Do the Unprecedented: Harnessing Technology to Improve Research and Care

CHeT's Ray Dorsey and Erika Augustine

Ray Dorsey, M.D., the director of the URMC Center for Health + Technology (CHeT) and David M. Levy Professor of Neurology is fond of the phrase “anyone, anywhere” and this mantra has become the guiding principal behind the Center’s efforts to create new technology-driven platforms to study neurological disorders and expand access to specialized care.

“The goal of the Center is to allow anyone, anywhere to participate in research, enable anyone, anywhere to benefit from the resulting advances, and allow anyone, anywhere to receive the care they need,” said Dorsey.

This convergence of technology and healthcare is possible due to the recent explosion of digital tools—such as smartphones, remote and wearable sensors, telemedicine, and artificial intelligence—that enable researchers and clinicians to develop a more robust understanding of the daily lives of patients with complex neurological diseases, like Parkinson’s, Huntington’s, and Autism. These efforts have taken on a greater sense of urgency, as new studies show that neurological disorders are the leading source of disability in the world today and there is a growing demand to accelerate the development of new treatments by employing clinical studies that involve a smaller number of people, are shorter in duration, and cost less.

“These technologies provide us with sensitive and objective measures and new insight into what was previously unmeasurable,” said Dorsey. “Until recently, we had no window, or a limited window, into understanding how people with these diseases function in the real world environment, which is 99 percent of the time that they're not in the clinic.”  

A prominent example of the power of technology to transform health research is the mPower iPhone app for Parkinson’s disease which was developed by Dorsey, Max Little, Ph.D., with Aston University in the U.K., and Sage Bionetworks.  The app—which allows Parkinson’s patients to track dexterity, balance and gait, voice, and memory on a daily basis—was highlighted by Apple during a project launch event in 2015. More than 2,000 people downloaded the app and signed up to participate in a Parkinson’s study on the first day it was available.  By comparison, the largest clinical trial ever conducted for the disease involved 1,700 people and it took more than two years for the study to meet its recruitment goals.  To date, 19,000 people have downloaded mPower and the app has served as a tool for multiple clinical studies.

The success of the mPower app was an “aha” moment for the team at CHeT.  Not only did it demonstrate the research potential of the suite of smartphone technologies that reside in our pockets, but also the ability to reach large numbers of study volunteers when participation can be accomplished remotely and on their own terms.   

“There are clear parallels between the way technology is now integrated into every aspect of our daily lives, and the potential that technology holds for being able to make care and research accessible and integrated into our daily lives, from common diseases to very rare diseases,” said Erika Augustine, M.D., the Robert Joynt Associate Professor of Neurology and associate director of CHeT.

Over the ensuing years, CHeT has blossomed into one of the nation’s leading centers for innovation and the application of new technologies to study neurological disorders, an effort that has brought together researchers from a wide swath of academia and industry. 

CHeT can trace its origins back to 1987 when URMC neurologists Ira Shoulson, M.D., and later Karl Kieburtz, M.D., M.P.H., formed what would eventually be called the Clinical Trials Coordination Center (CTCC).  The Center remains one of the only academic-based organizations with the expertise and infrastructure necessary to conduct multi-site clinical trials.  Since its inception, the CTCC has played a pivotal role in bringing seven new drugs to market – four for Parkinson’s, two for Huntington’s, and one for periodic paralysis.  This history, along with the leadership of Cynthia Casaceli, M.B.A. (director of the CTCC), Cornelia Kamp, M.B.A. (director of the Clinical Materials Services Unit), and Elise Kayson, M.S., R.N.C., A.N.P. (director of Clinical and Strategic Initiatives)—who between them have more than 60 years of experience in building international networks of researchers, working with industry, government, and foundation sponsors, and designing and managing clinical trials—has provided the foundation for current efforts to build new technology-based health tools.

In many respects, the digital transformation of research and care at the Medical Center began more than a decade ago with the creation of telemedicine programs in the Departments of Neurology and Pediatrics. Dorsey and Kevin Biglan, M.D., M.P.H., began connecting with nursing homes in upstate New York via web cameras to conduct virtual house calls with Parkinson’s patients.  Over the years the program has expanded and support from Greater Rochester Health Foundation and the Edmond J. Safra Foundation helped create the Parkinson’s Disease Care, New York (PDCNY) initiative which connects movement disorders specialists in Rochester with 400 patients from across New York State. 

