Clinical study investigates wearables and big data to treat Parkinson's
January 19, 2016
Cynapsus Therapeutics and the Michael J Fox Foundation (MJFF) are working together to incorporate wearable device technology and big data approaches into a clinical study to treat Parkinson's disease.
The phase three clinical study is of APL-130277, a sublingual (under-the-tongue) formulation of apomorphine to treat episodes in Parkinson's disease patients.
"This strategic alliance with the Michael J Fox Foundation and the use of technology-enabled research builds on our standing collaborative relationship as well as our individual commitments to change the lives of people with Parkinson's disease," said Anthony Giovinazzo, president and CEO of Canadian company Cynapsus. "Employing wearable technology to collect data in clinical trials has enormous potential to improve our understanding of how drugs and other treatments impact patients living with the debilitating symptoms of this disease."
This is a pilot effort to understand how clinical studies can harness data science approaches to measure disease progression objectively with the goal of speeding progress towards breakthroughs in drug development. The project builds on MJFF's ongoing data science partnership with Intel, launched in August 2014, to develop platforms for the storage of large volumes of patient-generated data and algorithms to glean insights from these data.
"Amassing valuable objective data and turning it into insightful information can lead to advances in how new therapeutics are developed,” said Intel fellow Eric Dishman. “This implementation of a consumer wearable and an analytics platform, developed by the Michael J Fox Foundation and Intel for use in Parkinson's disease research, is a great example of interdisciplinary collaboration harnessing the power of data to advance disease research while bringing value to patients."
As Parkinson’s disease progresses, the efficacy window of dopamine replacement medication (the gold standard treatment for the disease) shortens, and patients experience motor fluctuations known as off episodes. Apomorphine is a rescue therapy, approved in subcutaneous formulation, to bring patients back quickly to on. Since 2010, Cynapsus has been developing APL-130277, its fast-acting, easy-to-use sublingual thin-film formulation of apomorphine. While the MJFF funded earlier phases of clinical development, neither the foundation nor Intel are funding these APL-130277 phase three studies. Phase three results on the safety and efficacy of APL-130277 are expected in 2016.
"Clinical studies are the most expensive and time-consuming stages of drug development,” said Todd Sherer, chief executive officer of MJFF. “Data science approaches hold the potential to accelerate the pace of progress by allowing drug developers to objectively gather and analyse unprecedented volumes of data and more quickly reveal insights about a potential new treatment. We're optimistic about the potential of this technology to help speed breakthroughs patients need."
A subset of participants in the phase three safety study will take part in a data analytics sub-study. Through a wearable device and the Fox Insight smartphone application (developed jointly by MJFF and Intel), volunteers will contribute data on movement and medication effect. The data will be securely collected, de-identified and evaluated using analytics, then stored in a cloud platform that will allow researchers to potentially gain insights into Parkinson's disease, off episodes and the efficacy of APL-130277.
The technology platform and algorithms developed by Intel for the foundation are intended as a proof of concept, demonstrating that data science technologies can contribute to the objective measurement of Parkinson's disease in interventional clinical studies.
"The data analytics capabilities enabled by Intel and the Michael J Fox Foundation will allow us to better evaluate how APL-130277 is helping patients," said Albert Agro, chief medical officer of Cynapsus. “As our phase three clinical trials progress and we move towards gaining FDA approval of APL-130277, we plan to work closely with Intel and the Michael J Fox Foundation to use this technology to improve the lives of patients with Parkinson's disease.”
Cynapsus is a central nervous system pharmaceutical company developing and preparing to commercialise a fast-acting, easy-to-use, sublingual thin film for the on-demand management of debilitating off episodes associated with Parkinson’s disease. The company recently completed a phase two clinical trial for APL-130277, a sublingual formulation of apomorphine hydrochloride, or apomorphine. Apomorphine is the only molecule approved for acute, intermittent treatment of off episodes for advanced Parkinson’s disease patients, but is only approved as a subcutaneous injection in the USA.
APL-130277 is a turning-on medication designed to convert rapidly, safely and reliably a Parkinson’s patient from the off to the on state while avoiding many of the issues associated with subcutaneous delivery of apomorphine. It is designed to convert all types of off episodes, including morning off episodes, often considered the most difficult to treat.
The Michael J Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The foundation pursues its goals through an aggressively funded, highly targeted research programme coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers.
In addition to funding more than $525m in research to date, the foundation has altered the trajectory of progress towards a cure. Operating worldwide, it forges collaborations with industry leaders, academic scientists and government research funders, and increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder. It also promotes Parkinson's awareness through high-profile advocacy, events and outreach, and coordinates the grassroots involvement of thousands of Team Fox members around the world