On January 6, the Michael J. Fox Foundation (MJFF) hosted a webinar called “Ask Us Anything,” with members of their leadership available to answer questions about Parkinson’s disease. They tackled questions that are frequently asked, such as how close scientists are to a cure for PD, as well as more specific inquiries, such as the purpose of a DaTscan or the use of mannitol. We at Stanford Parkinson’s Community Outreach listened to the webinar and are sharing our notes.
Ask Us Anything: Our Doctors Take Your Questions
Michael J. Fox Foundation
By Lauren Stroshane
January 6, 2020
Panelists included Rachel Dolhun, MD, a movement disorders specialist and VP of Medical Communications; Brian Fiske, PhD, Senior VP of Research Programs and Therapeutics Portfolio Lead; and Mark Frasier, Senior VP and Biomarkers Portfolio Lead. The discussion began with a recap of notable projects the Michael J. Fox Foundation (MJFF) had worked on in 2019, then proceeded to cover questions submitted by the webinar audience.
MJFF hosts these webinars on the third Thursday of each month. The January 6 webinar was recorded. You can find the recording here.
If you have questions for MJFF about content in this webinar, you can contact them via email or via phone at 800-708-7644.
Question & Answer Session with the Panelists:
FREQUENTLY ASKED QUESTIONS
Q: How close are we to a Parkinson’s cure?
A: It depends on how you define a “cure.” One definition of a cure is whether we can help someone with the disease and address many of their symptoms; in this sense, we have advanced pretty far already, with the development of levodopa and other symptomatic treatments.
Another definition of a cure is the ability to slow progression of a disease. Here, too, we have made some advances: we have a lot of genetic and biological insights into the mechanisms in our bodies that, when disrupted, can lead to PD; we know that exercise can slow its progression.
A third definition of a cure is the capacity to restore health that has been lost to someone with substantial disease, by replacing, repairing, or otherwise fixing. Currently, Deep Brain Stimulation (DBS) is an attempt to go in and fix the “circuitry” problems in PD. Eventually, cell treatments may be able to repair diseased areas in the brain.
A fourth definition of a cure, and perhaps the ultimate one, is to prevent someone from getting PD in the first place, by identifying from risk factors or at very early stages of disease and offering intervention to prevent the disease from occurring. Scientists hope to one day have these capabilities.
Q: Why does someone need to see a movement disorders specialist, rather than a general neurologist?
A: A Movement Disorders Specialist is a neurologist with additional training (often a 2-year fellowship) and expertise in movement disorders such as PD, essential tremor, and dystonia. Those with PD typically report feeling more informed about their care when they have seen a specialist.
While a general neurologist can often provide care for someone with PD in the early stages of disease, Movement Disorder specialists are typically better equipped to handle complications or challenges that come up in PD, and are usually more up to date on the research.
It is often beneficial to see both! A local neurologist can provide good day-to-day care and handle medication refills, while a visit once or twice a year to a movement disorder specialist for “tune ups” can be the most well-rounded model of care.
Q: How do I access genetic testing for PD-linked mutations?
A: Genetic testing for personal reasons (i.e., to learn more about your disease) is different than genetic testing in a research setting. Clinical trials often allow you to provide your genetic material with the option not to learn the results yourself, if you prefer not to. Limited genetic testing is widely available now through services like 23andMe or through a doctor’s office, but does not always contain every gene that might be clinically relevant to your disease.
An important caveat: genetic counseling is really important, both before and after testing. There are certain things that the genetic tests can’t tell you about the disease and implications for yourself and your family. A genetic counselor will help you identify what you would learn by undergoing testing, and will help you understand the results if you decide to proceed. It is important to remember that, currently, genetic testing results won’t change your treatment at all.
However, there are clinical trials happening now that are testing drugs and therapies on people with certain genetic mutations. For instance, if you found out that you had a LRRK2 mutation, or GBA (two genes that, if mutated, are known to contribute to the development of PD), you might be eligible to participate in some clinical trials.
For more information about the LRRK2 gene, visit here.
For more information about the GBA gene, visit here.
Q: If I have family risk factors for PD, what can I do?
A: Monitor your own health, get lots of exercise, and stay abreast of the latest research and treatment options. Research studies greatly need people with PD but also health controls or with a family history of PD. See if you are eligible to participate in any clinical trials and know that you are helping further our understanding of the disease.
Q: What’s the latest in stem cells?
A: Stem cells are essentially cells in our body that have the potential to turn into other types of cells. Scientists have also made artificial stem cells in the body from other existing cell types, called “Induced Pluripotent” stem cells (IPSC’s). Therapeutically, we want to know if we can use these IPSC’s to restore some of the function that has been lost in neurons in PD. However, these technologies are years away from being available to patients. Eventually, these cellular treatments will probably useful, but may only be as good as levodopa or DBS. IPCS’s also have a lot of potential to assist with research into the basic biology of PD as well, and could be used to test the efficacy of new drugs as well.
Unfortunately, there are a lot of clinics out there in the world that claim to offer these types of stem cells therapeutically and “cure” your PD; these stem cell treatments are not currently approved by any regulatory body and may be fraudulent, or even dangerous.
The MJFF’s recent webinar, “Update on Stem Cell Treatments in PD,” can be accessed here .
Q: What is a DATscan, and should I get one if I have PD (or am at risk for PD)?
