While Parkinson’s disease (PD) research has generated a number of treatments aimed at treating symptoms of the disease, primarily by addressing dopamine shortages in the brain, there are currently no known treatments that stop the disease from progressing.
Existing therapies treat motor symptoms like tremor and dyskinesia and non-motor symptoms such as sleep disturbances, mood changes and trouble with memory. Finding therapies that can prevent, slow or stop Parkinson’s progression remains a critical goal for people with Parkinson’s.
That’s why The Michael J. Fox Foundation, in coalition with other nonprofits, hosted the Workshop on Assessing Cumulative Benefits of Disease-Modifying Therapies Targeting Synucleinopathies in New York City in November 2024. The workshop brought together representatives from academia, industry and advocacy, including both the patient and family perspective. Discussion centered on disease-modifying therapies (DMT), in development that can prevent, slow or stop PD as well as other biologically similar diseases called synucleinopathies, including dementia with Lewy bodies (DLB) and multiple system atrophy (MSA).
Key themes included:
- Similarities and differences between synucleinopathies
- How to measure long-term benefits of treatments that slow these disorders
- Clinical trial recruitment and retention
- Experience related to DMTs in other diseases
The workshop proceedings and a roadmap for research were published in May 2025. The report notes why it’s so important to fully comprehend the benefits of DMTs:
As of this writing, it is possible that one or more DMTs addressing synucleinopathies will obtain regulatory approval in the next several years. However, many other factors contribute to whether patients will have access to these novel therapies. Government and private payers will need to assess whether the expected benefits justify the costs. Clinicians and medical professional specialty societies will need to know how effective and safe treatments are, including for diverse patient populations and patients with complex needs. Patients and their families will want to understand how their lives could change from the benefits and risks of DMTs. Governments, private payers, clinicians, medical professional specialty societies, patients and care partners think about meaningful benefits differently from regulators, who focus on safety and efficacy.
This workshop tackled topics ranging from recruitment for DMT clinical trials to the experience of other disease communities (like Alzheimer’s disease, multiple sclerosis and ALS), where a DMT was introduced. Hearing from patients about their lived experiences and expectations was also a significant aspect of the workshop.
The workshop highlighted that alongside efforts to develop DMTs for synucleinopathies, advocates also need to develop strategies for communicating their benefits. The full conference proceedings, Assessing Cumulative, Meaningful Benefits of Disease-Modifying Therapies Targeting Synucleinopathies, is available for download.