Scientists take on 'moonshot' project mapping the human brain in hopes of fighting disease

To many other researchers, creating a map of the 86 billion neurons, or nerve cells, that make up the brain would be considered a nearly insurmountable project. However, researchers at the Allen Institute are pursuing that very task, saying it will lay the foundation for helping to better understand how the brain functions the way it does.

The Allen Institute, founded by Microsoft co-founder and philanthropist Paul Allen in 2003, was originally created to map gene activity in the mouse brain, but researchers quickly began including studies of the human brain in their work. The non-profit is the workplace of hundreds of researchers who, in large teams, launch aspirational ‘moonshot’ projects that could help solve the biggest questions regarding diseases afflicting millions of Americans.

"It does things a bit differently than other research institutions," Dr. Rui Costa, president and CEO of the Allen Institute, told ABC News. "We do big moonshot projects in life sciences that last sometimes 10, 15 years, where teams of interdisciplinary scientists answer a specific question, and then we share openly with the world."

Costa says these projects lay foundational work that can be used by other institutions. However, they are extremely complex and need to be done to scale, which requires either a multidisciplinary team, or even larger consortiums.

Dr. Ed Lein, senior investigator at the Allen Institute for Brain Science, said it was only recently that the tools existed to enable researchers to make high-resolution maps of the brain, which in turn can provide complete descriptions of all the cells that make up the organ.

He says the maps, described as a moonshot project, will be akin to sequencing a genome, which – when complete – describes a person’s complete genetic makeup.

"That becomes a really powerful reference for not only understanding the normal brain but understanding disease," Lein told ABC News. "So, we also have major efforts in trying to understand Alzheimer's disease, and the really specific types of cells that may be vulnerable in disease, as well as trying to create new tools that can target particular cell types that may actually be useful for gene therapies. "

"For example, if we can identify the vulnerable types of cells, we can now develop tools to check to start to target them,” Lein noted. “And so, it's a whole new research area that's been opened up by new technologies."

There are hundreds of brain diseases, meaning millions of American suffer from them. Alzheimer's disease, which is the most common type of dementia, affects about 6.7 million people. Parkinson's disease, a progressive disorder caused by degeneration of nerve cells in the part of the brain that controls movement, is expected to affect 1.2 million people by 2030.

Neither Alzheimer's nor Parkinson's disease has a cure, and most treatments focus on managing symptoms and maintaining quality of life. The Allen Institute team hopes its research can change that, and potentially lead to targeted therapies that help eliminate the diseases.

"To develop any type of pharmacologies or pharmacotherapeutics, or even understand the way diseases work and how they are damaging to the brain and cell types, we have to understand what are the basic core components of the cell type," Dr. Brian Lee, a senior scientist in the integrative cell physiology department at the Allen Institute, told ABC News. "So, this provides that information for people to start to do some targeted approaches to fixing or treating Alzheimer's or Parkinson's."

Lee explained that in the case of Parkinson's disease, there is a loss of cells in the basal ganglia circuitry, located near the center of the brain and which are responsible for controlling movement in the body.

However, it's unclear which types of cells are being affected by disease. A map of the brain’s cells could help answer this question.

"So, it's going to be super helpful, the more that we start to understand all the cell types in the brain and how different cell types are affected to disease, we can start taking a more a very more selected and targeted approach to treating it and, hopefully, eliminating it," Lee said.

Lein said similarly, for Alzheimer's, research has shown that specific types of cells are affected at various points in the disease’s progression and in different regions of the brain. He said the brain map can be used to map data on Alzheimer's to determine if and how these neuronal losses are leading to cognitive decline.

"I think this is just the beginning of this field so just like the genome, these maps will serve this role that now anyone studying any disease can come and get this high-resolution perspective on what's happening in that disease," Lein said. "And so, it's sort of a big community moonshot here, but it begins with this fundamental foundational atlas."

The Allen Institute is also working on other ways to try and get a better understanding of the brain. For example, Lee is working on a project that allows researchers to essentially probe or poke a single neuron to understand its functional properties. The cell is also filled with a dye to further determine its shape and function. Because there are thousands of cell types in the brain, all doing vastly different, complex tasks, Lee said this helps understand how each cell contributes to the brain’s make-up and function.

He added that the institute has also collaborated with local neurosurgeons who often must remove healthy brain tissue from patients during surgery, such as in cancer patients to reach a brain tumor. Researchers at the institute then collect some of those tissues and use the finite time they have while the tissues are alive to culture them and study individual neurons.

Allen Institute CEO Dr. Rui Costa said this research is not only setting a framework for a further and deeper understanding of the brain, but it’s also providing answers to what makes humans unique creatures.

"To understand the brain is something very important for humans, because the brain is essential to who we are, what we love, what we hate, what we remember, how we behave, how we feel," he said. "Everything that makes our persona, in a way, depends on how our brains evolve, and they experience things throughout life. So, to understand the brain is one of the big quests of humanity."