Study Rationale: Parkinson’s disease (PD) occurs because of the degeneration of the neurons that produce the neurotransmitter dopamine. This causes the disease signs and symptoms. Nerve cell destruction is linked to a dysregulated immune system causing certain T cells in blood to destroy the dopamine-producing nerve cells. Our group transformed the destructive T cells called (Teff) into neuroprotective regulatory cells that repair nerve cell damage and developed this as a therapy. We offer a means to build the numbers and function of the reparative cells and bring them to brain sub-regions where disease occurs.
Hypothesis: We theorize that we can control immune toxic reactions in PD known to affect the destruction of the dopamine neurons by neuroprotective regulatory cells (Tregs). Therapeutic outcomes of this cell therapy are facilitated by increasing the numbers and function of neuroprotective cells by bringing them from the blood to affected brain regions. We contend that this would reverse disease progression.
Study Design: We will stop Teff destructive activities which injure dopamine producing neurons. To achieve this, we will biochemically modify Tregs by a chemical process called fucosylation that will generate FUCO-Tregs. FUCO-Tregs will be injected to PD small pre-clincal models then travel to sites of dopamine neuron damage but stay there. Once there will positively transform nerve cell function leading to disease reversal.
Impact on Diagnosis/Treatment of Parkinson’s disease: Our therapy addresses the root cause of nerve cell injury in disease. The FUCO-Tregs can reverse cell damage seen as part of the disease course thereby filling a major unmet need for PD treatment.
Next Steps for Development: After affirming that FUCO-Tregs can reach and repair areas of nerve cell damage in animal models of PD, we will determine if this enhanced brain-directed Treg therapy can reverse the course of disease. Success of the animal model research will be transformed into a randomized Phase 2 clinical trial then to a larger registration trial for human use approvals.