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New drug study
Posted: Wednesday, November 29, 2017 6:49 AM
Joined: 5/20/2014
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Promising new Alzheimer's drug aims to reverse past failures

(Pixabay stock photo)

Jerry McLaughlin is in the business of hope. And in the not too distant future, he's looking to offer people with early signs of Alzheimer's disease something even better.

His company, AgeneBio, is about to launch HOPE4MCI, an eagerly anticipated study with the potential to provide the first medical treatment that can actually delay the onset of Alzheimer's.

When this clinical trial launches early next year, hundreds of people with mild cognitive impairment due to Alzheimer's will receive a drug called AGB101 over a period of 18 months, to see if it slows the progression of the disease, compared to patients taking a placebo.

There has not been a major breakthrough in Alzheimer's drug research in well over a decade, and there's currently no medication approved to treat people with mild cognitive impairment. AgeneBio seeks to change all of that in a single stroke by showing that a medication already used to treat epilepsy can help put the brakes on Alzheimer's by regulating the hyperactivity that occurs in the brain of people with this form of dementia.

"Part of the reason we're so excited is if we can intervene and restore this hyperactivity back to normal, you can preserve this area of the brain longer, and preserve memory and cognition longer," says McLaughlin, CEO of AgeneBio. "You may in fact be able to slow the underlying progression of the disease and hopefully a patient would be able to avoid the progression to Alzheimer's dementia."

Preliminary research on AGB101 has shown good results, reducing brain hyperactivity and significantly improving memory in a group of several dozen older adults with mild cognitive impairment due to Alzheimer's. The next step is a Phase III clinical trial involving a much larger group of patients.

The Alzheimer's Drug Discovery Foundation is a funder of this drug research, and optimistic about its chances of success.

"AGB101 has the potential to become the first drug approved for mild cognitive impairment," says Dr. Howard Fillit, founding executive director of the foundation. "The ADDF funded AgeneBio's work on this drug because we believed in its unique mechanism of action and the focus on MCI patients. We look forward to the results of the phase 3 trial."

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The recent history of Alzheimer's drug research has been disappointing and frustrating, with a failure rate of 99 percent. But most of those efforts have focused on trying to clear away the beta-amyloid "plaque" that accumulates in the brain of Alzheimer's patients. AgeneBio is taking aim at a different target, seeking to regulate hyperactivity in a part of the brain called the hippocampus.

McLaughlin says in the early stage of Alzheimer's, neurons begin to fire more frequently in the hippocampus, to the point where it becomes "hyper-excited." He compares this to a surge in the electrical current that powers your appliances.

"In your home, if you send too much electricity through the socket, you can have a surge and it can damage your TV or refrigerator," McLaughlin says. "It's much the same with the brain. Over time, you start to see a deterioration of these key areas of the brain that help regulate and help process new memory."

For a long time, this hyperactivity was presumed to be a good thing, revving up the brain to compensate for the memory loss that occurs with Alzheimer's. But Michela Gallagher, a professor of psychology and neuroscience at Johns Hopkins University and founder of AgeneBio, was the first researcher to observe that people with this hyperactivity were performing worse and worse on memory tests.

"She was the very first one to say, 'Wait, this over-activity is probably not beneficial, and in fact, it's causing memory impairment and neuron loss," McLaughlin says.

To halt this hyperactivity, AgeneBio developed AGB101, a low-dose formula of levetiracetam, an anti-epileptic drug that's been on the market for more than a decade.

"One of the things that's really promising about our therapeutic is that it has a very strong track record of safety and also it's a once-a-day oral tablet," McLaughlin says. "It can be really attractive as a therapy for patients."

The effort to test this medication on people with early signs of Alzheimer's is backed by a public-private partnership that includes the National Institutes of Health and Johns Hopkins. The Phase III trial is scheduled to begin early next year, targeting people who have been diagnosed with mild cognitive impairment due to Alzheimer's.

Mild cognitive impairment is not dementia, but rather describes people who some cognitive problem that's not normal for their age, but not to the degree that it interferes with their daily life. Some people with MCI have Alzheimer's, but some don't.

McLaughlin says the ability to diagnose Alzheimer's early has improved dramatically, and people who seek to enroll in the HOPE4MCI trial will be carefully screened to determine if they have Alzheimer's, using the combination of a cognitive exam called CDR-SB and a PET scan that detects beta-amyloid in the brain.

"We know if you have a positive diagnosis based on the CDR-SB rating and then on an amyloid test, you're above a certain threshold of amyloid in your brain, we have just about complete confidence you truly have Alzheimer's, and you are more likely to progress over the next year or two," he says.

The HOPE4MCI study will be conducted at several medical centers, although the locations haven't been announced yet. McLaughlin says the goal is to enroll a number of patients "in the hundreds." People who are interested in the trial can sign up for updates and submit information about themselves by visiting the study website at

McLaughlin says the name of the study purposefully reflects the sense of possibilities around this potential treatment.

"That really drives our vision," he says. "We want to give these patients with mild cognitive impairment hope that in a not-too-distant future, there will be a therapeutic available to them that can preserve their memory and perhaps slow the progression of their disease and give them more productive years with their family and more memories."

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He says it will take about a year to enroll participants, who will then receive the medication for 18 months. The findings are expected in late 2020 or early 2021.

"We are relentlessly focused on helping these patients and getting there as fast as possible, because every day matters," McLaughlin says. "That's the message to the patients. It won't be here tomorrow; it will take a few years, but if we are successful, we're very hopeful we can change their lives."

Tony Dearing may be reached at Follow him on Twitter @TonyDearing. Find on Facebook.

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