By Ramon A. Gil, M.D., Medical Director, Parkinson’s Disease Treatment Center of SWFL
Welcome to April: “Parkinson’s Disease Awareness Month”. It’s hard to believe it, but we made it through the month of March and here we are, trying to get our country and our lives back. The question now is: “Do you remember Parkinson’s Disease?”. I hope so, because we are bringing you good news in reference to the progress that basic science and clinical research has brought to the front of the battle with this chronic, progressive, but the most treatable neurodegenerative disease.
In the field of basic science, deeper understanding of the different mechanisms in which the nerve cells are damaged in Parkinson’s Disease (PD) has led to numerous research projects. Scientists are looking at multiple ways to prevent the formation, the accumulation and the rate of excretion of alpha synuclein (the protein associated with the death of nerve cells in the brain and in the periphery). In the future (and it may be sooner than we think) an effective and safe way to do that should be achieved. Once earlier identification of “pre-clinical” PD is achieved, we could picture a way to stop the disease before it gets to the brain and then we will be looking at a whole new definition of PD, particularly without the neurological motor problems that we are so familiarized with.
With the assistance of focused ultrasound (FUS) technology, the field of gene therapy continues to advance. FUS is now being used (experimentally) to open the blood brain barrier and allow the delivery of therapeutic substances into the brain. This includes gene vectors, with the idea of modifying the expression of the defective genes associated with the clinical expression of PD. This technology is also attempting to correct, or improve, the availability of the “natural healing substance” known as “glial cell derived neurotrophic factor (GDNF).” This could lead to the reactivation of the natural production of dopamine in the brain, with remarkable benefits for patients with PD.
Originally and for many years, PD was identified based on motor symptoms associated with dopamine deficiency. This neurotransmitter was and, in many ways remains, the center of the disease treatment. “Non-motor symptoms” (NMS) of PD were recognized and addressed relatively recently. Most of them are linked to deficiency of other neurotransmitters such as: noradrenaline, serotonin and acetylcholine.The improvement in the management of motor symptoms has made more apparent the impact that NMS has in the quality of life of patients with PD. An increasing number of these problems already have effective therapy with FDA approved drugs. Neurogenic Orthostatic hypotension with (droxidopa/
Northera), PD psychosis with (pimavanserin/Nuplazid) and drooling with (botulinum toxin type A: Myobloc, Xeomin) are some examples. We now have off-label ways to treat gastroparesis (slow emptying of the stomach). For constipation, experienced by 90% of patients with PD, an experimental drug ENT-01 (currently in phase II trials) appears very promising as an effective agent, hopefully better than the commonly used OTC and prescribed laxatives. Soon, we will start enrollment in a phase II clinical trial for mild cognitive impairment (MCI) in patients with PD. To this date, there is no approved drug proven effective for this very common problem. For the time being, physical exercise has been demonstrated effective (if the patients are willing and consistent with “the treatment”). Once the drug has been approved, nothing indicates that exercise and drug treatment are incompatible.
The number of new medications and reformulations of older drugs (to improve efficacy and tolerability) is too large to be described in this article. Levodopa (Rytary, Duopa, Inbrija), once a day amantadine (Gocovri), istradefylline (Nourianz) and safinamide (Xadago). These are some of the agents that you need to look into, as they have changed for good the outcome of treatment of patients with PD and their quality of life. Soon to be released, a new once a day medication (opicapone) will be available, it prolongs the duration of the effect of levodopa. New formulations of rasagiline and pramipexole, a sublingual form of apomorphine and a new dopamine agonist (tavapadon) are among those in the pipeline. With better delivery systems, some of them will have longer lasting effects. The advantages of these new drugs are countless and often make the use of older drugs obsolete. Just think for a moment what year is the car you drive? And then think how old is the drug that you are taking?
Finally, progress is not limited to pharmacologic therapy. Surgical treatment has seen a huge progress in the last 5 years. There are now two more systems of Deep Brain Stimulators available. They have the
ability to provide more selective stimulation to smaller areas of the brain, limiting side effects and reducing the amount of energy required to achieve optimal results with subsequent longer life of the battery. The battery life may soon be an irrelevant issue since the new rechargeable batteries are much better, easier to recharge and last up to 15 years. The concept of “directional leads” or “steering” describes the ability of these new systems to redirect the field of electrical stimulation on different directions without the needs to change the position of the intracranial lead.
Quoting Dr. A. Rana, founder of the “World Parkinson’s Program”: “those who fight Parkinson’s with knowledge always find solutions”. We thank you for reading these lines searching for knowledge and seeking for solutions to your problems. At Parkinson’s Disease Treatment Center of SWFL we are committed to deliver the state-of-the-art treatment for PD and work with you and your loved ones to optimize your quality of life and theirs.
We hope to see you in our upcoming educational program this coming Friday, April 17.
Ramon A. Gil, M.D.
– Diplomate American Board of Psychiatry and Neurology
– Diplomate American Board of Internal Medicine
– Former Clinical Associate National Parkinson Foundation
– Medical Director and Founder, Parkinson’s Disease Treatment Center of SWFL
Parkinson’s Disease Treatment
Center of SW Florida
4235 Kings Highway, Unit 102, Port Charlotte, FL 33980
415 Commercial Court, Suite E, Venice, FL 34293
8931 Colonial Center Drive, Suite 401, Fort Myers, FL 33905