October 15, 2019
Pam Sullivan is a 64-year-old grandmother living with Parkinson's disease. While receiving advanced care from Kansas City's experts, she's also helping to shape the future of Parkinson's disease treatment and bring new therapies to market. She's an enthusiastic participant in clinical trials and is beginning her third since receiving her diagnosis.
"Participating in Parkinson's disease research makes me feel like having the disease has a purpose," Pam says.
Pam was 55 years old when she experienced the first symptoms of Parkinson's disease.
"While I was doing fast, repetitive motions, like whisking eggs, my brain stopped talking to my hand. My hand just stopped working," she recalls.
And there were tremors. Pam loves to sew – at the time, clothing, wedding dresses and Halloween costumes for friends and family, and today projects for her young grandson – but uncontrollable shaking in her right hand began interfering with her hobby. Concern sent her to her doctor and later to a neurologist. Pam quickly learned the neurologist was not a specialist in movement disorders nor a Parkinson's disease expert.
"I struggled along, taking medication to try to relieve my symptoms, but it didn't really work," she says.
A second opinion
Seeking more effective care, Pam turned to experts in her own backyard.
"I thought if I'm going to get a second opinion, I should get it from a renowned specialist," she says.
The University of Kansas Health System has one of the largest Parkinson's disease centers in the country. It is recognized by the Parkinson's Foundation among just 31 centers of excellence in the United States. In partnership with the University of Kansas Medical Center, the Parkinson's disease and movement disorder center offers dozens of the latest clinical trials for all stages of the disease.
The health system's patients benefit from a multidisciplinary team that provides complete care for Parkinson's disease and its broader effects. In addition to specialized physicians including neurologists, neurosurgeons, general surgeons, neurophysiologists, neuropsychologists, neuroradiologists, ophthalmologists, psychologists, dermatologists, urologists and anesthesiologists, the team also features specially trained pharmacists, speech, occupational and physical therapists, social workers and nurses who focus exclusively on Parkinson's disease care.
As soon as Pam described her symptoms to health system neurologist Rajesh Pahwa, MD, he identified the disease. Dr. Pahwa is an internationally recognized expert in the diagnosis, treatment and research of Parkinson's disease and director of the center.
"There is no definitive test for Parkinson's disease," he says. "However, a disconnect between the brain and a person's arms or legs is a classic symptom."
Our specialists offer unique experience in recognizing the symptoms that lead to an accurate diagnosis.
Pam lives and works in Overland Park, Kansas, as a business analyst for a software firm. She teaches clients how to use the company's products. During a training session after her diagnosis, Pam began with an introduction about Parkinson's disease to avert any distraction her tremors might cause.
"I want you to know why my hand is doing what it's doing so that I don't have to worry it's a distraction," she told her class. "I don't want you to be uncomfortable about this personal information. I'm explaining more for my comfort than yours, but if you'd like to talk to after our session, I'd be happy to answer questions."
Under Dr. Pahwa's care, Pam began a combination of carbidopa and levodopa, an oral medication commonly used to treat Parkinson's disease.
The treatment worked for Pam – but only for about 2 hours per dose. She experienced "on time" when the medication controlled her symptoms, but also "off time," when symptoms returned before it was time for her next dose. The fluctuations and their interference with daily activities led Pam to participate in her first clinical trial for Parkinson's disease.
The only way clinical research can progress is if patients like Pam are willing to do them. And patients who participate in trials – even if they get a placebo or sugar pill – do better than patients who are not in trials. – Rajesh Pahwa, MDDirector, Parkinson's Disease and Movement Disorder Center
Benefits of participation
"Very few patients participate in clinical trials," Dr. Pahwa says. "The only way clinical research can progress is if patients like Pam are willing to do them. And patients who participate in trials – even if they get a placebo or sugar pill – often do better than patients who are not in trials."
Participants in research studies often try new medications 5-8 years before they are commercially available and can receive the medication at no charge. The first clinical trial Pam participated in was a double-blind study in November 2012. It tested an anti-aging supplement to change the disease's course.
According to Dr. Pahwa, advancing age is the most significant risk factor for developing Parkinson's disease. The average age of onset is 60 years.
Expecting positive outcomes from participation in a trial is something Pam cautions others about.
"The people on the placebo actually did better than people on the supplement," Pam says.
Pam participated in a second clinical trial. In this one, a pump continually injected the drug combination under Pam's skin and into her abdomen. The outcome was much different.
"It was amazing," she says. "I had no tremors."
One of the drawbacks, however, was having to wear the pump all the time.
"The pump was the size of a box of checks, and I carried it in a holster over my shoulder," she says. "The visible holster and size of the pump made it pretty apparent I had something going on, but the tremor is pretty apparent, too."
At the end of the month-long trial, Pam offered feedback about the pump size and her desire to conceal it. Her input helped shape the third clinical trial in which she took part. It involves the same medication but a new, smaller pump that can be worn on a belt, similar to a cellphone case.
"I am very excited," she says. "The medication is supposed to be a better formulation that does not have to be custom-mixed and can be delivered to me by a pharmacy."
Despite the opportunity to take the medication, Pam carefully considered her participation in the year-long trial before making the commitment.
"You have to consider how the trial fits into your life," she says.
For example, during the second trial, it would have been difficult for Pam to travel. The infused medicine had to be compounded weekly by a specific pharmacy and picked up at the health system's Landon Center on Aging in Kansas City, Kansas. The medication also had to be kept refrigerated. Fortunately, that trial ended in time for Pam and her husband, Terry, to be at their grandson's birth in Tucson, Arizona. Pam hopes the third trial offers more flexibility.
"I have to say with all the things that can happen to you when you have Parkinson's, I have been blessed to only have a tremor in my right hand," she says. "I have been very pleased with my treatment at the health system and my experience with clinical trials with the medical center. Everyone there is pulling for people with Parkinson's disease to have the best quality of life they can."
This individual participated in a clinical trial of an investigational treatment. Clinical trials are different from standard medical care. As with all research studies, clinical trial participant outcomes vary. Before participating in a clinical trial, it is important to discuss the risks and benefits with your healthcare provider.