AILSA CHANG, HOST:
Scientists who have spent decades learning how the brain works say they are ready to start fixing it when it breaks. A brain research center in Seattle called the Allen Institute is working to develop genetic therapies for diseases including Alzheimer's, Parkinson's, ALS and Huntington's. Here's NPR's Jon Hamilton.
JON HAMILTON, BYLINE: The effort is called the Brain Health accelerator, and it includes scientists like Jeff Carroll. When Carroll was a teenager, he found out his mother had Huntington's disease. It's a fatal inherited disorder that destroys brain cells.
JEFF CARROLL: The whole reason I'm in science started with this frustration with not being able to understand what was happening with my mom.
HAMILTON: Carroll learned that he, too, carried the Huntington's gene. He spent years at the University of Washington studying mice with the condition, which occurs when the faulty gene tells nerve cells to produce toxic levels of a certain protein. To Carroll, the solution seemed obvious.
CARROLL: Since we know all the bad stuff in Huntington's comes from this one gene, let's get rid of the levels of that gene.
HAMILTON: But finding a way to do that was beyond the reach of his own lab. So Carroll joined the Allen Institute.
CARROLL: It's difficult to do the scale of research that you need with a team of five or six or even 10 people, and so the hundreds of people they have here at the Allen Institute is just an entirely different approach to science.
HAMILTON: The scientist in charge of the Brain Health accelerator is Ed Lein. He says it's built on years of basic research.
ED LEIN: We now have a complete description of the types of cells that make up the brain and also the genetic underpinnings of their properties. This foundation then lets you study disease.
HAMILTON: By seeing how disease changes these cells - Lein says scientists at Allen already have some experience with this approach.
LEIN: And in the case of Alzheimer's disease, we can see that it's not only about plaques and tangles, but it affects very specific types of neurons that are lost early in the disease and then over the course of disease.
HAMILTON: A genetic therapy designed to protect these neurons might delay or prevent symptoms. Lein says other therapies might preserve the neurons affected by Parkinson's or ALS.
LEIN: And we can now turn on a therapeutic gene just in the right kind of cell with these types of technologies, and that opens up the possibility for very specific precision genetic therapies for brain disorders.
HAMILTON: One reason that's possible is research funded by the BRAIN Initiative, a program announced by President Obama back in 2013. John Ngai of the National Institutes of Health says it's given scientists new ways to observe the inner workings of the brain.
JOHN NGAI: I am shocked at how far we've come in the last 10, 12 years. It's just been beyond my wildest imagination, and I've been accused of having a pretty good imagination.
HAMILTON: Ngai says insights from the BRAIN Initiative have already helped researchers find new ways to use existing treatments. Deep brain stimulation, for example, got its start as a way to use electrical pulses to reduce the symptoms of Parkinson's disease.
NGAI: Now it's being applied to a whole range of disorders - epilepsy, treatment-resistant depression, OCD, PTSD, binge-eating disorder.
HAMILTON: Scientists at the Allen Institute say genetic therapies could follow a similar path. Jeff Carroll, the scientist with the Huntington's gene, says the approach has worked on children with spinal muscular atrophy. It's a genetic condition that destroys motor neurons in the spinal cord.
CARROLL: Every kid with this horrible mutation died when they were, like, 18 months for all of human history, and now they're going to high school. So things that were, like, unimaginable can just change.
HAMILTON: Carroll says his goal is to help accelerate that change. Jon Hamilton, NPR News.
(SOUNDBITE OF THE DARE SONG, "GIRLS") Transcript provided by NPR, Copyright NPR.
NPR transcripts are created on a rush deadline by an NPR contractor. This text may not be in its final form and may be updated or revised in the future. Accuracy and availability may vary. The authoritative record of NPR’s programming is the audio record.
