Roberta Diaz Brinton, PhD, director of SC CTSI Preclinical Translation and Regulatory Support, is one of 19 researchers to receive an award from the California Institute for Regenerative Medicine (CIRM) to assembe a research team to develop a clinical trial grant that could be worth up to $20 million.
Brinton’s proposal called for the founding of a disease team to develop a small molecule therapeutic, allopregnanolone, for the prevention and treatment of Alzheimer’s disease. Lon S. Schneider, professor of psychiatry, neurology and gerontology at the Keck School of Medicine of USC, is co-principal investigator on the project. The Clinical and Translational Science Institute Center for Scientific Translation and the USC NIA Alzheimer’s Disease Research Center are contributing partners.
“Allepregnanolone promotes the ability of the brain to regenerate itself by increasing the number and survival of newly generated neurons,” Brinton explained.
These newly generated neurons are associated with a reversal of cognitive deficits, restored learning and memory function in preclinical models of Alzheimer’s disease. In addition, allopregnanolone reduces the amount of Alzheimer’s pathology in the brain.
Brinton emphasized the fact that Alzheimer’s therapies like this urgently need to be developed.
“In the United States, 5.4 million people have Alzheimer’s and another American develops the disease every 69 seconds,” she said. “No therapeutic strategies exist to prevent or treat Alzheimer’s, and results of a recent two-year clinical study show that the currently available medications for managing symptoms are ineffective in patients with mild cognitive impairment or mild Alzheimer’s.”
Brinton, who believes that allepregnanolone has the potential to be effective for both the prevention of and early stage treatment of Alzheimer’s disease, will use the funding to assemble an interdisciplinary team of clinicians, scientists and experts in therapeutic development, data management and statistical analysis who will plan and implement clinical trials of the compound.
Brinton’s team will submit a full research application, which the CIRM governing board will vote on next summer.
The original article was published on USC.edu