By Andrew Pollack for the New York Times For several years, an experimental drug, secretin, has offered an unlikely ray of hope for some desperate parents of children with autism. Discovered accidentally by the mother of an autistic boy and licensed to a small biotechnology company led by the father of two autistic girls, secretin has advanced through clinical trials even as study after study showed it had little or no effect. Now, the largest and most definitive clinical trial of secretin has been completed, and it, too, showed that the drug was no better than a placebo in improving the social interaction of young children with autism. The failure, announced yesterday by the company, Repligen, of Waltham, Mass., casts doubt on whether secretin will ever get to market as a treatment, dealing a blow to scores of parents and some doctors who advocated its development. "We're horrified at the thought of this being dropped," said Jan Henry of Chattanooga, Tenn. The drug, which Ms. Henry said she bought from overseas sources and others at up to $10,000 a year, had helped her son Andrew speak and sleep. "It's life to us," she said. "My parents mortgaged their home for us," she added. The secretin story shines a light on the difficulty of developing a drug for autism, an often debilitating condition whose incidence seems to be rising sharply and for which there are no approved drugs. Experts estimate that the United States has more than 100,000 autistic children. Children with the condition have difficulty communicating or forming relationships, and they often have repetitive actions. The development of the drug has at times been a battle between the hopes of parents and the more dispassionate science of some experts. Some advocates say the experts too easily dismissed the drug because it was developed outside conventional academic channels or drug companies. "This is the type of thing you see all the time in medicine, where things that get strong patient and parental advocacy and don't come through the normal channels of discovery get dismissed," said Dr. Jeff Reich, a neurologist and senior analyst at Merlin Biomed, an investment fund in New York that owns shares of Repligen. Some experts say that parents see what they want to see and that the drug simply does not work when measured more objectively. "We now have multiple, multiple studies showing secretin is not better than placebo," Dr. Fred R. Volkmar, a professor of child psychiatry at Yale, said. "I would say it's time to shift our attention elsewhere." Secretin is a natural hormone that stimulates the pancreas to release juices that aid digestion. A synthetic version is approved for use in diagnosing pancreatic disorders. In 1996, Parker Beck, an autistic boy in New Hampshire, was given secretin to help diagnose gastrointestinal problems, which afflict many children with autism. His mother, Victoria Beck, soon noticed an improvement in his development and began to suspect the secretin. When the Becks tried to obtain more treatments for Parker and his medical records from the University of Maryland, where he had the diagnostic procedure, they were rebuffed, Mrs. Beck said. The family then learned that the university was applying for a patent on using secretin in autism. When the Becks told the university their history, the university listed her as an inventor and assigned her the patent rights. She in turn licensed them to Repligen, whose president, Walter C. Herlihy, has two autistic daughters. The news was reported on television and in newspapers in 1998 and 1999 and led to a flurry of interest in secretin. Some parents obtained the version for pancreatic disorders. Some ordered it from overseas. But some small studies sponsored by the National Institutes of Health showed that secretin had no benefit beyond that from placebos. Repligen conducted a larger trial that involved three doses. That test was also not convincing but the drug seemed to show some effect in younger children. The company began a Phase III study, usually the last stage before regulatory approval, involving 132 younger children, from 2 years 8 months to 4 years 11 months, who were given six doses each intravenously. The results announced yesterday showed that the recipients of the drug did not improve more than those who received the placebo when evaluated by parents or psychologists. Repligen stock lost more than 40 percent of its value, dropping $1.77, to $2.39. "It's a sad day," Dr. Herlihy, the chief executive, said in an interview. He said the company would decide whether to keep developing the drug for autism after further analyzing the data and conferring with the Food and Drug Administration in the coming months. But Dr. Herlihy and some doctors in the trial are not ready to concede. They said one reason that the trial might have failed is that it is difficult to measure improvements in autistic children. The drug did show an effect for a subset of children with higher I.Q.'s. Also, the term "autism" probably covers a wide range of conditions. Secretin may work for some patients, but the effects would be diluted in testing on a broad population. "I remain convinced there's a subset of children who benefit from secretin," said Dr. Paul Millard Hardy, a behavioral neurologist in Hingham, Mass., in the trial. One of his patients, Jeremy McGlone of Kingston, Mass., was in the trial. Jeremy's father, Shayne McGlone, acknowledged that he did not know whether his son received the drug or the placebo, although he speculated that it was the drug. "He started looking at me in the eyes a lot more," Mr. McGlone said. "As a father, that's something you want your kid to do." Parker Beck, 10, has improved greatly and no longer takes secretin, his mother said. Anticipating royalties from secretin sales, Mrs. Beck and her husband, Gary, set up the National Autism Outreach Foundation last summer in Highlands Ranch, Colo. But now the royalties will apparently not arrive. "We'll find other ways to raise
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