When Dorsey assumed leadership of CHeT in 2014, he championed the potential of technology to overcome the barriers of distance and disability and complement, accelerate, and improve traditional investigations into new drugs.   The name was changed from the original Center for Human Experimental Therapeutics to better reflect this expanded mission.  Augustine, who studies rare neurological diseases, and Chad Heatwole, M.D., who focuses on neuromuscular disorders, were subsequently appointed associate directors. 

CHeT also created new collaborations with Ehsan Hoque, Ph.D., and Gaurav Sharma, Ph.D., in the University of Rochester Goergen Institute for Data Science to develop new analytical tools and machine learning algorithms to help research sift through the large sets of data being generated by these technologies.

The Del Monte Institute for Neuroscience, in collaboration with CHeT, has developed a targeted pilot grant program to support technology-driven research projects.  David Dodell-Feder, Ph.D., in the UR Department of Clinical and Social Sciences and Psychology and Hoque received support for a project that will create computational models of human behavior.  Katy Eichinger, Ph.D., D.P.T., in the Department of Neurology, received a grant to employ wearable sensors to track the activity of patients with myotonic dystrophy.

It also became clear that these efforts would require close partnerships with other academic intuitions and industry.  In 2014, Dorsey and Avi Seidmann, Ph.D., the Xerox Professor of Computers and Information Systems and Operations Management at the Simon Business School, partnered to create the d.health Summit, an annual gathering now in its fifth year that brings together leading healthcare executives, physicians, entrepreneurs, policymakers, and technology pioneers to exchange ideas, develop partnerships, and foster disruptive technological and process innovations to improve the lives of the nation’s aging population.

The Medical Center’s long and successful track record in experimental therapeutics for neurological disorders, the emphasis on technology-enabled research, and the academic and industry relationships that were painstakingly created at the d.health Summit and in other forums culminated in URMC’s designation as a Morris K. Udall Center of Excellence in Parkinson's Disease Research by the National Institute of Neurological Disorders and Stroke in 2018. 

Parkinson’s disease is poised to reach pandemic proportions.  By 2040, it is estimated that more than 14 million people across the globe will have the disease and its rate of growth will outpace Alzheimer’s.  “In the face of this looming health care crisis, it is clear the status quo is not working,” said Dorsey.  “The medical community desperately needs to develop new approaches to better understand this complex disease, expand access to specialized care, and increase the speed and efficiency in how we bring new treatments to the market.”

Unlike other Udall Centers which focus primarily on basic science, the Medical Center’s emphasis is on the development of digital tools to enhance understanding of the disease, engage broad populations in research, and accelerate the development of new treatments.  URMC Udall Center projects will involve multi-institutional academic-industry research collaborations with:

• 23andMe to identify and follow individuals with a rarer inherited form of the disease;
• GNS Healthcare and Origent Data Sciences to develop advanced machine learning to create models that identify and predict the progression of the disease and the potential impact of new therapies;
• Johns Hopkins University, Aston University, and Sage Bionetworks to develop the next generation mPower app;
• The Goergen Institute of Data Science, MIT, the University of Michigan, Intel, and MC10 to develop and evaluate several remote monitoring and wearable technologies. 

CHeT has forged a number of additional research partnerships across the University and the application of digital technologies is providing new insight into diseases.  Dorsey is collaborating with David Mitten, M.D., with the Department of Orthopedics and the UR Health Lab, to develop a wearable device that is activated when someone speaks and counts the number of words they say over the course of a day.  The device will be used to measure cognitive impairment in Huntington’s, Parkinson’s, and Alzheimer’s diseases.  Research involving wearable sensors developed by MC10 has revealed that patients with Huntington’s disease often spend half the day lying down, potentially due to depression, social separation, or sleep disturbances, an occurrence that researchers would have never discovered through traditional clinical evaluations.     

“There's a real and growing need to pair clinical care with clinical research, to reach all individuals affected with a disease, including those with physical or geographic isolations, and to enhance the patient voice in care and research,” said Augustine.  “Emerging technologies are placing the patient at the center of information, decision-making, and choice, whether that is genetic test diagnostics ordered directly by patients, or a new wearable sensor for heart monitoring that ties in with your smartphone.  And as clinicians and scientists, we have a lot of catching up to do with respect to how we harness this new information, how we leverage data and research to improve care, and how we meet people where they are.”

This article appeared in the Spring 2019 issue of NeURoscience.