ARPA-H funds new project to deliver breakthroughs in the effective treatment of brain and nervous system diseases to improve health outcomes  

Project aims to develop cell-based therapies to deliver treatments to the brain, nervous system and other tissues, improving patient outcomes for hard-to-treat diseases, including glioblastoma, a deadly cancer 

Today, the Advanced Research Projects Agency for Health (ARPA-H) announced funding for the Cell Therapies for Neuroinflammation and Neurodegeneration (CT-NEURO) project, which aims to develop strategies for more effective tissue-specific delivery of therapies. Focusing first on brain diseases, the project’s goal is to develop an immune cell-based platform to provide therapy directly to the brain and nervous system. 

More than 7 million Americans are living with a neurodegenerative disease, such as Alzheimer’s, Parkinson’s, amyotrophic lateral sclerosis (ALS), or cancers of the brain, such as glioblastoma. These disorders destroy nerve cell tissue and are particularly difficult to treat due in part to the blood brain barrier, which prevents medications from reaching their intended targets. Other characteristics of the nervous system necessitate targeting treatment to impacted cells while sparing remaining brain and nervous system tissue. CT-NEURO aims to use immune cells that can already freely move about the body to deliver therapeutics directly and precisely to the tissue that needs it to improve patient outcomes for these devastating diagnoses. 

“If we can harness immune cells to shepherd therapies right to the source of disease, it would be a groundbreaking advancement in treating diseases and injuries in the brain,” said Program Manager Daria Fedyukina, Ph.D. “ARPA-H is focused on supporting platform technologies like this that could be applied to the treatment of multiple diseases and then expanded to other tissue types as well.”  

“CT-NEURO represents the agency’s first investment in therapeutics for neurodegenerative conditions,” notes ARPA-H Director, Renee Wegrzyn, Ph.D. “If successful, this cellular therapeutic platform could support better treatments and outcomes for brain tumors like glioblastoma, chronic disorders like multiple sclerosis, and Alzheimer’s disease.” The University of California San Francisco will lead the research effort with a budget of up to $35 million.   

CT-NEURO is one of multiple projects solicited through the agency’s Open Broad Agency Announcement (Open-BAA). The ARPA-H Open BAA seeks transformative ideas for health research or technology breakthroughs. Continued support of each award is contingent on projects meeting aggressive milestones. To learn more about projects as they are awarded, visit the Open BAA awardee page.