Woman's blindness reversed by stem cell treatment

Claire

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Woman's blindness apparently reversed by stem cell treatment

Regaining sight in stem cell study

Vanna Belton. local restaurant owner, regained much of her vision after participating in a stem cell study through NIH.

Meredith Cohn
The Baltimore Sun

She was blind and now can see, thanks to unexplained stem cell treatment.

Sitting on the front steps of her Cockeysville home a year and a half ago, Vanna Belton was startled and thrilled when her eyes focused on a car's license plate. Essentially blind for more than five years, she suddenly could read the numbers and letters.

"When I realized I could see the license plates, we started walking around the neighborhood reading them," said Belton, recalling the excitement she and her then fiancée felt at that moment. "We drove around and read store signs. The Pennsylvania Dutch Market. The tanning salon."

No one can explain exactly how Belton came to see again, not even the doctor who treated her as part of an unconventional stem cell study shortly before she regained limited vision.

Despite their promise, stem cell treatments often garner skepticism from experts who are still studying their safety and effectiveness. While stem cells can be grown into any type of cell in the body, scientists generally believe proving the cells can repair or cure anything is a ways off. The only U.S. government approved stem cell treatment involves blood clotting disorders, but that hasn't stopped those who can afford the treatments from seeking them.

Belton, now 29, was training to be a police officer in Washington in 2009 when, while stuck in traffic one day, she noticed the streetlights were blurry. Weeks later she had almost no vision and no explanation for why everything seemingly went dark.

She was diagnosed with a sudden and perplexing case of optic neuritis, a general term meaning optic nerve inflammation.

Belton's damaged optic nerve was not properly sending visual information to her brain. Doctors suspected the cause was multiple sclerosis, an autoimmune disorder, but Belton tested negative for it. They initially said her sight would improve, but after six months they stopped calling her blindness "temporary," she said.

Since doctors offered little hope and no treatment options, Belton turned to Chinese herbs and acupuncture and even made a trip to India seeking a stem cell treatment.

Stem cells for eyes

After relying on friends and family for help getting around, she learned to use a cane, public transportation and technological aids for the blind. One such aid helped her access a dating website and she met Julia Donoghue, who would become her girlfriend and later her wife.

Their first date was at Ruth's Chris Steak House, where Belton once worked. It was a familiar enough spot to initially hide the fact that she could see little more than shadows. Things went so well that she mustered the nerve to tell Donoghue about her condition. They began dating, enjoying meals and walks together with Donoghue holding Belton's elbow. They also devoured any research they could find online about her condition.

As Belton searched for alternatives, a doctor pointed her to clinicaltrials.gov, the National Institute of Health's clearinghouse of studies. There she found Dr. Jeffrey N. Weiss in Margate, Fla., who was enrolling blind patients in an unorthodox stem cell study.

He wasn't affiliated with a university or government institute, but he was taking on all those who could afford the roughly $20,000 to pay for the study and injecting stems cells into their eyes in one of three ways — around the retina, in the retina and directly into the optic nerve — in hopes of restoring some people's sight.

He made no promises.

Her local doctors advised against it; one doctor even called her stupid. Belton decided to go see Weiss anyway.

She paid for it with her wedding budget, opting instead for courthouse nuptials, and a few weeks later in early 2014, she had the surgery. During the four-hour procedure, Weiss and a medical team extracted bone marrow from Belton's hip, separated her stem cells in a machine and then injected the cells in and around her right eye's retina and directly into her left eye's optic nerve.

Weiss is not following the usual steps of clinical studies. 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.

Weiss, who is board-certified in ophthalmology and once taught at Harvard University, said he didn't have the patience for academic research, which is strictly governed by internal review boards and requires fundraising. Without a long history of stem cell research and a current academic appointment, he said, he sought legitimacy for his work by registering the trial with NIH, which scientific journals require to publish promising results.

The U.S Food and Drug Administration must permit the use of "investigational" drugs, and the NIH requires that researchers attest they have such permission to register a trial. While the FDA continues to tweak regulations, there are exceptions when stem cells aren't considered drugs, such as when they are minimally processed and taken from and used in the same person, a category in which Weiss said he falls.

The NIH also requires researchers to gain approval and oversight from an ethics review panel. Universities and government agencies have their own panels; Weiss tapped the International Cellular Medicine Society, an independent group that promotes stem cell therapies.

Weiss published a case study about Belton in the journal Neural Regeneration Research. However, he said, 60 percent of his 278 patients with macular degeneration, glaucoma and other diseases have regained some sight.

While he can't explain how it works, he believes that will become clear eventually.

"We didn't know how penicillin worked for many years, but it saved many lives in the meantime," Weiss said. "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."

No one disputes that Belton now sees well enough to read the menus in her new Mount Vernon restaurant, Flavor. She can navigate the eatery and the streets without the white cane she once used. And on her wedding day, just a week after she read the license plate, she could see her wife's face.

"She started crying when she realized I was looking in her eyes," Belton said.

Researchers believe stem cells can work in two ways, said Dr. Donald Zack, co-director of the Johns Hopkins Center for Stem Cell and Ocular Regenerative Medicine. They either revive sick cells or prompt them to perform better, or they replace the functions of the sick cells.

While calling Weiss' methods unconventional, stem cell researchers acknowledge results are what matter. There's not enough information to judge his research, said Dr. Henry John Klassen, associate professor and director of the Stem Cell & Regeneration Program at the University of California, Irvine.

