CHICAGO — A product developed by a retired University of Kentucky chemist and promoted to parents of children with autism is not a harmless dietary supplement, as claimed, but a toxic unapproved drug that lacks adequate warnings about potential side effects, the U.S. Food and Drug Administration says.
Potential side effects of the product, targeted for use by children, include hair loss and abnormalities of the pancreas.
The FDA sent Boyd Haley, the former UK chemist, a June 17 letter detailing five violations of the Federal Food, Drug and Cosmetic Act related to his product, OSR#1. Failing to correct such violations can result in fines, seizure of products and criminal prosecution.
The Chicago Tribune in January reported that the compound had been developed as an industrial chemical aimed at treating wastewater at sites such as acid mines and that it had not undergone rigorous testing to ensure it is safe and effective.
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The report was part of an investigation into risky, unproven autism therapies offered by health providers who say they can reverse the disorder.
OSR#1 is part of a family of chemicals called chelators originally developed for industrial purposes, according to a 2003 U.S. patent assigned to the UK Research Foundation.
The Web site of Haley's Lexington company, CTI Science, says Haley developed OSR#1 while working at UK, which licenses the underlying patent rights to his company.
Erin Holaday, a UK spokeswoman, confirmed Friday that the university receives royalties from the licensing agreement. She was not able to provide details about how much UK gets.
"While we might license to a company for medical-therapeutic uses, we do not in any way decide or endorse what approvals are needed or how they are used or marketed," Holaday said.
Haley supports the theory that autism can be caused by a mercury-based preservative that has been used in many vaccines given to children. That has made him a hero among parents who believe the theory, but repeated studies have found no connection between autism and vaccines.
Haley did not reply to repeated requests from the Chicago Tribune for an interview Wednesday. An FDA spokeswoman said the agency has not received any communication from Haley, who has 15 working days from the date of the letter to respond.
Last year, Haley told the Tribune: "I am not breaking any law. ... We are being very, very careful."
In the interview last year, Haley called the product "a food" that is "totally without toxicity." He said the compound had been tested on rats, and a food-safety study was conducted on 10 people. Asked to provide documentation of the research, he stopped communicating with the Tribune.
The FDA letter lists side effects recorded during Haley's animal studies: "soiling of the anogenital area, alopecia (hair loss) on the lower trunk, back and legs, a dark substance on lower trunk and anogenital area, abnormalities of the pancreas" and a rapid increase in normal cells contained in lymph nodes.
"It would be hard to imagine anything worse," said Ellen Silbergeld, an expert in environmental health who is studying mercury and autism at Johns Hopkins University's Bloomberg School of Public Health. "An industrial chemical known to be toxic — his own incomplete testing indicates it is toxic. It has no record of any therapeutic aspect of it, and it is being marketed for use in children."
OSR#1 has been promoted on autism Web sites including Age of Autism, where managing editor Kim Stagliano wrote of sprinkling the white powder on her three daughters' breakfast sandwiches and orange juice. "We've seen some nice 'Wows!' from OSR," she wrote.
In an e-mail, Stagliano wrote that she continues to support Haley, a regular speaker at autism recovery conferences.
"Having met Dr. Haley at conferences, including Autism One in Chicago last month, I continue to trust his science," she wrote on Wednesday. "I'm sure CTI Science will address the letter appropriately."
Dr. Arthur Grollman, a pharmacologist and director of the Laboratory for Chemical Biology at State University of New York at Stony Brook, said it is clear from the product's chemical structure that it is a "powerful chelator," a compound that binds to heavy metals such as mercury.
The FDA has approved several chelators as drugs to treat heavy-metal poisoning. Some doctors also use the drugs — which carry significant risks — to treat children with autism on the scientifically unfounded idea that their disorder is linked to toxic metals.
The magazine Medical Veritas in 2006 reported that Haley said he was interested in developing better chelators for people.
"We've made compounds that ... work tremendously" in a test tube, he was quoted as saying. "However, we've got to show that they're not toxic. That costs a lot of money, and it's very difficult to do; you have to have the right facilities. That's where we're hung up right now. The question is, 'How do we get somebody to do these studies?'"
In January 2008 Haley changed the name of his company from Chelator Technologies Inc. to CTI Science Inc., records show. Less than a month later, he notified the FDA he would be introducing the compound as a new dietary ingredient, a designation rejected by the FDA in its recent letter.
"Because OSR#1 does not bear or contain a dietary ingredient as defined (by the food and drug act), this product does not qualify as a dietary supplement," the letter states.
Instead, according to the letter, it is a new drug. Winning FDA approval requires proof of safety and efficacy through clinical trials, a process that can cost hundreds of millions of dollars and take many years.
"Anything might be a cure for anything else, but the odds are it will do nothing and it might very well be toxic," said Richard Mailman, a neuropharmacologist at Penn State University. "That is why drug discovery and development is so expensive."
Silbergeld said that the product represents a clear example of endangerment of public health and that the FDA should stop CTI Science from selling it immediately.
She drew a comparison to a city's drinking water system. If contamination is found, she said, "they turn off the pumps."
"They don't have to engage in a long discussion with you," Silbergeld said. "It would be hard to imagine a more clear example of immediate endangerment of public health. Turn off the pump."