School of Pharmacy scientists unearth a new target for treating Parkinson’s disease

Scientists at UC San Francisco have discovered how a particular protein regulates dopamine production, creating new possibilities for research into Parkinson’s disease, which involves the loss of dopamine-producing neurons.

The new research was partially funded by a Mary Anne Koda-Kimble Seed Award for Innovation, a grant given in honor of the former dean of the School of Pharmacy to support the pursuit of bold and risky projects that have the potential to move forward the mission of the School in new ways.

Parkinson’s disease, for which there is no cure, affects millions of people each year. Patients with Parkinson’s disease experience the gradual loss of certain dopamine-producing neurons in the brain, and researchers have not yet found a way of slowing or stopping the decline.

In findings published on March 7, 2019 in Cell Chemical Biology, the researchers uncovered how a protein that regulates dopamine production, as well as the survival of dopamine-producing neurons, can be triggered to increase dopamine levels. This protein, called Nurr1, is well-known for its role in Parkinson’s, but scientists previously lacked molecular tools, or drugs, for controlling its behavior.


Pamela England, PhD

“This molecule is widely regarded as one of the top therapeutic targets for Parkinson’s disease, but this is the first convincing evidence that it can be drugged,” senior author Pamela England, PhD, told UCSF News Center.

England is a faculty member in the School’s Department of Pharmaceutical Chemistry.

“We hope these insights will lead to drugs that for the first time can target the underlying causes of Parkinson’s disease,” said England. “But more immediately, this discovery will allow us to better understand Nurr1’s role in the earliest stages of the disease. As always, with understanding comes hope.”


‘Undruggable’ Parkinson’s Molecule Spills Its Secrets (UCSF News Center)

About the School: The UCSF School of Pharmacy is a premier graduate-level academic organization dedicated to improving health through precise therapeutics. It succeeds through innovative research, by educating PharmD health professional and PhD science students, and by caring for the therapeutics needs of patients while exploring innovative new models of patient care. The School was founded in 1872 as the first pharmacy school in the American West. It is an integral part of UC San Francisco, a leading university dedicated to promoting health worldwide.