The Parkinson’s research field is ready for precision medicine, four Parkinson’s experts stated in an editorial, suggesting that a switch from traditional disease concepts based on clustering of symptoms should be replaced by disease profiles built on knowledge of genetic and molecular changes.
The editorial, “Parkinson’s disease is ready for precision medicine,” published in the journal Future Medicine, outlined suggestions on how to overcome challenges linked to a precision medicine approach. They argued that researchers, clinicians, the pharmaceutical industry, and funding and regulatory agencies must find new ways to work together to reach these goals.
The idea that research into disease should not be organized according to traditional diagnoses is not new. In cancer research as well as treatment, the idea has become reality as oncologists increasingly rely on genetic or molecular data to choose the best treatment for a tumor, instead of grouping it into a treatment group based on the organ it came from.
The lead author of the editorial, Todd Sherer, PhD, and CEO of The Michael J. Fox Foundation, along with three colleagues believe it is high time for neurodegenerative diseases such as Parkinson’s to follow the same path.
People with Parkinson’s disease have a variety of symptoms, and it is likely these symptoms are caused by equally varied molecular changes. Symptoms tend to fluctuate over time, making it likely that drug development efforts that aim to find a single solution to such a complex issue will fail.
Instead, researchers must understand the underlying problems both before symptoms start showing and during the course of the disease. This is no easy task, Sherer and his colleagues admit, but with an exhaustive strategy at hand, they believe it is possible.
The strategy would need to include more extensive access to data and samples. Today, samples and data are often protected from use, significantly slowing progress, and changing this would require a shift in the mindset of everyone involved.
Funders, regulators, and publishers should put pressure on researchers to contribute data from their cohorts, and researchers need the motivation to do so, they stated.
The Michael J. Fox Foundation researchers also believe that partnerships with patients, families, and volunteers are crucial in collecting study groups that are truly representative of the population. Recruitment strategies need to be culturally relevant, and larger efforts to use technology to make it easier for people to participate, even if trials are geographically distant, are needed.
And importantly, patients participating in a trial should have access to study data and results at the end of the trial. “If patients are truly our partners, we should treat them as such,” Sherer wrote in the editorial.
Deeper molecular and genetic knowledge, linked to clinical data, is also a must. Again, these large amounts of data need to be handled correctly and made accessible to the research community. These efforts also need to include the discovery and validation of biomarkers that mirror the variability of Parkinson’s.
Finally, researchers need to rethink how drugs for brain diseases are developed, they argue. Molecular and genetic brain changes in Parkinson’s are often present in other neurodegenerative conditions. But drug development is often done within a single disease focus.
The four Parkinson’s experts believe it’s possible to develop both drugs and biomarkers to target biological anomalies instead of diseases. This type of precision medicine approach is more likely poised for success than current strategies, they argue.
The Michael J. Fox Foundation actively works to make these changes happen. By supporting research projects such as the Parkinson’s Progression Markers Initiative — collecting biological, imaging, and clinical data from varied Parkinson’s populations and making it available to the broader research community — the foundation hopes to make a difference.
“Patients play a role in directing that study and other efforts from our foundation,” Sherer told Parkinson’s News Today.
“With our growing understanding of the varied paths to Parkinson’s disease, we see potential routes to personalized medicine for our field,” Sherer said in a news release. “Increased attention and collaboration in this area will mean significant advancement in the identification and testing of new treatments to prevent, slow or stop disease.”