For thousands of people slowly losing their sight to a degenerative eye disease, it sounds like a miracle cure: a treatment devised by a Cuban opthalmologist that he claims can prevent retinitis pigmentosa from stealing their vision.
But the Baltimore-based Retinitis Pigmentosa Foundation is recommending against going to Cuba for the treatment -- despite patients who insist they left Havana with their vision improved considerably. The foundation's position, which has generated considerable controversy among RP patients, is based on what it insists is a lack of scientific proof the treatments work.
"Right now, we don't have any evidence of any improvement" in the vision of people who have undergone the treatments, says Robert M. Gray, executive director of the RP Foundation. "There's no evidence showing that it works. And until you've got evidence, medically and scientifically, we cannot go out and recommend this."
Retinitis pigmentosa affects an estimated 100,000 people in the United States. An additional 6 million are going blind due to other conditions that affect the retina, including Usher syndrome (which can also cause hearing loss). Patients lose their sight slowly, usually experiencing a narrowing of the field of vision to the point where they effectively are looking through a dark tunnel that lets in only a small beam of light. Some go completely blind.
There is no treatment for the condition, although some research suggests Vitamin A can help slow the degeneration.
In essence, the dispute pitting RP patients against an organization long in the forefront of searching for a cure boils down to a difference over what constitutes proof. For the scientific community, proof is accumulated over years: hundreds of tests performed on dozens of patients over thousands of hours in a controlled environment, with results published in scientific journals and subjected to peer review.
Researchers here say the Cuban treatment rests on outmoded theories about the cause of RP. They say the treatments could further damage a patient's eyes, and many believe research dollars could be better spent elsewhere.
But for the man with RP who knows that in a few years he won't be able to look into his wife's eyes, or the woman faced with never being able to see her child smile again, hearing people say the treatments have helped is all the proof they need.
While few are ready to proclaim the Cuban treatments a cure, they wonder why American researchers haven't paid more attention. Some note with disappointment, if not disgust, that no one associated with the RP Foundation visited Cuba until June -- about two months before an NBC newsmagazine segment spotlighted the controversial treatments.
A right to try it
"People have a right to [try] this, to find every avenue that they can explore -- for their lives and for their children," says Helen Harris, founder of RP International, a Los Angeles-based group that raises money for research into the disease and treatment for those who suffer from it. "I don't understand all this negativity about Cuba."
At the center of the controversy is Dr. Orfilio Pelaez, an unassuming, gray-haired doctor his proponents say is a dedicated surgeon who has succeeded where eye researchers throughout the world have failed. But his detractors warn that people traveling to Cuba not only risk fines and imprisonment -- traveling to the Caribbean island is still illegal for almost all Americans -- but also pay thousands of dollars for a treatment that may give them hope, but nothing more.
"We just can't recommend that people take their life savings or, worse than that, take very serious [health] risks for a treatment for which there is no scientific data to back it up," says Gordon Gund, owner of the Cleveland Cavaliers NBA basketball team and chairman of the board of the RP Foundation. "There are known examples of people who have had their sight damaged by this surgery."
But supporters decry the American medical community's refusal to take Dr. Pelaez seriously.
"The RP Foundation has not handled themselves well on this thing. They had not done anything" to try and substantiate Dr. Pelaez's claims, says Bob Stafford, a native Baltimorean now living in New Jersey. "There are too many people reporting good results" for the Cuban treatments to be ignored.
Mr. Stafford, who suffers from RP, says his vision increased substantially after being treated by Dr. Pelaez. While he can't swear the Cuban procedures work, he says the results should have been tantalizing enough to persuade the foundation to look into them more deeply. He's spent much of the past year doing just that.
"I have never claimed to have found a miracle in Cuba," he says, "only that people here need to get off their fannies."
Dr. Pelaez claims to have treated about 7,000 patients at the Cira-Garcia Clinic in Havana, using surgical procedures designed to increase the flow of blood to the retina. He claims a 70 percent success rate in halting the disease's progress -- provided treatments are started before the condition is too advanced.
Not a cure
Interviewed over the telephone from Havana, his associate, Dr. Mariadela Mendoza, stresses that Dr. Pelaez cannot cure RP. "The doctor is not a magician. He does not cure the disease, he contains it."
Dr. Mendoza admits her associate has been reluctant to reveal the details of the procedure, for fear other scientists may try to claim credit for its success. She says, however, that an international symposium has been set for Nov. 29-Dec. 1 in Havana, at which time full details of Dr. Pelaez's treatments will be revealed. Videotapes will be distributed, and six-, 12- and 18-month courses will be offered for ophthalmologists interested in learning the procedure.
"We're not trying to hide anything, we just want to make it public in an official way," she says.
