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What is the context of this research?

In most organs, neighboring cells mediate similar functions. However, brain cells are highly diverse and organized into hundreds of functionally distinct brain areas. Because of this diversity, a detailed comparison is necessary to assure the functional similarity between human and animal brains. The lack of comprehensive data sets prevented this comparison in the past.

A brain map is a data set of where and when genes turn on or off across the entire brain and how cells are interconnected. Our team has built and analyzed brain maps for over a decade. With this project, we plan to use these brain maps to develop a new approach for comparing brains between species. When scientists know how animal brains mimic human brains, they can make better predictions for a drug’s effects in humans.

What is the significance of this project?

Healthcare costs associated with brain diseases are steeply increasing. Dementia alone accounts for nearly 1 trillion dollars worldwide. Unfortunately, only 6% of drugs get FDA approval for the brain. It costs upwards of $2.5 billion to make a new drug. This causes the prohibitive rise in drug prices. Therefore, even small advances are invaluable.

When established, our analysis can help reveal a drug’s therapeutic potential and risk earlier in the development process. Further, it could distinguish drug targets among many candidates. With these results, drug companies can focus on experiments that help reduce potential risks prior to further investment. This would lower the amount of money and time needed to bring a drug to market, increasing accessibility for patients!

What are the goals of the project?

We are establishing a new analysis method to compare the brains of mice, monkeys, and humans. The resulting data set will cover up to 200 brain areas and multiple developmental stages. We will then evaluate more than 20 anti-epileptic drug target genes and assess the degree of similarity or difference between species. Next, we will test the utility of our analysis using clinical data from anti-epileptic drugs. We will look for associations between our results and clinical outcomes, such as efficacy and side effects.

This effort will serve as an exemplar as we continue to solicit funding from public agencies, private foundations, drug companies, etc. Following this study, we will apply our approach to drug targets implicated in other brain diseases. Click here for more details.