It’s called “the long good-bye” by Alzheimer’s disease sufferers and their caregivers—that heart-wrenching struggle of watching a loved one deteriorate one day at a time. First it’s names and faces that go. They forget who you are. They forget who they are. Eventually, the sufferer loses most or all cognitive ability and becomes a lifeless shell of the person you once knew.
Nearly 5.4 million people currently suffer from Alzheimer’s, and it’s become the sixth-leading cause of death of all Americans. But this cruel disease’s days look to be numbered, and a Sarasota organization is among those leading the death march.
The Roskamp Institute, a brain disease research center founded 15 years ago by local philanthropist Bob Roskamp, has recently begun clinical trials in Europe on a drug that may help end Alzheimer’s. The drug, called Nilvadipine, is already being used to treat hypertension, but Roskamp researchers say its anti-inflammatory properties can also attack the root cause of Alzheimer’s—beta-amyloid proteins, a kind of sticky plaque that builds in the brain and binds to and kills healthy cells and neurons.
The $10 million trials, launched in February with funding from a European Commission program, will study Nilvadipine’s effect in 500 Alzheimer’s patients in nine countries throughout Europe. Roskamp scientists believe the study will confirm the drug works the same in humans as it does in lab mice,
which have had their Alzheimer’s symptoms slow—and even reverse—after taking the drug. But it will likely take five years to extrapolate the necessary data from the trials and, if those trials prove successful, move the drug into clinical use.
The drug may well be a medical breakthrough, but it likely won’t be a magic bullet. Researchers say it will need to be combined with other therapies and medicines, such as Roskamp’s own anti-inflammatory supplement derived from nicotine. That supplement, anatabine, is already on the market.
There are also major regulatory hurdles to overcome before the drug can be sold in the United States, even after it’s shown to be effective in Europe and Asia. Funding is also a constant challenge for the nonprofit institute, with clinical trials and drug studies costing in the tens of millions of dollars, not to mention the day-to-day operations of the lab.
There’s plenty to be optimistic about, however. Many Alzheimer’s experts regard beta-amyloid science as one of the most promising areas of research in the disease’s history, and two other beta-amyloid drugs are in clinical trials around the world. Roskamp officials say whether or not a Sarasota clinic is remembered for ending the disease, 2012 could mark the beginning of “the long good-bye” to Alzheimer’s.
“There’s no question we will see the day when Alzheimer’s is cured,” says Dr. Michael Mullan, president and CEO of the Roskamp Institute. “Once we realized Alzheimer’s was a biological issue, it was just a matter of time. It’s not if it’s cured, it’s when.”
Mullan and the institute’s vice president, Fiona Crawford, were part of the pioneering British research team that in 1991 discovered the cause of Alzheimer’s—large amounts of beta-amyloids accumulated in the brains. Beta-amyloids occur naturally in all brains, but it’s still a mystery as to why they accumulate in Alzheimer’s sufferers, especially in younger people who have early-onset Alzheimer’s.
In 1996, Mullan and Crawford were tapped to head the newly formed Roskamp Institute at the University of South Florida. Sarasota developer Bob Roskamp birthed the institute through his Roskamp Foundation. He says he was inspired by his late brother, who suffered for years from schizophrenia.
“They discovered the cause of Alzheimer’s,” Roskamp says. “And when you get the cause, you can work on an antidote. I was excited to see what we could come up with.”
The institute left USF for Sarasota in 2003, in part to be free of the constraints of university-based research. “This is a very nimble, flexible organization. We can change direction quickly without wading through a large bureaucracy,” Mullan says. “You can’t find that in a university setting, where there is a divisional thinking of separate chemistry, biology and other departments.”
Today, the Roskamp Institute performs research on dementia, depression, head and traumatic brain injury, multiple sclerosis, Parkinson’s disease, Tourette’s syndrome and drug addiction. However, about 85 percent of all work is done on Alzheimer’s research.
The institute resides in a 48,000-square-foot facility on Whitfield Avenue north of Sarasota-Bradenton International Airport. More than 55 scientists and five doctoral students work in the hospital-like labs that contain more than $50 million worth of specialized equipment, some unique in the state.
The institute’s most important assets, however, are nearly 2,500 specially bred mice that are genetically predisposed to develop Alzheimer’s-like brain diseases. Mullan says Nilvadipine has cured hundreds of mice of their cognitive conditions—which makes a strong case for its efficacy in humans. His research team is also optimistic.
“I’ve been working here for 15 years, and we finally have a drug in phase three trials,” says Daniel Paris, lead researcher on Nilvadipine. “Some scientists never see their drug go into the market, and we are really close.”
