Roberta Diaz Brinton, the R. Pete Vanderveen Endowed Chair in Therapeutic Discovery and Development, is one of 19 researchers to receive a CIRM Disease Team Therapy Development Award from the California Institute for Regenerative Medicine (CIRM) to support the assembly of a research team to develop a clinical trial grant that could be worth up to $20 million.
“These planning awards continue CIRM’s record of requiring scientists to work in teams, sharing knowledge and speeding the time to new therapies,” said CIRM President Alan Trounson in a release. Brinton’s is the only award received by USC in this round of funding.
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, is co-PI on the project, and the Clinical and Translational Science Institute Center for Scientific Translation and USC NIA Alzheimer’s Disease Research Center are contributing partners.
“Allepregnanolone promotes the ability of brain to regenerate itself by increasing the number and survival of newly generated neurons,” explains Brinton.
These newly generated neurons are associated with a reversal of cognitive deficits and restored learning and memory function in preclinical models of Alzheimer’s disease. Further, allopregnanolone reduces the amount of Alzheimer’s pathology in the brain.
Brinton emphasizes the fact that Alzheimer’s therapies like this urgently need to be developed.
“In the US, 5.4 million people have Alzheimer’s and another American develops the disease every 69 seconds,” she says. “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 believes that allepregnanolone has the potential to be effective for both the prevention of and early stage treatment of Alzheimer’s disease, and will use the funding to assemble an interdisciplinary team of clinicians, scientists, and therapeutic development, regulatory, data management and statistical analysis experts that 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 in the summer of 2012.