A: The DaTscan is a brain scan that is currently approved by the FDA as a tool for diagnostic purposes. It involves the intravenous injection of a drug, Ioflupane, to create an image of the brain that shows activity of dopamine cells. In PD, one of the primary changes that occurs is the loss of dopamine-producing neurons in the brain. The DaTscan is a way to image these cells and get a sense of how much dopamine the brain is able to produce. It can also be a useful research tool for drug developers to incorporate in their evaluation of new drugs. However, the scan is extremely expensive and not always covered by insurance.
PD is a “clinical diagnosis,” meaning that the gold standard for diagnosis is your history and physical exam. Studies have shown the clinical exam is as effective as DaTscan for accurately diagnosing PD, and poses far less risk and expense.
The DaTscan can be useful for differentiating PD and essential tremor (ET), since the dopaminergic system is not affected in ET. However, atypical parkinsonian disorders such as Multiple System Atrophy, Corticobasal Syndrome, and Progressive Supranuclear Palsy will also have an abnormal scan result. Thus a DaTscan does not differentiate between PD and one of these diseases.
For more information about DaTscans, check out the APDA’s article on this topic.
Q: Is medical marijuana a good option for those with PD?
A: Some clinical trials of cannabinoids in PD have aimed to determine if marijuana is helpful for dyskinesia, tremor, and movement symptoms. The data so far has been inconclusive, with mixed results. One challenge is that these studies have looked at all different kinds of marijuana – THC, CBD, different doses & formulations –it’s not standardized.
We don’t have the data yet to support marijuana as a therapy for PD. Anecdotally, for non-motor symptoms like pain or sleep, some patients do find it helpful. Lots of people use it, but it has lots of potential side effects as well.
The MJFF recently produced a webinar on medical marijuana, accessible here.
Question & Answer Session with the Panelists:
LIGHTNING ROUND
Q: Are muscle cramps part of PD?
A: Typically, those with PD may experience muscle spasms (also called “dystonia”) rather than cramps. Dystonia pulls a body part into an abnormal position, such as toe curling or foot turning. This often occurs when PD medications are wearing off, and can be quite uncomfortable. Make sure to mention it to your Movement Disorders neurologist if you are experiencing this, as there are a number of strategies to try to address it, such as Botox injections, changing the medication schedule, and physical therapy.
Q: What is atypical parkinsonism?
A: This is a blanket term to describe movement disorders that are similar to PD (“parkinsonian”) but have a slightly different biologic cause and tend to progress differently than PD.
These include Multiple System Atrophy (MSA), Corticobasal Syndrome (CBS or CBD), and Progressive Supranuclear Palsy (PSP), among other diagnoses. They present early on with PD-like symptoms, but progress differently (sometimes more rapidly) over time. It can be a challenge for clinicians to diagnose atypical parkinsonism early on, because it resembles PD, which is far more common. As symptoms evolve over time, then the diagnosis usually becomes more definitive.
Further reading about atypical parkinsonism can be found at the APDA.
Q: Do some people have a less aggressive form of PD than others?
A: PD is a very heterogeneous disease – it’s different for everyone. Some people do progress at a slower rate than others. Historically, we lump people into very general categories:
- Tremor-dominant – tend to have slower progression
- Walking & balance issues – tend to have a little bit of faster progression
Those with genetic risk factors for PD might experience more cognitive impairment or earlier onset of the disease. Scientists are doing research to try to figure out ways to eventually predict if someone will get PD, what their symptoms will be, and how fast they will progress.
Q: Is mannitol a treatment for PD?
A: Mannitol is a sugar substitute, often used as a sweetener for those with diabetes. Researchers identified mannitol in a study that seems to affect alpha-synuclein clumping. The idea is maybe mannitol could make its way into the brain and help prevent this clumping; however, since it was only tested in lab models for safety and tolerability so far, we don’t yet know if this actually affects people with PD. The takeaway: mannitol won’t harm you as long as you stick to reasonable amount guidelines, but there’s no evidence yet that it will have any effect on your disease.
Q: When should I start on levodopa?
A: This is a difficult question to answer because it is very individualized. Levodopa (Sinemet is the brand name) is the gold standard of treatment for PD. There are no hard and fast rules around the use of this medication, and no “cook book.” Medications for PD all treat the symptoms but cannot slow or stop the disease itself.
When symptoms get in the say of things you want to do, such as travel, playing with grandkids, or exercise, then you should start on medication.
There is a history of people being reluctant to start on Sinemet due to the potential for developing dyskinesias, which are uncontrolled movements often triggered by medication. But not everyone gets dyskinesias; for those who do, it’s not always bothersome, and we have ways to manage it. As they say, “don’t save your umbrella if it’s raining” – it’s best to start medication sooner rather than sacrificing your quality of life out of concerns for a hypothetical complication down the line.
Q: What is young-onset PD versus early-onset PD?
A: These mean the same thing! Young-onset PD (YOPD) or early-onset PD is typically defined as a diagnosis before the age of 50. Early stage PD refers to the period within the first few years of diagnosis, sometimes before medication has been started.
Q: Is there a connection between gut bacteria and PD?
A: There’s an idea that maybe there is early PD pathology in the gut, before other symptoms arise in the brain. Many people with PD have constipation, for instance. If we can target the microbiome (the microbes that populate our intestines), then maybe this could help treat PD. Additionally, for those who take oral medication, sometimes there is concern that the intestines are not able to absorb the medication well. Maybe targeting in the microbiome could help improve drug absorption.
An article in the Scientific American on what we currently know about this question.