Public-Private Effort Seeks to Expedite Discovery of Autism Drugs

Under a contract with the institute, U.C.L.A. will form a network of researchers at other academic centers that will try to identify promising new and older drug compounds quickly, and conduct early tests to see if they merit additional investment.

The program, part of the “Fast Fail” initiative at the institute, aims to determine within weeks whether a drug works, rather than the years it traditionally takes to evaluate a new drug.

“The whole idea is just getting much better in these early phases at identifying drugs that are going to be efficacious and safe, and thereby greatly speeding the development of effective new therapies and reducing the overall cost,” said Dr. James McCracken, who is leading the effort at U.C.L.A. as director of the division of child and adolescent psychiatry at the Semel Institute for Neuroscience and Human Behavior.

The number of diagnosed cases of autism, Asperger’s syndrome and related disorders in children has been growing in recent years, largely because of increased awareness. A recent report by the Centers for Disease Control and Prevention and the Health Resources and Services Administration concluded that one in 50 children aged 6 to 17 had been found to have autism or a related disorder, a 72 percent increase since 2007.

Although more cases are being diagnosed, no drugs are approved to treat the core symptoms of the disorders, which are characterized by delays in developing effective communication and social skills. Other drugs often prescribed to people with the disorders treat difficult behaviors like aggressiveness, hyperactivity and irritability.

Dr. McCracken said developing effective treatments had been difficult because the underlying causes were poorly understood until the last few years, and some prominent efforts had failed. In 2004, the experimental drug secretin, developed by RepliGen, did not show that it worked in an advanced clinical trial, disappointing parents of children with autism who had placed their hopes in the drug.

Several major drug companies, including GlaxoSmithKline and AstraZeneca, have scaled back their research in the neurosciences because of the high failure rate, Dr. McCracken said.

Developing drugs to treat neurological disorders is difficult, in part because brain science is still evolving. The field is littered with drugs that scientists had hoped would be effective against diseases like Alzheimer’s and schizophrenia but that performed poorly in clinical trials.

Despite the setbacks, scientific advances in understanding the genetic underpinnings of autism have accelerated, leaving the door open for new drug discoveries, said Robert H. Ring, vice president of translational research at Autism Speaks, a patient advocacy group.

“Autism spectrum disorder is the brave new world of medicine development, and most companies out there — despite a lot of the retraction you’re seeing — they do recognize autism as a clear area of opportunity,” said Mr. Ring, who serves on a committee that helps select which compounds the U.C.L.A. program will test.

Some companies are pursuing treatments. Seaside Therapeutics, a private company in Cambridge, Mass., is developing drugs to treat autism and a form of mental retardation known as fragile X syndrome in a partnership with Roche.

“The approach that people have taken over the years is, ‘This person looks anxious, I’ll give them a drug I use to treat anxiety,’ ” said Dr. Randall L. Carpenter, a co-founder of Seaside and its chief executive. “We hope to treat the underlying molecular abnormality.”

Dr. McCracken said the program would identify four to eight compounds and run them through small trials in humans, testing how the drugs are absorbed and how they affect brain wave patterns that scientists say they believe are linked to autism.

“It’s taken a really long time to kind of crack open and begin to understand part of the disorder of brain biology that underpins autism,” Dr. McCracken said. “This is, to me, the most exciting time because we understand so much more than we did even five or 10 years ago.”

Public-Private Effort Seeks to Expedite Discovery of Autism Drugs

Under a contract with the institute, U.C.L.A. will form a network of researchers at other academic centers that will try to identify promising new and older drug compounds quickly, and conduct early tests to see if they merit additional investment.

The program, part of the “Fast Fail” initiative at the institute, aims to determine within weeks whether a drug works, rather than the years it traditionally takes to evaluate a new drug.

“The whole idea is just getting much better in these early phases at identifying drugs that are going to be efficacious and safe, and thereby greatly speeding the development of effective new therapies and reducing the overall cost,” said Dr. James McCracken, who is leading the effort at U.C.L.A. as director of the division of child and adolescent psychiatry at the Semel Institute for Neuroscience and Human Behavior.

The number of diagnosed cases of autism, Asperger’s syndrome and related disorders in children has been growing in recent years, largely because of increased awareness. A recent report by the Centers for Disease Control and Prevention and the Health Resources and Services Administration concluded that one in 50 children aged 6 to 17 had been found to have autism or a related disorder, a 72 percent increase since 2007.

Although more cases are being diagnosed, no drugs are approved to treat the core symptoms of the disorders, which are characterized by delays in developing effective communication and social skills. Other drugs often prescribed to people with the disorders treat difficult behaviors like aggressiveness, hyperactivity and irritability.

Dr. McCracken said developing effective treatments had been difficult because the underlying causes were poorly understood until the last few years, and some prominent efforts had failed. In 2004, the experimental drug secretin, developed by RepliGen, did not show that it worked in an advanced clinical trial, disappointing parents of children with autism who had placed their hopes in the drug.

