The Ministry of Health, Labour and Welfare (MHLW) is deliberating whether to approve the use of a new drug for Alzheimer's disease jointly developed by Japan and an American pharmaceutical company.
If approved, drugs that directly act on and eliminate the causative agent of Alzheimer's disease will be manufactured and sold in Japan for the first time.
table of contents
Deliberations begin at the expert committee
Expected to have the effect of slowing the progression of the disease itself
Voices of expectation at specialized clinics
What is the development status of therapeutic drugs that suppress the progression of Alzheimer's disease itself?
What are the challenges after approval of a new drug?
Experts "welcomed a new stage of treatment"
Open Table of Contents
table of contents
table of contents
Deliberations begin at the expert committee
Expected to have the effect of slowing the progression of the disease itself
Voices of expectation at specialized clinics
What is the development status of therapeutic drugs that suppress the progression of Alzheimer's disease itself?
What are the challenges after approval of a new drug?
Experts "welcomed a new stage of treatment"
Deliberations begin at the expert committee
Recanemab, a new treatment for Alzheimer's disease, one of the causes of dementia, is a drug that has been developed by the Japan pharmaceutical giant Eisai in collaboration with Biogen of the United States.
It is expected to suppress the progression of symptoms by removing an abnormal protein called "amyloid β" that accumulates in the brains of Alzheimer's patients, and Eisai applied to the Ministry of Health, Labour and Welfare for approval of "recanemab" in January.
The Ministry of Health, Labour and Welfare (MHLW) is deliberating whether to allow the use of drugs at an expert committee that began at 1 p.m.
According to research groups such as Eisai, the results of the final stage of clinical trials showed that patients who received this drug had about 1% less cognitive decline after one and a half years than patients who received the fake drug, and it was confirmed that it was effective in slowing the progression of the disease.
In the United States, it was approved as a treatment in early July, and if approved by Japan, a drug to eliminate the causative agent of Alzheimer's disease will be manufactured and sold in Japan for the first time.
Expected to have the effect of slowing the progression of the disease itself
Recanemab is a drug for the treatment of Alzheimer's disease developed by the pharmaceutical giant Eisai in collaboration with the American pharmaceutical company Biogen.
In the brains of Alzheimer's patients, abnormal proteins called "amyloid β" accumulate, which are thought to cause nerve cell breakdown.
"Recanemab" binds and removes artificially created antibodies at a stage called "protofibrils" before "amyloid β" becomes a fibrous mass, and is expected to have the effect of preventing nerve cells from breaking down and slowing the progression of the disease itself.
The trial found that the group that received the drug once every two weeks by intravenous drip significantly reduced the amount of amyloid β accumulated in the brain, and that cognitive decline after one and a half years was reduced by about 2% compared to the group that received a drug without an active ingredient called a "placebo."
According to research groups such as pharmaceutical companies, the results of this trial suggest that early patients can delay progression to more severe stages of symptoms by an average of two to three years.
However, since it is not possible to regenerate broken nerve cells, it is important to administer drugs at the stage of "mild cognitive impairment" before the onset of dementia and at an early stage after the onset of dementia.
On the other hand, as for side effects, cerebral hemorrhage was reported in 1.1% of patients receiving the drug, and swelling of the brain was reported in 27.2%.
Most of them were relatively mild side effects, but the research group will continue to confirm their long-term safety.
Voices of expectation at specialized clinics
At a dementia clinic in Tokyo, patients and doctors were expecting approval for recanemab.
A 79-year-old woman who attends the clinic has been diagnosed with "MCI = mild cognitive impairment," which is considered to be a precursor to dementia, so she said, "If I have dementia, my daughter will have a hard time caring for her, so if the progression can be suppressed, I would like to use it even if it is expensive."
An 81-year-old man suffering from forgetfulness said, "I often forget the most recent things and I don't have confidence in myself, so I have high hopes for medication."
The man's wife (78) said, "I hope my husband can stay at home without going to the facility.
Dr. Hirai Arai, director of Alz Clinic Tokyo, which has been diagnosing and researching dementia for more than 40 years, said, "Until now, it has been used as a symptomatic drug, but being able to slow its progression is of great significance, and it is a very big step in the history of Alzheimer's disease treatment. However, it is important to make patients and their families understand that they do not have excessive expectations about the effects. I believe that we must properly assess the risks and use them while ensuring safety."
What is the development status of therapeutic drugs that suppress the progression of Alzheimer's disease itself?
Since the first cases of Alzheimer's disease were reported about 120 years ago, research on the causes and treatments has been conducted around the world.
So far, four types of Alzheimer's disease drugs have been approved in Japan, and although they temporarily alleviate symptoms by enhancing the function of remaining nerve cells in the brain, they have not been able to stop the breakdown of nerve cells in the brain.
