$200M effort aims to move from mapping the brain to treating disease – GeekWire
The organization that Paul Allen founded to map and understand the complexity of the human brain is now looking to turn that hard-earned knowledge into treatments for brain diseases.
The Allen Institute announced Tuesday that it is launching a new program with $200 million in initial funding and the ultimate goal of developing compounds to treat neurodegenerative diseases such as Alzheimer’s, Parkinson’s, Huntington’s, and ALS.
The program, called the Brain Health Accelerator, is a newly created unit within the Allen Institute in Seattle, which is part of its Brain Science division. Starting with around 60 people, it is expected to expand to a 200 person effort over time.
It was the first time this center was founded in 2003 by the late Microsoft founder that curing diseases has become a goal, building on his long-standing work in brain imaging.
The goal is “a new type of therapy that, instead of targeting a protein, targets cells in the brain that are involved in disease,” said Ed Lein, executive vice president of the Allen Institute and director of the Brain Health Accelerator, in an interview with GeekWire this week.
“This could be a revolution in both neuroscience and the way we think about treating brain diseases, because this is not happening now,” Lein said.
How it is funded: The accelerator’s $200 million core funding comes from the Science and Technology Fund, Allen’s facility that launched an initial $3.1 billion fund to support work in bioscience, the environment, and AI.
Allen, who died in 2018, directed that money from his estate go to charity. His sister, Jody Allen, who chairs the foundation, is overseeing the sale of merchandise including his sports, the Seattle Seahawks and Portland Trail Blazers, with a large portion of the proceeds expected to flow into the foundation’s science and technology work.
The commitment to the new Brain Health Accelerator is for 14 years, and Lein said he expects the entire accelerator’s funding to grow as the program adds partners.
It will also attract federal funding, including grants from the National Institutes of Health. Asked about the impact of pressure on federal science funding, Lein described the relationship with NIH as mutually beneficial: philanthropy builds the research infrastructure, federal grants help run it at a high level, and the resulting data becomes a public resource.
“You can even call it a public-private partnership,” he said. “I won’t say we can’t do it without them, but I can say we can’t do much without them.”
From brain maps to medicine: The initiative builds on the Allen Institute’s more than two decades of brain mapping work. Advances in single-cell genomics in recent years allow researchers to catalog the human brain at a resolution never before possible, describing thousands of different cell types by their genes.
Lein called it “the equivalent of the human genome meets Google Earth” – a reference map that allows scientists to see diseases at the level of specific cells and circuits for the first time.
The same map offers new possibilities for treating diseases. It shows genetic changes that turn on genes in certain types of cells, allowing researchers to develop tools to target those cells. A possible outcome is gene therapy that works only on the cells the disease affects.
What’s coming: The stated goal of the accelerator is to reach a clinical trial within five years.
Lein was careful not to say which disease might be the subject of the first trial, but admitted that ALS looks promising. Researchers already know which cells are affected by this disease – motor neurons in the spinal cord and cortex – and in some cases its genetic cause. ALS also progresses rapidly, which makes patients more willing to try experimental treatments.
“We’ve never done this before,” Lein said. “We’ve been describing our environment all our lives, and now we want to try to do something with that knowledge and those tools.”
The role of artificial intelligence: Recent advances in AI, including basic models, are giving researchers new ways to discover patterns and show how disease develops.
“The scale of the data is really going to be unlike anything we’ve ever done before, and that fits well with the foundation of modeling and new approaches in AI,” Lein said.
Partners in the program include Amazon Web Services, with whom the Allen Institute has worked for years. Lein said the institute is starting to work with the Allen Institute for AI, or Ai2, a Seattle-based nonprofit that Allen founded separately.
Accelerator participants include more than a dozen universities and research institutions, including the University of Washington, Stanford, MIT, Fred Hutch, and the patient advocacy group EverythingALS, and international partners such as the Sanger Institute in the UK and Riken in Japan.
Allen’s legacy: Lein has been at the Allen Institute since 2004, when he joined as the first neuroscientist. He said the project is beginning to fulfill Allen’s biggest ambitions.
“I feel really happy, and I’m really sad that Paul isn’t here to tell you,” Lein said. “He was full of curiosity, but also ambitious, and hopeful that eventually we would achieve things that impact people’s lives. I think we are finally there.”
