — even the doctor who conducted the procedure, ophthalmologist Jeffrey N. Weiss, can’t really explain how it works.
"Weiss is not following the usual steps of clinical studies," reports Meredith Cohn, a writer for the Baltimore Sun. "Among other things, he didn't test his treatment theories first on lab animals or using computer models, or randomize his trials by using either stem cells or placebos in study participants. He didn't test the procedure for safety on a small group before moving to a larger trial."
The process to get to clinical trials can take years, so Weiss decided to find a way around the traditional system in order to start testing the stem cell treatment on his own terms.
Since stem cells aren’t classified as a drug, Weiss was able to register his human trials with the US National Institutes of Health (NIH) without getting approval from the Food and Drug Administration (FDA). Stem cells are extracted from the patient’s body and then used on that same patient, so he also didn’t need approval from an ethics review panel.
Instead, he got oversight from the International Cellular Medicine Society, which is an independent, pro-stem cell therapy group.
Weiss wasn’t affiliated with a government institute or a university and made no promises about the efficiency of his stem cell treatment, but he took on 278 patients who could afford to pay about $20,000 for the procedure in his study. The patients were injected with stem cells in one of three places in the eye — in the retina, around the retina, or directly into the optic nerve.
Belton’s procedure took roughly 4.5 hours as Weiss extracted stem cells from bone marrow in her hip and injected them into different areas of each eye. Amazingly, Belton gradually regained some of her sight.
"When I realised I could see the license plates, we started walking around the neighbourhood reading them," she told the Baltimore Sun, noting that she’s been able to navigate her way around without a cane for the first time since 2009.
Additionally, she can now read menus and street signs, and has seen her wife’s face for the first time in five years.
Weiss reports that 60 percent of the 278 patients regained some sight following his stem cell procedure — they’d gone blind due to various diseases like macular degeneration and glaucoma. He’s also published a case study on Belton in the journal Neural Regeneration Research.
There’s still a lot of further research to complete in order to fully understand Weiss’ treatment and how it works. For example, Belton regained sight in both of her eyes despite the fact that Weiss injected the stem cells into her right eye’s retina and her left eye’s optic nerve — does it matter where the cells are injected? Or was Belton a peculiar case?
However, as long as the treatment works, Weiss isn’t overly concerned about figuring out all of these details just yet.
"We didn't know how penicillin worked for many years, but it saved many lives in the meantime," Weiss told the Baltimore Sun. "It is hubris to think that something can't work until you understand how it does. ... It is more important what the patient sees, not what I see."
It will certainly be interesting to see how Weiss’ research pans out in the future and if his patients continue to regain their sight. Even though it’s unclear how this stem cell treatment truly works and whether continuous treatments would be beneficial or detrimental for patients’ sight, there is at least a new sense of hope for people who are blind to regain some of their sight.
"I'm happy to be a guinea pig," Belton said. "Tell me what you wouldn't do."