Boston’s Autism Mini-Cluster: New Drugs and Diagnostics Target Mysterious Brain Disorders

Xconomy Boston — 

One day in my high school Spanish class, a kid who I was told had autism started hitting himself on the head repeatedly until our teacher sent him to the nurse. Looking back, I wonder whether he understood how abnormal his behavior was. But 17 years later, I’m encouraged by the growing cluster of outfits in the Boston area that are developing a host of new treatments and medical tests for people like my former classmate.

Autism spectrum disorders—which are brain disorders that make socializing difficult, among other symptoms—are a growing health concern in the United States. And it’s viewed as a huge potential market for drugs and diagnostics. There are an estimated one in 110 children in the country who have autism spectrum disorders, according to the U.S. Centers for Disease Control and Prevention. To date, there are neither approved drugs nor any easy ways to diagnose autism. Yet there have been discoveries at MIT and other research centers in the Boston area in recent years that have given people hope of improved treatments for autism—and reasons for investors to pump capital into startups focused on advancing these treatments.

“What we’re hoping to do is step into a complete void out there in terms of proven medical treatments for the condition,” says George Evans, the chairman and chief executive of Beverly, MA-based Cellceutix, which is seeking capital to advance its experimental compound for autism.

In the Boston area, which is rich with renowned academic centers and biotech companies, there are clusters of companies in almost every kind of disease: cancer, autoimmune diseases, cardiovascular conditions, and on and on. However, there appear to be a particularly high proportion of recent entrants in the area’s budding autism cluster, which tells me that I probably would have had trouble compiling much of a list of such firms back, say, in the early 1990s.

Here’s an alphabetized list of startups and research organizations in the Boston area that are trying to address the unmet medical needs of people with autism:

Waltham, MA

This MIT spin-off is developing the “Q Sensor,” which detects electrical signals on the skin to measure people’s emotional states. The sensor is being used in an autism sleep disorder study, according to the company. The firm, which revealed it raised $1.7 million in November 2009, says it sprouted from research at the MIT Media Lab aimed at helping people with autism. Its technology is also being developed as a marketing tool to detect consumers’ emotional states, according to its website.

Autism Consortium
Boston, MA

Housed at Harvard Medical School, this nonprofit group fosters collaboration among academics, families, and others with a stake in advancing research of autism spectrum disorders. There are a pair of familiar biotech investors on the group’s board of directors: Peter Barrett, a partner at Atlas Venture, is chairman of the consortium; and Alan Crane, a general partner at Polaris Venture Partners, is vice chairman. The group is hosting its second annual symposium that covers some of the latest innovations in autism research on October 26 at Harvard Medical School.

Beverly, MA

This small drug developer aims to make a big impact on the lives of people with autism. In a study done in rats, the firm’s experimental drug, KM-391, corrected abnormal behavior, improved brain plasticity, and increased serotonin levels. Earlier this month, I spoke to the company’s CEO, George Evans, a former general counsel for the prescription drug unit of the pharmaceutical giant Pfizer (NYSE: PFE). He and his team say the firm aims to launch initial human studies of its autism drug next year. Yet the firm’s lead drug candidate is an anti-cancer compound. (BusinessWeek wrote a feature in January about the drug industry’s increased interest in autism, though it didn’t mention Cellceutix’s nascent efforts.)

Harvard Medical School
Boston, MA

Watch for major papers on autism floating down from the ivory towers of this hub of medical research. Scientists at multiple local research institutions—Massachusetts General Hospital, the Broad Institute of Harvard and MIT, and others—are affiliated with Harvard or its Autism Consortium. For example, Harvard scientists were behind the recent discovery of genetic and chromosomal patterns behind certain cases of autism and related brain disorders.

Cambridge, MA

At MIT, there are researchers from multiple departments advancing autism research. Perhaps most notably, MIT professor and Nobel laureate Susumu Tonegawa led a study that showed that blocking a certain enzyme in mice reversed the symptoms of Fragile X syndrome, thought to be a cause of autism. The San Diego-based biotech startup Afraxis is in human testing of a drug that targets such enzymes as a potential treatment for Fragile X and autism.

Seaside Therapeutics
Cambridge, MA

This biotech startup, co-founded by the MIT neuroscientist Mark Bear in 2005, is also advancing treatments for autism and Fragile X syndrome. The company’s lead drug, STX209, is in mid-stage clinical trials for both Fragile X and autism spectrum disorders, according to a government website that tracks clinical trials. In September 2009, Luke covered the firm’s $30 million round of financing from a family investment fund. At that time, the firm had raised $66 million from the family fund, the National Institutes of Health, Autism Speaks, and the Fragile X Research Foundation, company CEO Randy Carpenter told Xconomy.

Waltham, MA

If I were asked to make a short list of the Boston area’s top diagnostics entrepreneurs, Stan Lapidus would definitely be on it. Lapidus—who is a founder of Cytyc, a Massachusetts provider of women’s health diagnostics that was acquired by Bedford, MA-based Hologic (NASDAQ: HOLX) for $6.2 billion in 2007—has recently formed SynapDx to develop blood-based tests for autism. In June, the firm announced that it had raised $9 million in a Series A funding round. Lapidus is president and chief executive of the firm.

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