PARKINSON’S DISEASE
Streamlined Clinical Trials, From a Home Computer
by JENNIFER COUZIN
A Parkinson’s research and treatment center and the genetic testing company 23andMe, both in California’s Silicon Valley, are experimenting with an unusual new approach to clinical trials: have participants assess themselves from their home computers potentially using everything from videos of tremors to a mouse that senses motor abilities. If it works—still a big if—the strategy could greatly reduce the need for doctors’ visits and make trial participation vastly cheaper and possible from anywhere in the world.
Researchers have long considered how the Web might enhance clinical trials, in particular its ability to aggregate data from the thousands of people needed for studies tracking genetic and environmental factors linked to common diseases. But the new alliance comes with many uncertainties. Will there be a bias in who completes the assessment? Can the approach capture subtle changes in disease symptoms? Will participants understand what they’re signing up for, and how will the data they supply be used?
“Can you really do valid clinical research via the Web?” asks Katie Hood, chief executive officer of the Michael J. Fox Foundation for Parkinson’s Research in New York City, which is funding the project with a $625,000, 2-year grant. “That’s what this will show.”
The Parkinson’s Institute and Clinical Center in Sunnyvale, California, will recruit 150 people, half with Parkinson’s disease and half without, who recently participated in a traditional study examining occupational risk factors behind Parkinson’s; through that study, researchers will have amassed a wealth of data on them. 23andMe will help design or adapt computer technologies and questionnaires for the 150 participants to see whether they accurately capture their health and health history. If they do, the tools could be
expanded to much larger populations and to other research groups. Volunteers will also submit saliva samples to 23andMe, and those samples, like all supplied to the company, will be sequenced for more than 580,000 single-base variations. Eventually, their genetic information will be matched with information they provide online and, say the study leaders, stored securely.
Getting creative. Could Michael J. Fox and others with Parkinson’s assess symptoms themselves online? “We want to build this engine basically to be able to power genomewide association studies,” says Linda Avey, a co-founder of 23andMe. Such enormous studies compare the genomes of those with a particular disease to the genomes of those without. To accomplish this, 23andMe needs to collect large online cohorts of people with different ailments, including Parkinson’s disease. Eventually, 23andMe might sell information on participants, with their consent, to pharmaceutical companies looking to recruit for clinical trials. Avey notes that the company is considering similar strategies in other diseases, although she declined to say which ones.
“We’re all trying to come up with new clinical approaches that can help us get large cohorts,” says Kenneth Marek, a neurologist at the Institute for Neurodegenerative Disorders in New Haven, Connecticut. Marek is experimenting with another in-home assessment for Parkinson’s disease, independent of the 23andMe venture: a sniff test that includes 40 different odorants. One of the first symptoms of the disease is a diminished sense of smell. Marek and his colleagues have begun recruiting 10,000 people whose family members have Parkinson’s to see whether the disease can be detected in its earliest stages.
One concern, says Marek, is that relying on volunteers to run through such tests out of sight of clinicians may lead to bias. People who struggle with the tasks could “get frustrated and may be less willing to complete this at home than they would be in a clinic,” he says. “We have some sense that that might be happening,” but more information is needed. A question for 23andMe is whether participants in a disease-focused study will be ready for all the other genetic information the company offers when it surveys their genomes. Will someone in a Parkinson’s trial want to know whether they—and by extension, their family members—are at increased risk for heart attacks or prostate cancer? The project leader, Parkinson’s Institute scientific director and CEO J. William Langston, expects that the institutional review board considering the ethics of the venture, which could start later this year, will scrutinize it closely.
As that process gets under way, Langston and 23andMe are bringing together technology companies to explore how far computers can be pushed to assess Parkinson’s disease from a person’s home. The Michael J. Fox Foundation, meanwhile, is gearing up to spend as much as $1 million more on other Web- based assessment tools: It put out a call for applications in March.
Downloaded from www.sciencemag.org on May 30, 2008
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