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

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

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

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

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

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

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

Illustration of an open book with the pages creating a brain shape, and with a tassel resembling a DNA sequence.

Open-access neuroscience comes to the classroom: Q&A with Liz Kirby

Neuroscience textbooks can be prohibitively expensive for some undergraduate students. A new open-access alternative seeks to change that.

By Francisco J. Rivera Rosario
13 December 2024 | 6 min read
Black-and-white image of cubes floating out of the bottom half of a human head.

Solving intelligence requires new research and funding models

Our research ecosystem isn't built to deliver the breakthroughs needed to understand intelligence at scale. We need a dedicated research institution to take up the task.

By David A. Markowitz
13 December 2024 | 6 min read
Research image of brain scans.

Impaired molecular ‘chaperone’ accompanies multiple brain changes, conditions

Rare genetic variants in a protein-folding complex contribute to a spectrum of phenotypes that encompass brain malformations, intellectual disability, autism and seizures, according to a new “hallmark” study.

By Holly Barker
12 December 2024 | 5 min read