Ending Alzheimer's

By Hannah Wallace October 31, 2004

When Venkat Mathura slides a disk into a computer at The Roskamp Institute, thousands of tiny, glowing white lights pop up on a black screen. Incredibly, each of the 23,000 dots represents a human gene, and the disk, about the size and shape of a disk that fits into a child's portable GameBoy, contained the RNA of a single individual. "We look like a galaxy of stars," exclaims a visitor. "Exactly!" Mathura says, just as awe-inspired by the complexity and mystery of our genetic makeup. Mathura moves a cursor onto one of the dots and clicks. To the untrained eye, this dot looks no different than any other on the screen, but Mathura, a bioinformatician, knows it represents a specific genetic characteristic such as eye color, height, a tendency for breast cancer or, more importantly at The Roskamp Institute, a gene that is implicated in the incidence of Alzheimer's Disease. This breakdown of our selves into genetic bits-only possible in the last decade-still seems fantastical. That this sophisticated genetic research is being conducted in an industrial area in south Manatee County, far away from any major scientific center is, well, astonishing.

The Roskamp Institute, which relocated from USF in Tampa to the former Bausch & Lomb building on Whitfield Avenue about 18 months ago, is conducting groundbreaking genetic research to cure Alzheimer's. It's an ambitious goal, especially for a small nonprofit foundation with an annual budget of about $4 to $5 million. Medical breakthroughs are often discovered at major universities or research centers with prestigious names like Harvard, Johns Hopkins and Scripps, places that employ hundreds of clinical staff and scientists, and possess the institutional muscle to win major federal grants.

But inside this tidy, yellow building with a low, white picket fence, 35 scientists from around the world are trying to unlock the mystery of how genes are associated with diseases of the brain. And when it comes to Alzheimer's they say they're ahead of other researchers all over the world. "If we're not at the head of the pack, we're pretty close to it," says Bob Roskamp, a well-known Sarasota philanthropist and developer of retirement communities, who is funding this institute out of pocket. So far, he's donated $xx million.

Other scientists are impressed. "The research that is being done there is of the highest quality and is also very unique," says Dr. Rudy Tanzi, a geneticist and professor of neurology at Harvard Medical School who has isolated a number of Alzheimer genes. "It's not derivative as so many other studies are in this field."

It's somewhat unusual for philanthropists to establish a research institute. Most philanthropists establish foundations and distribute money to a variety of institutions-think of Michael Milken and his foundation to cure prostate cancer, for example, or the Bill and Melinda Gates Foundation, which distributes tens of millions of dollars to various organizations around the world to treat AIDS and other infectious diseases. But the model that Roskamp has created is seeing results.

"It is an extraordinary institution, world-class, on the par of any university institution," says Dr. Steven Brem, director of the neuro-oncology program at H. Lee Moffitt Cancer Center in Tampa, which is collaborating with Roskamp scientists on the connection between an Alzheimer's protein and its ability to inhibit cancerous tumors. "It is receiving high marks from the National Institutes of Health based on rigorous peer review. It is a sterling example of a strong academic-private-public partnership, a model for [the] biotechnology field, and a great boost to the prestige of medical research in Florida."

Roskamp, a former high school physics teacher who made his fortune developing upscale retirement communities such as the Sarasota Bay Club (and whose name is on a number of high-profile nonprofit buildings thanks to the millions of dollars he has contributed to arts and human services in this area), says his interest in Alzheimer's dates back to the early '80s, when he founded a research lab at USF in memory of his brother who suffered from schizophrenia and committed suicide. His wife Diane had a mother who died from Alzheimer's. The couple settled on Alzheimer's as a cause they would devote their rest of their lives to. "That's why I work now," he says. "We don't need anymore for ourselves. Our kids are OK. We've pulled back from Sarasota and we're focusing on this."

In 1998 Roskamp donated $5 million to USF to form The Roskamp Institute, which recruited such top researchers as molecular biologists Michael Mullan and Fiona Crawford, who were among a team of researchers in London to first identify the gene that caused a form of early-onset Alzheimer's back in 1991 by locating a Swedish family with a history of the disease. It was an earthshaking discovery; to this day, Alzheimer's researchers refer to the Swedish mutation. The Institute seemed to be doing well, with scientists winning federal grants and accolades in the field, when Roskamp pulled his money and most of the scientists, including Mullan and Crawford, out of USF and moved the institute near his own home base. "We had outgrown USF," he says.

