Posts tagged with "Alzheimer’s Association"

In controversial decision, FDA approves new Alzheimer’s disease drug

June 9, 2021

On June 7, the U.S. Food and Drug Administration approved the use of the experimental drug aducanumab for patient treatment during the early phases of Alzheimer’s disease—overriding the conclusion of an FDA advisory committee last year that there was not enough evidence to support the effectiveness of the treatment.

According to a report by CNN, the drug was developed for patients with mild cognitive impairment, not severe dementia, and intended to slow progression of Alzheimer’s disease —not just ease symptoms.

The FDA has not approved a novel therapy for Alzheimer’s disease since 2003.

The FDA approved aducanumab, also known as Aduhelm, using its “accelerated approval” program, which allows for the earlier approval of a drug for a serious or life-threatening illness even though more study into the drug’s benefits may be needed.

“There has been considerable public debate on whether Aduhelm should be approved. As is often the case when it comes to interpreting scientific data, the expert community has offered differing perspectives,” Dr. Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, said in Monday’s announcement.

“At the end of the day, we followed our usual course of action when making regulatory decisions in situations where the data are not straightforward,” she said, noting that the FDA ultimately decided to use accelerated approval and “concluded that the benefits of Aduhelm for patients with Alzheimer’s disease outweighed the risks of the therapy.”

“FDA will continue to monitor Aduhelm as it reaches the market and ultimately the patient’s bedside,” Cavazzoni said.

In November, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee was asked to vote on several questions about evidence of the drug’s effectiveness. In response to a question about whether it was reasonable to consider data from one positive study as the primary evidence of aducanumab’s effectiveness for the treatment of early Alzheimer’s disease, none of the committee members voted yes; ten voted no and one was uncertain.

The committee’s opinions were then left with the FDA as the agency mulled whether to approve the drug or pump the brakes.

“In all studies in which it was evaluated, however, Aduhelm consistently and very convincingly reduced the level of amyloid plaques in the brain in a dose- and time-dependent fashion,” Cavazzoni said on Monday. “It is expected that the reduction in amyloid plaque will result in a reduction in clinical decline.”

The pharmaceutical company Biogen and its Japanese partner Eisai developed aducanumab—administered through intravenous infusion to treat early Alzheimer’s disease. The drug was developed for patients with mild cognitive impairment, not severe dementia.

Dr. Richard Isaacson, director of the Alzheimer’s Prevention Clinic at Weill Cornell Medicine and NewYork-Presbyterian in New York, who had patients in the original aducanumab clinical studies, told CNN the drug targets the earliest symptomatic phase of the disease, called mild cognitive impairment due to Alzheimer’s. Treatment of the pre-dementia period was the focus of the FDA’s decision.

“We have to really temper expectations and explain to people that this drug is meant for the earliest symptomatic phases,” he said. “It pains me to say this, but if I have a severe Alzheimer’s patient that can no longer speak or interact much with others and their family member is begging me to give them this drug, I won’t be able to do it.”

Some groups, including the nonprofit Public Citizen’s Health Research Group, argued that the FDA should not approve aducanumab for treatment of Alzheimer’s disease due to lack of evidence of its effectiveness.

There have also been concerns around cost: Biogen announced on Monday that the wholesale cost of treatment with aducanumab—which requires an infusion once every four weeks—is about $4,312 per infusion, making the annual cost around $56,000 for a high dose.

However, the participating companies said, “Biogen and Eisai are committed to providing access to ADUHELM for patients across a spectrum of financial situations,” the company noted in its announcement. “For qualified, commercially insured ADUHELM patients, co-pay and infusion cost assistance programs may reduce out-of-pocket costs to as low as $0. Patients who are covered by Medicare through a Medicare Advantage plan have a maximum annual out-of-pocket cap.”

In May, the Institute for Clinical and Economic Review released a draft report estimating that the drug should cost between $2,560 to $8,290 per year, and noted that “the evidence is insufficient to conclude that the clinical benefits of aducanumab outweigh its harms or, indeed, that it reduces progression” of Alzheimer’s disease.

