IBJNews

Firm joins race with IU autism drug

Back to TopCommentsE-mailPrintBookmark and Share

Dr. Craig Erickson, a pediatric psychiatrist at the Indiana University School of Medicine, has found initial success using an alcohol-dependency drug to improve the language and social skills of patients with autism.

Now IU has licensed Erickson’s patent on the use of the drug to Indianapolis-based startup Confluence Pharmaceuticals Inc., which aims to bring the medication, called acamprosate, to market.

Confluence founders Boyd Sturdevant and Steve Johns are pitching their company to various investors, trying to raise the $1.5 million they need to move the drug into the first round of human testing.

Erickson Dr. Craig Erickson is director of Christian Sarkine Autism Treatment Center.

It’s a local example of what is a growing push globally to develop drugs to treat autism. Major drugmakers like Novartis, Pfizer and Roche, as well as small outfits like Confluence and Massachusetts-based Seaside Therapeutics, are all racing to find the first drug to significantly improve the core symptoms of autism.

Indianapolis-based Eli Lilly and Co. says its scientists are working with academic institutions to understand autism, but the company has disclosed no drugs in development to treat the disease.

Winning the autism drug race would be a financial boon for IU and the Christian Sarkine Autism Treatment Center, which Erickson directs. Market research conducted by a class of MBA students at IU’s Kelley School of Business predicted sales of acamprosate could reach as high as $400 million a year in the United States and even more if launched worldwide.

“The clinic here would stand to make considerable money,” said Erickson, 34, from his office on the fourth floor of Riley Hospital for Children in Indianapolis. The center funds a significant portion of the salaries of its 15-person staff through a series of short-term contracts with drug companies and federal, state and foundation grants.

According to the U.S. Centers for Disease Control and Prevention, one in every 110 children in the United States has an autism spectrum disorder, a phrase that includes related developmental disorders such as Asperger’s syndrome.

Patients with autism often have difficulty speaking and with social skills, struggling to make eye contact with others and to say things appropriate to an ongoing conversation.

The CDC estimates that 730,000 Americans under the age of 21 have an autism disorder. Even greater prevalence of the disease has been documented in areas of Japan, Sweden, the United Kingdom and Norway.

Researchers have little understanding of what causes autism, making it difficult to develop drugs to treat it. Antipsychotic drugs like Risperdal and Abilify are approved to treat aggression in autistic patients. But for improving the core symptoms of the disease, behavioral therapy and diet changes are some of the most common treatments.

Erickson’s research has focused on patients with a genetic mutation called fragile X syndrome. It is the leading known cause of autism, but accounts for only roughly 5 percent of all cases.

In the past decade, researchers found that fragile X syndrome causes a neurotransmitter in patients’ brains to be overly active. To reduce the activity of this chemical, called glutamate, researchers have tried to develop drugs that block a portion of brain neurons, called the mGluR5 receptor, where glutamate attaches itself.

In late 2008, Erickson read journal articles on alcohol treatment research, which had determined that acamprosate blocked the mGluR5 receptor. So he decided to try the drug in his patients, including those with fragile X and those without.

The results have been “remarkable,” according to Erickson. All patients showed improvement in language skills, speaking in longer sentences, more appropriate to the social setting. Eye contact improved in a couple of patients, but not in several others.

In Erickson’s case study notes, he describes a 23-year-old male, called C, who took acamprosate for 16 weeks:

“C’s language pragmatics were described as improved, marked by an increased ability to convey his needs and interests in socially appropriate ways. He was described as asking more appropriate and not just repetitive questions, and generally ‘talking more on topic.’”

The psychiatrist also has given acomprosate to patients as young as 6 years old, reporting positive effects. In only one case so far, an 11-year-old boy with autism, did the improvements wane during the course of taking acamprosate.

Erickson has tried acamprosate in only two dozen patients so far. Several large, expensive clinical trials lie ahead before it could be approved by U.S. regulators for sale.

He was confident about trying the drug in the first place because it had already proved safe for decades as a dampener of withdrawal effects in alcoholics trying to beat a drinking addiction.

But the patents on acomprosate, also known as Campral, have now expired, making it widely available in a cheap generic form. Drugstore.com lists 180 tablets of the drug for a price of $161.24.

That fact was enough to scare off the half dozen or so pharmaceutical and biotech firms who expressed an interest in licensing Erickson’s patent, according to Brad Fravel, a business development manager in the IU Research & Technology Corp., which handles IU’s patenting and licensing work.

“The commercial potential on it has always been a little bit of a double-edged sword,” Fravel said. “If you have something [for autism], it’s almost a no-brainer that there’s going to be a market for it.” But, he added, “A generic is a challenge.”