For this reason, research institutes and pharmaceutical companies around the world have been working on the development of therapeutic drugs that can suppress the progression of Alzheimer's disease itself, but recently there has been a succession of development of therapeutic drugs to remove amyloid β, which is thought to be one of the causes of Alzheimer's disease.
Prior to recanemab, the therapeutic drug aducanumab, developed by pharmaceutical giant Eisai and American pharmaceutical company Biogen, took advantage of a system called "rapid approval" established in 4 to provide early treatment to patients with serious illnesses. US FDA = Conditionally approved by the Food and Drug Administration.
However, while "aducanumab" has been shown to be effective in reducing "amyloid β" in clinical trials, it has not been approved in Europe or Japan because sufficient data have not been shown to suppress cognitive decline.
In addition, even this year, the American pharmaceutical giant "Eli Lilly" has similarly announced that the treatment drug "donanemab" to remove "amyloid β" in clinical trials is compared to the group that received this drug called "placebo" compared to the group that received a drug that did not contain an active ingredient. The results show that cognitive decline was suppressed by about 1% after one and a half years.
The drug has been submitted to the FDA for approval, and it is expected to be applied for approval by the end of the year in Japan.
In addition to these drugs, the development of drugs for Alzheimer's disease is active worldwide, and according to the Alzheimer's Association of the United States, as of January 2021, 1 types of therapeutic drugs are being conducted worldwide.
What are the challenges after approval of a new drug?
Even if recanemab is approved in Japan, it will not be available to all Alzheimer's patients.
According to the results of clinical trials, the drug can be expected to be effective in patients with Alzheimer's disease in the early stages of Alzheimer's disease, whose symptoms are relatively mild and "amyloid β" has accumulated in the brain.
People with dementia caused by causes other than Alzheimer's disease and patients with moderately advanced Alzheimer's disease symptoms are not expected to be eligible for the drug.
Therefore, the challenge is to accurately identify patients with Alzheimer's disease at an early stage that is expected to be effective.
PET that can photograph tissues in the body
Currently, a device called PET (PET) is used to search for patients with Alzheimer's disease by showing images of how much amyloid β has accumulated in the brain, but the device itself is large and expensive, and the medical institutions where it is installed are concentrated in urban areas.
In addition, there is a method to check whether "amyloid β" has accumulated in the brain by examination using cerebrospinal fluid, but there is a problem that the burden on the patient's body is high because the sample is collected by inserting a needle into the lower back.
Currently, none of these tests are covered by public insurance to diagnose Alzheimer's disease, making them expensive.
As a simpler and less burdensome diagnostic technology, several companies in Japan have developed a technology to estimate how much amyloid β accumulates in the brain from a small amount of blood, but it has not yet been put to practical use as a technology to confirm the diagnosis.
Another challenge is the price of the drug itself. The price in Japan has not yet been determined, but in the United States, where it has already been approved, the drug is set at an average of $1,2 per person per year, or about 6500.385 million yen per Japan yen.
In Japan, Eisai, which developed the drug, is expected to apply for public medical insurance coverage in the near future, and within 60 days in principle, and within 90 days at the latest, the Chuikyo = Central Social Insurance Medical Council will make a conclusion on insurance coverage and price.
While there are concerns that if covered by insurance, depending on the number of patients covered, it will lead to a tightening of insurance finances, but there is also a view that it will lead to a reduction in the burden of long-term care, and future discussions will be closely watched.
Experts "welcomed a new stage of treatment"
Regarding the new treatment drug "recanemab" for Alzheimer's disease, Professor Ken Ikeuchi of the Brain Research Institute, Niigata University and board member of the Japan Society for Dementia said, "I think it has been proven that removing amyloid β in the brain is one of the important targets of Alzheimer's disease treatment in the sense that it has shown that it leads to the suppression of cognitive deterioration.
"Until now, treatment methods have been positioned as symptomatic treatment, but recanemab will become a treatment that directly works on the cause, and there is a possibility that the way we think about the treatment of dementia will change. Being able to reduce symptoms and extend the state in which people can live independently can be expected to have the effect of reducing the cost of supporting people with dementia in society."
On the other hand, he pointed out that there are many challenges to dissemination, and said of the patients targeted by this drug, "It is thought that the effect can be expected in patients at an early stage, and in patients with moderate or higher progression, the effect will be limited.
In addition, regarding the system for diagnosing patients who are subject to drugs, he said, "It is necessary to conduct a test to confirm the presence of 'amyloid β in the brain of a person with early symptoms, but there is a problem that it is not an easy test because facilities with equipment are limited and it must be performed by skilled doctors. We must establish an inspection system."
Regarding the price of the drug, he said, "The price in Japan will be determined from now on, but in any case, it will not be a low price, and it may affect insurance finances. In the future, it is necessary to verify whether the effect is really commensurate with the price of the drug from various angles, such as reducing the burden of family care."