Potential Parkinson Targets to Improve Care

Neepa J. Patel, MD, a movement disorder specialist in the Henry Ford Health System

Neepa J. Patel, MD

Although there are plenty of available therapies in the movement disorder space for symptom management, there remains an unmet need that area in Parkinson disease.

Specifically, there is a need to address gait problems and balance issues for patients with Parkinson disease, according to Neepa J. Patel, MD, a movement disorder specialist in the Henry Ford Health System. She told NeurologyLive in an interview that there are plenty of therapies in the pipeline that are exciting, but to truly tackle these problems additional research needs to be done.

With years of experience in the field, Patel provided further insight into the treatment and diagnosis of movement disorders, expressly for Parkinson disease.

For Parkinson disease, what could be making a splash in the pipeline?

Neepa J. Patel, MD: We have some great symptom-based therapeutics, but we certainly don’t have a treatment that helps all the symptoms of Parkinson. Right now, some of the things that are really missing in addressing very specific symptoms are gait abnormalities and balance problems. Carbidopa/levodopa can help to some point, but at a certain point, it doesn’t help enough for the freezing of gait and postural instability. In the short term it’s a great solution, but long term it still seems to plague a lot of patients with Parkinson. There isn’t anything specifically in development yet, we are looking at a couple of drugs that have other purposes and that have a hint of perhaps helping with walking and balance in Parkinson.

In the scope of deep brain stimulation, we’re looking at new potential targets like the PPN, pedunculopontine nucleus, as an area that might help walking and balance more so than our traditional targets. No formal studies, no large-scale clinical trials are being done in this, but small little pocket observations, small little studies of small numbers of groups in a blinded and unblinded fashion are being studied and reported out in the literature.

Another area—I think that’s the most important area in Parkinson research—is we don’t have any therapies that are disease-modifying, that change the course of Parkinson in any meaningful way. This is a huge area of focus in the research community, and right now there are a lot of different novel compounds being looked at for the opportunity to change the course of the disease. They’re still in phase II, going into phase III, clinical trials so the jury is still out on the effectiveness, but I will say there’s at least 3 or 4 different compounds being looked at in large-scale randomized clinical trials. And a lot’s being done to develop new drugs with the potential opportunity to do this.

Is any one of those compounds of particular interest to you?

For me, one of the exciting things coming out is that we are looking at some alpha-synuclein (α-synuclein) targeting therapies, which are not necessarily antibodies but help clear the abnormal buildup of α-synuclein in the neurons. If it were to work, I think it would get at the heart of the problem, probably, most effectively. Whether or not it actually clears it out in human beings in meaningful ways has yet to be seen, but the animal data looks really interesting. This is still in its very preliminary stages, but it appears to be relatively safe. We now just have to watch long term and see if it’s really working. That to me is one of the more exciting drug trials that we’re looking at.

Another one is looking at more of reducing the inflammation that comes with the irritation in the buildup of the α-synuclein. It’s kind of the same idea in the cancer research of creating a less hostile environment so these neurons can survive and function. We’re looking at the cancer drugs that’s gotten some popularity as of late. This might be very interesting as well, just to help these neurons survive longer. For me, those are probably the 2 I’m paying most attention to, but there are a few other novel compounds being investigated looking at other ways of modulating neuronal health so that these neurons don’t die as quickly.

Is there anything else that’s needed to improve Parkinson care?

The last area of research that is really a priority for us is finding a better way to diagnose Parkinson—a biomarker’s for Parkinson. Also, a biomarker to monitor progression, more a laboratory objective test, rather than just a subjective clinical evaluation. There are a variety of studies going on nationally and internationally trying to identify biochemical signatures for 2 reasons. To identify patients who are at risk for developing Parkinson before they display their symptoms because we do believe that Parkinson starts at least 5 to 10 years before they develop the clinical symptomatology. The other would be to monitor its progression with a laboratory test so that when we develop these disease-modifying therapies, we can get it to people before they are symptomatic and disabled.

Transcript edited for clarity.