Other organizations, such as the Alzheimer’s Association, have supported approval of the drug.

“This approval is a victory for people living with Alzheimer’s and their families,” Harry Johns, the association’s president and CEO, said in a post on Twitter on Monday.

Research contact: @CNN

Closer to a cure: United Neuroscience tests Alzheimer’s vaccine

January 18, 2019

Today, 5.7 million Americans are living with Alzheimer’s—and, every 65 seconds, someone in the United States develops the disease, according to the Alzheimer’s Association.

For the past 20 years, biotech companies have been striving to tackle Alzheimer’s—with little success.  However now, Bloomberg reports, a four-year old Dublin-based biotech team—comprising leaders in neurology, vaccines, drug development, and disruptive ideas—believes it may be on to something.

To be clear, the news outlet says, United Neuroscience hasn’t solved Alzheimer’s yet, nor has it claimed to. But previously unreported results from a small, recent United clinical trial find that 96% of patients responded, without serious side effects, to the Alzheimer’s vaccine the company calls UB-311. The researchers describe the drug as “a novel synthetic peptide vaccine targeting beta amyloid [the main component of the amyloid plaques found in the brains of Alzheimer patients] in the treatment of Alzheimer’s.”

The patients demonstrated improved brain function and showed a reduction in the protein plaque gumming up their neurons, the company’s report says.

“The positive results show that we can safely raise and maintain [anti-beta amyloid] antibody titers in a predictable and sustained manner,” said Peter Powchik, EVP of Research and Development at UNS, in a company release.

“High response rates, reproducibility of response, and generation of antibodies directed to relevant toxic protein species are key elements of an effective therapeutic vaccine for neurodegenerative conditions. The UNS platform is proving that it can deliver on these requirements,” Powchik claimed.

Indeed, Bloomberg explains, United’s vaccine stimulates the patient’s own immune system to attack amyloid, which some researchers believe to be the leading cause. The vaccine’s job is to slow the proteins’ clumping and, if possible, reverse some damage and restore brain function.

United’s clinical trial, a Phase II study completed last year, tested the vaccine with a group of 42 patients who had mild cognitive impairment and appeared to be in the early stages of Alzheimer’s.

One set of patients was in the control group and received a placebo; while two other groups received three shots of the vaccine and then boosters either every three or six months over the course of a 18 months.

Although the small number of patients prevents United from drawing any major statistical conclusions, the company has been encouraged enough to move ahead with development of the vaccine, possibly with a larger partner, according to CEO Mel Mei Hu.

For now, United says it’s focused on raising capital to fund a more conclusive UB-311 study and to keep refining its widening range of vaccines. The 35-person company is gearing up to start trials of UB-312, aimed at Parkinson’s disease, and a second Alzheimer’s vaccine meant to combat tau [a protein that causes tangles in the brain].

“They have taken thoughtful initial steps with this very promising technology,” Eric Reiman, a leading Alzheimer’s researcher and an adviser to United Neuroscience, told Bloomberg. “But this is still the beginning of the beginning.”

Research contact: @UNSTechBio

All is not lost: When dementia patients wander, GPS devices can locate them quickly

August 27, 2018

Now where was I? That’s a phrase many of us use when we lose track of our thoughts for a moment. However, for people suffering from Alzheimer’s disease or dementia, that question often should be taken much more literally.

The disorientation that comes with these diseases often results in wandering—a common and serious concern for caregivers, who may fear that their loved ones are oblivious to their surroundings, or frightened and even in danger, according to Alzheimers.net.

