advances now in the genetic identification of potential new targets. I think it’s going to open up a new phase that should be exciting and interesting,” Maniatis says.
Indeed, the last few years have witnessed several discoveries in ALS. Maniatis says ALS researchers have identified more than a dozen genetic mutations that contribute to the disease by influencing RNA metabolism and the misfolding of key proteins. “Now we’re trying to understand how protein misfolding generates neurodegeneration, and how RNA metabolism is involved,” he says. “They’re undoubtedly connected.”
In addition to supporting that research, Prize4Life manages several other programs related to ALS. In 2009, it formed a partnership with the Alzheimer’s Research Forum to create a website called the ALS Forum, which provides research news and resources for scientists. Prize4Life also manages a database that amalgamates results from ALS clinical trials. “The idea is that analysis of the data might reveal interesting insights that can become the basis for designing new trials,” Maniatis says.
Kremer continues to act as Prize4Life’s CEO, despite having lost most of his motor functioning to ALS, Maniatis says. He communicates through slight head movements that allow him to operate a cursor on the computer. “He’s become extremely proficient at this and runs meetings from his apartment,” Maniatis says. “He really is deeply engaged at every level. It’s remarkable.”
Kremer says that Maniatis has helped attract employees, board members, and donors to Prize4Life, as well as a level of notoriety that might have otherwise been difficult to attain. “For a young nonprofit with a disruptive model, having a heavy hitter like Tom on board adds a lot of credibility,” Kremer says.
Maniatis believes Prize4Life’s work is helping move ALS closer to the forefront of drug development. He points out that several major drug companies have initiated studies of new ALS treatments, including Biogen Idec (NASDAQ: [[ticker:BIIB]]), which is in late-stage trials of a novel drug to treat the disease. “ALS used to be in the backwater of the medical community,” Maniatis says. “Now it’s very much at the head of the pack because of breakthroughs in genetics and the amount of information that’s accumulating.”
