UNMC scientists achieve research milestone with Parkinson's disease
Drug that transforms immune system may predict motor improvement
In an early phase human clinical trial, researchers at the University of Nebraska Medical Center tested a drug that transforms the immune system for diagnostic and therapeutic gain in Parkinson's disease (PD).
Press Kit Documents
Video Soundbites and B-roll
Howard Gendelman, MD, professor and chair, UNMC Department of Pharmacology and Experimental Neuroscience
0:00 – 0:19
Parkinson’s disease, like Alzheimer’s, is a progressive, degenerative disorder. Right now has no cure and has very limited therapies – most of which are symptomatic. There is nothing that currently exists that can change the course of any of these neurodegenerative disorders.
0:20 - 0:55
Many years ago we felt the immune system in one way or more was part of the disease process. We know that PD has effects due to genetics, due to environment, due to exposure to herbicides, and pesticides, due to mechanisms that are involved in what we call ongoing, misfolded and aggregated proteins that may effect communications in the brain or how nerve cells communicate with one another.
1:38 – 1:55
Can they change what they do by using pharmacological approaches? And the answer was yes. And we saw that something in model systems of Parkinson’s disease where we can take a toxic cell in the body, in the blood and transform it into something good, something that will nourish and repair the brain.
3:06 – 3:23
We’re able to transform the immune system from something quite harmful to something that was repairative, supportive to the brain in Parkinson’s disease….essentially change the environment of where disease was actually ongoing, occurring.
3:24 – 3:58
And we were buoyed to see that several of our patients had a modest improvement in neurological function. Now, we can say and be very excited that the drug changed behavior, changed movement, changed motor skills, but it’s still early. This excitement must be translated to larger numbers of patients, so we can be secure that we really do indeed have a drug that is helpful in humans.
4:01 – 4:19
The breakthrough is in the new strategy for Parkinson’s disease. The idea of transforming the immune system in ways that could be a therapeutic gain is indeed a milestone or breakthrough in how we think about the disease.
5:01 – 5:27
We’ve demonstrated the possibility that these effects can cause physiological improvements, and that in a small number of patients that demands validation. That we have early evidence that there is the strong possibility that these kinds of drugs, this kind of format, this kind of direction will have broader positive outcomes.
Pamela Santamaria, MD, neurologist, Nebraska Medicine, founder, Neurology Consultants of Nebraska
“It’s been a very exciting study. My patients have been very excited to participate in it. They were looking forward to participating in it. I think it’s exciting to be in a study that may actually may help to reverse some of the symptoms of Parkinson’s disease or potentially help slow down the rate of progression of Parkinson’s.”
“We saw patients that were on medication as well as patients that were on a placebo who were not on medications, and so those patients that received medications -- they showed an improvement in walking, some showed an improvement in tremors. Not everybody had as robust a response, but it was really exciting. The patients were able to recognize that they were getting better. Even my staff, some of the staff who did not know who was getting medicines, I didn’t know who was getting medicines, but a lot of our staff would say, ‘I think that patient’s on medication, I can just tell because they’re walking better coming into clinic.’ ”
“They were very excited. Their families were excited. A lot of them I would ask them, ‘What can you do now that you couldn’t do before?’ Someone would say, ‘I can button my shirt a little better. I can walk a little faster. I feel like I can get from point A to point B a little quicker.’ And so they were really excited.”
“I think we’re eagerly awaiting the next phase. My patients are eagerly waiting to be able hopefully to participate in the next stage as well. I think it holds promise. It was a small study, and so going forward we’re hoping to expand it to have more patients, but I think it’s a great stepping stone to going forward to helping patients.”