August 19, 2016
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| Searching all over the world for Improved Treatments |
Introduction
In late June of this year, 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 meeting's focus: precision, or personalized, medicine.
As we learn more about Parkinson 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.
Designing Trials for Personalized Medicine
At the international congress, Susan Fox, PhD, of the University of Toronto, noted[1] that clinicians already individualize treatment: choosing medication or another intervention on the basis of a person's age and 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:
Finding Tools to Help Potential Therapies Succeed
Unfortunately, several therapies for Parkinson disease have failed to show efficacy in clinical trials. Reviewing the drug development pipeline,[2] David Standaert, MD, PhD, of the University of Alabama, Birmingham, and a member of the Michael J. Fox Foundation (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 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 preclinical models that more closely mimic human disease, especially genetic forms of Parkinson disease, which will help medications enter clinical testing with more confidence in their potential neuroprotective effects.
We are also getting closer to Parkinson disease biomarkers (biological measures associated with disease risk, onset, or progression). Researchers are pursuing such markers 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 that it is effective for people with Parkinson 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 Wearables and New Data
Another means toward a better understanding of Parkinson 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, whereas 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[3] 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 disease 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 2 study[4] of a pump/patch delivering levodopa and carbidopa continuously to control motor symptoms. The MJFF 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[5] on its phase 1b 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 MJFF funded work in this area at the University of California, San Francisco, which entered into an agreement with Voyager to advance its study.
"Of note, the use of real-time, intraoperative 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 toward potentially improving patients' response to levodopa, the standard of care treatment for Parkinson disease, and will allow us to begin to more fully assess the impact of VY-AADC01 on patients' motor symptoms."
http://www.medscape.com/viewarticle/867450
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