The 16th annual Parkinson’s Disease Therapeutics Conference (PDTC), held on October 17, 2024 in New York City, brought together more than 300 research and industry leaders from over 170 institutions who shared cutting-edge advancements in Parkinson’s disease (PD) treatment.  

Hosted by The Michael J. Fox Foundation, the meeting featured reports on promising therapies in the pipeline and advancements in how Parkinson’s is staged and managed. It was packed with informative sessions around disease progression and biomarkers and addressed challenges and opportunities in clinical trial innovations, including new approaches to integrating patient perspectives.  

"PDTC was truly inspiring, with some of the brightest minds in the field sharing research insight and forward-thinking approaches,” said Sohini Chowdhury, MJFF’s chief program officer. “The depth of discussion around clinical trials and the development of new therapies was particularly encouraging. We’re excited to see how these new discussions may encourage future collaborations, drive progress in the road ahead and translate into real-world impact.” 

PDTC Conference Highlights 

Highlights from the day-long meeting include: 

• LRRK2 is a prominent target in Parkinson’s research. Angela Cacace, PhD, reported on an experimental drug that reduces LRRK2 overactivity, with positive effects on cellular waste disposal and inflammation levels.   

• Martin Gill, PhD, presented on the development of a treatment that activates a key regulator of autophagy and lysosomal function, TRPML1, to help brain cells clear out waste more effectively, which is a key challenge in PD. If successful, this research could lead to a safe, targeted therapy that not only helps with Parkinson’s symptoms but may also slow disease progression.  

• Daniel Huddleston, MD, presented on the importance of neuroimaging to detect PD and track its progression for clinical trials. Work is being done to develop specific MRI neuroimaging techniques that bring down the cost of imaging and increase its efficiency compared to other brain imaging techniques currently in use.  

• In 2023, scientists validated a biomarker test for PD biology, but that test is only currently validated in cerebrospinal fluid obtained by a lumbar puncture (sometimes referred to as a spinal tap). Roland Heym, PhD, covered research efforts on using skin samples for biomarker detection as a less invasive option.  

A Patient-Focused Approach to Research 

This premier meeting not only showcased the latest in Parkinson’s therapeutics development but also reinforced the importance of keeping people with PD and their loved ones at the center of these efforts. In our panel discussions, field leaders and patients shared their own insights and practical perspectives on priorities for therapeutic development, providing an opportunity for drug developers and experts in the field to devise patient-centric strategies around disease management and informed care.  

A highlighted discussion touched on the Neuronal alpha-Synuclein Disease Integrated Staging System (NSD-ISS); a staging system proposal focused on the biology of neurodegenerative disorders. Panelists emphasized the importance of sharing resources and tools amongst organizations targeting disorders with overlapping pathology to help characterize the disease, develop biomarkers and build clinical approaches.  

“It’s incredible to see how far Parkinson’s research has advanced during my career, leading therapeutics development,” said Bruce Morimoto, PhD, vice president, Alto Neuroscience. “As a scientific advisor at MJFF, I’ve experienced first-hand the urgency to find effective treatments and ultimately a cure for Parkinson’s. PDTC continues to excel at bringing innovative discussions to the table so that the science community can work on delivering results to patients’ hands.” 

MJFF launched PDTC with the goal of providing a platform for field leaders to share new and published results and for fostering new relationships and collaborations. In the years since, it has been a dynamic hub for PD research, knowledge sharing and therapeutic development discussions.