The first patient took a new drug for Alzheimer’s. Doctor: We don’t know if it works

Alzheimer’s is an irreversible brain disease that gradually takes away memory and the ability to live independently. There is no cure for it, but on June 7 it was worded that the US approved a drug that slows brain damage. The first patient just got it. However, the drug aducanumab is controversial. “This is probably the worst drug approval decision in recent US history,” says Prof. Aaron Kesselheim. How does this drug work and where do these critical opinions come from?

  1. Alzheimer’s disease is an irreversible brain disease that gradually destroys neurons and, consequently, memory, thinking abilities, and the ability to perform the simplest tasks
  2. June 7 this year The FDA has approved the first drug to slow the progression of Alzheimer’s. On June 16, the first patient received the preparation (apart from the clinical trial)
  3. Some experts argue that the agency’s decision is based on uncertain data on the drug’s effectiveness
  4. Criticism is also raised by the price and its impact on the insurance system. The cost of the annual treatment for one patient is estimated at PLN 56. USD
  5. More current information can be found on the Onet homepage.

The first patient took a new drug for Alzheimer’s. How does aducanumab work?

Alzheimer’s disease is an irreversible, progressive disease of the brain that gradually destroys neurons (the brain gradually shrinks) and thus memory, thinking abilities and, finally, the ability to perform the simplest of tasks. 52 million people are already struggling with alzheimer’s disease, and in 30 years there will be three times more. These numbers and the consequences of the disease explain why it is considered one of the most serious health crises of the XNUMXst century.

  1. It takes away the memory, it destroys the brain, it cannot be cured. What is Alzheimer’s Disease?

This also explains the uproar caused by the information that the US Food and Drug Administration (FDA) has approved a new drug for alzheimer’s – a preparation containing aducanumab developed by aBiogen Inc. «(…) This is the first approved drug for Alzheimer’s disease since 2003, but above all, it is the first treatment to target the cause of the disease, not just the symptoms it causes» – communicated the FDA. Doctors reminded that this is not yet a cure for Alzheimer’s disease. “We hope the drug will slow the progression of the disease,” said Ronald Petersen, an Alzheimer’s disease expert at the nonprofit Mayo Clinic.

Nine days from the announcement of the FDA’s decision, The first patient received aducanumab outside of a clinical trial. He is a 70-year-old man from Rhode Island (Northeast United States). The drug “changes the way we treat Alzheimer’s disease,” said Dr. Stephen Salloway, director of neurology at Butler Hospital, the facility where the senior was given the new drug, at a press conference. How does it work?

The preparation contains the substance aducanumab, which removes sticky deposits of a protein called beta amyloid from the brains of patients in the earlier stages of Alzheimer’s disease (they disrupt the proper functioning of neurons, eventually leading to their death). In this way, the processes of brain destruction that include memory loss and the ability to take care of yourself are to be stopped. The drug is administered intravenously once a month.

Controversy over the drug for alzheimer’s disease. “We don’t know if it works, the side effects can be significant”

The US Food and Drug Administration’s decision, however, is controversial – the main accusation being that it was made on the basis of uncertain data. Moreover, the agency decided on the “accelerated approval” of the use of the drug, despite the almost unanimous negative opinion of the advisory commission (its recommendations, however, are not binding). US media reports that at least three of its members resigned in protest against the FDA’s decision.

One of them, prof. Aaron Kesselheim from Harvard Medical School in his resignation letter even stated that “This is possibly the worst drug approval decision in recent US history.” It is worth mentioning that in the fall, the expert committee rejected the authorization of aducanumab, considering that the studies did not sufficiently prove its effectiveness. This argument returns today. “We don’t know if the drug works, and the side effects can be really significant,” said Matthew Schrag, a neurologist at Vanderbilt University Medical Center in Nashville, quoted by nationalgeographic.com. Speaking of side effects, critics of the drug indicate that approx. 35 percent. all patients taking aducanumab experienced brain edema.

Another controversial issue is the price of the drug. According to estimates, the treatment will cost almost 56 thousand. USD per year. And how much an Alzheimer’s patient will pay out of pocket will depend on their insurance. The non-governmental organization KFF predicts that aducanumab will ultimately result in an increase in health contributions, and some patients using it will still have to pay up to 11,5 thousand zlotys for it. dollars a year.

“This is a huge amount of money for a drug that we are not sure if it works for a disease that affects millions of people in the US,” comments internal medicine specialist David Rind. To his surprise, the FDA made the drug available to all Alzheimer’s patients, although clinical trials only included people with mild cognitive symptoms.

A new drug for Alzheimer’s disease. FDA position

How does the US Food and Drug Administration respond to these arguments? In FDA-assessed clinical trial data, aducanumab was effective in reducing amyloid protein accumulation in the brain and showed signs of marginal cognitive decline. This means that, unlike previously approved treatments, the drug may slow the progression of the disease rather than simply targeting symptoms. “Although data on aducanumab are complex in terms of clinical benefit, the FDA has determined that there is substantial evidence that the drug reduces the amount of beta-amyloid plaques in the brain, which is likely to benefit patients,” the FDA said in a statement.

These results were commented on by neurologist Matthew Schrag: – There is no doubt that this is a statistically proven effect (slowing down the deterioration of cognitive functions), but it is doubtful whether it is clinically significant – noted the doctor. This means that the slightly smaller cognitive decline noted in the study does not necessarily improve patients’ memory.

As for the price of the preparation, AP reports that it has been determined after careful research, and the company has committed not to be increased for the next four years. “We priced the drug at about a third of the price of cancer immunotherapy,” said Biogen director Chirfi Guindo. He added that this price seems reasonable and sustainable by the system (according to PAP)

– I’m afraid there are many more nuances and details here that the average person, desperately seeking treatment options, won’t look – comments Ellie McBroom, who looks after her mother suffering from Alzheimer’s. – We want to support scientific progress, but rashly using expensive therapies that may not bring any proven results is risky.

Experts also hope such measures will not stifle research into other drugs related to Alzheimer’s disease.

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