Allen Institute Launches Genetic Therapy Program Targeting Alzheimer's, Parkinson's, ALS and Huntington's

The Allen Institute in Seattle launched the Brain Health accelerator to develop genetic therapies for Alzheimer's, Parkinson's, ALS, and Huntington's. The program will use gene editing and other genetic approaches to target specific genes linked to these disorders.

Ed Lein, who directs the institute's brain health programs, said the latest genetic treatments allow scientists to control the activity of particular genes.

"That opens up the possibility for very specific precision therapies for brain disorders," he said. The accelerator is an outgrowth of the BRAIN Initiative, a public-private partnership unveiled by President Obama in 2013. The initiative aimed to create tools to see the brain's inner workings and develop treatments.

John Ngai, a senior investigator at the National Institutes of Health who directs the BRAIN Initiative, said progress has exceeded expectations. "I am shocked at how far we've come in the last 10, 12 years," Ngai said. " Jeff Carroll joined the accelerator after working at the University of Washington.

He learned as a teenager that his mother had Huntington's Disease and later discovered he carries the gene. "The whole reason I'm in science started with this frustration with not being able to understand what was happening with my mom," Carroll said. He added that because all the damage in Huntington's stems from one gene, the approach is to remove that gene.

Carroll said the scale of work at the Allen Institute differs from smaller university labs. "It's difficult to do the scale of research that you need with a team of five or six or even 10 people," he said. " He pointed to genetic therapies already used for spinal muscular atrophy, noting that children with the mutation once died around 18 months old and now attend high school.

The Allen Institute was founded in 2003 by Paul Allen and Jody Allen. Scientists there have completed a description of the types of cells that make up the brain and the genetic basis of their properties. They have begun studying how Alzheimer's disease changes nerve cells and affects specific types of neurons lost early in the disease.

Lein said genetic therapies designed to protect these neurons might delay or prevent symptoms, and similar approaches could apply to Parkinson's or ALS. The institute's policy of sharing its databases means scientists worldwide can contribute to identifying targets and developing therapies.