Some 25 years ago, a young Parkinson鈥檚 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鈥檛 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鈥檚 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鈥檛 necessarily seem surprising. But it contradicted the prevailing view that tremors in Parkinson鈥檚 only occurred when the patient鈥檚 arms were in a resting position 鈥� 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.
鈥淎fter the presentation,鈥� he recalled recently, 鈥渕ultiple leading senior experts stepped to the microphone to explain why the results were wrong and noted that the tremor of Parkinson鈥檚 disease is a rest tremor so therefore doesn鈥檛 cause disability.鈥�
Dr. Hauser remembers being so unnerved by the senior experts that he thought he would never be able to have the results published. 鈥淪o I foolishly never wrote the manuscript describing the study and its results,鈥� he said.
That is, until now 鈥� with an updated, comprehensive exploration recently published in the with Dr. Hauser as first author.
The new study surveyed 634 people with Parkinson鈥檚 disease, or PD. It finds that people with PD report that tremor is one of the most troublesome symptoms 鈥� and, in fact, that PD tremor 鈥渋s often more than a rest tremor and frequently impacts function and activities of daily living.鈥�
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, 鈥渦ntil now, there has not been much research done to understand, from the patient鈥檚 perspective, how living with tremor impacts their ability to do simple things and their overall quality of life.鈥�
In one sense, the paper serves as dual validation 鈥� 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.
鈥淔or 25 years, I鈥檝e been listening to patients say, 鈥楬ey, what can you do for this tremor? I don鈥檛 like it鈥� and it takes me back to those experts telling me, 鈥業t just occurs during rest and doesn鈥檛 interfere with function 鈥� it鈥檚 no big deal,鈥� 鈥� he said. 鈥淲ell, they were wrong.鈥�
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鈥檚 tremor: psychological and physical.
鈥淔rom a psychological perspective, patients don鈥檛 like the way it looks,鈥� Dr. Hauser said. 鈥淚t 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鈥檚 get anxious, the tremor gets worse, so it鈥檚 a 鈥榯ell,鈥� adding to the psychosocial component. And from a physical standpoint, it鈥檚 uncomfortable and fatiguing. So tremor affects all three domains: It interferes with function. It has a psychological impact. It affects how a patient feels 鈥� and all three ultimately interfere with quality of life.鈥�
The study also looked at the efficacy of medications available to treat Parkinson鈥檚. Dr. Hauser鈥檚 findings show that available medications do a good job in controlling tremor in only about a quarter of cases.
鈥淚n another quarter of cases, they help a little bit, and that leaves about half the time that medications don鈥檛 work at all,鈥� he said. 鈥淪o, this article sends a message to the pharmaceutical community 鈥� current medications aren鈥檛 that good for tremor. We need better ones.鈥欌��
Many drug companies, Dr. Hauser explained, are still rooted in the belief that tremor does not interfere with function.
鈥淚n that case, why would a pharmaceutical company develop new medications for Parkinson鈥檚 Disease tremor?鈥� he said. 鈥淲ell, they wouldn鈥檛. So, part of the message from this study is: 鈥楾his is a big problem. Patients are complaining to me about it every day, and I don鈥檛 have good pharmaceutical treatments for it 鈥� so come on, we need research and development of medications to help this problem.鈥�
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鈥檚 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.
鈥淚 was so intimidated by these senior experts that I thought we would never be able to get the results published 鈥� and therefore I foolishly never wrote the manuscript describing the study and its results,鈥� he said. 鈥淚 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.鈥�
鈥淚n retrospect, now that I鈥檓 a senior investigator, I know that when people are telling you you鈥檙e wrong, you may well be on to something.鈥