Several major drug companies, including GlaxoSmithKline and AstraZeneca, have scaled back their research in the neurosciences because of the high failure rate, Dr. McCracken said.

Developing drugs to treat neurological disorders is difficult, in part because brain science is still evolving. The field is littered with drugs that scientists had hoped would be effective against diseases like Alzheimer’s and schizophrenia but that performed poorly in clinical trials.

Despite the setbacks, scientific advances in understanding the genetic underpinnings of autism have accelerated, leaving the door open for new drug discoveries, said Robert H. Ring, vice president of translational research at Autism Speaks, a patient advocacy group.

“Autism spectrum disorder is the brave new world of medicine development, and most companies out there — despite a lot of the retraction you’re seeing — they do recognize autism as a clear area of opportunity,” said Mr. Ring, who serves on a committee that helps select which compounds the U.C.L.A. program will test.

Some companies are pursuing treatments. Seaside Therapeutics, a private company in Cambridge, Mass., is developing drugs to treat autism and a form of mental retardation known as fragile X syndrome in a partnership with Roche.

“The approach that people have taken over the years is, ‘This person looks anxious, I’ll give them a drug I use to treat anxiety,’ ” said Dr. Randall L. Carpenter, a co-founder of Seaside and its chief executive. “We hope to treat the underlying molecular abnormality.”

Dr. McCracken said the program would identify four to eight compounds and run them through small trials in humans, testing how the drugs are absorbed and how they affect brain wave patterns that scientists say they believe are linked to autism.

“It’s taken a really long time to kind of crack open and begin to understand part of the disorder of brain biology that underpins autism,” Dr. McCracken said. “This is, to me, the most exciting time because we understand so much more than we did even five or 10 years ago.”

Site Insight: Are Big Clients Worth the Effort for This Web Start-Up?

For me, writing this column brings two fascinating benefits: the people I get to meet and the information they share. Because my days at AbesMarket.com are filled with e-commerce decisions, I appreciate the value of baseline data, and knowing how visitors act on other sites helps me gauge our performance. I hope you, too, can benefit from these ideas and statistics.

In last week’s post, we met Recruiterbox, a three-year-old start-up that organizes the recruiting process. Having grown through smart and frugal marketing, Raj Sheth and his co-founders are debating how to guide their company to the next set of customers. Their focus has been on companies with fewer than 50 employees. Larger companies have more to spend on recruiting, but also demand a more resource-intense sales approach.

We left off asking readers for feedback on which market Recruiterbox should emphasize. We also asked whether you thought the site was easy enough to understand.

One comment suggested focusing on the potential of the small- to medium-size business market. In fact, the commenter went so far as to suggest focusing on start-ups and small enterprises in India and Silicon Valley. His point was that the $100-per-month cost would be easy for these companies to digest given the value Recruiterbox offers. I agreed wholeheartedly that the company should avoid the temptation of catering to larger companies. While the appeal of increasing revenue per client can be tough to resist, it comes with three key obstacles.

Larger companies mean more decision makers, which translates into a significantly longer sales cycle.Recruiterbox does not have an outbound sales team to sell into that market. This is a significant cost savings but makes outreach to bigger companies tougher.Large companies are often swayed by reputation, and because Recruiterbox is relatively new to the market, it does not have the corporate credibility that a human resources executive may be expecting.

Other comments provided feedback on how the site functions. I challenged Mr. Sheth with these thoughts, and he shared some of his core site statistics to support his answers.

Is the Try It for Free button too easy to miss?

We used color to help make that button stand out. It is the only blue button on the site. Eighty-four percent of the people who hit the homepage end up clicking on the button. That works for us although we also want people to watch the video to get a feel for the product. On average 7 percent of the visitors that click on the Try It for Free button sign up for the trial version.

At three minutes, isn’t the video too long?

We have remade this video three times. The actual site demo takes 15 to 20 minutes, so it was quite an effort to edit that video down to three minutes. Seven percent of our visitors watch the whole video — or roughly 2,000 of our 30,000 monthly visitors. We don’t have stats on the number of people who start to play it and then stop in the middle. We haven’t made the investment in producing a one-minute concept video although we do understand the value of one. The positive side is that after watching the current video many of our potential customers can jump right in because they understand how to use the application.

On the homepage, why not have the Reasons You’ll Love It section follow the video instead of the Social Proof? Why not put some of that information on a separate page.

This isn’t a focus because so few people scroll down below the fold. Approximately 90 percent of the visitors click on either the Try It for Free button or the video. As to potentially separating those sections into different pages, we believe having all of the key information on the homepage raises our search engine optimization ranking.

Can the text on the homepage explaining what you do be more concise?

We have struggled with that line, appreciate that feedback and agree it could be improved.

Why don’t you advertise on Monster.com or other job sites?

