Ignacio Mata, PhD, on the Challenges of Developing New Therapies in Parkinson Disease

“I think the closest that we are right now to something that may slow or stop the progression are those that are targeted for people that have a genetic form of PD, where the biology is at least mostly understood and is caused in some of those cases by 1 single variant. [W]e can understand what is happening biologically and we can try to compensate what the deficit is.”

Parkinson disease (PD) is a highly complex disorder that is still not well understood by medical science. Current treatment options for PD are very limited, and despite numerous attempts at clinical trials for novel therapies over the past few decades, little progress has been made in introducing new treatment options as of late. As such, great unmet need for patients with PD persists.

PD research is further complicated by the fact that there are different forms of PD that likely have different pathophysiologies, and thus may need independent approaches to effective treatment. That said, there is interest among some companies and institutions in applying gene therapy approaches to PD, especially for the forms of PD that seem to have a genetic basis.

CGTLive® recently reached out to Ignacio Mata, PhD, an associate professor of neurology at the Cleveland Clinic Lerner Institute, to get his perspective on the challenges of developing new therapies for PD. Mata pointed out that often by the time patients are recognized to have PD by the appearance of distinct movement disorder symptoms, the disease has already been developing for 10 to 20 years. As such, these patients have already sustained significant deterioration from the disease that would likely be very difficult to reverse with therapeutics. Mata thus emphasized the need to identify definitive biomarkers so that PD can be diagnosed earlier, and patients can potentially receive treatment before the disease has caused as much damage to patients’ dopaminergic neurons. He also highlighted that genetic forms of PD are more well-understood than other forms of PD and as such targeted therapies for these genetic forms may be a desirable starting point for the development of new therapies for PD.