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In the fight against Alzheimer's, a need for speed

The Herald-Tribune - 9/29/2018

Sept. 28--For the researchers at Sarasota'sRoskamp Institute, the years since its founding in 2003 have been an exercise in frustration -- with glimmers along the way of new paths forward in the painstaking search for Alzheimer's disease treatments.

Now the team led by Mike Mullan and Fiona Crawford is engaged in another waiting game, anticipating the publication of results from a clinical trial of a drug they have scrutinized for decades. The Phase III trial in Ireland -- a large human study of safety and efficacy that is necessary before a drug can reach the market -- yielded tantalizing indications of where their research should go next, Mullan and Crawford told a roomful of donors at the institute's "Grey Matters" symposium Friday. The details remain under embargo, Mullan added, but already he and colleagues are designing a followup trial using a special version of Nilvadipine, a blood pressure drug prescribed in Europe.

"The way science works is we have to take this now; we have to build on it," Mullan said of the study. "We are planning second- and third-generation drugs that can be more effective. This is just one of the drugs that we work on. We can do this trick over and over again, and we can do it better and better, because we're learning so much."

Well-publicized failures of large-scale tests of new Alzheimer's medications developed by pharmaceutical giants have been an important and perhaps necessary process of elimination, Mullan told the audience. But neurologist Andrew Keegan, medical director of the institute's clinic, acknowledged that patients with the disease now don't have many treatment options.

"For 15 years I've been almost embarrassed to tell my patients, 'No, there's nothing new,'" Keegan said. "There's a desperate need for something new."

Prescription drugs currently available for Alzheimer's only manage some symptoms of cognitive decline, and only for a short period, he added: "We tell patients, maybe for the first six months, we'll get some stability. They work, but only mildly so."

Crawford told the story of how she and Mullan collaborated 30 years ago in England to publish the first definitive evidence that build-up in the brain of beta amyloid proteins are crucial in causing Alzheimer's. That discovery made possible the mouse models on which most research into the disease has been based. About 10 percent of support for the institute's work, she said, still comes from the Roskamp Foundation, and the rest is provided by highly specific research grants that dictate the terms of their scientific exploration. Applying for such grants is often a two-year process, she said.

"Our patients don't have that time," Crawford emphasized. "We need to be able to move today, if we find something in our research. Philanthropic funding is critical -- that's the money that funds our discoveries."

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