Life-saving GPS devices can help caregivers to quickly track and find wanderers, before they go too far astray. Among those recommended by Alzheimers.net are the following:

  • AngelSense is a device that can be attached to a patient’s clothing and can only be removed by the caregiver. It provides a daily timeline of locations, routes, and transit speed—and sends an instant alert, if a loved diverts from a safe radius. Caregivers can use the device to listen in to what is happening around their loved one; to receive an alert if the patient has not left for an appointment on time; or to communicate with a lost person, wherever he or she may be.
  • GPS Smart Sole fits into most shoes and allows caregivers to track their loved one from any smartphone, tablet, or web browser. The shoe insert is enabled with GPS technology and allows real-time syncing, provides a detailed report of location history, and empowers users to set up a safe radius for their loved one.
  • iTraq can be used to track pretty much anything—from loved ones to luggage. This tracker pairs with a smartphone app and, for seniors, includes a motion or fall sensor that will send an alert if a fall is detected. It also has a temperature sensor. The company’s newest device, the iTraq Nano is marketed as “the world’s smallest all-in-one tracking device that has global tracking, two months of battery life, is water and dust resistant, and is able to be charged wirelessly.” The device also has an SOS button that will send an instant alert to friends and family, notifying them of their loved one’s precise location.
  • MedicAlert Safely Home originally was created to help emergency responders treat patients who could not speak for themselves. Today, the device also helps people with dementia who wander. The device is worn as a bracelet and—when a loved one goes missing—caregivers can call the police and have the police call the 24-hour hotline to get the location of the missing person. Caregivers also can call the hotline themselves to get information. In addition to a tracking device, the bracelet has important medical information engraved on it.
  • Mindme offers two lifesaving devices—one,a location device; and the other, an alarm. The alarm allows the user to alert a Mindme response center, in case of a fall or other emergency. The locator device is specifically designed for people with dementia or other cognitive disabilities. The simple device works as a pendant that can be put in a bag or pocket and allows caregivers to track the user online at any time. Caregivers also can set a radius for the user and receive an alert if the person travels outside that zone.
  • PocketFinder was founded in 2005 by a single parent who wanted to know the whereabouts of his young son. Their slogan, “If you love it, locate it!” says it all. Tracking everything from luggage to pets to children to seniors, the company offers a wide range of emerging technological products. PocketFinder is designed to be the smallest tracker on the market: The device can fit in the palm of your hand. It has a battery life up of to one week and allows caregivers to track wearers through a user-friendly app. The device was updated in January 2017 and now includes three location technologies—including GPS, Cell ID, and Google Wi-Fi Touch. It now also has an SOS button.
  • Project Lifesaver provides enrolled seniors with a personal transmitter that they wear around an ankle. If they wander, the caregiver calls a local Project Lifesaver agency and a trained team will respond. Recovery times average 30 minutes and many who wander are found within a few miles of their homes.
  • Revolutionary Tracker has location-based systems to keep tabs on seniors who may wander. This GPS-enabled personal tracker features an SOS button for emergencies and offers real-time tracking ability. The device allows multiple seniors to be tracked at the same time and syncs directly to a caregiver’s smart phone or computer.
  • Safe Link, also GPS-enabled, is a small device carried by the person who may wander. The device periodically sends its geographic coordinates to central server; and family members and caregivers can view the wearer’s location via website. The device needs to be charged and worn at all times. All devices have an SOS button for emergencies.
  • Trax is touted by the company as “the world’s smallest and lightest live GPS tracker.” The device sends position, speed, and direction through the cellular network directly to your app on a smartphone. Trax comes with a clip that is easy to attach to a loved one. The app allows caregivers to set “Geofences” and will send an alert if a loved one enters or leaves a predetermined area. Trax Geofences have no size limit: Caregivers can create as many fence areas as needed, and can schedule when those virtual fences are in effect.

According to the Alzheimer’s Association, an estimated 5.7 million Americans of all ages are living with Alzheimer’s dementia in 2018. This number includes an estimated 5.5 million people age 65 and older and about 200,000 individuals under age 65 who have younger-onset Alzheimer’s. One in 10 people age 65 and older has Alzheimer’s dementia.

Research contact: @alzassociation