But close may be relative, as Bob Roskamp knows all too well. He says that dealing with the U.S. Food and Drug Administration has been the biggest obstacle in the past 15 years to getting a drug to market.
That’s why the institute is pursuing clinical trials in Europe, where it is easier to bring a drug to market and then easier to bring the drug back home to America.
“That’s part of my role, is to be the business side of whatever it is we are doing,” Roskamp says. “I want to find out how we get our product, which we’ve known for six years that it works, into the hands of Alzheimer’s victims. I’m more encouraged today with our progress than ever before.”
Roskamp still guides the nonprofit as board chairman, but he’s also been its chief financier for most of its history. The institute’s budget this year is about $6.5 million, with nearly $2.5 million coming from private donations, including $1.4 million from the Roskamp Foundation Irrevocable Trust. The institute did receive $4 million from research grants from groups like the National Institutes of Health, Department of Defense and private foundations like the Alzheimer’s Association and American Health Assistance Foundation.
Alzheimer’s Association senior associate director Heather Snyder, who oversees the group’s international research grant program, says the Roskamp Institute routinely qualifies for grants based on the type of new research proposed and the institute’s track record of having the resources to complete the work.
“We find the best science that is out there,” Snyder says. “We have a peer review process that uses reviewers from 55 countries, and the projects that rise to the top are funded. The Roskamp Institute has qualified for several grants.”
However, Bob Roskamp’s goal was not to have the institute survive on the mercy of grants and donations, but rather from the profits of the drugs and medicines developed by the institute itself. To that end, the institute in 2008 spun off the for-profit company Archer Pharmaceuticals, which shares office space in Sarasota. Archer owns intellectual rights to drugs produced by the institute, including Nilvadipine, and will use sales profits to pay investors and fund further research.
Snyder says such partnerships between nonprofit research organizations and for-profit pharmaceutical companies “aren’t unusual” in the research world, which is increasingly competitive. That’s particularly true in Florida, where the biotechnology industry has been courted heavily in the last five years.
But for Archer, such profits might be years off as Nilvadipine slowly—if ever—makes its way onto the market. In the meantime, Roskamp has made a recent push for new private donors, mainly large benefactors.
Mullan says that some recent research has been funded by such large donors, who generally have an interest in making a contribution to science and medicine and not just a tax write-off.
“We are working on these things because people in the community want to advance an area of research,” Mullan says. “Sometimes they have funds, and sometimes they help us look for funds.”
The institute earlier this year hired development director Steve Klindt to increase awareness of Roskamp in Sarasota. Klindt says he aspires to the model set by Mote Marine Laboratory, which uses its aquarium to draw attention and donations to fund the real work behind the scenes.
“Mote is 85 percent fish science and 15 percent aquarium, but in most people’s minds it’s the other way around,” Klindt says. “A new area for us is getting education out to the public, and that takes a plan and some backing.”
Roskamp has started a speaker’s bureau of its scientists who lecture for local groups and civic clubs. Direct mailings of Alzheimer’s facts and tips and other promotions are part of the grassroots awareness campaign. However, Klindt hopes the public face of the institute will be its memory clinic, which provides free memory screenings and referral services for treatment.
Sarasota is likely the perfect place for such a clinic, says Chuck Albrecht, chief operating officer for the Alzheimer’s Association’s Florida Gulf Coast chapter. He cites statistics that show one in 58 people, regardless of age, are living with Alzheimer’s in the U.S. In Sarasota, that ratio is one in 18, or some 22,000 people.
“I can tell you the Roskamp Institute is a valuable asset in the community of Sarasota, and not many people really know what they do,” Albrecht says. “There is a shame felt by many Alzheimer’s sufferers about having this disease, and that can prevent them from seeking out all the great resources that are out there.”
Mullan agrees that there is an embarrassment and shame surrounding people with the disease, which can prolong their decision to get diagnosed and seek treatment. Mullan says if a drug like Nilvadipine is to work, it will have the best chance on patients who detected the disease early.
“Our memories are so closely associated with who we think we are as people,” Mullan says. “If we are to help people get that back, it will be with multiple approaches and multiple assaults on the disease. This drug is a big part of that, and it’s close.”
Back to Life
One man’s story shows there’s new hope for memory disorders.
It was early 2010 when Philip Briganti first saw the warning signs. His uncle, always good about remembering birthdays and holidays, stopped sending cards.
Then Briganti noticed his uncle repeating himself, even duplicating entire conversations five minutes later. His uncle would get lost while driving and sometimes couldn’t find his way home. He was also malnourished and sick from forgetting to eat and take his medications.
