When doctors and health policy experts debate the advantages of Aduhelm (the first new drug approved for Alzheimer’s disease in 18 years), patients want to know: “Does this drug help me? How much does it help?”

Doctors who explain the advantages and disadvantages of Aduhelm will not give clear answers. “Personally, this is absolutely impossible to predict,” said Dr. Allan Levey, director of the Goizueta Alzheimer’s Disease Research Center at Emory University.

Levey points out that cognitive decline differs greatly between people who are beginning to have memory and thinking problems or who are in the early stages of Alzheimer’s disease-these patients are tested by Aduhelm.

“The nature and speed of progress vary widely, and we don’t know when to treat someone [with Aduhelm] If their progress is fast, slow or average-we just can’t say,” Levy explained.

Nor can it specify how much the drug will affect a particular patient. “Trying to tell individuals how much delay they will experience [if they take Aduhelm] It’s simply something we can’t do,” said Dr. Jason Callaway, a professor at the Perelman School of Medicine at the University of Pennsylvania and co-director of the Pennsylvania Memory Center.

Uncertainty about the potential benefits of Aduhelm, where Get conditional approval The news from the Food and Drug Administration on June 7 is quite impressive. A phase 3 drug trial found that taking a high dose within 18 months can reduce cognitive ability for about 4 months; the second clinical trial did not show any effect. The FDA requires pharmaceutical manufacturers Biogen and Eisai Inc. to conduct post-approval trials to provide more data, but the final results may not be available until February 2030.

Due to many unresolved issues regarding the approval of Aduhelm, the House Oversight and Reform Committee has launched an investigation. Faced with criticism of insufficient guidance, the US Food and Drug Administration revised the drug’s label on Thursday to narrow its potential use. “ADUHELM should be started in patients with mild cognitive impairment or mild dementia, and these patients were started in clinical trials,” it now reads.

These developments make it extremely difficult to educate patients and their families about Aduhelm and recommend or oppose the work of doctors.

Karawish said that given the complexity of the information that needs to be conveyed, the dialogue will be “very challenging.” The following are the main points that the experts aim to explain.

The effectiveness has not been proven. Aduhelm has been shown to be very effective in removing ?-amyloid from the brain of patients, which is a hallmark of Alzheimer’s disease. This clump of protein, called amyloid plaque, is thought to be involved in the underlying disease process. However, clinical trials of other drugs that remove amyloid plaques have not proven that they can effectively prevent the progression of Alzheimer’s disease.

Although the data from the two clinical trials of Aduhelm are inconsistent, the FDA has accelerated the approval of the drug, noting that it “may bring clinical benefits.” But this is speculative, not a confirmed result.

The potential benefits are small. Dr. G. Caleb Alexander, co-director of the Center for Drug Safety and Effectiveness at Johns Hopkins University, is a member of the FDA Advisory Committee, which reviewed Aduhelm, and the group recommended against approval of the drug.

He described the positive results of a clinical trial by Aduhelm as “minor clinical changes.” On the 18-point scale used to assess cognition and function, patients who responded to medications had a 0.39 slower rate of decline within 18 months. Roughly speaking, this means that the subtle symptoms are delayed by four months.

Neuropsychological tests that assess cognitive ability usually require patients to copy charts, subtract 7 from 100 and spell back a word, among other tasks. Dr. Samuel Gandy, professor of neurology and psychiatry at Icahn College at Mount Sinai, said: “But managing daily life is much more complicated, and it’s completely unclear whether Aduhelm’s so-called benefits are enough to affect an individual’s daily life.” medicine.

The disease progression will continue. “Suppose someone has cognitive impairment or other dysfunction and decides to take Aduhelm. Will they return to normal? There is no evidence that this will happen,” Dr. Henry Paulson, professor of neurology and director of the Michigan Center for Alzheimer’s Disease Says Disease Center.

“The expectation must be that disease progression will continue,” Levey of Emory University agreed.

