Welcome to the July issue of Going on Trial, a monthly newsletter covering clinical trials and drug development for autism and related conditions. This month, we dig into a cell culture model that could reduce the frequency of failed drug trials, and we review early results from an antisense oligonucleotide treatment for Dravet syndrome. Roughly 90 percent of candidate drugs share the same problem: They appear promising in cell culture and animal studies, only to prove ineffective or unsafe in people. A cell culture model called an organ-on-a-chip, or tissue chip, could help smooth the transition by weeding out toxic or other problematic drug candidates before they're tested in people. Last month, researchers met at the Microphysiological Systems World Summit in Berlin, Germany, to discuss plans to shepherd the chip models into the drug development process. The models involve culturing human cells in a device containing microfluidic channels that emulate the flow of blood or air through an intact tissue or organ. For example, one blood-brain-barrier-ona-chip contains two stacked chambers, separated by a thin porous membrane; astrocytes and pericytes grow in the base layer, and gatekeeping endothelial cells grow in the upper compartment. The device pumps fluid through tiny channels atop the endothelial cells to provide them with fresh oxygen and induce mechanical stress, similar to the environment of a real blood-brain barrier. Scientists have created a menagerie of tissue-chip models -mimicking the liver, lungs, heart and neuromuscular junction -but the blood-brain-barrier model may prove particularly useful for drug screening, says YongTae Kim, associate professor of mechanical engineering at the Georgia Institute of Technology in Atlanta and chief executive officer of MEPSGEN, a company that develops and sells tissue-chip models.
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