That makes sense to Dr. Jeffrey T. Apter. It’s why he and other researchers are looking to enroll people with mild to moderate Alzheimer’s in clinical trials for a promising new drug being developed by Athira Pharma, a Seattle-based biotech company that focuses on small-molecule therapies for dementia.
“Athira has something very unique; it’s not like anything else in the pipeline,” says Apter, founder and director of the Princeton Medical Institute. “I’m a great skeptic on a lot of the drugs currently in development. We need a fresh approach and I think this is very exciting.”
The past approach has focused on trying to clear away the beta amyloid “plaque” that gums up the brain of people who have Alzheimer’s. But Apter says the effort to target amyloid “has not yielded significant results.”
The last drug to be approved by the FDA for the treatment of Alzheimer’s was Namenda, and that was in 2003. That long dry spell may end in June, when the FDA announces whether it will approve a hotly debated drug targeting amyloid that was abandoned at one point, but then revived when evidence emerged to suggest it might benefit some people with Alzheimer’s when given at a higher dose.
In the meantime, Alzheimer’s research continues to broaden beyond the so-called “amyloid theory” as scientists seek other answers, such as the Athira drug.
Apter was involved in research that led to the approval of Namenda. So was Dr. Hans Moebius, who now serves as chief medical officer for Athira Phama and is credited with developing Namenda and winning approval for it in Europe and then the U.S.
That success is one Apter is hoping to replicate by teaming with Moebius to help test ATH-1017, a novel treatment being developed by Athira. The drug has shown promise in a small study, and now research is expanding with a pair of Phase 2/Phase 3 clinical trials designed to include more than 300 patients at more than 50 research sites across the United States.
The Princeton Medical Institute is the only research center in New Jersey chosen to participate in the trial. “They are very picky about sites they go to,” Apter says. “We were very fortunate to be picked.”
The Athira drug takes a radically different approach to the treatment of Alzheimer’s. It doesn’t go after either amyloid “plaque” or the “tangles” that are the hallmarks of the disease.
Instead, it seeks to activate an obscure enzyme — Hepatocyte Growth Factor (HGF) — that is known to repair cell damage in the brain and strengthen the connectivity between brain cells.
As Alzheimer’s advances, brain cells begin to die, and the ability of remaining brain cells to communicate with each other withers.
The research so far suggests that when people with mild to moderate Alzheimer’s take the Athira drug ATH-1017, within days they begin to experience better neural connections, and that in the following weeks and months, the drug could begin to block neural degeneration and allow damaged brain cells to repair themselves.
That’s an innovative way to potentially slow down or turn back Alzheimer’s, and Apter finds it promising.
“We’re looking for drugs that may work with a different mechanism of action,” he says. “We have to find a general mechanism to stop neurodegeneration and promote neuron growth.”
Apter says he already has a few patients in the Athira trial, and he’s looking to enroll about 20.
To be eligible, a person must be between the ages of 55 and 85 and have mild or moderate Alzheimer’s disease. Participants must have a support person or caregiver, and will be considered if they either are receiving a stable dose of an Alzheimer’s drug such as Aricept or if they are not currently taking another Alzheimer’s medication.
The study involves taking the drug through an injection once a day for 26 weeks. After a patient has been evaluated and enrolled in the trial, they receive the first injection at the clinic. After that, either they or their care partner administer the injection at home.
“We give the patient and caregiver training in that,” Apter says. “We give them several sites where they can administer the medication. It’s usually not an issue for a patient after we show them how to do it.”
Anytime someone receives a medicine, there is the potential for side effects. Apter says that is discussed with the patient prior to enrolling them. The early research on ATH-1017 indicated the drug can be taken safely, with minimal side effects. “We haven’t seen anything in Phase 1 studies that would be alarming,” Apter says. “It seems to be well-tolerated.”
Conducting studies of this scale has been a challenge over the past year due to COVID and patient safety concerns.
Apter says his staff is following CDC guidelines to screen patients for COVID symptoms or exposure. Patients are asked to wear a mask and use hand sanitizer, and social distancing and isolation are maintained during their visit. With the proper protocols in place, he says, patients have continued to participate in studies without interruption.
“All of our patients have been coming in, even during COVID,” he says. “We haven’t closed for a single day.”
If you would like to be considered for the Athira study, or to learn more about it, you can visit the website of the Princeton Medical Institute or call them at (609) 921-6050. The clinic also encourages people who are experiencing memory problems or other cognitive issues to call for information about other clinical trials, or to receive a free memory screening.
“If people are interested, I would encourage them to call,” Apter says. “If they’re not eligible for this study, we may be able to find another study they quality for.”