The University of Maryland Medicine https://www.umm.edu is leading a phase 3 study to test the safety and efficacy of using MRI-guided focused ultrasound on the brain to treat Parkinson’s disease (PD). This study is the final step before FDA will consider approving the new technology for widespread use as a nonsurgical treatment option to eliminate key motor symptoms of this common neurological condition.
Howard M. Eisenberg MD, Professor and Chair of Neurosurgery at the University of Maryland School of Medicine and Chair of Neurosurgery at the University of Maryland Medical Center, reports, “The goal of the focused ultrasound treatment is to both lessen the main symptoms of PD which includes tremors, rigidity and slow movement, as well as treat the dyskinesia that is a medication side effect. The eventual goal is to use less medication on patients.”
This is the second clinical trial for the MRI-guided focused ultrasound for PD. The original pilot study was led by the University of Maryland Medicine and focused on a subset of patients who had symptoms that were worse on one side of the body, as well as severe dyskinesia where medication had failed. The patients received focused ultrasound and most experienced significant improvement in their tremors.
This new larger phase 3 study wants to include 80 to 100 participants, and is designed with a softer inclusion criteria, so that more patients will be eligible. The multi-site PD study is expected to begin in 2018 with UMMC as the primary treatment site with Insightec https://www.insightec.com funding the study. For more information about either trial, contact Charlene Aldrich RN, Clinical Research Manager at 410-328-5332.
Another clinical trial has been launched as part of the NIH Accelerating Medicines Partnership for PD (AMP-PD) program to improve success of clinical trials for patients with PD. NIH https://www.nih.gov is teaming with government, biopharmaceuticals, life science and non-profit organizations to advance promising treatments for PD.
AMD-PD will focus on identifying and validating promising markers of disease called biomarkers that may be useful in tracking the progression of PD and could serve as biological targets for the development of new drugs.
AMP-PD partners include Celgene https://www.celgene.com, GlaxoSmithKline https://www.gsk.com, and The Michael J. Fox Foundation (MJFF) https://www.michaeljfox.org for Parkinson’s Research, along with Sanofi https://www.sanofi.com, and Verily https://verily.com
These organizations will invest a combined total of $12 million over five years through the “Foundation for the National Institutes of Health” (FNIH) https://fnih.org to provide management for the project.
The $12 million includes $2 million of in-kind contributions in software and services from Verily https://verily.com. NIH’s National Institute of Neurological Disorders and Stroke (NINDS) https://www.ninds.nih.gov will match the private sector funds with an additional $12 million contribution pending availability of funds. FDA https://www.fda.gov will also be a partner in providing regulatory guidance.
These studies will provide access to the AMP PD Knowledge Portal. With its development through Verily, the Knowledge Portal will enable the sharing of de-identified data and findings among all the AMP PD partners and the research community.
The partners will analyze combined datasets from more than 3,000 PD cases and 1,700 healthy controls from studies funded by NINDS and MJFF including the Parkinson’s Progression Markers Initiative. The goal will be to determine which biomarkers show the most potential for predicting disease progression and prognosis. Results from the analyses will provide the opportunity to conduct genome-wide analyses that could not be performed by a single partner.