Scientists who've spent decades learning how the brain works say they're now ready to start fixing it when it breaks.
That's the premise of the Brain Health accelerator, a collaborative effort launched by the Allen Institute in Seattle, which has become a major player in brain research.
The $400 million initiative includes plans to develop new genetic therapies — a term that includes gene editing as well as traditional gene therapy — for diseases including Alzheimer's, Parkinson's, ALS, Lewy body dementia, and Huntington's.
"The latest genetic treatments allow scientists to control the activity of particular genes," says Ed Lein, who directs the institute's brain health programs. "That opens up the possibility for very specific precision therapies for brain disorders."
The accelerator is an outgrowth of the BRAIN Initiative, an ambitious research program unveiled by President Obama in 2013. The goal of this public-private partnership was to create tools that would allow scientists to see the brain's inner workings, and, eventually, to develop treatments.
But the effort has progressed far faster than many scientists expected.
"I am shocked at how far we've come in the last 10, 12 years," says John Ngai, a senior investigator at the National Institutes of Health who directs the BRAIN Initiative. "It's just been beyond my wildest imagination — and I've been accused of having a pretty good imagination."
Support for the accelerator includes a $200 million commitment from the Allen Institute, $100 million from the Bezos family, and $100 million from sources including the National Institutes of Health, Amazon Web Services, and EverythingALS. Collaborators include a wide range of hospitals, universities, and research centers from around the world.
From mice to humans
The Allen Institute's Brain Health accelerator is attracting a range of scientists intent on translating their knowledge about the brain into treatments for its disorders.
Jeff Carroll, for example, was a teenager when he learned that his mother had Huntington's Disease, a fatal, inherited disorder that destroys brain cells.
"The whole reason I'm in science started with this frustration with not being able to understand what was happening with my mom," he says.
Carroll eventually learned that he, too, carried the Huntington's gene.
He spent years at the University of Washington studying mice with the condition, which causes nerve cells to produce toxic levels of a certain protein.
To Carroll, the solution seemed obvious: "Since we know that all the bad stuff in Huntington's comes from this one gene … let's get rid of that gene."
But finding a way to do that was beyond the reach of his own relatively small lab at a university. So Carroll joined the accelerator effort.
"It's difficult to do the scale of research that you need with a team of five or six or even 10 people," he says. "The hundreds of people they have here at the Allen Institute [allow for] an entirely different approach to science."
And one of the first targets for that approach will be Huntington's disease.
Carroll says he's optimistic.
He notes that genetic therapies have already had success treating at least one nerve disorder, a rare genetic condition called spinal muscular atrophy. It's caused by a gene mutation that leads to the death of motor neurons in the spinal cord.
"Every kid with this horrible mutation died when they were like 18 months [old]," Carroll says, "and now they're going to high school. So things that were unimaginable can change."
Carroll says his goal is to help accelerate that change.
Big science, shared
The Allen Institute was founded in 2003 by the late Paul Allen of Microsoft fame, and his younger sister, Jody Allen.
Its goal was to speed up science research. And it has done so by developing technologies that allow scientists to rapidly characterize and map millions of cells.
"We now have a complete description of the types of cells that make up the brain, and also the genetic underpinnings of their properties," Lein says. "This foundation then lets you study disease."
Allen scientists have already begun studying how Alzheimer's disease changes nerve cells in the brain.
"It affects very specific types of neurons that are lost early in the disease and then over the course of the disease," Lein says.
Genetic therapies designed to protect these neurons might delay or prevent symptoms, he says. Similar approaches might also work to preserve the neurons affected by Parkinson's or ALS.
Ultimately, though, the Allen Institute may not be the place that develops these therapies. Its policy of making its databases available to anyone means that scientists around the globe can help look for answers.
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