When developing fetuses are exposed to alcohol, they are at risk for a variety of irreversible birth defects, and it seems that any amount of alcohol could lead to fetal alcohol spectrum disorder (FASD). Alcohol consumption during pregnancy can lead to cognitive and intellectual deficits, decreased brain volume, and brain damage. While it is unclear exactly what mechanisms underlie the disorder, researchers have been able to correct motor deficits in a pre-clinical model of FASD using a drug made from scorpion venom. The research has been reported in Nature Neuroscience.
In this work, by the team of Kazue Hashimoto-Torii, Ph.D., a principal investigator at the Center for Neuroscience Research at the Children's National Research Institute, the researchers assessed how neural progenitor cells, which would be found in the developing brain, reacted to alcohol exposure. The model tested the impact of alcohol at embryonic day 16 and 17; this is when an important region is growing that is related to motor abilities, and the early gestation of humans.
Significant deficits were seen in these models after exposure, and the researchers found that the alcohol was immediately triggering a cellular response known as heat shock, causing cells to generate protective proteins. The cells produced them in a seemingly random fashion, and not throughout the whole population. Gene expression was assessed, and the activity of 93 genes had changed in the cells. One gene, Kcnn2, produces a potassium channel that has been linked to learning and memory, and when overexpressed, abnormal signaling in cells.
The scientists blocked this channel with a drug called Tamapin, which is a type of toxin derived from Indian Red Scorpion venom. The drug caused cells that were altered by alcohol to go back to normal. Pre-clinical models showed improvements in motor skills when they received the therapy at 30 days of life. It may be possible to treat children impacted by FASD in a similar way.
Learn more from the video about a different study that showed that for fetuses, there is no safe level of alcohol exposure
Hashimoto-Torii and colleagues have created a biotech company to study whether this drug will work in people.
"Usually investigators looking for the molecular mechanisms behind disease stop there, but we want to move forward to have a real impact on public health," she said. "We really want to give patients the hope of having a better life through treating the neurodevelopmental problems caused by FASD."