The treatments, which Dr. Pelaez claims to have developed over 30 years of treating patients with RP, involve three steps:
Patients first are treated with a mild electric current through electrodes attached to the head. Then ozone gas, which cannot legally be used for medical treatments in the U.S., is mixed with the patients' blood. Finally, surgery is performed on the patients' eyes to help increase the flow of blood to the retina.
The treatments, performed over a three-week period, cost $7,040, Dr. Mendoza says, including food and lodging. The only additional cost involves getting to Cuba, which for most Americans means first traveling to Mexico City, where they can then board a plane for Havana.
RP Foundation officials say the treatments are based on disproven theories about the cause of RP and use procedures that have no proven medical benefit. What's worse, the treatments have never been subjected to the sort of rigorous evaluation that medical researchers traditionally require: careful, analytic record-keeping, with results published in scientific journals, where they can be reviewed and tested by other scientists.
"If I thought for a minute this treatment or any treatment anywhere throughout the world was real, I'd do anything for them," says Dr. Alan Laties, a professor at the University of Pennsylvania who was one of five men sent to Cuba by the RP Foundation in June to investigate the procedure. nnTC Dr. Laties, an ophthalmologist for 30 years and chairman of the RP Foundation's scientific advisory board, bristles at the notion that American researchers have been ignoring the Cuban research in deference to their own work. Any advances in RP research would be welcomed by the foundation, he insists, regardless of their origin.
"Bless anyone who can help," Dr. Laties says. "I've had children in my care in which the child was taken from his own class to a special class for the partially sighted. I don't know if you've ever seen that, had to take a child away from his playmates. If that doesn't tear your heart out, nothing will."
Last month, a segment on Dr. Pelaez and his RP treatments was broadcast on NBC's Now newsmagazine. The piece featured interviews with Dr. Pelaez and three of his American patients, all of whom claimed to see better after undergoing surgery in Cuba.
Melissa Cornick, who produced the piece for Now, says the show was not trying to comment on the treatment's effectiveness one way or the other. But it did raise the question of why the RP Foundation, which last year distributed $6 million in research grants throughout the world (but primarily in the U.S.), had never seriously investigated this procedure.
Pursuing answers
The answer, according to RP officials: They've been trying to find out more about the procedure since 1988, largely without success.
"Since 1991, we've been trying to get a team of researchers into Cuba, to see the studies, to check the files, look at the evidence, to see [if there is] anything valid about this treatment," Mr. Gray of the RP Foundation says.
In June, Dr. Pelaez agreed to work with the foundation to try and document the effectiveness of his procedures. Beginning in January, researchers will spend 12 to 18 months monitoring patients before and after the treatments. Last week, the foundation's board approved a grant for the University of Miami to study the results.
In a printed response to the NBC program, RP Foundation officials downplayed Dr. Pelaez's methods. Preparing the patient by using mild electric shocks "has no sound underlying medical or scientific basis," they said. Ozone is so unstable that it breaks down before blood can be pumped back into the body. And increasing blood circulation to the eye as a treatment for RP is based on outdated theories on the cause of the genetic condition.
According to the foundation, RP is caused not by damaged blood vessels in the eye or inadequate blood circulation, but rather by defects in the cells that make up the retina, or back of the eye.
"These are wonderful people," Dr. Laties says of his Cuban hosts. "I think honestly they've convinced themselves they're doing some good. Ask them if they were happy with the treatments, and the patients say yes."
'No difference'
Unfortunately, Dr. Laties says, patient charts, some of which are 40 pages long and go back 20 to 30 years, suggest the treatments have been ineffective.
"I turned every page," he says. "I saw the typical sad progression of RP, that these people had lost their vision. I could see in those charts no difference from the patients I've seen in the United States. No difference."
Although ophthalmologists here say they would love to see the treatment work, some also resent the spot Dr. Pelaez and the clamor generated by the Now segment have put them in. Traditionally in medicine, the burden of proof has been on the researcher doing the work. In this case, they say, he has done the work, while the rest of the medical community is being asked to provide the proof.
"It's the responsibility of people claiming the good results" to supply objective data, says Dr. Morton Goldberg, director of Johns Hopkins Hospital's Wilmer Eye Institute. "If it is true, the burden of proof is on them."
But Mr. Gund, 55, understands what motivates people to risk so much on an unproven medical technique administered in a country they're not legally allowed to enter.
Diagnosed with RP in 1965, he is now nearly totally blind, able simply to distinguish light from dark.
"You want to hang your hopes on any possible threads you can," says Mr. Gund, who was lured to Russia in 1970 by claims that doctors there could preserve his sight, "because you don't want to lose your sight and lose the hope you might be helped."