New Effort to Identify
Clemens Scherzer, MD
Last month, the National Institutes of Health announced a
new collaborative initiative that aims to accelerate the search for biomarkers in
Parkinson's disease, in part by improving collaboration among researchers and
helping patients get involved in clinical studies. As part of this program,
launched by the National Institute of Neurological Disorders and Stroke
(NINDS), part of the NIH, Clemens Scherzer, MD, a neurologist and researcher at
BWH, was awarded $2.6 million over five years to work on the development of
biomarkers and facilitate NINDS-wide access to one of the largest data and
biospecimens bank in the world for Parkinson's available at BWH.
"There is a critical gap in the research that leads to lack
of treatment for diseases like Parkinson's," said Scherzer. "Biomarkers are
desperately needed to make clinical trials more efficient, less expensive and
to monitor disease and treatment response. We are hopeful that this initiative
will fast-track new discoveries in this area."
According to Scherzer, most of our knowledge of the human
brain is based on the analysis of just 1.5 percent of the human genome that
encodes proteins. The first part of Scherzer's project will examine the
function of the remaining 98.5 percent of the genome that, so far, has been
unexplored in the human brain. While this remainder had been previously dismissed
as "junk," it is now becoming clearer that parts of it actively regulate cell
biology. Scherzer and colleagues
believe that "dark matter" RNA transcribed from stretches of so called "junk" DNA
is active in brain cells and contributes to the complexity of normal dopamine
neurons and, when corrupted, Parkinson's disease.
"This offers a potentially ground breaking opportunity for
biomarker development. Initially, the team will search for these RNAs
associated in brain tissue of individuals at earliest stages of the disease.
Then, this team will look for related biomarkers in the bloodstream and
cerebrospinal fluid in both healthy brains and those with Parkinson's,"
Scherzer's lab has been spearheading biomarker research in
this field since 2004 and the team already has 2,000 patients enrolled and
being followed in a longitudinal study with rich clinical data and one of the
largest biobanks in the world for Parkinson's tissue with support from the Harvard NeuroDiscovery Center. The biobank was designed as an
incubator for Parkinson's research and until now was chiefly available for
research collaborations within the Harvard-affiliated community. As part of this new project, this vast
resource will be open to all NIH-funded investigators.
"Our ultimate goal is to personalize treatment for our
patients with Parkinson's," said Scherzer. "By opening up this vast collection
of specimens, we are exploding the resources that are available to NIH-funded
investigators looking at this disease. We hope to harness the power of
collaboration to speed up biomarkers discovery."