Some 25 years ago, a young Parkinson’s disease investigator, Robert Hauser, MD, stepped to the podium at an American Academy of Neurology Conference to present findings from his patient survey that didn’t quite jive with classic beliefs. And he was about to discover that it can be hard to stand your ground as a newcomer challenging the status quo.
Dr. Hauser, who today serves as director of the USF Parkinson’s Disease and Movement Disorders Center and professor of Neurology in the USF Health Morsani College of Medicine, had asked 176 patients to list their top three chief complaints from the condition. The No. 1 response: tremor.
Dr. Robert Hauser
On its face, that didn’t necessarily seem surprising. But it contradicted the prevailing view that tremors in Parkinson’s only occurred when the patient’s arms were in a resting position — and thus would not hinder a patient engaged in movement, such as typing, using cooking utensils or keeping a key steady to open a door.
“After the presentation,” he recalled recently, “multiple leading senior experts stepped to the microphone to explain why the results were wrong and noted that the tremor of Parkinson’s disease is a rest tremor so therefore doesn’t cause disability.”
Dr. Hauser remembers being so unnerved by the senior experts that he thought he would never be able to have the results published. “So I foolishly never wrote the manuscript describing the study and its results,” he said.
That is, until now — with an updated, comprehensive exploration recently published in the Journal of Parkinson’s Disease with Dr. Hauser as first author.
The new study surveyed 634 people with Parkinson’s disease, or PD. It finds that people with PD report that tremor is one of the most troublesome symptoms — and, in fact, that PD tremor “is often more than a rest tremor and frequently impacts function and activities of daily living.”
Furthermore, the study concludes that medication is not an adequate treatment in about half the cases, underscoring the point that tremor is an important unmet need. The findings also pointed out that while tremor is one of the most common PD symptoms, “until now, there has not been much research done to understand, from the patient’s perspective, how living with tremor impacts their ability to do simple things and their overall quality of life.”
In one sense, the paper serves as dual validation — both for Dr. Hauser, who was able to validate the results of his original study, and for patients who feel heard in a fundamental way.
“For 25 years, I’ve been listening to patients say, ‘Hey, what can you do for this tremor? I don’t like it’ and it takes me back to those experts telling me, ‘It just occurs during rest and doesn’t interfere with function — it’s no big deal,’ ” he said. “Well, they were wrong.”
In addition to determining that tremor is far more of a quality-of-life issue and that medications to treat it are frequently insufficient, the study underlined other aspects of Parkinson’s tremor: psychological and physical.
“From a psychological perspective, patients don’t like the way it looks,” Dr. Hauser said. “It reminds them of their disease. For people who have to interact with clients, it undermines their ability to generate confidence. And when people with Parkinson’s get anxious, the tremor gets worse, so it’s a ‘tell,’ adding to the psychosocial component. And from a physical standpoint, it’s uncomfortable and fatiguing. So tremor affects all three domains: It interferes with function. It has a psychological impact. It affects how a patient feels — and all three ultimately interfere with quality of life.”
The study also looked at the efficacy of medications available to treat Parkinson’s. Dr. Hauser’s findings show that available medications do a good job in controlling tremor in only about a quarter of cases.
“In another quarter of cases, they help a little bit, and that leaves about half the time that medications don’t work at all,” he said. “So, this article sends a message to the pharmaceutical community — current medications aren’t that good for tremor. We need better ones.’”
Many drug companies, Dr. Hauser explained, are still rooted in the belief that tremor does not interfere with function.
“In that case, why would a pharmaceutical company develop new medications for Parkinson’s Disease tremor?” he said. “Well, they wouldn’t. So, part of the message from this study is: ‘This is a big problem. Patients are complaining to me about it every day, and I don’t have good pharmaceutical treatments for it — so come on, we need research and development of medications to help this problem.”
The current study was funded by one such company, Jazz Pharmaceuticals, and was conducted in association with the PMD Alliance, an organization that promotes Parkinson’s education and care. Dr. Hauser also notes that his original study from 1997, though never published, was done with the help of then-medical student Cathy Wendell.
But Dr. Hauser holds onto a lesson learned from that early setback and hopes it will benefit others.
“I was so intimidated by these senior experts that I thought we would never be able to get the results published — and therefore I foolishly never wrote the manuscript describing the study and its results,” he said. “I now describe my failure to publish that study in manuscript form as my biggest academic regret and use it to teach residents not to be intimidated, but rather be spurred on when established experts tell them their results are incorrect.”
“In retrospect, now that I’m a senior investigator, I know that when people are telling you you’re wrong, you may well be on to something.”