The Michael J. Fox Foundation for Parkinson's Research

The Michael J. Fox Foundation for Parkinson's Research is using its 2021 distribution to fund:

PROJECT TITLE: Understand Me for Life: An Intelligibility App for Parkinson’s Disease

Investigator/Author:Gemma Moya-Gale, Long Island University

Objective:The goal of this project is to help improve intelligibility in people with PD to allow them to effectively communicate in their daily environment, especially in noisy situations, and to self-monitor their voice changes over time from their home.

Background: Approximately 90% of individuals with Parkinson’s disease develop speech and language problems during the course of the disease and more than half of these speakers experience problems with intelligibility (their ability to be understood). This reduced ability to effectively communicate in social settings, especially in noisy environments, together with the frustration and effort needed to overcome their communicative limitations, frequently results in social withdrawal and isolation. This project will develop an in-home intelligibility treatment program to help speakers with PD improve their ability to be understood in daily settings.

Methods/Design:A total of 150 people with PD will be randomly assigned to three possible groups, the intelligibility-treatment group, a voice-focused treatment to serve as control or to no-treatment. Those with no treatment will receive speech therapy after completion of the clinical trial. The intelligibility-treatment group will self-conduct their speech sessions at home using the Understand Me app, an app designed to target intelligibility problems in Parkinson’s disease. Ten licensed speech and language therapists will conduct the control treatment through videoconferencing. Voice recordings of people with PD will be completed before and immediately after treatment, as well as at six- and twelve-months post-treatment.

Relevance to Diagnosis/Treatment of Parkinson’s Disease: This is the first large-scale speech treatment study to exclusively focus on improving intelligibility in speakers with PD in real-life situations (including those speakers with young disease onset as well as those with advanced symptoms). It is also the first study of its kind to provide follow-up data over a one-year period to examine speakers’ intelligibility gains through the use of a specialized app to increase their social participation and reduce social isolation.

When efficacy of the in-home intelligibility treatment is established, further testing will be implemented nationally and further development of the Understand Me app will be tailored towards personalized user characteristics.

PROJECT TITLE: The Heads-Up Trial Sleeping in a Head-up Tilt Position to Treat Orthostatic Hypotension, Supine Hypertension and Nocturia in PD

Investigator/Author: Roland Thijs, Leiden University Medical Center

Objective: Autonomic dysfunction is common, disabling and often underrecognized in Parkinson’s disease (PD). Orthostatic hypotension (OH) affects up to a third of patients with PD. About half of them also exhibit supine hypertension (SHT). In current clinical practice, both OH and SHT are undertreated. Importantly, the common co-occurrence of OH and SHT complicates pharmacological treatment, as improvement of one can be accomplished only at the expense of the other. The Heads-Up trial aims to evaluate a well-known, straightforward but understudied and widely underutilized intervention for autonomic dysfunction in PD: sleeping in head-up tilt (SHU).

Background: The concept of SHU is based on clinical observations made 70 years ago. Based on several small-scale studies and expert opinion, SHU has been proposed as a first-choice treatment for OH for over three decades, but it is often not advised to patients in daily practice. Moreover, when it is recommended, most physicians prescribe low tilt angles which have no effect on symptoms. The aim of this study is to provide a robust evidence base for SHU, by studying the feasibility and efficacy of SHU and optimizing the conditions for best treatment effects.

Methods/Design: 60 PD patients with OH will be tested in a well-designed randomized controlled trial. The primary aim is to determine the optimal angle of SHU. The trial will adopt a unique personalized approach with angles adjusted to OH severity. The hypothesis is that SHU will improve SHT and OH (primary aim), and thereby have a positive effect on clinically relevant outcomes, including motor symptoms, nycturia, sleep quality and number of syncopal falls (secondary aim). In addition, the project will create an animation for patients, explaining the underlying mechanisms, and develop a practice guideline of SHU for PD-OH.  

Relevance to Diagnosis/Treatment of Parkinson’s Disease: This project addresses an unmet need for PD patients by testing a nonpharmacological intervention for troubling yet difficult to treat symptoms. If the intervention proves feasible and effective, the plan is to conduct a definitive phase 3 randomized controlled trial, informed by the crucial preparatory work proposed here. Ultimately, this could lead to changes in standard of care and improve the quality of life for PD patients.