Measuring the outcome of clinical trials

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Several arrows have fallen short of their target, which is a pill on a bullseye. This image suggests that a drug targeted towards autism's symptoms has not yet been made.

Why don’t we have better drugs for autism?

Clinical trials for autism drugs have been plagued with problems: bad design, the wrong measures, too broad a range of participants. All that is finally starting to change.

By Rachel Zamzow
15 February 2017 | 18 min read

Questions for Gahan Pandina: New tool may aid autism trials

A new ‘knowledge engine’ collects reams of behavioral and sensory data to create highly sensitive outcome measures for autism drugs.

By Nicholette Zeliadt
14 February 2017 | 6 min read

Despite setbacks, fragile X drugs file into clinical trials

Our infographic displays efforts to develop treatments for fragile X syndrome. So far, none of them have passed muster in clinical trials.

By Jessica Wright
30 November 2016 | 3 min read

From the archives

Someone writes descriptions on gridded paper, the paper is in the shape of a child's silhouette.

The innovators: How families launch their own autism studies

Some parents are starting ‘N-of-1’ studies for autism, but their efforts don’t always get taken seriously.

By Carrie Arnold
28 September 2016 | 20 min read
Spectrum from The Transmitter.

Questions for James McPartland: Biomarkers for better trials

A $28 million initiative aims to develop objective tools for tracking social skills in children with autism. The initiative’s leader outlines its approach.

By Rachel Zamzow
22 September 2015 | 5 min read
Spectrum from The Transmitter.

Devising spectrum of tests for different types of autism

Finding biomarkers for different forms of autism may lead to more effective treatments for each.

By Eva Loth
16 February 2016 | 5 min read
Spectrum from The Transmitter.

Better behavioral tests may save trials of autism treatments

Trials to test drugs for autism suffer from subjective measurements and placebo effects. Helen Tager-Flusberg outlines how to ferret out the true effects of potential autism therapies.

By Helen Tager-Flusberg
21 April 2015 | 1 min read

Some people in ‘failed’ drug trial for autism showed benefit

An experimental drug called arbaclofen improved autism features in about 13 percent of people who took it in a shuttered clinical trial.

By Jessica Wright
14 December 2016 | 4 min read
Spectrum from The Transmitter.

Questions for Eric Klann: Translating treatments for fragile X

Treatments for fragile X syndrome may be more successful if they block direct targets of the key missing protein, says Eric Klann.

By Jessica Wright
30 March 2015 | 4 min read
A pill opens up to reveal a puff of smoke, suggesting that pharmaceuticals aren't offering much as promised.

Why trials of autism treatments have a placebo problem

People with autism — and their family members — are susceptible to powerful placebo effects. Some researchers are using the problem to better understand this mystifying phenomenon.

By Erik Vance
30 November 2016 | 17 min read
Spectrum from The Transmitter.

Questions for Elizabeth Berry-Kravis: Measuring drug effects

Drugs designed to treat fragile X syndrome have yet to show substantial benefits in people. But rather than abandon them, child neurologist Elizabeth Berry-Kravis suggests a new way to measure their effectiveness.

By Nicholette Zeliadt
17 February 2015 | 5 min read
Spectrum from The Transmitter.

Better tools needed to assess clinical trials

The past few years have seen an unprecedented number of clinical trials for experimental drugs to treat autism-related disorders, most notably for fragile X syndrome. But as the trials progress, scientists are calling for better methods to measure the drugs’ effectiveness.

By Emily Singer
14 May 2012 | 6 min read

Explore more from The Transmitter

Close-up of high-resolution fMRI images.

Functional MRI can do more than you think

Recent technological advances provide a range of new and different information about brain physiology. But taking full advantage of these gains depends on collaboration between engineers and neuroscientists.

By Laura Lewis
14 April 2025 | 6 min read
People help each other climb up a supersized human brain.

As federal funders desert mentorship programs for marginalized students, trainee-led initiatives fill the gap

Grassroots organizations, led by graduate students and postdoctoral researchers, are stepping up to provide neuroscience career training and guidance for students from marginalized backgrounds—and they need your support.

By Christian Cazares, Maribel Patiño
11 April 2025 | 5 min read
Research image of two mouse brain slices.

Split gene therapy delivers promise in mice modeling Dravet syndrome

The new approach overcomes viral packaging limitations by delivering SCN1A piecemeal and stitching it together in target cells.

By Holly Barker
10 April 2025 | 5 min read