New Approaches to Treating Movement Disorders

The Ohio State University Wexner Medical Center’s Movement Disorders Division offers advanced therapies to treat more than 100 patients a year with movement disorders like Parkinson’s disease, essential tremor, dystonia and Tourette syndrome. These advanced therapies include deep brain stimulation, MRI-guided focused ultrasound, gene therapy and infusion therapies.

“Advanced therapies can provide meaningful benefits to patients,” says Aristide Merola, MD, PhD, a former neurologist at Ohio State. “Patients can expect a 30% to 50% improvement in quality of life compared to oral medication.”

Deep brain stimulation

Deep brain stimulation (DBS) is a common surgical procedure to treat patients with Parkinson’s disease, essential tremor or dystonia. It addresses a variety of disabling neurological symptoms associated with these conditions, including tremor, slowed movement, rigidity and stiffness.

During the procedure, electrodes are implanted into the brain and connected to a pacemaker-like device that is implanted into the chest or abdominal wall. The electrodes send electrical signals to the brain to calm abnormal signals, thereby reducing disabling symptoms and restoring physical function.

“At The Ohio State University, we use the highest standards for DBS surgery. To improve the accuracy of DBS, we use tractography to integrate a detailed study of each patient’s individual anatomy into the surgical planning,” Dr. Merola, who is an associate professor – clinical at the College of Medicine, says.

While many health care systems offer DBS, few offer the treatment using a tractography-based approach. With tractography, neurosurgeons are able to target and treat a single system of fibers and steer away from systems of fibers that may cause side effects. The goal is to minimize side effects and maximize benefits to the patient.

MRI-guided focused ultrasound

The team also provides magnetic resonance imaging (MRI)-guided focused ultrasound, a system that is similar to radiotherapy but does not involve the use of X-rays. With this approach, physicians convey ultrasounds to target the area of the brain that is responsible for symptoms. These ultrasounds can safely generate thermal ablation to treat the area without needing to open the skull.

This Food and Drug Administration (FDA)-approved procedure is currently available for patients with essential tremor and Parkinson’s disease. The Ohio State team is working to improve the outcomes of this innovative approach with tractography-based MRI approaches and to extend its indication to new diseases.

Gene therapy

“We are deeply invested in cutting-edge research such as gene therapy,” Dr. Merola says. “Gene therapy is a revolutionary approach to treating neurodegenerative disorders.”

With gene therapy, the focus isn’t only on managing symptoms, but also on providing disease-modifying treatment, he says.

Ohio State is currently in phase I of a clinical trial for patients with Parkinson’s disease and multiple system atrophy. The team is trialing the ability of viruses to infect neurons with DNA that produces glial cell-derived neurotrophic factor (GDNF) that helps neurons grow, so they are protected from the effects of neurodegeneration.

GDNF is a compound produced by the glial cells in the brain. The function of these cells is to support and provide a nourishing environment for neurons. If they degenerate, this nourishing environment goes away.

One goal of gene therapy is to boost the production of this nourishing environment, so even though the brain is aging, it can still have the protection and support of neurotrophic factors to help prevent neurodegeneration.

“We are at the very beginning of this line of research, but with the support of our institution, collaboration with the Department of Neurosurgery and excellent scientific research, we are extremely positive about the results that might be obtained,” Dr. Merola says.

Infusion therapies

Ohio State offers infusion therapies when oral medications no longer offer patients the best results.

The first is an FDA-approved formulation of carbidopa/levodopa that continuously delivers a liquid form of antiparkinsonian medications through a pump into the bowel. This approach guarantees clinical results that are similar to DBS with a less invasive surgical approach.

Also available is a subcutaneous infusion, which was already developed for diabetes and offers continuous delivery of medications so patients have consistent performance during the day. This system is still in a phase of research, but as an academic institution, Ohio State can offer leading-edge treatments that often aren’t available at non-academic medical centers, Dr. Merola says.

The benefits of advanced therapies

At the beginning stages of a neurological movement disorder, most patients can be treated with oral medications. However, when the disease progresses, the medications gradually become less effective.

Larger doses of medications can come with more side effects, resulting in abnormal movements, drowsiness and confusion. In addition to these side effects, patients still have debilitating symptoms for most of the day.

“We consider patients for more advanced treatments when they no longer have good control of their symptoms with oral medications and when they have side effects,” Dr. Merola says.

“When a patient has to make more social or working adjustments, we want to be aggressive in the timing of surgery or advanced treatments. Our goal is to treat them before social adjustments occur. We want to make sure they don’t have to give up their lives, friendships or activities. Thanks to advanced treatments, they can remain functional in those activities for a longer period of time.”

A team approach

“We truly believe in the importance of making an accurate selection of therapy for our patients,” Dr. Merola says. “Our team discusses every case in a combined surgical meeting of neurologists, neurosurgeons, neuropsychologists and neuroimaging experts to identify what treatment is best for each patient. We don’t believe in a one-size-fits-all approach.”