Xconomist of the Week: Tom Maniatis on Prize4Life and ALS Research

Xconomy National — 

A few years back, molecular geneticist Tom Maniatis was approached by a Harvard Business School student with a heart-wrenching story. The student, Avichai Kremer, then 29, had been diagnosed with amyotrophic lateral sclerosis (ALS), otherwise known as Lou Gehrig’s disease. Kremer had an unusual idea: He wanted to advance ALS research by offering million-dollar prizes to scientists who made meaningful contributions to research into the disease.

Maniatis was both skeptical and intrigued. He had lost his sister to ALS, and was the longtime chair of the research and drug-development committees for the Amyotrophic Lateral Sclerosis Association. “The prize model had never been tried in life sciences,” says Maniatis, who is now a professor and chair of the department of biochemistry and molecular biophysics at the Columbia University College of Physicians and Surgeons (and one of our Xconomists). “At the time Avi entered the scene, the ALS community was pretty small. And the drug companies did not see it as a profitable pursuit.

As it turns out, Maniatis says, ALS was a perfect model for a prize-based research approach. In 2006, Kremer’s idea became Prize4Life, a Cambridge, MA-based nonprofit that is now one of the most influential forces in ALS research. In addition to offering a $1 million research prize each year—-the latest of which will be announced at a fundraising gala in New York on June 6—Prize4Life has spearheaded several programs designed to mobilize and energize scientists working in ALS. Kremer and a handful of his HBS classmates are running the entire endeavor, says Maniatis, who is a member of Prize4Life’s scientific advisory board. “They had such an affection and respect for Avi that many of them put off jobs to stay in Cambridge and work for this nonprofit,” he says.

Kremer and his classmates spent more than 1,000 hours talking to experts from the drug industry, academia, and nonprofits, before officially launching Prize4Life’s model. “What inspired me was the success of the Ansari X-Prize,” Kremer says in an e-mail, referring to the California organization that provides incentives to researchers in education, energy, the environment, life sciences, and exploration. “It proved to me that a prize can correct a market failure.” In Prize4Life’s case, he explains, that market failure is the lack of capital “to focus innovation coming from academia and small biotechs on ALS,” he says.

This year’s award will mark the third effort by Prize4Life to support ALS scientists with $1 million in research funding. In 2010, 33 teams of scientists—about half of whom came from academia and the other half from pharma companies—competed for the organization’s first $1 million award by proposing treatment protocols meant to extend the lives of mice with ALS by 25 percent. Ultimately, none of the teams met the goals set out by Prize4Life, and the $1 million prize went unclaimed, though several of the applicants are continuing their research with the organization’s support. (Prize4Life also sponsors a number of smaller research grants.)

Last year, Prize4Life awarded $1 million to Seward Rutkove, co-founder of Woburn, MA-based Convergence Medical Devices, for his work developing a biomarker that can measure the progression of ALS in patients. The tool is designed to make clinical trials of potential new drugs more efficient.

This year’s major prize will be a revival of the inaugural award’s goal—the promise of $1 million for research that demonstrates 25 percent survival in ALS mice. “In the previous round, no one was able to achieve that, but there are so many 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.

Tom Maniatis chairs the department of biochemistry & molecular biophysics at Columbia University and is on the scientific advisory board of Prize4Life

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: 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.”

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