We have tried partnering with job sites but have not made progress. We aren’t relevant to the job seeker, only to the employer, so we are looking to structure a relationship that only shows our ads to employers.

How do you use customer feedback?

We receive a few hundred e-mails per month from paying and trial customers. This is in addition to the feedback we get during our two to three demo calls every day. It is a big advantage to be this close to our customer. We are careful to filter the feedback based on the size of the company — large companies provide different feedback than their smaller counterparts. We compile the feedback, and it drives our development road map, what features we add and when.

Would you like to have your business’s Web site or mobile app reviewed? This is an opportunity for companies looking for an honest (and free) appraisal of their online presence and marketing efforts.

To be considered, please tell us  about your experiences — why you started your site, what works, what doesn’t and why you would like to have the site reviewed — in an e-mail to youretheboss@abesmarket.com.

Richard Demb is co-founder of Abe’s Market, an online marketplace for natural products that is based in Chicago.

Site Insight: Are Big Clients Worth the Effort for This Web Start-Up?

For me, writing this column brings two fascinating benefits: the people I get to meet and the information they share. Because my days at AbesMarket.com are filled with e-commerce decisions, I appreciate the value of baseline data, and knowing how visitors act on other sites helps me gauge our performance. I hope you, too, can benefit from these ideas and statistics.

In last week’s post, we met Recruiterbox, a three-year-old start-up that organizes the recruiting process. Having grown through smart and frugal marketing, Raj Sheth and his co-founders are debating how to guide their company to the next set of customers. Their focus has been on companies with fewer than 50 employees. Larger companies have more to spend on recruiting, but also demand a more resource-intense sales approach.

We left off asking readers for feedback on which market Recruiterbox should emphasize. We also asked whether you thought the site was easy enough to understand.

One comment suggested focusing on the potential of the small- to medium-size business market. In fact, the commenter went so far as to suggest focusing on start-ups and small enterprises in India and Silicon Valley. His point was that the $100-per-month cost would be easy for these companies to digest given the value Recruiterbox offers. I agreed wholeheartedly that the company should avoid the temptation of catering to larger companies. While the appeal of increasing revenue per client can be tough to resist, it comes with three key obstacles.

Larger companies mean more decision makers, which translates into a significantly longer sales cycle.Recruiterbox does not have an outbound sales team to sell into that market. This is a significant cost savings but makes outreach to bigger companies tougher.Large companies are often swayed by reputation, and because Recruiterbox is relatively new to the market, it does not have the corporate credibility that a human resources executive may be expecting.

Other comments provided feedback on how the site functions. I challenged Mr. Sheth with these thoughts, and he shared some of his core site statistics to support his answers.

Is the Try It for Free button too easy to miss?

We used color to help make that button stand out. It is the only blue button on the site. Eighty-four percent of the people who hit the homepage end up clicking on the button. That works for us although we also want people to watch the video to get a feel for the product. On average 7 percent of the visitors that click on the Try It for Free button sign up for the trial version.

At three minutes, isn’t the video too long?

We have remade this video three times. The actual site demo takes 15 to 20 minutes, so it was quite an effort to edit that video down to three minutes. Seven percent of our visitors watch the whole video — or roughly 2,000 of our 30,000 monthly visitors. We don’t have stats on the number of people who start to play it and then stop in the middle. We haven’t made the investment in producing a one-minute concept video although we do understand the value of one. The positive side is that after watching the current video many of our potential customers can jump right in because they understand how to use the application.

On the homepage, why not have the Reasons You’ll Love It section follow the video instead of the Social Proof? Why not put some of that information on a separate page.

This isn’t a focus because so few people scroll down below the fold. Approximately 90 percent of the visitors click on either the Try It for Free button or the video. As to potentially separating those sections into different pages, we believe having all of the key information on the homepage raises our search engine optimization ranking.

Can the text on the homepage explaining what you do be more concise?

We have struggled with that line, appreciate that feedback and agree it could be improved.

Why don’t you advertise on Monster.com or other job sites?

We have tried partnering with job sites but have not made progress. We aren’t relevant to the job seeker, only to the employer, so we are looking to structure a relationship that only shows our ads to employers.

How do you use customer feedback?

We receive a few hundred e-mails per month from paying and trial customers. This is in addition to the feedback we get during our two to three demo calls every day. It is a big advantage to be this close to our customer. We are careful to filter the feedback based on the size of the company — large companies provide different feedback than their smaller counterparts. We compile the feedback, and it drives our development road map, what features we add and when.

Would you like to have your business’s Web site or mobile app reviewed? This is an opportunity for companies looking for an honest (and free) appraisal of their online presence and marketing efforts.

To be considered, please tell us  about your experiences — why you started your site, what works, what doesn’t and why you would like to have the site reviewed — in an e-mail to youretheboss@abesmarket.com.

Richard Demb is co-founder of Abe’s Market, an online marketplace for natural products that is based in Chicago.