What side effects are associated with this type of intervention?

Common treatments for Parkinson's Disease include medications like Levodopa, dopamine agonists, MAO-B inhibitors, and enzyme replacement therapies. Levodopa can cause side effects such as nausea, dizziness, and dyskinesia. Dopamine agonists may lead to compulsive behaviors, hallucinations, and sleepiness. MAO-B inhibitors can cause headaches and joint pain. Enzyme replacement therapies, like the intracerebral delivery of GCase, aim to improve cellular function but may carry risks such as infection, inflammation, and immune reactions at the injection site. It is essential to discuss these potential side effects with a healthcare provider to tailor the treatment plan to individual needs.

What prior FDA approvals exist?

FDA-approved treatments for Parkinson's Disease primarily include medications that manage symptoms rather than modify the disease. These include Levodopa combined with Carbidopa, dopamine agonists like pramipexole and ropinirole, MAO-B inhibitors such as selegiline and rasagiline, and COMT inhibitors like entacapone. While these treatments focus on dopamine replacement or preservation, there are no current FDA-approved therapies specifically targeting glucocerebrosidase (GCase) pathways similar to the investigational MRgFUS delivery of GCase.

What other types of research exist?

Promising novel alternatives for Parkinson's Disease treatment in 2024 include: 1) GLP-1 analogues such as semaglutide and liraglutide, which have shown neuroprotective effects and improvement in motor impairments in the MPTP mouse model of PD. 2) Epigallocatechin-3-gallate (EGCG), which has demonstrated potential in reducing neuron apoptosis and improving motor function in Parkinson's models. 3) Antisense oligonucleotide therapies targeting specific genetic mutations associated with PD, which are emerging as potential disease-modifying treatments.

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Enzyme Replacement Therapy

MR-Guided Focused Ultrasound + GCase for Parkinson's Disease

N/A

Recruiting

Research Sponsored by InSightec

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

No Placebo-Only Group

Summary

This trial aims to test a new treatment for Parkinson's Disease by delivering an enzyme directly into the brain using MRI and focused ultrasound. It targets patients with genetic or unknown causes of Parkinson's. The enzyme may help reduce harmful substances in the brain and improve symptoms. Focused ultrasound combined with microbubbles is becoming a promising technology for the treatment of central nervous system diseases, including Parkinson's Disease.

Who is the study for?

This trial is for men and women aged 35-75 with Parkinson's Disease diagnosed at least 2 years ago, at stages 1-3 on medication. Participants must have a positive DAT SPECT scan, be on stable PD meds for over 90 days, and either carry a GBA mutation or not, depending on the study arm. Exclusions include pregnancy, severe kidney issues, other clinical trials participation, hypersensitivity to certain agents used in the study or MRI contrasts.

What is being tested?

The trial tests if it's safe and doable to deliver GCase directly into the brain using MR-guided Focused Ultrasound (MRgFUS). This method has shown promise in animal studies for improving Parkinson’s symptoms by exposing part of the brain called putamen to GCase.

What are the potential side effects?

Potential side effects are not detailed but may relate to MRgFUS procedure complications like discomfort or pain at the target site. There could also be reactions related to GCase delivery such as allergic responses or infusion-related reactions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Adverse Events

Secondary study objectives

Feasibility of MRgFUS BBB opening for GCase brain delivery

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Idiopathic PD PatientsExperimental Treatment1 Intervention

Idiopathic PD patients receiving 3 cycles of BBBO. There is option to include simultaneous GCase delivery after safety profile review by Data and Safety Monitoring Board (DSMB).

Group II: GBA PD PatientsExperimental Treatment1 Intervention

GBA PD patients receiving 3 cycles of BBBO. There is option to include simultaneous GCase delivery after safety profile review by Data and Safety Monitoring Board (DSMB).

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Parkinson's Disease (PD) focus on managing symptoms and slowing disease progression. One emerging treatment involves the intracerebral delivery of glucocerebrosidase (GCase) via MR-guided focused ultrasound (MRgFUS). GCase breaks down glucocerebroside, reducing its accumulation and improving PD pathology by potentially decreasing alpha-synuclein aggregation, which is toxic to neurons. This is crucial for PD patients as it targets a root cause of neurodegeneration. Other treatments include dopamine replacement therapies like Levodopa, which replenishes dopamine levels to improve motor function, and deep brain stimulation (DBS), which modulates brain activity to reduce symptoms. These treatments are essential as they help manage the debilitating symptoms of PD, improving patients' quality of life.

Gami-Chunggan Formula Prevents Motor Dysfunction in MPTP/p-Induced and A53T α-Synuclein Overexpressed Parkinson's Disease Mouse Model Though DJ-1 and BDNF Expression.Development and biochemical characterization of a mouse model of Parkinson's disease bearing defective glucocerebrosidase activity.Neuroprotective Effects of Sulphated Agaran from Marine Alga Gracilaria cornea in Rat 6-Hydroxydopamine Parkinson's Disease Model: Behavioural, Neurochemical and Transcriptional Alterations.