Confluence’s strategy around that problem is to reformulate acomprosate so it can be more easily absorbed by children and into more kid-friendly doses. The drug currently is poorly absorbed, meaning Erickson had to give his patients a lot of it to have an effect. Some patients developed side effects, like nausea and vomiting.

Reformulating the drug should allow Confluence to win a patent on the new drug’s “composition of matter,” a version of a patent much harder to challenge in court than the use patent Erickson currently holds.

Fravel was worried acamprosate would die after the drug companies all said no. But then Sturdevant and Johns approached IU about a licensing deal.

Sturdevant, a Bloomington resident, founded and ran Employee Counseling of Indiana, which provided mental health and “human problem-solving” services to Midwestern employers, their workers and their families. He sold the company in 2003 and has done consulting work since then.

Johns was CEO of Carmel-based eGix Inc., which sold complex telephone systems, from 1990 to 2008.

Sturdevant and Johns spent a year studying acamprosate and its potential markets before saying yes. They intend to apply for FDA approval for acamprosate as an orphan drug for a rare disease—fragile X syndrome. That could allow them to enroll fewer patients in clinical trials, saving money and time. It also makes them eligible for grants and tax incentives.

At the same time, they liked that the drug already has proved safe in humans and has a patient population eager for a treatment.

“This was a market that had an unmet need, was highly motivated and highly organized,” Johns said of autism. “Nobody tried to talk us out of it.”•

ADVERTISEMENT

Post a comment to this story

COMMENTS POLICY
We reserve the right to remove any post that we feel is obscene, profane, vulgar, racist, sexually explicit, abusive, or hateful.
 
You are legally responsible for what you post and your anonymity is not guaranteed.
 
Posts that insult, defame, threaten, harass or abuse other readers or people mentioned in IBJ editorial content are also subject to removal. Please respect the privacy of individuals and refrain from posting personal information.
 
No solicitations, spamming or advertisements are allowed. Readers may post links to other informational websites that are relevant to the topic at hand, but please do not link to objectionable material.
 
We may remove messages that are unrelated to the topic, encourage illegal activity, use all capital letters or are unreadable.
 

Messages that are flagged by readers as objectionable will be reviewed and may or may not be removed. Please do not flag a post simply because you disagree with it.

Sponsored by
ADVERTISEMENT

facebook - twitter on Facebook & Twitter

Follow on TwitterFollow IBJ on Facebook:
Follow on TwitterFollow IBJ's Tweets on these topics:
 
Subscribe to IBJ
  1. By Mr. Lee's own admission, he basically ran pro-bono ads on the billboard. Paying advertisers didn't want ads on a controversial, ugly billboard that turned off customers. At least one of Mr. Lee's free advertisers dropped out early because they found that Mr. Lee's advertising was having negative impact. So Mr. Lee is disingenous to say the city now owes him for lost revenue. Mr. Lee quickly realized his monstrosity had a dim future and is trying to get the city to bail him out. And that's why the billboard came down so quickly.

  2. Merchants Square is back. The small strip center to the south of 116th is 100% leased, McAlister’s is doing well in the outlot building. The former O’Charleys is leased but is going through permitting with the State and the town of Carmel. Mac Grill is closing all of their Indy locations (not just Merchants) and this will allow for a new restaurant concept to backfill both of their locations. As for the north side of 116th a new dinner movie theater and brewery is under construction to fill most of the vacancy left by Hobby Lobby and Old Navy.

  3. Yes it does have an ethics commission which enforce the law which prohibits 12 specific items. google it

  4. Thanks for reading and replying. If you want to see the differentiation for research, speaking and consulting, check out the spreadsheet I linked to at the bottom of the post; it is broken out exactly that way. I can only include so much detail in a blog post before it becomes something other than a blog post.

  5. 1. There is no allegation of corruption, Marty, to imply otherwise if false. 2. Is the "State Rule" a law? I suspect not. 3. Is Mr. Woodruff obligated via an employment agreement (contractual obligation) to not work with the engineering firm? 4. In many states a right to earn a living will trump non-competes and other contractual obligations, does Mr. Woodruff's personal right to earn a living trump any contractual obligations that might or might not be out there. 5. Lawyers in state government routinely go work for law firms they were formally working with in their regulatory actions. You can see a steady stream to firms like B&D from state government. It would be interesting for IBJ to do a review of current lawyers and find out how their past decisions affected the law firms clients. Since there is a buffer between regulated company and the regulator working for a law firm technically is not in violation of ethics but you have to wonder if decisions were made in favor of certain firms and quid pro quo jobs resulted. Start with the DOI in this review. Very interesting.

ADVERTISEMENT