Potential side effects are commonAccording to a study, 41% of patients treated with the highest dose (10 mg) of Aduhelm experienced cerebral hemorrhage and swelling. This subgroup showed some reactions in a clinical trial. FDA issued documentsA brain scan revealed that 30% of the cases were mild, 58% were moderate, and 13% were severe. Most cases were resolved within three months without serious accidents.

The FDA recommends that patients taking Aduhelm have at least 3 brain MRI scans in the first year to check for side effects. Doctors and the health system are discussing what security protocols are needed in addition to these scans.

Other tests will be necessary. Aduhelm tested patients with ?-amyloid deposits in the brain, which have been confirmed by positron emission tomography brain imaging.

Experts agree that in clinical practice, only patients with these deposits can use Aduhelm and imaging to confirm that this should be necessary. But this is a problem for many patients. Because of their age, most of them are underwritten by Medicare, which does not pay for PET imaging outside of the research environment. Instead, most medical centers will rely on amyloid cerebrospinal fluid tests obtained by spinal puncture.

A form of genetic testing Apolipoprotein E gene APOE4Gandy suggested that its presence increases the risk of Alzheimer’s disease and may also cause problems. Patients with the APOE4 gene are more likely to respond to Aduhelm; at the same time, he pointed out, they are more likely to have cerebral hemorrhage and swelling. However, medical insurance does not pay for APOE4 testing or related consultations, and a positive test may significantly affect the patient’s family. “Once you find the APOE4 genotype, the first-degree relatives of all these people are at risk,” Gandy pointed out. “You will immediately change the psychology of a family.”

Treatment will be expensive. Medical insurance and private insurance companies have not yet decided whether to restrict who can obtain Aduhelm insurance, which will be managed by monthly infusions at the medical center. Considering only the annual list price of the drug is US$56,000, KFF researchers estimate that some Medicare beneficiaries can pay up to US$11,500 to cover their co-insurance obligations.

Some experts suggest that, in addition to this, the cost and expense of brain scans, monthly infusions, doctor services, amyloid testing, and APOE4 genetic testing may approach $100,000 per year.

“The most urgent thing we need is to understand how this drug is paid,” said Dr. Aaron Ritter, a dementia specialist at the Lou Ruvo Brain Health Center at the Cleveland Clinic in Las Vegas. “Many patients will receive a fixed income and have limited ability to pay large expenses.” He pointed out that the more than 1,000 patients in the clinic are ideal candidates for Aduhelm.

Experts will not prescribe for all Alzheimer’s patients. Although doctors can prescribe approved drugs for anyone they think will be helpful, dementia experts say that Aduhelm should only be used on patients similar to those in clinical trials: mild cognitive impairment (will not interfere with their memory And thinking problems) and early Alzheimer’s disease (when the symptoms are still mild but the function is impaired).

Dr. Maria Torroella Carney, director of geriatrics and palliative medicine at Northwell Health, New York’s largest healthcare system, said: “We will start small and work slowly until we have a better understanding of medications and patient responses.”

Several experts said that since Aduhelm is not tested on people with moderate or severe Alzheimer’s disease, it should not be used for these patients. Paulson of the University of Michigan said: “If patients in these later stages require this drug, we will say that we have no evidence that it works for you, and we cannot reasonably provide it to you.”

The doctor will respect the wishes of the patient. Even doctors who are worried that the potential harm of Aduhelm may outweigh the potential benefits said that they will carefully and carefully consider prescribing drugs. Callaway from the University of Pennsylvania is one of them.

“Now that this drug is available, I must adhere to the core ethics of medical practice, which is to respect patient autonomy,” he said. “If the patient and the home care staff ask to use Aduhelm after a thorough discussion, I will prescribe it reluctantly.”

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KHN (Kaiser Health News) is a national newsroom that provides in-depth reports on health issues. Together with policy analysis and voting, KHN is one of the three major operating projects in the United States. KFC (Caesar Family Foundation). KFF is a funded non-profit organization that provides information about health issues to the country.