Charlene Ouellet wants to clearly see her grandchildren one day, and she’s willing to do whatever it takes – including spending $21,000 – on an experimental and controversial stem cell treatment that she hopes will improve her vision.
“It’s a big gamble,” said her brother, Rick Ouellet, who’s accompanying her from Maine to Florida for the treatment. “It was now or never.”
Ouellet’s surgery is scheduled for Aug. 23, when doctors in Margate, Florida, will extract adult stem cells from bone marrow in her hips and inject them into her eyes. Ouellet is legally blind and suffers from Usher Syndrome, a degenerative condition that causes vision and hearing loss. Ouellet said her hearing is OK thanks to effective hearing aids, but her vision is severely compromised.
The procedure is generating criticism from some scientists and bioethicists, who say that it’s of dubious therapeutic value, could be bankrupting desperate people and is designed to avoid federal rules that would require more government oversight. The Florida doctor performing the procedures, Dr. Jeffrey Weiss, defended the practice because he said the clinic has helped hundreds improve their vision since he saw his first patient in 2012.
“The first question shouldn’t be ‘Are you charging money for this?’ but rather, ‘Are the patients seeing?’ ” Weiss told the Portland Press Herald in a telephone interview. “We’ve only received criticism from jealous people who have never spoken to us and don’t know what we’re doing.”
Weiss said 60 percent of his 370 patients have improved vision, but he acknowledged those numbers have not been submitted for peer review by other scientists. Weiss said he’s working on three research papers to be published in scholarly journals. He has previously had two individual cases of vision improvement published in medical journals.
LEGAL AND ETHICAL GRAY AREA
The U.S. Food and Drug Administration is hosting hearings in September in part to determine whether the stem cell procedures need to be more tightly regulated. The clinic is registered on www.clinicaltrials.gov – the official government site for research trials – but the clinical trial is not funded or officially recommended by federal health agencies. The trial is registered with the National Institutes of Health, according to the clinic’s website.
Bioethicists say research trials like the ones at the Florida clinic fall into a legal and ethical gray area.
Jonathan Kimmelman, associate professor of biomedical ethics at McGill University in Montreal, said he questions the 60 percent figure touted by Weiss if it hasn’t been reviewed by other scientists. For all the public knows, it could just as easily be 10 percent or 1 percent, Kimmelman said.
“For every patient who has been helped, there could be 100 or 1,000 who have not seen any benefits, and we don’t know whether some patients are being harmed,” he said. “The whole setup is a way for people to be bamboozled.”
Kimmelman said adult stem cells have been proven to have therapeutic value when treating leukemia, but research into using stem cells for the eyes and other diseases is “in its infancy.”
Ouellet, of Brunswick, said her vision has worsened significantly since she was in her late 20s.
She has tunnel vision, and her vision is so poor that she is considered legally blind. She can’t drive and can only read magnified print for a short amount of time because of eye strain.
“It’s like you’re wearing dark sunglasses indoors and there’s always big smudges on the lenses,” Ouellet said. “By the time I’m in my 70s I won’t be able to see at all anymore.”
Ouellet, 53, said her deteriorating condition – for which there is no known cure – motivated her to try the experimental treatment.
“I have a lot of faith this is going to work,” said Ouellet, who has two adult children and three toddler grandchildren. “I have nothing to lose, and everything to gain.”
Ouellet, who works part time at Village Candle in Wells, said she was willing to take out a personal loan for the $21,000, but fortunately an Internet fundraising campaign is covering her costs.
NOW WAITING FOR AGENCY APPROVAL
Bioethicist Art Caplan said charging patients substantial fees for treatment is a red flag. Most legitimate clinical trials have government or private funding so that the patient doesn’t pay any money.
“If it can’t attract funding, it’s really suspicious. It’s a big danger sign,” said Caplan, a professor of bioethics at New York University. Caplan said if patients have to pay, it contributes to the bias of the study.
“Nobody is going to spend $20,000 to be part of a placebo group,” Caplan said.
Caplan, who said he had no personal knowledge of Weiss’ Stem Cell Ophthalmology Treatment Study – or SCOTS – said some doctors are cashing in on the popularity of stem cells with treatments that have little proven scientific value.
“There is so much hype over stem cells,” Caplan said. “Stem cells are not this magic bullet for everything. It’s easy to persuade desperate people to try almost anything.”
Weiss said that the bureaucracy of trying to acquire government funding is cumbersome, especially when the procedure is not tied to the profit motives of the pharmaceutical industry.
Meanwhile, he said he could help patients now.
“We didn’t really know how penicillin worked when it was first developed, but we still used it because it works,” said Weiss, former chief of retinal surgery at the Joslin Diabetes Center in Boston. “Stem cells work, but we don’t know exactly why.”
Weiss said they are screening patients, but treating people with many different eye conditions – about 50 separate eye diseases – makes it difficult to conduct population studies. Weiss said he’s currently working on a research paper with a small population of six to eight patients who have the same eye disease.
But Kimmelman said the fact that Weiss is treating people with so many varied eye diseases is another warning sign. The proper way to conduct a study would be to control as many variables as possible, Kimmelman said, and one variable that can be controlled is to only treat people with the same eye disease. If that works, then try different diseases.
“It’s like he’s doing medicine entirely by individual anecdote,” said Kimmelman, who is advocating for more regulation of stem cell clinics.
Kimmelman said the practice as it’s currently designed can “fly under the radar” of FDA regulations. If the stem cells were processed in any way – such as through genetic modification – the clinic would have to operate under much more stringent federal regulations. Kimmelman said it’s probably not a coincidence that clinics like SCOTS instead do “minimal processing” in order to be less regulated. But does that mean the “minimal processing” is the best way to treat patients with stem cells, or merely the most convenient, Kimmelman said.
“The idea that one could simply extract cells from one part of the body and then squirt them into the eyes, and it would work, that sounds pretty outlandish,” he said.
PATIENTS REPORT BETTER VISION
But some people have come forward and said Weiss’ clinic has improved their vision, including one Maryland woman who was profiled in a Baltimore Sun article this year.
And Jennifer Carden of San Diego said she was one of four patients who received treatment through the Stem Cell Ophthalmology Treatment Study who traveled to Washington in June to lobby Congress in favor of the treatments. Carden said she has talked with two other patients on the phone who have seen their vision improve after going to the same stem cell clinic.
Carden said she spent $20,000 – with about half defrayed by fundraising – and it was worth it to improve the clarity of her vision and her peripheral vision.
“Am I willing to wait 10 years and my vision would just keep getting worse and worse?” said Carden, who has retinitis pigmentosa, a degenerative eye disease. “What is your vision worth? If I could do it again, I would, but I don’t have another $20,000.”
Carden said some patients can be helped more by additional stem cell treatments.
Another warning sign, Kimmelman said, is that there was no research conducted by Weiss on animal models, to determine the basic safety of the procedure.
Weiss said the animal models would have had no scientific value.
Rick Ouellet, Charlene’s brother, said the family is aware of the risks and the controversy.
“It’s very courageous for her to do this,” Ouellet said.
Charlene Ouellet said she’s ready for the treatment and aware that it may not work.
“I’m comfortable with this decision. I want to be able to see across the room, to be able to drive again,” she said.