Since then, The Roskamp Institute, with more than 20 patents to its name, has won significant grants and has had nine articles published in prestigious medical and scientific journals. The research that is being conducted is exciting. Crawford recently discovered a gene that is associated with a form of Tourette's, a syndrome that causes tics and uncontrollable verbal outbursts. Research by the Institute's Daniel Paris, recently published in Angiogenesis, one of the most prestigious journals in cancer research, shows that some of the same research the institute is conducting on Alzheimer's has exciting implications for inhibiting the growth of cancerous tumors, specifically melanoma. Scientists have already stopped the growth of tumors in mice. The Institute also won a $6-million grant from the federal government's Counterdrug Technology Assessment Center to study the genetic underpinnings of drug addiction.

Still, the heavy emphasis is on Alzheimer's. The Institute's primary focus is looking at a toxic protein called "amyloid precursor protein" that is found naturally in the body but seems to go awry and form sticky plaques within the brains of Alzheimer's patients. Mullan's goal is to stop the accumulation of this destructive protein; Harvard's Tanzi says Mullan's work is "one of the most unique programs in how this works. His science is top notch and easily on a par with any of the better researchers at Harvard."

To grasp the concept behind his research, Mullans says it's easiest to think of the brain as a sink: "There's either too much protein flowing into the sink-our brain-or the sink isn't draining properly. We either have to turn the tap off or increase the clearance from the brain." He and other scientists at the Institute are testing ways of controlling the flow of amyloid on the Institute's mice, specially bred to carry the Alzheimer's gene (and about as costly as a waterfront mansion). Already, Mullan's says, "If you're a mouse at The Roskamp Institute, you'll never suffer from Alzheimer's again."

The amyloid hypothesis is one of the hottest research topics in the field of Alzheimer's research. Thousands of researchers are racing to find a cure, or least find a way to halt the progression of the disease. At stake are the lives of millions who have Alzheimer's or are bound to get the disease; 4.5 million people in the United States have Alzheimer's, a number that is expected to balloon to 16 million by 2050. And while researchers collaborate by building on each other's published results, they are also in competition. Whichever scientist, university or research center discovers a drug or vaccine for Alzheimer's could make a fortune. "It [the discovery and resulting money] would be the blockbuster of the century in my opinion," says Dr. Claudia Kawas, professor of neurology and neurobiology and behavior at UC Irvine who is an Alzheimer's researcher. "It would stagger the imagination." At least six companies, Eli Lilly & Co. and Merck & Co. among them, are trying to develop drugs right now that would prevent the buildup of amyloid in the brain.

For Roskamp, 66, winning the big prize is also a way for him to ensure that the Institute survives him. The Institute is gearing up to do human clinical trials on amyloid in the near future, with a hope that researchers may have results in three years. If The Roskamp Institute succeeds in creating a cure, the drug could be worth billions to a pharmaceutical company; the royalties, often in the range of 5 to 10 percent, would go to The Roskamp Institute and its scientists and help to continue their work.

And while the small institute is competing against giant departments in major universities or centers such as the Salk Institute and Scripps, it has some advantages. Unlike university scientists, researchers at The Roskamp Institute don't have time-consuming, distracting teaching duties, and yet they are paid salaries competitive with those at major institutions. They also don't have university bureaucracies to navigate when they request funding, additional staff or equipment since Roskamp holds the purse strings. They're also free to move in whatever direction their research takes them. "We're geared specifically to research," says Mullan. "Universities have multiple missions." Grant writing, the Institute's scientists say, is smoother since there are fewer levels of approval to go through. For other researchers the fact that Mullan and the other scientists don't have to depend on grants is a blessing. "They have the advantage of hard money," says Tanzi. "They're very fortunate to have Mr. Roskamp. At Harvard, we rely completely on grants, even for our salaries."

Roskamp, always the entrepreneur, says there's more freedom in hiring and firing scientists than in the academic world. "Most people aren't self-directed and motivated," he says. "We're looking for the entrepreneurial type who sees this as their joy and spirit." Eventually, he's hoping to expand his 42,000-square-foot facility, which is only 40 percent occupied right now, to 100 scientists and clinical staff.

Of course, there's a danger in working for a wealthy philanthropist as well, as many scientists have discovered when their funding was pulled away after slow or disappointing scientific results in their research. Entrepreneurs tend to set goals and timetables that may be unrealistic in science. But Roskamp says curing Alzheimer's is his dream. "This is what I want to be remembered for," he says.

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