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Hope for Alzheimer’s treatment in Washington University trials

5:59 am by | 0 Comments


Brent Whitney, 34, found out two years ago he has the same inherited gene mutation that caused early onset Alzheimer's disease and took the lives of his father, grandmother and 10 of his grandmother's 13 siblings.

His voice cracks as the married father of two talks about his father and how "the man I thought was Superman could no longer figure out how to do something like tie his shoes or read a phone book."

But there is hope he can avoid the same fate, or at least help others who carry the genetic mutation. Whitney wants to be chosen for a clinical trial at the Washington University School of Medicine that holds promise for helping scientists prevent Alzheimer's in patients who carry the mutation. Led by Dr. Randall Bateman, a neurologist, the study will use patients like Whitney, who are at an extremely high risk of developing the disease at an early age because of the mutation.

The study will be closely watched worldwide because instead of testing the drugs' effectiveness on Alzheimer's patients, it will test the ability to prevent the disease from afflicting people who were destined to develop it otherwise.


"We'll try to prevent them from getting it," Bateman said.

Patients chosen for the trial -- called Dominantly Inherited Alzheimer Network Trials Unit -- will test drugs that may prevent the buildup of plaques in the brains of Alzheimer's patients. The plaques, made up of proteins called amyloid betas, are toxic to thinking cells called neurons. As the plaques build up in an afflicted brain, they block signals that can be sent between neurons, leading to memory loss, dementia and death.

"It was one of the worst things I've ever seen," said Whitney, who lives in Grove, Okla., with his wife and son, 13, and daughter, 12. His father, Roger, died in 2001 at the age of 55 after first experiencing symptoms seven years earlier. "I'm hoping my kids don't have to go through that."

Drugs to be used in the trial have already demonstrated an ability in animal and human brains to clear those amyloid beta buildups, Bateman said in an interview Friday.

Alzheimer's caused by the genetic mutation is extremely rare. Most Alzheimer's sufferers develop the disease in old age, without any family history. But Bateman believes the treatments developed through the trial could be used to stall noninherited Alzheimer's, too. Despite the different causes, buildup of amyloid betas are common to each type of patient.

The time may come when people who have no symptoms of Alzheimer's take preventive drugs to block the buildup of plaques in their brains, he said. The drugs have few side effects.

Bateman likened the concept of preventing Alzheimer's to preventing heart disease. "We don't wait until people have heart attacks or strokes. We try to prevent it and we do that by controlling high blood pressure or cholesterol levels."

But just as no cures exist for heart attacks or strokes, the treatments in the trial shouldn't be considered cures for Alzheimer's, Bateman said. While the drugs may improve conditions for someone suffering from disease, "Having their brain function return to normal and having all their memories come back -- I don't think that's likely."

The trial is funded by a $1.5 million federal grant, which could amount to as much as $6 million over four years of the study, as well as the largest grant the Alzheimer's Association has ever given -- $4.2 million. The drugs' manufacturers, Roche and Eli Lilly & Co., also donated the treatments.

The two drugs that have been selected for the trial are gantenerumab and solanezumab, antibodies that bind to forms of the aggregated amyloid beta and help remove them from the brain. The drugs failed to be effective in trials involving mid-stage Alzheimer's patients, but the hope is they will be effective on presymptomatic patients. Bateman said other drugs will be added to the trials.

"Some will be excellent and superior and move forward," he said. "Some may not work so well, so they may not continue."

Alzheimer's sufferers and their supporters will be watching the trial closely. "We've had our hopes raised before for things like a vaccine," said Jan McGillick, education director of the Alzheimer's Association's St. Louis Chapter. "We're very hopeful that with the amount of dollars involved, we can begin to unravel the causes of this illness."


To participate in the trial, a person must have at least a 50 percent chance of having an Alzheimer's disease gene mutation. That means the person has a parent or sibling who has a known mutation, or has had a positive genetic test. Participants are not required to know their mutation status to participate, but researchers will know and keep that confidential.

They must be between the ages of 18 and 80. They also must be from 15 years younger and up to 10 years older than the age at which their parent or sibling first showed symptoms of Alzheimer's. Certain medical conditions may also prevent participation.

Participants who have the mutation will have a 75 percent chance of being assigned to the group that receives one of the drugs. It's a requirement in clinical trials that some participants receive a placebo designed to resemble the test drug.

Not everyone with a family history of the disease wants the confirmation that comes with a genetic test, but Whitney and his family wanted to be mentally and financially prepared. Knowing, however, has taken its toll.

"Ever since I found out, almost 10 times an hour, every hour, every day, every week since then, there hasn't been one time I haven't thought about what I have and who this is going to affect," Whitney said. "Every little mistake I make, every time I forget to make a turn or stop in the middle of a sentence because I forget what I'm saying, it scares me."

To cope, he volunteers for the Alzheimer's Association and speaks at fundraisers to raise money for research. He's also working to improve his fitness. Participation in the clinical trial requires a healthy weight; Whitney said he's lost 35 pounds from his high of 345 pounds and is shooting for 275. He hopes that reducing his weight will also reduce his elevated liver enzymes, another condition for participating in the trial.

He could be among the numbers that are required to take a placebo.

"I can say for my entire family that because of the ... study, we have more hope now than ever before," Whitney said. "I am hopeful that these drugs will produce results, if not for me, for my children and their children." ___

Copyright 2014 MedCity News. All rights reserved. This material may not be published, broadcast, rewritten or redistributed.

By Kohler, Michele Munz and Jeremy

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