It’s a Promising Time for Parkinson’s Disease Research – Here’s Why
It's an exciting time to consider participating in Parkinson's research, says Katie Kopil, PhD, Director of Research Partnerships at The Michael J. Fox Foundation for Parkinson's Research (MJFF).
Thanks in part to work from MJFF, Parkinson's research opportunities increasingly reflect real patients needs, such as addressing motor and non-motor Parkinson's disease symptoms. There are also several different trials in the research pipeline that aim to slow or stop Parkinson's, and better understand the role genetics plays in the disease.
There are also more ways to get involved than ever before. The Foundation's online research study Fox Insight gives anyone — people with or without Parkinson's — the chance to participate by answering questions about their daily lives. So far, 32,000 people have signed up to contribute, and the study is hoping to recruit tens of thousands more.
We talked with Kopil about how patients can evaluate their options and take part, and how including the patient voice has improved research options for everyone living with Parkinson's.
Antidote: One common misconception around clinical research is that trials that test new drugs are the only option for patients. What other kinds of trials can patients join?
Katie Kopil: Research can encompass a lot of different things. Most obviously research makes you think of scientists with Bunsen burners and chemical goggles in the lab. But the process of understanding more about a disease, and in particular Parkinson's disease, comes by actually engaging people with and without Parkinson's and inviting them to participate in research in a number of ways. One way is recruiting patients to clinical trials to see if a new treatment is safe and effective. But there are also opportunities to learn about a disease by inviting people to share information about themselves and to test non-drug interventions.
For example, there are studies that are not trying a new drug, but are assessing if a different type of therapy is effective. People can participate in exercise studies to see if tai chi versus running could improve symptoms of Parkinson's disease. There are plenty of opportunities for people to participate in research and see a symptom benefit.
Antidote: Tell me more about the importance of patient centricity in drug development and what's been missing in the past.
Katie Kopil: Oftentimes there's been a paternalistic approach that scientists or doctors know better than patients. But in a disease like Parkinson's, where often we say, if you've met one person with Parkinson's, you've met one person with Parkinson's, symptoms can be very diverse. Listening to individual people about their experiences with Parkinson's across the journey allows us to better understand a heterogeneous disease and think about how to develop drugs or new treatments for people who have different journeys.
One of the challenges in research — of conducting patient-centered drug development — is really understanding the heterogeneity of the disease, which you can't understand by gathering information from only 100 people. To be able to understand the diversity of disease experience, it has to be done in a way that collects data from tens of thousands of individuals to really get a representative sample.
What's exciting about Fox Insight and its technological approaches to clinical research is that by allowing people to participate in an online study you can find individuals who are geographically remote or who might not be able to get to a physical center. To date, over 32,000 individuals have enrolled in the study. So with that kind of number, in a diverse disease, you can really start to understand what treatments might work for people who have certain disease courses. Technology has democratized what it means to engage with research, and has given more people the chance to contribute.
Antidote: What are some current Parkinson's research projects that you're personally excited about?
Katie Kopil: One of the great success stories about Parkinson's research participation comes from the recent drug approval of Inbrija from Acorda. To bring this new drug to market, it involved research participation from hundreds of individuals over many years. It's really exciting when there's a tangible result and a patient can say, my participation in this study actually helped get a new drug out there that's going to be able to treat tens of thousands of people with Parkinson's.
As far as active studies, I'm extremely optimistic about the development of new treatments that could potentially slow or stop Parkinson's disease. Many of these studies — that are moving into or are already in clinical trials — are focused around key therapeutic targets. One of those is a protein called alpha-synuclein that in everyone with Parkinson's seems to clump up in the brain and cause problems between neurons communicating with one another. There are many ongoing therapies that are targeting this sticky protein.
Another area of continued advancement is personalized medicine — the idea that a person with Parkinson's can be uniquely qualified to receive a particular treatment because of their genetics. In the last ten years alone, we've come to know so much more about genetics in Parkinson's. There are a couple of different targets — LRRK2 is one and another is called GBA. Upcoming trials will start recruiting individuals with Parkinson's who carry one of these two mutations. And if these trials are successful, they could prove useful for everyone with Parkinson's — not just those with a mutation.
Antidote: Are there any trials looking into treatments for specific symptoms that you would like to touch on?
Katie Kopil: There's a lot of focus currently on the non-motor symptoms of Parkinson's. That's a big need. Many people think of Parkinson's disease as a movement disorder, and that's what you can see externally. But there are many symptoms that are not externally obvious, including changes in cognition, constipation, challenges with over salivation. Scientists have been able to identify these lesser known challenges because people have shared their experiences with Parkinson's. This is another reason why participating in observational studies and in studies like Fox Insight is such an important way of identifying unmet needs in the Parkinson's community.
Antidote: For patients who are interested in joining a trial, what questions do you recommend they ask the research team before deciding to join?
Katie Kopil: Anyone interested in joining a trial, should first talk about it with their care partners and families. What makes sense for you and your family, could be dependent on many things, including the amount of time it takes to participate in a particular study, what outcomes you're looking for from participation, etc. While it's a personal decision, research should be part of every patient's care. At the Foundation we encourage everyone — people with Parkinson's and their care team — to get involved.
When talking to doctors about participating in a clinical study, it's important to talk about what the expectations are in terms of time commitment, the potential benefits and any potential risks. Be clear about your expectations, what you're looking to get out of participating in any study. There are more studies available than there are patients to fill them, so it's a buyer's market for patients who are interested in research. I would tell patients to put your desires first and figure out what's the right study because there are so many to choose from.
Interested in participating in research? Start your clinical trial search below.