Posts tagged with "Weill Cornell"

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

Your ‘wildest’ dreams could offer an early clue to Parkinson’s disease

October 2, 2018

In July, 82-year-old actor Alan Alda revealed that he has Parkinson’s disease—a progressive nervous system disorder that affects movement—and in an interview with CBS This Morning, he revealed that an unusual dream helped lead him to his diagnosis. 

Alda, best-known for his portrayal of Army Captain “Hawkeye” Pierce in the TV series M*A*S*H (1972-1983), said he asked his doctor to test him for the disease after reading an article about how physically acting out your dreams can be one of the earliest precursors of the neurological disorder.

“I asked for a scan because I thought I might have it,” Alda said. “I read an article by Jane Brody in The New York Times that indicated that if you have — if you act out your dreams, there’s a good chance that might be a very early symptom, where nothing else shows,” Alda told CBS News.He recognized that what the story described had happened to him.

“By acting out your dreams, I mean I was having a dream where someone was attacking me and I threw a sack of potatoes at them, and what I was really doing was throwing a pillow at my wife,” Alda explained.

At that point, he had no other sign of illness. “The doctor said, ‘Why do you want a scan? You don’t have any symptoms,'” Alda recalled. “And I said, I want to know if there’s anything I can do—I want to do it.”

About 60,000 Americans are diagnosed with Parkinson’s disease each year, and this number does not reflect the thousands of cases that go undetected. An estimated 7 million to 10 million people worldwide—and about 1 million in the United States—are living with Parkinson’s disease, according to the Parkinson Association of the Carolinas..

Catching the disease in its early stages can be beneficial for a number of reasons, Dr. Claire Henchcliffe, director of the Parkinson’s Disease & Movement Disorders Institute at Weill Cornell Medicine and New York-Presbyterian told the network news outlet.

“There are modifiable lifestyle factors that could make a difference, for example exercising and diet. While these are not proven to delay onset of Parkinson’s there is considerable optimism about their role,” she told CBS News. “Making the diagnosis also means that if a person wants to get involved in clinical studies then they can make a real contribution to developing better understanding of and treatments for Parkinson’s.”

Henchcliffe notes that it’s been well documented that sleep disturbances, including having problems falling asleep or staying asleep, and restless legs syndrome, are common in people with Parkinson’s. Over the years there’s been some debate over whether sleep trouble is a complication of Parkinson’s or a precursor of the disease — an early warning sign that surfaces well  before other symptoms set it.

“What’s really turned out to be a critical link is the recognition that certain specific sleep disorders [such as REM sleep behavior disorder, or RBD], not only affect people with Parkinson’s but in fact show up in some cases many years earlier than the movement symptoms that lead to diagnosis,” Henchcliffe said. “So while for some types of sleep disturbances we might still debate whether they are precursors or complications, for RBD there is now extremely strong evidence that it can be a harbinger of Parkinson’s disease that will manifest some years down the line.”

Carlos L. Rodriguez, MD, a sleep medicine doctor at Cleveland Clinic Sleep Disorders Center, told CBS News that he saw a patient with RBD several years ago who was dreaming that he was playing high school football again in the defensive end position. “He had a clear avenue of attack straight to the quarterback and was rushing aggressively to tackle him when he awoke to find that his head had rammed through the drywall of his room,” Rodriguez told CBS News.

Rodriguez explains that RBD is usually caused by neurodegeneration within the brainstem, which disables the mechanisms responsible for immobilizing muscles during REM sleep—the cycle of of sleep in which we dream. This is what enables someone to literally act out what’s happening in their dreams.

The sleep disorder has been connected to other neurodegenerative diseases, including multiple system atrophy, and dementia, including Alzheimer’s disease and Lewy body disease. In one small study of 26 patients with RBD, fully 80% went on to develop Parkinson’s or another one of these diseases.

Henchcliffe emphasizes that not everyone with RBD is destined to develop Parkinson’s.

“But I do think that acting out dreams in RBD warrants a visit to a doctor to figure out what the cause may be,” she said. ”

Alda told CBS This Morning that one of the reasons he decided to speak out about his medical condition was to send a message of hope to others who might be facing the disease. The actor is still extremely active, taking boxing lessons three times a week, and he recently launched a podcast called Clear+Vivid that explores all the ways people communicate with each other.

“In the very beginning, to be immobilized by fear and think the worst thing has happened to you — it hasn’t happened to you. You still have things you can do,” he said.

Research contact: @Ashley_LizWelch