A Path for Hope

Roberta Diaz Brinton ’79 ’81 ’84 believes regenerating the brain means regenerating a life. Brinton is the director of the University of Arizona Health Sciences Center for Innovation in Brain Science, and her life’s work and research at the UArizona bring hope to people with Alzheimer’s disease.

Her research team has developed a new therapy designed to restore cognitive function in patients in the early stages of Alzheimer’s. Their research has found that the neurosteroid allopregnanolone, or allo — used in high doses to treat women with postpartum depression — promotes the connectivity between neural networks required for cognitive function by generating new neurons and synapses in early-stage Alzheimer’s patients.

The therapy is proceeding through a Phase 2b clinical trial, now enrolling patients. Phase 2b trials involve rigorous testing for efficacy in groups of 100 to 300 patients. Working with Tech Launch Arizona, the office that commercializes inventions stemming from UArizona research, the team has launched NeuTherapeutics LLC to advance the technology toward being available for patients.

“Starting up NeuTherapeutics is so exciting because we are creating the path to bring the first regenerative therapeutic for Alzheimer’s disease to those who need it. We believe that treatment with allo will restore brain function and independence for patients,” says Brinton, a Regents Professor of pharmacology and neurology and a member of the BIO5 Institute. 

The UArizona drug development team recently launched the Phase 2b trial after securing more than $37 million in funding from the National Institute on Aging and approval from the U.S. Food and Drug Administration. This trial follows a successful three-month Phase 1b/2a trial, which developed an optimal treatment regimen and dose and monitored patient progress through MRI brain imaging and blood tests.

“We are encouraged that the MRI brain imaging provided early evidence of a regenerative response in Alzheimer’s patients treated with allo, confirming years of preclinical data,” says Brinton. “Importantly, the Phase 1 data indicated a strong safety profile while demonstrating none of the brain bleeds associated with recently approved Alzheimer’s treatments.”

Approved as safe for medical use in 2019, allo is a naturally produced steroid. To promote regeneration in brains affected by Alzheimer’s, the medication is delivered at a low dose once a week.

NeuTherapeutics is developing novel allo formulations and delivery strategies to ensure that treatment is easy, efficient and affordable while also following up on preclinical data that suggest it may be effective in treating other neurodegenerative diseases, such as Parkinson’s disease.