This One Is For Amy

Amy Shimberg is the Grande Dame of our Parkinson’s support group at Florida Hospital Tampa. She is also matriarch of the civicly active Shimberg family, a crowd of Tampa doers and builders.

Amy is the person I quoted in my last post wanting the most up-to-date books on Parkinson’s disease to read. So this post is for you Amy.

The prolific Dr. Michael Okun, prominent University of Florida neurologist and researcher, has come out with a new book about Parkinson’s: “10 Breakthrough Therapies for Parkinson’s Disease.” It is a follow-up to his earlier and very popular “Parkinson’s Treatment: 10 Secrets to a Happier Life.”

On my first, quick read, I thought the new book might be too technical for the lay reader. A second, closer read changed my mind. Okun is exceedingly skilled at writing first a general-interest narrative, then diving deep into the same material for the more technical reader.

Chapter titles give a flavor of the book’s range of topics: disease modifying drugs and biomarkers; coffee, tea, exercise, interdisciplinary teams and caregivers; extended release/novel third delivery systems, to name a few.

Okun’s definition of a breakthrough is a bit broader than I’m used to. “A breakthrough is defined as a sudden increase in knowledge, improvement in technique, or for fundamental advancement and understanding,” he writes. “Often breakthroughs occur when a formidable obstacle is penetrated.”

Okun is unquestionably optimist about progress in understanding and treating Parkinson’s and the possibilities of new therapies to slow or stop Parkinson’s. But he is also a realist about the many dead ends research has produced.

“I always share with patients and families the simple truth that our knowledge of the brain is advancing rapidly,” Okun writes. “Despite the expected failures in the majority of Parkinson’s disease clinical trials, these failures will guide us to the breakthroughs.”

A rather large bone I have to pick with Okun is his description of the sophistication of the Parkinson’s patient. “I am convinced that the Parkinson’s disease patient is possibly the most sophisticated consumer of healthcare.”

Patients he sees at his Center of Excellence in Gainesville may be that way. But patients I have encountered in Tampa do not fit that description. They are very frustrated, for the most part, with what they don’t know about treatment options.

I ran into fun facts in the book. One in particular drives my wife nuts. “Smokers have been observed in multiple studies have a lower risk of developing Parkinson’s disease, and there has been ongoing interest in nicotine as a disease modifying approach in Parkinson’s disease patients.” She is no fan of my two-cigar a day ritual. Okun’s words deflate her arguments, only in part, of course.

Okun endorses the benefits of moderate exercise. He says “… low intensity workout on the treadmill paired with resistance exercises have the potential to be the most beneficial combination for Parkinson’s disease.”

I was encouraged to read Okun’s endorsement of team-based approaches to PD patient care. It is powerful support for emerging ideas from the palliative care movement. He likes a team that includes neurologist, psychiatrist, social worker, health counselor and counseling psychologist.

All Parkinson’s disease patients should also have a general practitioner and a dermatologist involved with patient care. “The reason for involving other doctors is because with adequate Parkinson’s treatment the patient will be more likely to encounter difficulties with other medical illnesses (heart disease, prostate cancer, breast cancer, melanoma, etc.) Melanoma,for example, occurs more frequently in Parkinson’s disease patients.”

I was most surprised at what Okun had to say about the use of marijuana in PD treatment: “There is mounting evidence that marijuana may be useful for the treatment of Parkinson’s disease symptoms,” he writes. “The fundamental issue in the field is that we do not yet know which symptoms of Parkinson’s disease patients medical marijuana may be useful.”

He concludes: “I always share with patients and families the simple truth that our knowledge of the brain is advancing rapidly. Despite the expected failures in the majority of Parkinson’s disease clinical trials, these failures will guide us to the breakthroughs.”

Further: “A good method to monitor drugs that may be available as part of research studies is using the website http://www.clinicaltrials.gov. Though the cost in time and money can be tremendous for Parkinson disease drugs, there is hope that new symptom-specific approaches will be introduced to address bothersome and previously drug resistance symptoms (such as walking, talking and thinking). There is also a hope that a new future drug will meaningfullly slow disease progression.”

Okun welcomes communication. His email is Michaelokunmd@Gmail.com. Twitter is @Michaelokun. Website is http://parkinsonsecrets.com.

USF’s Hauser Explains the Possible Role of alpha-synuclein in Parkinson’s

Dr. Robert A. Hauser wrote recently in the prestigious medical journal Lancet about the role of the protein alpha-synuclein in the Parkinson disease process. Excerpts follow.

“When patients asked me when we will have a cure for Parkinson’s disease, I still say that I don’t know. But unlike a few years ago, I now add that scientists have uncovered the major part of the disease process and we can now envision what a cure might look like and what needs to be done to get there. I am referring to the fact that propagation of alpha-synuclein missfolding and aggregation seems to be at the heart of most types of Parkinson’s disease and that this process presents several attractive opportunities for therapeutic intervention…

“Nonetheless, the demonstration that aggregated alpha-synuclein is the main component of Lewy bodies, even in patients without mutation in the alpha-synuclein gene, widely implicated alpha-synclein as central in the disease.

” … Many preclinical studies have suggested that alpha-synuclein can behave in a prion like fashion, with misfolding and aggregation, and propagation from neuron to neuron by a templating process.

“…Because synuclein targeting and other potential disease modifying therapies are now rapidly being developed, we need ways to assess their effectiveness in patients and we need a pathway to regulatory approval.

“… Substantial progress is being made in our efforts to provide patients with a robust symptomatic antiparkinsonian response through the day. New medications in development include a longer acting oral levodopa formulations and acute, intermittent therapies to treat “off” episodes, such as inhaled Levodopa and sublingual asomorphine. Additional options for patients with uncontrolled motor fluctuations include deep brain stimulation, levodopa-carbidopa intestinal infusion, and asomorphine or levodopa-carbidopa subcutaneous infusion. By contrast, highly effective symptomatic therapies for disabling long term features such as balance impairment and cognitive dysfunction seem beyond our grasp, thereby really re-emphasizing the need to slow and ultimately stop progression of this disease.

“Much work needs to be done, and we should not become overconfident or complacent. However the ongoing unraveling of the alpha-synuclein story allows me to tell patients that we appear to be on the cusp of seeing disease-modifying therapies emerge from preclinical work. Seems like we are on the right track.”

Hauser is a Professor of Neurology, Molecular Pharmacology, and Physiology at the University of South Florida Morsani College of Medicine, in Tampa. He serves as Director of the Clinical Signature Interdisciplinary Program in Neuroscience and as Director of the USF Health Byrd Parkinson’s Disease and Movement Disorders Center.


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