In your opinion, what is the single biggest challenge for researchers investigating neurodegenerative disorders?
Parkinson’s disease is complex but we’re learning a lot about its possible causes and underlying biology and this, in turn, is feeding a robust therapeutic pipeline. But getting these ideas to the clinic doesn’t happen easily. If you were to ask many researchers, no doubt they would highlight inadequate access to funding as a major challenge. And certainly without funds, science cannot move forward. But more money is not the sole solution to curing disease. In our view, some of the biggest challenges are seen in how science is done: too little collaboration due to competition and a mindset of secrecy, as well as data sharing and publication models that leave valuable information (often negative data) buried in lab notebooks where they offer little help to the field. Issues like these represent some of the biggest challenges to developing and delivering new treatments to patients.
Why do you think there has been so little progress, if any, in developing a drug that can slow disease progression?
There are numerous potential disease-modifying drug candidates being developed for Parkinson’s disease. And organizations like ours continue to support and fill that pipeline each year. The real challenge is in making the transition from the preclinical space to the clinical one. Much of this has to do with the fact that we don’t have great ways of measuring the underlying disease process and ultimately whether a drug is modifying that process in people. Current trials for Parkinson’s disease use mostly clinical scales, which give you only a superficial view of a patient’s disease. We need biomarkers of the disease itself that can not only help us select the right patient for the right drug trial but also tell us when a drug is having a real biological impact on the disease. This is why The Michael J. Fox Foundation has made such a significant investment in biomarkers, including our flagship Parkinson’s Progression Marker Initiative. These and other efforts are paving the way for the day when we will be able to truly develop drugs to slow, halt or even prevent Parkinson’s disease.
Are you discouraged by the progress that has been made, particularly after the failure of recent drugs?
We’re optimists – something we learned from our founder, actor Michael J. Fox. Although negative results can certainly be disappointing, they are also opportunities to learn what to do, or not to do, the next time. We are also risk takers, which by definition means we may fail. But if no one else is willing to try, it’s up to us to keep pushing ahead.
What needs to happen before we can successfully treat the individual causes of neurodegenerative disorders?
To make all this work you really need three things: good drugs, a good plan and the right people. We need a robust pipeline of drugs built on solid data and rigorous understanding of not only disease biology but also how a drug targets and ultimately alters that biology. We also need to have clearer understanding of how to design informative clinical trials to test those drugs, including having the right biomarkers for selecting patients, tracking disease and measuring drug actions. Finally, we need engaged research, industry, regulatory, funding and patient communities all working together to move things along quickly and efficiently.
How can people with neurodegenerative disorders contribute to the prioritization of research?
The best way to contribute is to simply get involved. You can do this in so many ways. Start by educating yourself and bringing awareness to others about your disease. You can also donate to organizations like The Michael J. Fox Foundation who can act as your eyes and ears in the research effort to find a cure. Our people come into work every day thinking and strategizing about how to cure Parkinson’s disease, and we couldn’t do this if we didn’t have the Parkinson’s community and the generosity of our donors to keep us pushing ahead. But probably one of the biggest contributions people can make is to get involved in research directly. Participating in a clinical study or therapeutic trial is one of the most important impacts you can make. Without these volunteers, new drugs will never make it to pharmacy shelves, no matter how promising they may be. That’s why we’ve put a lot of effort in educating the community about study participation, including developing tools like Fox Trial Finder to help match people to the trials that need them.
In your opinion, what would the benefits be for organisations like Michael J. Fox Foundation of partnering with JPND?
We will never solve the problem of how to cure neurodegenerative diseases without collaboration. The challenges are too big for any one group to overcome. Sharing of data and information, as well as leveraging resources and funding, are some of the best ways to make progress. Being part of a network of like-minded partners through JPND can only benefit our shared goals and help reduce wasteful duplication of effort.