Study Rationale: Parkinson's disease (PD) is characterized by the death of dopamine neurons, which control movement. We have generated a novel therapeutic agent — an antibody that activates a receptor called TrkB — that can shows neuroprotective effects. Delivery of this therapeutic antibody is hampered by its ability to cross the blood-brain barrier. To ferry our antibody across this biological barrier, we have coupled it with a "brain shuttle" system that ferries it into the brain. In this study, we will determine whether this enhanced, “shuttle equipped” therapeutic can protect neurons and boost dopamine production in a preclinical PD model.
Hypothesis: We hypothesize that our novel, shuttle-equipped therapeutic can enter the brains of preclinical PD animals, where it will protect neurons and boost dopamine production.
Study Design: We will determine whether our therapeutic is effective in a preclinical PD model in which the animals display symptoms similar to people with PD, including neuronal cell death and decreased dopamine production. If the treatment works in a mouse model, we will test it in nonhuman primates to see how it affects their brains and neurons.
Impact on Diagnosis/Treatment of Parkinson’s disease: This new treatment might help slow down the disease and potentially improve how well people with PD can control their movements.
Next Steps for Development: If successful, we'll conduct additional studies to make sure our treatment is safe for humans and, in just over two years, start testing it in people with PD.