Dundee scientists to lead new global Parkinson’s disease initiative
LRRK2 Investigative Therapeutics Exchange (LITE) will support new approaches targeting LRRK2, the most common cause of inherited Parkinson’s disease
Programme to focus on bridging basic science advances into industry-led drug development
Programme to be led by Professor Dario Alessi, a global leader in kinases like LRRK2, of the University of Dundee, along with colleague Dr Esther Sammler
The Michael J. Fox Foundation (MJFF) has launched a major new initiative to rapidly expedite development of therapeutic strategies targeting LRRK2. Called the LRRK2 Investigative Therapeutics Exchange (LITE), the program, which will include tens of millions of dollars of grant support, focuses on bridging basic science advances to industry-led drug development.
LRRK2 is a gene that makes a protein that helps control different activities inside our cells, like how they communicate and clean up waste. Mutations in the LRRK2 gene were first linked to Parkinson’s 20 years ago, and they are now understood to be the most common causes of inherited Parkinson’s. The mutations hyperactivate LRRK2, triggering cellular dysfunction that leads to the disease. Researchers are looking for strategies to reduce LRRK2 hyperactivity, which would help the four percent of people with inherited LRRK2 mutations. Research also suggests LRRK2 therapies could have much broader use, helping people without mutations as well.
LITE will be led by Professor Dario Alessi, a global leader in the study of kinases, a class of cellular proteins that includes LRRK2. Professor Alessi runs a lab focused on kinase research at the University of Dundee, and directs the Division of Signal Transduction Therapy, a collaboration between leading researchers in the University of Dundee’s School of Life Sciences and global pharmaceutical companies. Professor Alessi, the winner of the 2023 Robert A. Pritzker Prize for Leadership in Parkinson’s Research, also leads LRRK2 work for the Aligning Science Across Parkinson’s (ASAP) Collaborative Research Network (CRN), creating a point of collaboration across organisations as he continues to build on this work in the space.
“LRRK2 presents key opportunities to the field, both for better understanding Parkinson’s and for treating it,” said Professor Alessi. “The LITE initiative gives us a chance to clarify key points of understanding and use that knowledge to inform drug development.” Dr Esther Sammler, a neurologist at the University of Dundee who also has LRRK2 expertise, will serve as co-principal investigator for LITE, focusing on efforts to identify critical biomarkers for testing LRRK2-targeted therapies in clinical trials. The University of Dundee’s Dr Paul Davies and Dr Francesca Tonelli will join them as part of the study’s leadership. The team will work with numerous academic and industry collaborators to support their success. Several existing initiatives will contribute to and support the LITE program, including Aligning Science Across Parkinson’s (ASAP) and a number of ASAP-supported programs, such as the Collaborative Research Network (CRN), the Parkinson’s Progression Markers Initiatives (PPMI) and the Global Parkinson’s Genetics Program (GP2).
“We’re building a translational research engine that diversifies ways to target LRRK2, improving confidence and clarity in the most promising approaches to targeting the pathway. This program will make therapeutic development faster and more informed while ‘de-risking’ industry investment,” said Shalini Padmanabhan, PhD, MJFF’s head of translational research.
Several Parkinson’s drugs targeting LRRK2 are currently in clinical trials, and several other strategies to target LRRK2 have been identified and await further evaluation. The LITE program will focus both on supporting therapeutic approaches as well as identifying LRRK2-specific biomarkers, which could measure the effect of potential LRRK2-based treatments.
“By building a cohesive, collaborative approach to discovery and development, LITE expedites efforts to leverage the enormous potential of LRRK2-targeted therapies,” said Todd Sherer, PhD, chief mission officer at MJFF. “Through the initiative, we see the potential for transformative advances in understanding Parkinson’s, intervening in the disease process and, we hope, stopping Parkinson’s disease in its tracks.”
About The Michael J. Fox Foundation for Parkinson’s Research
As the world’s largest non-profit funder of Parkinson’s research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson’s patients, business leaders, clinical trial participants, donors, and volunteers. In addition to funding $1.75 billion in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson’s research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; creates a robust open-access data set and biosample library to speed scientific breakthroughs and treatment with its landmark clinical study, PPMI; increases the flow of participants into Parkinson’s disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson’s awareness through high-profile advocacy, events, and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world. For more information, visit us at www.michaeljfox.org, Facebook, Twitter, LinkedIn.
