Study Rationale: Preclinical and human studies have demonstrated the potential of using granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of Parkinson’s disease (PD). GM-CSF is a protein that activates T cell immune responses in a manner that prevents nerve cell loss. In this study, we will produce a blood test that evaluates T cell immunity in PD while confirming the disease-modifying potential of long-acting GM-CSF. At the same time, we will evaluate therapy-linked biomarkers that can be used to assess immune-based therapies in future clinical trials.
Hypothesis: We hypothesize that long-acting GM-CSF will modify the microenvironment of the PD brain, modifying the local immune response, protecting dopamine-producing neurons and improving clinical outcomes that can be scored by measuring biomarkers designed to assess clinical therapeutic responses.
Study Design: We will conduct the study in two phases. In the first phase, we will test the therapeutic potential of long-acting GM-CSF in a preclinical model of PD. We will examine the ability of the drug to transform pro-inflammatory neurotoxic activities into responses that spare neurons normally lost in PD. We will also confirm these changes in white blood cells isolated from people with PD. In the second phase, we will validate our prior “proof of concept” findings by developing a blood test that can be used to screen people with PD for entry into clinical evaluations.
Impact on Diagnosis/Treatment of Parkinson’s disease: Our study seeks to better define the therapeutic potential of GM-CSF for PD and will allow the development of immune-based biomarkers for clinical testing.
Next Steps for Development: If successful, we will develop a blood test that can be used to determine a positive response to GM-CSF treatment. The resulting blood test will be utilized in future clinical trials and patient assessments.