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A potentially game-changing set of compounds that can inhibit premature cell death has jumped from the Harvard University lab where they were first discovered and are being prepared for commercial application. These compounds, called necrostatins, could be used to treat a wide range of ailments, including traumatic injuries and cardiovascular diseases.
The necrostatins have already been licensed by a biopharmaceutical company and are being tested on animals at Boston-area research institutions, with human clinical trials projected for 2010. The market could be worth many billions of dollars annually and be a major biopharma breakthrough, say advocates.
“It could be a revolutionary platform,” said Michael Whalen, an independent researcher at Massachusetts General Hospital in Boston. “It’s still in the discovery phase, but I believe it could be a significant contributor to medicine. Although a lot more work needs to be done to bring these recent discoveries to the clinic, it is a very exciting field to explore, in large part because necrotic cell death has generally been thought to be untreatable.”
Cells are naturally pre-programmed to die in a process called “necroptosis” when affected by such things as inflammation, or through the cutoff of blood supply from a trauma. However, according to researchers, necrostatins interfere with a cell’s self-destruct signal, preventing cells from committing suicide. Diseases and injuries related to this type of cell death include spinal cord injury, stroke, advanced macular degeneration, amyotrophic lateral sclerosis and multiple sclerosis. Others include wound and burn healing, acute organ failure and stem cell transplantation.
“There are a huge number of diseases and injuries where cell death plays a key role,” said John Gill, president and CEO of Pennsylvania-based biopharmaceutical TetraLogic Pharmaceuticals Corp., which specializes in creating drugs that modulate cell death. In January, it acquired exclusive worldwide rights to develop and commercialize the necrostatin compounds from Harvard.
The research has been ongoing since the early 1990s, said Junying Yuan, one of the lead necrostatin researchers from the Harvard Medical School’s department of cell biology. “Necroptosis was supposed to be unregulated cell death from overwhelming stress,” she said. “It wasn’t regulated, so therefore could not be inhibited with drugs.”
While this is a new and promising technology, it still requires more testing to prove it can do what these researchers claim, said Devasis Chatterjee, assistant professor in medicine at Brown University in Providence. Other similar cell death inhibitors have had only “limited success.”
TetraLogic plans to file an application with the U.S. Food and Drug Administration next year, said Gill. A handful of animal studies are ongoing or about to start at Harvard Medical School, Mass. General, Tufts University School of Medicine, and the University of Massachusetts Medical School in Worcester. The studies will cover stroke, traumatic brain injury, acute liver failure, and retinal diseases and injuries.
Michal Preminger, senior director of business development at the Harvard University Office of Technology Development, handled the negotiations with TetraLogic. She called the necrostatin discovery “revolutionary.”
The project also involved scientists from other institutions in the research, including Boston-based Brigham and Women’s Hospital, Boston University, and the Shanghai Institute of Organic Chemistry.
There are a variety of areas for necrostatin exploration, said advocates. Research indicates the compounds prevent cognitive malfunctions after brain trauma, although just how isn’t clear, said Whalen. In collaboration with the Harvard researchers, he is preparing a grant application for submission to the U.S. Department of Defense to fund research around necrostatins’ effect on the brain. “There may be functions to necrostatins other than strictly sparing cells,” said Whalen.
Necrostatins at a glance
• Discovered by Harvard researchers
• Can inhibit programmed cell death
• Vast range of potential applications — cardiovascular, neurological, organ failure and more
• Licensed by TetraLogic, which will file FDA application in 2010
• Several animal trials now under way in Boston area
• Human trials slated for next year
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