Experimental Treatment Only at UK Targets Parkinson’s Disease Symptoms


At the UK HealthCare Brain Restoration Center (BRC), a team of physician-scientists and researchers is working to forever change the lives of patients with Parkinson’s disease. The BRC team has embarked on a first-of-its-kind clinical study aimed at stopping or reversing the degenerative effects of the movement disorder.

The study combines the established treatment for advanced Parkinson’s – an electrode-implanting surgery known as Deep Brain Stimulation, or DBS – with an experimental nerve-grafting procedure. The nerve cells are transplanted during DBS surgery, meaning patients do not have to undergo additional procedures.

In this combined approach, called DBS+, the surgeon transplants peripheral nerve tissue into an area of the brain where neurons are dying. The grafted cells are being tested for their ability to release chemicals believed to rejuvenate the brain’s weary dopamine-producing neurons.

Peripheral nerves – which lie outside the brain and spinal cord – have regenerative qualities that nerves inside the brain do not. The UK team hopes to leverage those regenerative effects within the brain, potentially halting or reversing nerve damage caused by Parkinson’s.

“While the peripheral nervous system can repair itself, the central nervous system does not do a very good job of it,” said neurosurgeon Craig van Horne, MD, PhD, co-director of the BRC and the study’s principal investigator. “So the question is can we tap into the ability of the peripheral nervous system’s response for repair. Can we bring that to the central nervous system?”

To test the effect of the graft, researchers can simply turn off the DBS pulse generator and evaluate patient’s symptoms at a baseline level.

The team’s vision is to alter the course of Parkinson’s. “With Parkinson’s disease, there’s no treatment to change the course of the illness because it’s a progressive disorder – there’s no treatment that does that. Our concept for DBS+ – the ‘plus’ part being the nerve grafting – is disease modification,” van Horne said. “Previously, all of the other transplant models were looking at symptoms and not disease progression and from that standpoint, that’s where we can say the DBS+ has its big advantage.”

DBS+ has been performed in 60 patients, from across Kentucky and from as far away as Florida, Texas, California and Canada. Initial results of the Phase 1 study are highly encouraging.

“We’ve seen good outcomes from a safety and feasibility point of view,” said George Quintero, PhD, clinical trial manager. Additionally, early participants have shown a clinically important improvement in symptoms, marked by a decrease in scores on the UPDRS – the Unified Parkinson’s Disease Rating Scale – the most widely used clinical rating scale for Parkinson’s. “We do see a significant change from baseline at one year in the participants that we’ve observed,” van Horne noted. “And the ones that we’ve followed out for two years, that change has increased and is sustained.”

Quintero said the team continues to bring a methodical approach to examining efficacy, with an eye to one day taking DBS+ into a multi-center, randomized, double-blind controlled trial.

Van Horne hopes DBS+ will eventually become the new “standard of care” for advanced Parkinson’s, improving the quality of life for his patients. “I’d really like to see this through to its fullest potential,” he said.