In June scientists and physicians from around the world gathered at the annual International Congress of Parkinson’s Disease and Movement Disorders in Berlin, Germany to discuss emerging trends in care and research. This year’s focus: precision, or personalized, medicine.
As we learn more about Parkinson’s disease, can specific treatments be targeted to those individuals who are most likely to respond? The ability to do so would improve clinical care and accelerate research. Watch a webinar on personalized medicine to learn more.
Designing Trials for Personalized Medicine
At the meeting, Susan Fox, PhD, of the University of Toronto, noted that clinicians already individualize treatment: choosing medication or another intervention based on a person’s age, other medical conditions, and which symptoms are most bothersome. Knowing more about the subtypes of disease – such as those based on clusters of symptoms or genetics – can help us target treatments to be more precise, said Dr. Fox.
She reported that this precision approach is possible, but the field needs to focus on a few aspects of clinical trial design to learn more:
- pharmacogenomics: how one’s genes dictate their response to medication
- trial exclusivity: larger trials including a broad population vs. smaller trials targeting a specific group
- revisiting failed therapies: testing drugs in a population that may be more likely to respond
Finding Tools to Help Potential Therapies Succeed
Unfortunately, a number of Parkinson’s therapies have failed to show efficacy in clinical trials. Reviewing the drug development pipeline, David Standaert, MD, PhD, of the University of Alabama, Birmingham, and a member of the MJFF Scientific Advisory Board, said at the congress, “It looks like a linear process – you follow the steps and get to a therapy, but that’s just not true. It’s a pretty leaky pipeline.”
“It’s often efficacy, not safety, that’s been our problem in Parkinson’s disease. Drugs in testing didn’t really have a big safety liability; they just didn’t work,” he said.
How will we do better? The field is creating pre-clinical models that more closely mimic human disease, especially genetic forms of Parkinson’s, which will help medications enter clinical testing with more confidence in their potential neuroprotective effects. We’re also getting closer to Parkinson’s biomarkers (biological measures associated with disease risk, onset or progression). Researchers are pursuing markers such as protein levels in imaging scans and fluids/tissues, although Dr. Standaert admitted the latter has been “a tougher nut to crack than any of us thought.”
Still, he is hopeful that with these tools we can cross the chasm between identifying a potential drug and proving it is effective for people with Parkinson’s disease.
“Can we build a better bridge over the valley of death? I think the models and the biomarkers are likely the critical factors there,” he said.
Embracing Wearable Devices for Research and Patient Care
Another means toward a better understanding of Parkinson’s disease and more effective testing of new therapies is wearable devices. On the congress’ “Tech Tuesday,” more than 30 posters covered the many ways researchers are developing this area of technology to objectively measure symptoms of disease. Some are used to remotely monitor patients outside the doctor’s office for input on clinical care, while others help people manage their disease or are integrated into trials to measure the impact of therapies in testing.
Lauren Bataille, MJFF associate director of research partnerships, presented three posters on the Fox Insight Wearables study and mobile application, developed in partnership with Intel. She reported that the wearables study has enrolled 650 people with Parkinson’s and compliance is high, demonstrating people’s willingness and commitment to enrolling in long-term mobile device studies. Another poster reviewed the Fox Insight application features, and the third touched on the ongoing work to develop algorithms to measure levodopa response with a mobile device.
Presenting Data on MJFF-Funded Projects
Companies also provided updates on their therapeutic trials at the congress. Neuroderm presented on the design of its Phase II study of a pump/patch delivering levodopa and carbidopa continuously to control motor symptoms. The Michael J. Fox Foundation funded an earlier trial of the therapy. The company also presented data that continuous carbidopa delivery under the skin improves availability of levodopa, giving rationale for development of its liquid levodopa/carbidopa formulation.
Biotech Voyager Therapeutics presented interim surgical and safety data on its Phase Ib study of 10 patients who have received a gene therapy (VY-AADC01) designed to help the brain convert levodopa into dopamine. Top-line results are expected later this year. The Michael J. Fox Foundation funded work in this area at the University of California, San Francisco, which entered into an agreement with Voyager to advance its study.
“Importantly, the use of real-time, intra-operative MRI-guided delivery allowed the surgical teams to visualize the delivery of VY-AADC01, administer higher infusion volumes, and achieve greater coverage of the putamen, the brain region that we are targeting with our gene therapy program,” said Bernard Ravina, MD, MS, vice president of clinical development at Voyager Therapeutics. “Obtaining sufficient coverage of the putamen with VY-AADC01 is a key step towards potentially improving patients’ response to levodopa, the standard of care treatment for Parkinson’s disease, and will allow us to begin to more fully assess the impact of VY-AADC01 on patients’ motor symptoms.”
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