The vibrant, active and sharp man Briganti knew was disappearing. It was like watching him die, Briganti says. But now, that man is slowly reappearing. After finding treatment through doctors and specialists at the Roskamp Institute, Briganti’s uncle is coming back to life.
“There is hope now—hope he’ll live out a meaningful life,” says Briganti, who lives in Pennsylvania but monitors the progress of his uncle, 77, in Sarasota. “I don’t expect him to have the capabilities that he used to, but he’ll be happier and more apt to live up to his potential.”
The cognitive decline of Briganti’s uncle is a common story for the country’s elderly. More than 4.5 million Americans currently suffer from Alzheimer’s disease, and millions more live with dementia and other memory disorders. Last year, nearly 15 million family members and other unpaid caregivers provided 17 billion hours of care—worth some $202 billion—to these sufferers, the Alzheimer’s Association estimates. But another increasingly common story is that many are slowing down their disease and returning to a more normal way of life. Medical breakthroughs in drugs, therapies and disease detection have increased, and there is hope from research groups like the Roskamp Institute that a cure is not too far off.
For Briganti’s uncle and many others with brain diseases, the road to recovery began by admitting there was a problem.
“He wouldn’t go to his doctor,” Briganti says. “It wasn’t that he was embarrassed. When you have a memory issue, you don’t remember that you say the same thing over and over or that you forget to take your medication. He wasn’t even aware he had a problem.”
It took a year to confront his uncle with the evidence, but Briganti managed to get him to his doctor this past summer. The doctor ruled out Alzheimer’s—a big relief, but one that opens up even more questions.
Briganti was referred to the Roskamp Institute, where cognitive neurologist Dr. Michael Hoffman administered a memory test and recommended an MRI brain scan to detect any medical issues that could be causing the problem. The MRI determined there was a vascular issue preventing blood flow to the brain—a common and often treatable condition if detected early. New medications, reducing the dosage of his blood pressure drugs and improvement in his overall health have helped to slow the symptoms and even begun to reverse them, Briganti says.
“The doctor made him feel at ease and gave him confidence,” Briganti says. “By then, he was able to buy in to the fact that he had a problem and started to cooperate with treatment. He wanted to get better.”
Briganti’s uncle is not cured, but he’s better. “He still has a problem with his short-term memory, but he doesn’t repeat things as much. He just seems to think a lot more clearly,” Briganti says. “What sticks in my mind is that MRI. If he would’ve had it done maybe five or six years ago, things could have been a lot different.”
Test Your Memory
A 10-minute test can spot—and often help—memory problems.
If Alzheimer’s disease and other memory-related disorders are detected early, the chances of managing the symptoms increase exponentially. For more than five years, Roskamp Institute’s memory clinic has offered free memory screenings—a simple 10-minute test that can determine if a person is having memory issues and needs further care.
Although the test is offered to anyone, Roskamp recommends it is taken once a year by people 60 years and older, much like an annual physical. “Early detection is absolutely the key,” Janette Girard, research coordinator at the clinic, says. “A couple of medications and some lifestyle changes can slow the disease and make it manageable.”
The memory screening actually begins before the test, when Girard talks to patients to gauge their conversation skills. This can be tricky, she says, as some people learn to mask their symptoms well.
“Sometimes I am completely fooled,” Girard says. “It’s amazing how their personalities can allow them to converse and talk about current events, but when they take the test, those memory deficiencies show up.”
The standardized test, developed by neuropsychologists, gauges attention, memory, language, visual construction, visual thinking and orientation. (The type and nature of the questions are kept confidential, as the questions must be completely new to the person taking it.) If the test catches something, the patient is asked to see their primary care physician, who will perform further diagnostic tests. Sometimes, patients return to Roskamp for treatment under in-house neurologists.
Some patients are also asked to participate in clinical trials of new drugs and therapies administered by Roskamp’s research wing. Roskamp also offers referral services to other specialists and forms of treatment, as well as support programs and education information for caregivers of those with memory diseases.
“We still have a lot of patients who unfortunately don’t have happy endings, but we do catch things and see those people starting meds and getting help from families,” Girard says. “All that begins with the test.”
For more information and appointments, contact Roskamp Institute Memory Center, 2040 Whitfield Ave., Sarasota, FL 34243. (941) 256-8019; firstname.lastname@example.org.
Journalist David Ball was recognized by the Florida Press Association in 2010 for his investigative series about a Monroe County School District financial scandal. He also wrote “I’m Being Punished for Teaching These Kids” in our 2011 summer issue.