At the end of March, Parkinson Canada hosted a two-day discussion on the current and future states of Parkinson’s research. The meetings brought together clinicians, researchers, people living with Parkinson’s and other Parkinson’s organizations, with the aim to achieve the following:
- Identify current and emerging trends in basic and clinical Parkinson’s research
- Explore strategies to leverage our research investments and increase impact, possibly through partnerships with other Parkinson’s organizations
In upcoming editions, we’ll share more on the outcomes of the meetings including the resounding takeaway that having people with Parkinson’s involved at all stages of research was important to its success. In this post, we review a presentation shared by Dr. Maxime Rousseaux on the ideal future of Parkinson’s treatment. His summary on the “not-so-distant future of Parkinson’s treatment” follows.
I think many goals of basic science researchers like myself are focused on disease modifying treatments. But, the biggest issue we have is that we can’t test these on people who are well into the progression of their disease. In order to find treatments that can help these people, we need to first test whether we can delay the progression or otherwise alter the course of Parkinson’s on a patient before their symptoms progress to that level.
I think the biomarker argument is kind of obvious – we need to find one to test these treatments, but also so that we can diagnose the disease earlier.
When we achieve this, I think it’ll probably be feasible to halt neurodegeneration so you know if an individual, let’s say you can manage their symptoms using a certain dose of Levodopa and at least we can just stop the neurodegeneration.
That’s probably going to be one of the earlier milestones that that might be seen.
The main focus of my own research is doing two things, figuring out how Alpha-synuclein is toxic and then trying to find a treatment that will clean that toxicity. There are a few different ways we could go about that. An easy example to understand is to try to find something that’s a traditional target for drug therapy that may already have a medication designed to reach that enzyme. We could attach something to it to decrease the negative effects of Alpha-synuclein. We’re working on a goal now that if you target it, either genetically or with a drug, it can help to limit its toxicity.
Ultimately, the goal is the “statin model”.
In the case of cholesterol and statins there are commonly accepted indicators of risk or likelihood to develop an issue, there is an easy way to test and confirm a diagnosis and a readily available medication to start to treat the symptoms before they become a true problem.
It all starts with our ability to diagnose the disease. If you want to catch the bulk of people, you would follow what we know now to be the longitudinal progression of Parkinson’s. Some of that stuff might actually be informed by common screening tests for symptoms or preclinical markers. If those screening tools help us to identify something is at a certain progression of risk, it would be someone who qualifies for more detailed testing.
The four steps I presented in this model are:
- Someone goes to the doctor’s office for a regular visit (pre-symptomatic) and that doctor may have a screening tool to help identify their risk for Parkinson’s
- They’ll order bloodwork, which can use some combination of biomarker and genetic indicators to suggest a diagnosis of Parkinson’s
- Within a few days, that diagnosis is confirmed
- The patient can begin to take medication to treat the disease, including potentially by subtype, before the onset of symptoms using some disease modifying therapy
What it boils down to is that we need an easy test, or a set of uniform criteria that help to indicate who is at a greater risk of developing Parkinson’s. There is a lot of research in this area already, including the impact of loss of sense of smell; gastric motility; sleep factors and much more. If we have a commonly agreed upon set of criteria to assess which combination of factors are indicators of increased risk – and I know a number of researchers including former colleagues who are working on these – we can then identify people most likely to develop Parkinson’s. Then, we move on a test to confirm diagnosis. This is the area that I’m most hopeful about.
From there, I think that treatment by subtype – for specific genetic deficiencies or in response to symptoms that people exhibit – can really make a difference in the ability of medications to control symptoms and perhaps even progression. It’s going to take some time to collect the data, test medications, and put it into practice but I really do think this type of treatment is an eventuality.
Every milestone in our research is meaningful and it really keeps you going. It’s the one thing that keeps you motivated when you do speak to patients. You can run the risk in research of seeing the condition you’re investigating as a statistic, but with Parkinson’s its not like that for me because I see how it impacts people.
I want to be careful that people don’t read this and think that the cure is around the corner, but I do hope that it shows progress and provides some hope.