I want to say "Let's take medicine quickly", but ... January 30, 18:43

A 9-year-old boy was found to have a brain tumor.

He says it can't be removed surgically.

As the disease progressed, his right hand became more paralyzed and he threw up more often.

One day he asked me crying.

"Is this happening more often?"

"It's okay, it's okay," his mother said.

Some drugs are developed abroad.

However, it cannot be used in Japan.

"Let's take medicine soon," she said, but couldn't.

(Chiba Broadcasting Station Reporter Shintaro Sakurai)

"Brain Stem Glioma" Suddenly Struck

My son's name is Kaichi Daimon and he is 9 years old.

His favorite food is the takoyaki that my mother makes.

He likes soccer and dancing and grew up full of energy.

So the onset of this disease was really abrupt.

On the morning of May 23, 2022, Kaichi collapsed at his home.

He regained consciousness quickly, but he was taken to the hospital just in case.

A week later, his doctor told him he had a brain tumor.

why?

Even though he was in good spirits.

In the confusion, I was confronted with a disease name I had never heard of.

A type of childhood cancer called “brain stem glioma”.

Tumors form in the "brain stem," which controls breathing and heart movement, and depending on the degree of progression, it can be life-threatening.

Even after receiving an explanation from the doctor, my anxiety only increased.

Because the tumor is deep in the brain, it cannot be removed by surgery, and anticancer drugs cannot be expected to be effective.

I was told that it would take less than half a year at the earliest.

I can't say "Let's take medicine quickly"

Fortunately, Kaichi's condition did not deteriorate rapidly.

However, the disease is definitely progressing.

The paralysis in my right hand became so strong that I couldn't even open the lid of a plastic bottle.

There are also more cases of vomiting because the saliva cannot be swallowed properly.

One day, Kaichi asked while crying.

"Is this going to increase?"

"It's okay, it's okay."

I wanted to say, "Let's take medicine quickly," but I couldn't.

Even though there are miraculous medicines

I can't do surgery, and I can't expect anticancer drugs.

Even if you say so, I can't give up.

As a father, I have been researching treatments.

While working as a physiotherapist, I consulted doctors, read articles, and came across a drug.

"Dabrafenib"

is one of the "molecular-targeted drugs" that target cancers caused by mutations in specific genes.

A Canadian paper reported a case in which a child with the same tumor as Kaichi had a mutation in the gene targeted by dabrafenib showed a dramatic effect.

It was a 2016 paper.

First, Haichi's genetic test was required, which was not easy either.

Part of the tumor must be removed by surgery, and there is a risk of complications and sequelae.

The operation lasted about 9 hours.

And as a result of the test, it was confirmed that Kaichi also had the gene mutation.

Now you should be able to use the medicine.

If this was America or Canada.

However, in Japan, its use for pediatric tumors is not covered by insurance, so it cannot be used immediately.

I also inquired with the pharmaceutical company that is developing the product, but they replied that they had not submitted an application for approval to the government.

The big wall we face is called the "drag lag".

This is the difference in the time it takes for a new drug that can be used overseas to become available in Japan.

I took the risk and had the surgery.

Even though mutations in the genes that cause cancer have been discovered, and miraculously there is a drug.

It's painful that I can't let Kaichi take medicine.

I haven't even stood on the starting line

I face the harsh reality that I can't use medicine somehow, and I'm looking for the remaining means.

Dabrafenib is already approved in Japan for adult cancer.

Therefore, you can obtain and use it at your own expense.

However, it takes a lot of money continuously, and even if serious side effects occur, it will be self-responsibility.

The clinical trial conducted by the company had already closed for recruitment.

There is also a system called the "Patient Requested Treatment System," which can be used by patients who want to use unapproved drugs. .

I have heard from my doctor that Hokkaido University Hospital is currently proceeding with the plan, and I would like to participate.

Kaichi is now receiving anti-cancer drug treatment once a week.

Even though it doesn't seem to have much of an effect.

This is because the use of an unapproved drug must be ineffective in treatment with an already approved drug.

It's been more than half a year since I found out about Kaichi's illness, but he hasn't even made it to the starting line.

I can't help but think that if Kaichi's condition had progressed so quickly that the doctor's words that he wouldn't last half a year had come true.

