9/16/2020 Aaron Seidlitz, Illinois CS
Funding from the Aligning Science Across Parkinson’s (ASAP) initiative will amount to $8.95 million over three years to understand the way stem cells can reveal more about risk factors.
Written by Aaron Seidlitz, Illinois CS
Illinois CS professor and Willett Faculty Fellow, Jian Peng, PhD, is now part of a multi-institutional research team of scientists that earned a grant from the Aligning Science Across Parkinson’s (ASAP) initiative to use stem cells to study how risk factors accumulate and interact to drive Parkinson’s disease (PD).
The team is led by Lorenz Studer, MD, of Memorial Sloan Kettering Cancer Center. In addition to Dr. Studer and Peng, the team also includes Gist Croft, PhD, of The New York Stem Cell Foundation Research Institute (NYSCF); Vikram Khurana, MD, PhD, of Brigham and Women’s Hospital; and Joseph Powell, PhD, of Garvan Institute of Medical Research.
The grant funding totals $8.95 million over three years for their project titled, “Defining the cellular and molecular determinants of variable genetic penetrance in Parkinson’s disease.” The Michael J. Fox Foundation for Parkinson’s Research is ASAP’s implementation partner and issued the grant.
The project will progress along two primary components. First, the group will seek to better understand the risk factors that contribute to disease progression. Second, researchers will build a computational network model of these factors causing PD.
This is where Peng’s expertise in machine learning will help identify patient subgroups, reveal new disease mechanisms and forecast therapeutics.
“This artificial-intelligence-driven model will help us identify new targets for PD drugs or gene therapies,” Peng said. “Once we have those, we can evaluate therapeutic options in our patient-derived stem cells, giving us a better idea of what types of treatments will work for which subgroups of patients.”
Peng's research lab has developed numerous computational approaches for biological data analysis, including the application of deep learning techniques to protein structure prediction and the invention of a novel computational framework for simultaneous dimensionality reduction of multiple heterogeneous biological networks.
This background interacts particularly well with this project, as PD is an especially tricky disease to study. While scientists have identified many genetic risk factors, many people who carry these risk factors do not develop PD — a phenomenon known as ‘variable penetrance.’ It is still unclear how exactly PD is triggered and how brain cells become diseased as patients age. These gaps in knowledge are a major reason why there are no disease-modifying treatments that can slow, stop, or reverse PD.
“We believe that single genetic risk factors are probably not enough to cause PD on their own,” said Dr. Studer, Director of the Center for Stem Cell Biology at Memorial Sloan Kettering Cancer Center. “More likely, PD is triggered by a combination of genetics, age-related factors, and their effects in different brain cells. Stem cells give us the perfect opportunity to look at the interplay of these factors in the different cell types of the brain.”
The team hopes that their study will provide an entirely new level of understanding regarding the interplay between genetics, different brain cells, and the way aging shapes individual disease risk.
“With more information about the complex mechanisms that make someone at a higher risk for developing PD, we can start working toward strategies for early diagnosis and personalized therapeutic interventions that halt or reverse the disease,” said Dr. Studer. “I am delighted to be working with such an exceptional, collaborative team to take on these important challenges and ultimately improve the lives of patients.
See the complete list of ASAP Grantees for 2020 and NYSCF's press release.