Emerging Therapeutic Strategies and Trial Innovations in Parkinson Disease: Mariana Hernandez Gonzalez-Monje, MD, PhD

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"One of the most exciting is the development of what we call disease-modifying strategies in Parkinson disease. Now, with these approaches, the idea is to target the fundamental disease mechanisms rather than merely controlling the symptoms of our patients."

Recent literature reflects the rapidly evolving therapeutic landscape in Parkinson disease (PD), with advances spanning pharmacologic, surgical, and device-assisted strategies. A prior review published in Cureus synthesized current evidence across medical, procedural, and assistive approaches for both motor and nonmotor symptoms, underscoring the continued central role of dopaminergic therapy in addressing dopamine deficiency.¹ It also highlighted the growing use of advanced interventions such as deep brain stimulation, levodopa-carbidopa intestinal gel infusion, and ablative therapies in progressive disease, while placing these developments within the broader historical evolution of PD management.

Building on this clinical framework, a recent review published in Neuroscience examined evolving insights into PD pathogenesis and their implications for therapeutic development.² The review summarized established symptomatic treatments and detailed emerging strategies, including gene therapy, stem cell transplantation, immunotherapy, gene editing technologies, and multi-target approaches. Although current treatments remain symptomatic and no disease-modifying or curative therapy has been established, the recent study highlighted ongoing translational research aimed at informing future therapeutic directions.

To further contextualize the evolving therapeutic landscape of PD, NeurologyLive^® spoke with Mariana Hernandez Gonzalez-Monje, MD, PhD, a movement disorder specialist at Northwestern Medicine, who outlined the latest key developments. Throughout the discussion, she emphasized the growing momentum behind disease-modifying strategies targeting underlying mechanisms of PD. She also described the emergence of multiarm, multistage adaptive platform trials in the UK designed to evaluate multiple therapies more efficiently. Furthermore, Gonzalez-Monje pointed to near-term advances likely to impact patient care, including novel levodopa delivery systems, adaptive DBS, and expanded targets for MR-guided focused ultrasound.

REFERENCES
1. Alotaibi S, Alfayez L, Alkhudhair M. Parkinson's Disease: Current Treatment Modalities and Emerging Therapies. Cureus. 2024;16(12):e75647. Published 2024 Dec 13. doi:10.7759/cureus.75647
2. Zuo Y, Ding X, Liu Z, Xie Y, Liu G, Liu C. Diverse pathways for the treatment of Parkinson's disease: Integration and development of traditional and emerging therapies. Neuroscience. 2025;568:388-398. doi:10.1016/j.neuroscience.2025.01.045