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 anticholinergics. These treatments can have side effects such as nausea, dizziness, hallucinations, and dyskinesia. Treatments similar to GM-CSF, such as Granulocyte Colony-Stimulating Factor (G-CSF), are used to stimulate white blood cell production and modulate immune response. Known side effects of CSFs include bone pain, fatigue, fever, and injection site reactions. In some cases, they may also cause spleen enlargement and, rarely, splenic rupture.

What prior FDA approvals exist?

FDA-approved treatments for Parkinson's Disease primarily focus on managing symptoms through dopaminergic therapies, such as Levodopa, dopamine agonists (e.g., pramipexole, ropinirole), and MAO-B inhibitors (e.g., selegiline, rasagiline). While these treatments do not directly modulate immune response, recent research has explored immunomodulatory agents. For instance, studies have investigated the potential of drugs like sargramostim (GM-CSF) to enhance immune function and reduce neuroinflammation in PD. However, as of now, no immunomodulatory agents have received FDA approval specifically for PD treatment.

What other types of research exist?

Promising alternatives to Leukine for Parkinson's Disease patients include: 1) Mesenchymal Stem Cell (MSC) therapy, which has shown potential in modulating immune responses and reducing inflammation in neurodegenerative diseases. 2) Anti-TNF agents like infliximab or etanercept, which have been effective in other inflammatory conditions and may offer benefits in PD by reducing neuroinflammation. 3) Dimethyl fumarate, used in multiple sclerosis, which has immunomodulatory and anti-inflammatory properties that could be beneficial in PD.

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Cytokine

Sargramostim for Parkinson's Disease

Phase 1

Waitlist Available

Led By Howard E Gendelman, MD

Research Sponsored by University of Nebraska

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 every 6 months during the course of treatment, up to 48 weeks until drug cessation

Awards & highlights

No Placebo-Only Group

Summary

This trial tests Leukine, a medication to boost the immune system, in patients with Parkinson's Disease. It aims to see if Leukine can improve their immune function and motor control over several months.

Who is the study for?

This trial is for people aged 35-85 with Parkinson's Disease (PD) who've had symptoms like slow movement, tremors or stiffness for at least 3 years and are not beyond stage 4 on the Hoehn and Yahr scale. They must be able to get pregnant but agree to avoid it during the study. Excluded are those with other neurological disorders, certain medical treatments or conditions, allergies to yeast products, recent flu shots, substance abuse issues, or previous GM-CSF therapy.

What is being tested?

The trial tests Sargramostim (Leukine), given as a weight-based dose over a period of 48 weeks. It aims to extend prior biomarker evaluations from an earlier study by observing clinical signs through well-being assessments and blood tests. The impact on motor control and mobility will also be measured using UPDRS scores both off treatment and while receiving Leukine.

What are the potential side effects?

Potential side effects include immune system reactions due to stimulation by Sargramostim which could lead to inflammation in various organs. There may also be risks associated with leukapheresis (a procedure that separates white blood cells), such as bleeding or bruising at the needle site.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ every 6 months during the course of treatment, up to 48 weeks until drug cessation

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~every 6 months during the course of treatment, up to 48 weeks until drug cessation

This trial's timeline: 3 weeks for screening, Varies for treatment, and every 6 months during the course of treatment, up to 48 weeks until drug cessation for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Change in incidence of adverse events over time

Secondary study objectives

Change in Treatment Biomarkers over time

Side effects data

From 2021 Phase 4 trial • 87 Patients • NCT04326920

22%

Infectious disorder (not COVID-19)

15%

Constipation

Cardiac disorder

Epistaxis

Multi-bacterial bacteremia causing hemorrhagic shock

Pneumonia

Respiratory failure

Hypoxia

Thormboembolic event

Aspergillus infection

100%

80%

60%

40%

20%

Study treatment Arm

Control Group

Active Sargramostim Treatment Group

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: Leukine TreatmentExperimental Treatment1 Intervention

48 week regimen of Leukine administered as a weight-based dose at 3 µg/kg/day for 5 days (week), followed by a 2-day holiday (weekend)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Sargramostim

2006

Completed Phase 4

~880

Do I qualify?Apply below to find out

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) include medications like Levodopa, which replenishes dopamine levels, and dopamine agonists, which mimic dopamine's effects. Additionally, treatments like MAO-B inhibitors prevent dopamine breakdown. Recently, immune-modulating therapies such as Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) are being studied. GM-CSF, similar to Leukine, stimulates the production and function of white blood cells, potentially modulating immune response and reducing inflammation. This is significant for PD patients as neuroinflammation is believed to contribute to neuronal degeneration. By targeting immune pathways, GM-CSF could offer a novel approach to slowing disease progression and improving motor control and mobility.

Recombinant human granulocyte-macrophage colony-stimulating factor stimulates superoxide anion and hydrogen peroxide production in human neutrophils.Efficacy and tolerability of recombinant human granulocyte-macrophage colony-stimulating factor in patients with chemotherapy-related leukopenia and fever.Modulation of functions of granulocytes by recombinant human GM-CSF and possible complications of GM-CSF therapy.