5/25/2010 Eric Schoeniger

Connecting the Dots to Treat Autism

A new drug developed by Joan Fallon '79 could prove to be a life-changing treatment

Autism: It's a diagnosis no parent wants to hear. But around 20 years ago, Joan Fallon, D.C., '79 began to see more and more autism symptoms among patients in her pediatric chiropractic practice. "These were young children coming in with symptoms like no eye contact, lack of speech, socialization issues," Fallon says. "Clearly, it was autism."

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Other than this diverse set of symptoms, though, nothing appeared to link the children. But soon Fallon noticed a common trait: Many of these patients avoided foods rich in protein. Subsequent testing revealed that a large number lacked a protein-digesting enzyme. As a result, they could have low levels of amino acids, the building blocks of neurotransmitters and neuroreceptors in the brain.

For Fallon, it was a profound insight—and one that launched her journey from chiropractor to drug clinician to researcher to entrepreneur. Today her company, Curemark, is in Phase III research trials with a new drug that could bring life-changing treatment to thousands of children with autism.

Linking Symptoms with Physiology

Autism is a range of developmental brain disorders that typically involve language delays, communication problems and impairment of social interaction. Symptoms usually appear before age 3, and they usually last a lifetime. About 1 percent of children in the United States will be diagnosed with some form of autism, according to the Centers for Disease Controland Prevention.

As many as 85 percent of kids with autism have gastroin-testinal problems such as colitis, chronic constipation and recurrent abdominal pain. Many are picky eaters. Among her own pediatric patients, Fallon noticed that many, if not most, avoided protein. She began to wonder if there was a connection between diet and disorder.

Eventually, Fallon ran tests on hundreds of children with and without autism. What she found was compelling. A large percentage of children with autism, and especially those who did not have autism present with another type of disorder, had abnormally low levels of a protease, or protein-digesting enzyme, produced by the pancreas.

The results suggest that these children cannot digest protein and therefore could lack sufficient amino acids. Amino acids are the building blocks of new proteins, including neurotransmitters and neuroreceptors, the brain's chemical messengers. Because some amino acids can be obtained only by ingesting proteins, Fallon hypothesized that the children who had autism likely would have low levels of these amino acids. It was this deficiency, Fallon believed, that could be contributing to the autism symptoms she saw in her pediatric patients.

"The fact that child after child after child came back with the same positive findings, that was a real 'aha' moment," Fallon says. "The physiology of these children was matching up with their symptoms."

Finding Renewed Purpose

Fallon was running a private pediatric practice with three New York offices. But now she had a decision to make. She could continue her successful career. Or she could devote her energy to potentially developing a treatment for children for whom effective treatments were painfully few.

"My practice was treating children with problems," Fallon explains. "To see a child with some sort of impairment was not unusual. But it was the constellation of these symptoms that was unusual. And the opportunity to help so many children at one time became paramount."

Fallon began to examine enzyme replacements. Existing enzyme replacements were designed primarily for fat digestion to treat individuals with cystic fibrosis. "There was nothing like this on the market to treat autism," Fallon says. "So, we simulated a high-protease product and started giving these children enzymes with what I thought they needed. We did this work with a lot of children and a lot of physicians."

The results were "extremely positive," according to Fallon. She went back to school and took classes toward a master’s degree in clinical investigation from Harvard Medical School’s joint program with Massachusetts General Hospital. At the same time, she began the development work for a treatment based on her discovery.

Fallon collaborated with experts in biotechnology and drug research and delivery to formulate a drug that could be brought to market. The next step was to found her company, Curemark, to develop and, she hoped, commercialize an enzyme that could help children with autism. The drug she developed is called CM-AT.

Curemark, based in Rye, N.Y., now has 10 full-time employees "and is partially virtual," Fallon says. "We have lots of regulatory people and consultants on the outside." The company has been issued four patents and has applied for nearly 40 more, and it has raised $6.5 million in private funding. Fallon is Curemark's CEO.

On the Fast Track

CM-AT is now in Phase III trials at 12 locations across the country, including Mount Sinai School of Medicine in New York and Hershey Medical Center in Pennsylvania. The drug is being tested in 170 children ages 3 to 8. The trials are randomized, double-blind, placebo-controlled tests in which test subjects are randomly assigned to the experimental group and the control group, and neither the test subjects nor the researchers know who belongs to which group.

The U.S. Food and Drug Administration (FDA) allowed CM-AT to move directly to Phase III trials. In February, Curemark reported that CM-AT had been designated by the FDA as a Fast Track drug. The Fast Track process is intended to facilitate the development and expedite the review of drugs that treat a serious disease or fill an unmet medical need. The goal is "to get important new drugs to the patient earlier," according to the FDA website.

The trials are expected to wrap up at the end of 2010. Pending the results of the data, Curemark hopes to submit a New Drug Application for FDA approval in 2011.

Coming Full Circle

In many ways, Fallon's path from chiropractor to researcher to entrepreneur started at Franklin & Marshall. "A liberal arts education was a defining factor, because it underscores the possibilities," she says. "I was a biology major heavily rooted in physiology, and that was very transportable."

But there is another F&M connection: Curemark's vice president of operations is Elisa Zinberg, D.C., '83.

Zinberg was running a successful chiropractic practice in midtown Manhattan when she broke her wrist, "which is fairly impractical for a chiropractor," she jokes. Unable to continue her practice, she became the first female consultant for The Master's Circle, a health and wellness practice-management firm. She soon became the company's first chief operating officer and helped expand its business worldwide.

"When we started Curemark and started to raise some money, it was clear I needed a businessperson to run the company," Fallon says. "Lisa had been very successful as COO in her former company. She took it from a small company to a large one with worldwide presence. That is what I needed, an individual with business acumen and an understanding of a startup." She approached Zinberg about coming on board.

"I thought, what a wonderful opportunity," Zinberg says, "to do something that could have an impact on the world, to help these children with autism and their families. So I said yes."

Both Fallon and Zinberg knew of each other's work. "If you're a chiropractor and you have a pulse, then you know what Joan has done in the profession," Zinberg says. But at the time, neither was aware they were both F&M graduates.

Zinberg echoes Fallon when she talks about the impact F&M had on her career. "My liberal arts education helped me with how I think and how I look at things," she says. "And my psych degree helped me think in a way that's logical and detailed-oriented and results-driven."

Zinberg also highlights her experiences as a student-athlete. "Playing sports had a big role, in retrospect, in helping me become who I've become," she believes, "because of the team interaction, because of the competition and because it requires you to be organized and disciplined and prioritize what you need to do." Fallon also was a student-athlete at F&M.

It is that kind of focus that helped Fallon connect the dots between diet and autism, between symptom and physiology. And it is a single-mindedness both Fallon and Zinberg will need as they work to grow a drug company. That may be especially true for a treatment targeted at autism, as many in the autism community have grown skeptical of new drugs after years of failed "cures" and unfulfilled promises.

But Fallon, for one, is not worried about skepticism. "It's all about the data," she insists. "The double-blind, randomized, placebo-controlled study is the gold standard for anything like this. It doesn't matter whether people are skeptical or not skeptical. The bottom line is, we're doing the gold standard of studies. And we hope it shows the drug to be efficacious."
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