Three MGH projects named among Top 10 Clinical Research Achievements of 2012

Top 10 achievement awards presented at annual meeting of Clinical Research Forum

Three projects led by Massachusetts General Hospital (MGH) investigators have been named among the Clinical Research Forum's Top 10 Clinical Research Achievements of 2012. The MGH-led teams are being honored for work to develop a system allowing people with paralysis to control computerized equipment via a small device implanted into their brains, for a clinical trial showing that a combination of two targeted treatment drugs significantly delays the development of treatment resistance in a common form of melanoma, and for a new approach to diagnosing hard-to-find chromosomal abnormalities that can provide critically important information.

"There's never been a moment in the history of biology that's more optimistic for spectacular breakthroughs to happen. However, it will require strategic investments at a most difficult time in our history," said William F. Crowley Jr., MD, founder and past chairman of the Clinical Research Forum and director of the MGH Clinical Research Program. "America is a world leader in biomedical research and if we are to retain that leadership role globally, we have to continue making these national investments." The awards are being presented today at the Clinical Research Forum's annual meeting in Washington, D.C.

MGH award recipients are:

• Leigh Hochberg, MD, PhD, MGH Neurology, for a Nature paper describing how the investigational BrainGate System - developed through a continuing collaboration with colleagues at Brown University and the Veterans Administration Medical Center in Providence - allowed two patients with paralysis affecting all four limbs to reach for and grasp objects using robotic arms controlled directly by their brain activity. Both study participants have been paralyzed for several years by brainstem strokes. This work is also receiving the Clinical Research Forum's Herbert Pardes Clinical Research Excellence Award as the most outstanding project nominated for this year's Top 10 Awards. More information on BrainGate is available at http://www.braingate2.org.

• Keith Flaherty, MD, MGH Cancer Center, for a New England Journal of Medicine paper reporting that combined treatment with two kinase inhibitors - dabrafenib and trametinib - delayed the development of treatment resistance in metastatic melanoma patients with tumors driven by mutations in the BRAF gene, which accounts for about half the cases of the deadly skin cancer. The phase I/II study, which found that combination treatment delayed resistance about four months longer than treatment with dabrafenib alone, is being followed with a larger phase II trial. A news release describing this study may be accessed at http://www.massgeneral.org/about/pressrelease.aspx?id=1274.

• Michael Talkowski, PhD, MGH Center for Human Genetic Research, for two papers. The first, published in Cell, identified 33 genes associated with autism and related disorders, 22 for the first time, using a gene-sequencing method that detects the location of abnormalities in which DNA segments are moved within the same chromosome or exchanged with segments in other chromosomes, leaving the overall size of the chromosomes unchanged. A news release describing this paper may be accessed at http://www.massgeneral.org/about/pressrelease.aspx?id=1460. The second paper, published in the New England Journal of Medicine, described use of the sequencing method to accurately determine the genetic basis of a prenatally detected structural abnormality.

Additional award recipients are from Yale University School of Medicine, Columbia University, Johns Hopkins School of Medicine, the University of North Carolina at Chapel Hill, the University of Pennsylvania and the University of Pittsburgh. Awardees were selected from nominated projects by the Clinical Research Forum's Board of Directors, senior leaders at some of the country's top academic health centers, all of whom recused themselves from discussing and judging submissions from their own institutions.