Currently, the likelihood of a promising new drug or therapy for a neuroscience-related disorder moving from a laboratory model, to a human trial, all the way to FDA approval is about 8%, strikingly low compared to other fields, such as cardiology, where rates are as high as 20%.
The reasons for this discrepancy are many, says pediatric neurologist Erika Augustine (MD ’03), MS, who works closely with URMC’s Center for Health + Technology director Ray Dorsey, MD, MBA, to carry out the center’s mission of supporting researchers in developing treatments more efficiently and safely, and in the most meaningful way for patients.
“Neurological diseases are chronic, disabling, cause multiple symptoms, and have a high impact on patients and families,” says Augustine. “That makes it hard to measure disease and disease burden. How do you quantify all of that together with their movement and cognitive problems? It’s not as objective as measuring blood pressure or glucose, and day-to-day fluctuations make it even more difficult.”
Making sure neuroscientific clinical trials in the academic arena succeed has been the center’s goal (formerly known as the Center for Human Experimental Therapeutics) since it was founded in the 1980s by neurologist Ira Shoulson (MD ’71, Res ’73, Flw ’77). During his time at URMC, he established two global academic networks to support this effort—The Parkinson Study Group in 1985 and The Huntington Study Group in 1994—and created an infrastructure that supported collaboration with industry. This work expanded under the direction of neurologist Karl Kieburtz (MD ’85, MPH ’85), and is supported by current URMC Clinical Translational Science Institute leadership.
“We were one of the first academic research centers that helped study groups develop scientific questions, and execute and operate multi-center trials,” says Augustine. “The relationships are here, the expertise and experience are all here. Now we’re looking at what’s next…what can we do even better?”
Many trials still fail because they fail to recruit, she says.
“The parameters may be too narrow in scope in terms of geography or the type of patient who can qualify,” she says. “For neuroscience, we have to cast a bigger net and reach more people, and make it equitable so that anyone who wants to participate can.”
The other aspect is improving how neuroscience diseases are measured.
“We still don’t do it very well,” she says. “For example, in a two-year study we may be getting data from a patient once every three months in an office after they’ve traveled, parked, and they’re tired, stressed. Measuring that one day is not going to give you a full picture of the burden of their disease. So we are trying to do a better job of meeting people where they are.”
Today the work pioneered by center director Dorsey is doing that and more. It uses web-based, mobile technologies to monitor patient symptoms in their homes, at work, and even while they’re sleeping. For example, the Parkinson’s iPhone App, mPower, co-developed by Dorsey, provides data on patient dexterity, balance, gait, and memory multiple times a day, that would otherwise be impossible to harness. In just its first year, the app was downloaded more than 60,000 times by participants in all 50 states, and represents the largest-ever study of the disease.
“With mPower, the patient is at the center of the study, representing a disruptive model for conducting research that has applications well beyond Parkinson’s disease,” says Dorsey.
Augustine points out the technology heralds a huge leap forward in patient
“Across the country, there’s a shortage of neurologists and millions of people who can’t access the care they need,” she says. “Technology like this makes it possible for doctors to not only monitor symptoms but intervene when necessary. Our larger vision today, which is reflected in our new name, is to enable anyone, anywhere to receive care, take part in research and benefit from its advances. That’s what we’re working toward.”