Weiss' inclusion of so many diseases and treatment methods could make conclusions tough, Klassen said. About a dozen other stem cell studies are now taking place in humans with eye diseases, all focused more narrowly, he said.

"I don't want to play down the results of this patient," Klassen said. "It's always great when a patient seems better. ... If 60 percent of his patients have improved, that would be a strong claim, and that's what he should be presenting."

For Belton, it was about trying, even if she defied her doctors.

Dr. Alexis G. Malkin, who examined Belton before and after the procedure, still would hesitate to recommend it to other patients. While Belton's improvement has been dramatic, she said, she's still legally blind and sees only "islands" amid blind spots.

"What was the mechanism?" said Malkin, who left Johns Hopkins' Wilmer Eye Institute to become a clinical assistant professor at the New England College of Optometry. "It's hard to say; we don't know why she lost her vision in the first place."

But she added, "Had she done nothing, would she randomly have gotten better on her own? That seems exceptionally coincidental."

Belton said she plans to return to Florida in March for another treatment. She could improve — or even lose ground.

"I'm happy to be a guinea pig," she said. "Tell me what you wouldn't do."
 

barbara

Pioneer Founding member
Another story about her. I'm proud to say that I serve on the ICMS IRB Board that approved this study.


http://www.sciencealert.com/a-blind-woman-has-regained-sight-thanks-to-a-controversial-stem-cell-treatment

A blind woman has regained sight following a controversial stem cell treatment
This is incredible.
29 FEB 2016

Vanna Belton from Baltimore has been blind for more than five years, but after undergoing surgery where stem cells extracted from her bone marrow were injected into her right eye's retina and left eye's optic nerve, she has 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, adding that for the first time since 2009, she’s been able to navigate her way around without a cane.

While Belton’s recovery is certainly remarkable, it’s also incredibly perplexing. The doctor who performed the stem cell treatment, ophthalmologist Jeffrey N. Weiss, cut so many corners to get her and 277 other patients into surgery, even he can’t explain why it works.

"Weiss is not following the usual steps of clinical studies," Meredith Cohn reports for the Baltimore Sun. "Among other things, he didn't test his treatment theories first on lab animals or using computer models, or randomise 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."

Essentially, the 'traditional route' of lab-to-animals-to-clinical-trials takes too long, Weiss says, so he figured out how to game the system and start testing his new treatment on his own terms.

All he had to do was register his human trials with the US National Institutes of Health (NIH), which usually requires approval from the Food and Drug Administration (FDA) before any new or established drugs can be tested on humans.

The catch is that stem cells aren’t classified as a drug - they’re extracted from a patient's body, go through very little processing, and are used on that same patient, so Weiss was able to bypass FDA approval to register his NIH trials. He did need approval and oversight from an ethics review panel, and got his from the International Cellular Medicine Society, an independent, pro-stem cell therapy group based in Nevada.

So Weiss, who is practicing independently of any university or research institution, is now free to charge around US$20,000 per patient to perform his stem cell treatment, and offers no promises of a cure to his patients.

But according to Cohn, Weiss reports that 60 percent of his 278 patients - who have gone blind because of diseases such as macular degeneration and glaucoma - have regained some sight following the procedure. He’s published a case study on Belton in the journal Neural Regeneration Research.

Belton lost her sight in a matter of weeks in 2009 due to a severe case of optic neuritis - inflammation of an optic nerve that causes progressively blurred vision because the nerve could no longer communicate with the brain. Until recently, dark shadows were the only thing should could make out, and she used a cane and various technological devices to help her function.

After undergoing Weiss's 4.5-hour surgical procedure, in which stem cells extracted from bone marrow in her hip were injected into a different area in each eye, she gradually regained some of her sight. She can now read menus and street signs, get around without her cane, and has seen her wife's face for the first time in five years.

We should point out that the procedure is by no means a cure - Belton is still considered to be legally blind, and rather than seeing everything now, she can only make out 'islands' of sight in a sea of nothing, Cohn reports. But you'd be hard-pressed to find someone who wouldn’t pay $20K to get at least some of their sight back.

The problem - likely confounded by the fact that Weiss cut so many corners - is that no one can explain how the treatment actually works.

For example, how does he explain the fact that Belton has regained sight in both her eyes, despite the fact that the stem cells were injected into her right eye's retina and left eye's optic nerve? Does that mean it doesn’t matter where the stem cells are injected into the eye, and if that’s the case, how does that even work?

Weiss says he or someone else will figure that out eventually, but previous research points to two possibilities - either the injected stem cells revive malfunctioning stem cells in the eye, or they replace them.

"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."

Right now, there are a number of clinical studies in the US that are also investigating the potential of stem cell treatments, but the earliest results of those are not expected until later this year. And late last year, we reported on the first person in the UK to receive stem cell treatment for their blindness (the results are still yet to be announced), so we are definitely going to hear much more about this in the near future.

What other researchers in the field are really hoping for is a proper report on Weiss's 278 patients, so it can be peer-reviewed and scoured for clues that would explain the mechanism that's restoring sight to some, but not others.

Another drawback of pursuing results before fully understanding the science is that it’s not clear if further treatment will hurt or hinder Belton and Weiss’s other patients’ progress. Belton’s scheduled to receive more stem cell injections, but has been warned that while it could give her more sight, it could also take away the sight that’s been restored.

"I'm happy to be a guinea pig," she told Cohn.
 
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