Parkinson's Disease Research Is About to Get Exciting: 4 Clinical Trials That Could Change Treatment

Four major clinical trials currently underway could fundamentally change how doctors treat Parkinson's disease, offering hope for the 1 million Americans living with this progressive neurological condition. These studies are testing everything from gene therapy that delivers protective proteins directly to the brain to medications that target the genetic roots of the disease.

What Makes These Parkinson's Trials Different?

Unlike previous studies that focused mainly on managing symptoms, these trials are investigating treatments that could actually slow down or modify the disease's progression. The research spans from early-stage patients to those with advanced Parkinson's, covering a broad spectrum of potential breakthroughs.

The LUMA study represents one of the most ambitious efforts, testing BIIB122 in 650 participants across 113 trial sites. This investigational drug is designed to penetrate the central nervous system and inhibit a protein called leucine-rich repeat kinase 2 (LRRK2), which plays a key role in Parkinson's development. The trial will run for up to 144 weeks, measuring changes in the Movement Disorders Society-Unified Parkinson's Disease Scale, the gold standard for tracking disease progression.

Which Patients Could Benefit Most?

The ACTIVATE trial is specifically targeting people with a particular genetic variant—those with pathogenic changes in the glucocerebrosidase 1 (GBA1) gene. This affects about 10-15% of Parkinson's patients and often leads to more aggressive disease progression. The study of 237 patients will test BIA 28-6156, which works by targeting the GCase enzyme. Early safety data looks promising, with the most common side effects being manageable issues like headache and fatigue.

Meanwhile, researchers are also exploring whether lithium—yes, the same medication used for bipolar disorder—might help protect brain cells in Parkinson's patients. A smaller study of 20 early-stage patients will test whether 20 mg daily of lithium can reduce brain inflammation and preserve nerve function, using advanced MRI scans to track changes.

How Does Gene Therapy Fit In?

Perhaps the most futuristic approach involves AB-1005, a gene therapy that uses a modified virus to deliver protective proteins directly to brain cells. The REGENERATE-PD trial will test this approach in 87 patients across multiple countries. In earlier studies, this treatment successfully covered 63% of the targeted brain region called the putamen, exceeding researchers' goals.

The gene therapy works by introducing genetic instructions for producing glial cell line-derived neurotrophic factor (GDNF), a protein that helps protect and repair the dopamine-producing neurons that are lost in Parkinson's disease. This represents a completely different approach from traditional medications that simply replace the missing dopamine.

These trials also include important practical considerations for patients and families:

• Study Duration: Most trials run 18 months to nearly 3 years, requiring significant commitment from participants
• Age Requirements: Studies typically include adults aged 30-80, with some focusing specifically on early-stage disease
• Safety Monitoring: All trials include extensive safety follow-up periods, with researchers tracking both effectiveness and potential side effects
• Geographic Access: Trial sites span multiple countries, though availability varies by specific study

The timing of these results couldn't be more critical. Current Parkinson's treatments primarily focus on managing symptoms rather than slowing the disease itself. If even one of these approaches proves successful, it could represent the first true disease-modifying therapy for Parkinson's—something researchers and patients have been hoping for decades.

Most of these trials are expected to report initial results by mid-2026, with some extending into 2027. The diversity of approaches—from small molecule drugs to gene therapy—increases the chances that at least one will show meaningful benefits for patients facing this challenging disease.