Allen Institute scientist Aaron Garcia (right) and executive vice president Ed Lein (left) examine a section of human brain in the lab. The institute has launched the Brain Health accelerator, which will focus on using genetic therapy to develop treatments for a range of brain disorders.
Erik Dinnel/Allen Institute
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Erik Dinnel/Allen Institute
After years of studying the brain, scientists believe they are now on the verge of addressing its malfunctions.
This forms the basis of the Brain Health accelerator, an initiative by the Allen Institute in Seattle, a prominent figure in brain research.
The project aims to develop genetic therapies, including gene editing and traditional gene therapy, to treat diseases such as Alzheimer’s, Parkinson’s, ALS, and Huntington’s.
“Current genetic treatments enable scientists to modulate specific gene activity,” explains Ed Lein, who leads the institute’s brain health programs. “This paves the way for precise therapies for brain disorders.”
The accelerator is an extension of the BRAIN Initiative, a groundbreaking research program introduced by President Obama in 2013. Its aim was to develop tools to visualize the brain’s workings and eventually create treatments.
The progress of this initiative has exceeded the expectations of many scientists.
“I’m amazed at our progress over the last decade,” says John Ngai, a senior researcher at the National Institutes of Health directing the BRAIN Initiative. “It’s surpassed even my most optimistic projections.”
From mice to humans
The Brain Health accelerator at the Allen Institute is drawing scientists eager to convert their knowledge of the brain into treatments for its disorders.
Jeff Carroll discovered his mother had Huntington’s Disease during his teenage years, a fatal hereditary condition affecting brain cells.
“I ventured into science to grasp what was happening with my mom,” he shares.
Carroll later found out he carried the Huntington’s gene.
He dedicated years at the University of Washington to studying mice with the disease, which induces nerve cells to produce excessive amounts of a specific protein.
Carroll thought the answer was clear: “If all the issues in Huntington’s stem from one gene, why not eliminate that gene?”
However, executing this was beyond the capabilities of his small university lab, prompting Carroll to join the accelerator initiative.
“A small team can’t achieve the required research scale,” he notes. “The Allen Institute’s extensive team allows a different scientific approach.”
The primary focus of this approach will be Huntington’s disease.
Carroll remains hopeful.
He points out that genetic therapies have successfully treated spinal muscular atrophy, a rare nerve disorder caused by a gene mutation affecting spinal cord motor neurons.
“Children with this mutation used to die by 18 months old,” Carroll says, “but now they live to attend high school. What seemed impossible is changing.”
Carroll is committed to accelerating these changes.
Big science, shared
The Allen Institute was founded in 2003 by Paul Allen, of Microsoft, and his sister, Jody Allen.
Its mission was to accelerate scientific research, achieved by creating technologies to swiftly map and describe millions of cells.
“We now have a comprehensive understanding of brain cell types and their genetic bases,” Lein comments. “This foundation enables disease study.”
Allen scientists have started examining how Alzheimer’s alters brain nerve cells.
“Specific neurons are affected early and continuously in the disease,” Lein explains.
Genetic therapies could protect these neurons, potentially delaying or preventing symptoms, and similar methods might preserve neurons impacted by Parkinson’s or ALS.
Ultimately, while the Allen Institute may not create these therapies, its open database policy invites global scientists to seek solutions.
