The $10 million that former Governor Paul Cellucci has pledged to raise for UMass Medical School could fast-track aggressive research into a treatment and -- eventually -- a cure for Lou Gehrig's disease, said lead researcher Dr. Robert Brown. The Champion Fund will provide a pot of money that is not subject to the often drawn-out timeline of federal grant-making.
"The important point about having a discretionary fund is that it allows one, in a rapid manner, to undertake experiments and programs which may entail risk but which have a high potential to be beneficial," said Brown, who is Cellucci's personal physician.
Cellucci planned to take the field at Fenway Park tonight to officially launch the fund. He announced in January that he was diagnosed two years ago with the disease formally called amyotrophic lateral sclerosis, or ALS.
“I want to take the diagnosis and try to do good,’’ Cellucci told the Globe's Frank Phillips. “I believe they are getting very close to a significant breakthrough. That’s what gives me hope, and that it is going to help me in my lifetime.’’
Securing funding for a project from the National Institutes of Health can take a year or more. Because it doles out taxpayer money, the federal research center takes a conservative approach and may pass over projects that show strong promise but come with risks, Brown said.
"The whole question is, can one move to a new level?" Brown said. "Can we do better, faster?"
This money will help UMass do that, he said. While Brown is the face of the effort, he said the fund will support work by a cadre of investigators there at the forefront of research on ALS and other neurodegenerative diseases. Among them are neurobiologist Marc Freeman, neurologist John Landers, and a group of researchers studying RNA biology, including Melissa Moore, Victor Ambros, Phillip Zamore, and 2006 Nobel Laureate Craig Mello.
ALS is incurable and current treatments only slow the disease progression slightly. But better treatments are within reach, Brown said. He led a team of researchers who, in 1993, were the first to identify a gene mutation associated with familial ALS. Since then, much of his research has been focused on turning that gene off or stopping the production of toxic proteins it creates.
"It's taken a lot longer than anybody would have thought," Brown said. "None of that diminishes my optimism."
While a complete cure may be further off, he said, some significant therapies may be ready for clinical trials within a few years. Getting there will require a team effort, he said, and the fund will play an important role.
About white coat notes
|White Coat Notes covers the latest from the health care industry, hospitals, doctors offices, labs, insurers, and the corridors of government. Chelsea Conaboy previously covered health care for The Philadelphia Inquirer. Write her at email@example.com. Follow her on Twitter: @cconaboy.|
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