By Mary Welch

Repligen Corp. acquired exclusive worldwide rights to patent applications for the use of secretin, a natural hormone that some parents say is helpful in treating their autistic children.

The Needham, Mass.-based company, which acquired the patents from Victoria Beck and the Autism Research Institute, of San Diego, intends to start clinical trials within the year, pending FDA approval.

¿There are about 400,000 to 500,000 [autistic] patients in the U.S.,¿ said Walter Herlihy, Repligen¿s president and CEO, ¿and if secretin works and is given to patients on a frequent basis ¿ which would be similar to administering insulin or growth hormones ¿ then this could be a significant market.

¿There are toxicity issues that we will want to see,¿ he added. ¿But we understand secretin already, because it is in use for other indications. We do have to find the optimal dose and level of dosing. One could argue about a low dose given daily being better than one given every couple of weeks or months. That¿s one of the things we will determine.¿

Repligen¿s stock (NASDAQ:RGEN) vaulted 37 percent Tuesday, closing at $2.312. A front-page story in The Wall Street Journal explained the research into secretin and Repligen¿s plan to develop the hormone.

The company, which reported fiscal year 1998 revenues of $2.38 million, will also look into ways to formulate secretin so it can be taken orally, a preferable way for children. Herlihy said no decision has been made about the number of children or sites participating in the trials, which are expected to last at least a year.

So far, no criteria have been established ¿ although in trials under way elsewhere, more severely autistic children are being treated rather than those who are higher-functioning. ¿That does make sense, to perhaps continue doing trials with the more severely autistic, the nonverbal, but nothing has been decided,¿ Herlihy said.

Nor has the company decided whether to pursue a fast-track designation with the FDA. ¿It¿s hard to judge that now,¿ Herlihy said. ¿It certainly is an unserved patient population.¿ There is no medical treatment for autism.

Secretin is a naturally occurring intestinal hormone that aids in the digestive process. It is isolated from pigs and used to diagnose certain gastrointestinal disorders. Part of Repligen¿s challenge is to develop a synthetic human form of secretin.

The hormone¿s potential use in treating autism has been the subject of the well-publicized effort by Victoria Beck, of New Hampshire, who discovered the potential secretin-autism connection when her autistic son was treated with the hormone for severe gastrointestinal problems.

When the boy suddenly developed speech and a better ability to relate to people, Beck sought to convince the medical community of the connection. Met with apathy and resistance, she posted her findings on the Internet. Eventually, a national TV news magazine did a story on her situation ¿ after which parents, trying to get secretin for their children, inundated doctors and medical centers.

Scores of physicians have since administered secretin ¿off-label¿ to more than 1,000 patients. Positive results ¿ such as better gastrointestinal function, improved sleep, better socialization skills and eye contact ¿ have been widely reported. Improved speech, general awareness and increased capacity to learn were seen in some patients.

Some Studies Already Under Way

Herlihy learned about secretin as a means of helping the gastrointestinal function in his seven-year-old autistic daughter, and became interested in the hormone. ¿We learned about it on the Internet and heard reports about marvelous improvements in gastrointestinal functions,¿ he said. ¿Our ears perked up because of our daughter. This was pre-Dateline¿ [the television show that reported the story]. We were convinced there was an effect that was not imagined.¿

Several studies are under way at major medical centers, including Scottish Rite Hospital, in Atlanta, in an attempt to better understand the effectiveness of secretin in the treatment of autism.

¿No question about it,¿ Herlihy said. ¿After that television show, secretin became a hot potato. Everybody wanted it. It¿s the type of thing people would be willing to mortgage their house for.¿

Autism, the third most common developmental disability in the U.S., is a neurological disorder that appears during the first three years of life. It more commonly afflicts boys, and sufferers have difficulty with verbal and nonverbal communication, social interaction and imaginative activity at play.

The disorder is becoming better known, thanks in part to publicity efforts by football quarterbacks Dan Marino and Doug Flutie, both of whom have sons with autism.

¿I don¿t know why autism has been ignored [by the research community],¿ Herlihy said. ¿Perhaps it is because it is such a complex disease, there has not been a lot of funding for research.¿ n