UF scientist’s Parkinson’s work selected for innovative drug development effort
University of Florida (UF) Health News
By Stacey DeLoye
Jupiter, Florida — People with Parkinson’s disease need better treatment options, and they need them fast. A California nonprofit organization, SPARK NS, announced on Tuesday, August 20, 2024 that it has selected University of Florida chemist Matthew Disney, Ph.D., for an ambitious two-year effort to move promising Parkinson’s disease discoveries to patients.
Disney, chemistry department chair and Institute Professor at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology, has pioneered ways to create RNA-targeted medicines. He is one of five scientists nationally whose projects were selected. The nonprofit will supply each scientist with up to $2 million and the expertise of more than 50 scientific and business advisers experienced in bringing new medicines to patients.
“A typical drug development process takes 10 to 12 years and costs more than a billion dollars,” said Daria Mochly-Rosen, Ph.D., chief scientific and education adviser for SPARK NS, and a professor of systems biology at Stanford University. Furthermore, she said, only about 14% of drugs tested in clinical trials become approved medicines.
SPARK NS said its model for surrounding investigators with industry experts and training has a 50% success rate advancing academic projects to licensing or clinical trials.
“Funding is essential, but it’s not enough,” Mochly-Rosen said. “SPARK NS programs provide comprehensive resources and collaborative experiences that prepare academic researchers for the challenging process of drug development.”
Parkinson’s damages and ultimately kills brain cells that make dopamine, one of the brain’s vital signaling chemicals. Over time, those with Parkinson’s may develop stiffness, moving and balance difficulty, tremors, and difficulties with speech, sleep, pain and fatigue. Medications and procedures can help with the symptoms, but the disease worsens over time, and a cure remains elusive.
An estimated 65,000 Floridians have Parkinson’s and more than 3,000 people a year die from it. Nationally, scientists estimate that 1 in 500 people over age 65 have Parkinson’s.
Disney’s lab develops potential medications designed to act on disease-causing RNA. RNA carries out much of the work inside cells. It reads the genetic code of DNA, and it helps carry out those genetic instructions to build proteins and regulate cellular processes. Disney’s group has found that RNA itself can cause disease, so creating innovative medicines to act on it could be an important new strategy for treating otherwise hard-to-treat disease.
“I know how much people with Parkinson’s and their families want and deserve better treatments,” Disney said. “This project will enable us to devote more resources to creating RNA-targeted small molecule medicines.”
The SPARK NS mission is to help academic researchers take discoveries to the next stage in their path to commercialization. The neuroscience-focused organization said each year, it will launch a two-year translational research program. SPARK NS does not own intellectual property rights or take a financial stake in the projects it supports.
Other scientists selected for this year’s Parkinson’s project include Gary W. Miller, Ph.D., and David Sulzer, Ph.D., both of Columbia University; Alice S. Chen-Plotkin, M.D., of the University of Pennsylvania; and James H. Hurley, Ph.D., of the University of California, Berkeley.