Study Rationale:
Emerging evidence suggests that the modulating the peripheral immune system can be a powerful disease-modifying approach for the treatment of Parkinson's disease (PD). We have developed PDM608, a long-acting granulocyte-macrophage colony-stimulating factor (GM-CSF) for PD. GM-CSF is a substance that helps make more white blood cells. PDM608 leverages the positive clinical trial results from testing another GM-CSF (sargramostim) in PD and builds in extended half-life, likely increasing patient compliance and leading to fewer side effects. Preclinical testing has demonstrated robust and durable expansion of T cells and neuroprotection following a single dose of long-acting GM-CSF in Parkinson’s models.
Hypothesis:
PDM608 treatment will result in durable effects following chronic repeated administration.
Study Design:
This project will provide critically important information necessary for determining the optimal dose and dosing frequency in humans to maximize chances of a successful clinical outcome.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
PDM608 has the potential to be transformative in the treatment of PD by mustering a regulatory immune response that protects from neuro-inflammation and restores cell function. The main innovation PDM608, would deliver is an extended half-life, resulting in reduced dosing frequency and potentially reduced overall dose level required for clinical efficacy. We expect this improved pharmacokinetic profile to result in enhanced safety and efficacy. Compliance is also expected to greatly increase with a long-acting GM-CSF that can be dosed once or twice per month instead of daily.
Next Steps for Development:
Once the data from these studies are in-hand, we will complete manufacturing of the drug for use in preclinical toxicology studies and a clinical trial. Altogether, the data package will then be submitted to the Food and Drug Administration for evaluation and subsequently dosed to humans in a Phase 1 clinical trial.