The New “Drag Lag”

Mr. Kyohei Daimon and Mr. Shinya, and Mr. Kaichi, kindly accepted our interview.

Mr. Kaichi is a boy with a strong core who silently undergoes examinations and consultations.

My two-year-old brother also seems to love Kaichi.

The more I listened, the more I felt the unreasonableness of not being able to use drugs that were expected to be effective.

This "drug lag" has been seen as a problem in the past, and has been shortened by improving the review process for drugs for adults.

However, a situation that can be called a "new drug lag" is currently occurring, and it is particularly noticeable in the field of childhood cancer.

In fact, this situation has arisen precisely because of the widespread use of "molecular-targeted drugs."

“If the system does not change, the gap will continue to widen.”

Dr. Chitose Ogawa of the National Cancer Center points out Japan's lack of legislative development regarding the development of molecularly targeted drugs for pediatric cancer.

Because the number of patients with pediatric cancer is small, it is difficult to generate profits from drugs, and the development of new drugs for pediatric cancer has been slower than for adult cancer in Europe and the United States.

However, in 2017, a new law was enacted in the United States, making it obligatory to develop drugs for children when developing molecular-targeted drugs for adults.

Genetic mutations that cause pediatric cancer are listed, and drugs that must be developed are clarified.

Since that year, 34 new childhood cancer drugs have been approved in the United States.

However, only seven of them have been approved in Japan.

There is also a big difference in the number of clinical trials aiming for drug approval.

As of March 2022, there are 38 clinical trials targeting pediatric cancer in Japan.

In comparison, the number of clinical trials in the United States is 321.

Dr. Ogawa

: “The number of molecular-targeted drugs developed in the field of pediatric cancer has become significantly different between Japan and Europe and the United States. I think it's just one way to go."

Creating a profitable system for the development of pediatric cancer drugs

Dr. Ogawa points out that it is important to create a profitable system so that pharmaceutical companies can actively develop drugs for pediatric cancer.

In the United States, developing a drug for a rare disease in children gives the right to shorten the approval review period for another drug.

Companies will be able to sell drugs quickly, which will make it easier for them to make a profit.

This right can be bought and sold between companies and can be used to review drugs for adults.

Therefore, if it is used for drugs that are expected to sell in large numbers, there is a great advantage.

In the United States, a company that developed a pediatric cancer drug sold the rights to another pharmaceutical company for $350 million.

In Japan, there is a system that adds to the price of drugs developed for children, but it is not designed to attract pharmaceutical companies, and Dr. Ogawa says that a drastic review of the system is necessary. .

Dr. Ogawa

: “If we simply require the development of pediatric drugs by law, the cost burden will increase and there is a risk that even drugs for adults will not be developed in Japan. , we need to think about a profitable mechanism within the Japanese system.”

There is also a new movement toward a solution to Japan that is not interested

In addition, some voices point out another background.

It is the development of molecular-targeted drugs by venture companies.

In recent years, with the dramatic progress in technologies such as genetic analysis, the development of new drugs is no longer the domain of major companies, and many venture companies have entered the market.

However, they are reluctant to conduct clinical trials in Japan, regardless of whether they are for adults or children, because they do not have the financial resources of major companies.

A person in charge of a domestic company that supports the development of pharmaceuticals said, "In Japan, there is a disadvantage that pharmaceutical companies cannot decide the price of medicines. I think it is necessary to take measures to improve the attractiveness of the Japanese pharmaceutical market." .

Aiming to solve the drug lag, patient groups, doctors, pharmaceutical companies, etc. have set up an organization called the "National Conference on Pediatric Cancer Control" and are invigorating the movement by making proposals to the government.

The National Cancer Center has also created a project team to play the role of a control tower that promotes the development of treatments for childhood cancer.

The government also recognizes the drug lag of childhood cancer as an issue.

In the draft basic plan for new cancer control compiled in December 2022, it is written as follows.

“In pediatric cancer, even if a therapeutic drug candidate is found, there are few drugs that can be used under medical insurance, and there are few clinical trials in which people can participate. Improving access to drugs is an issue.

” is open for public comment until February 18, 2023, seeking input from the public.

Give your child medicine as soon as possible

Because it is a difficult problem, I wanted to carefully convey the current situation that one child is facing, so I asked for an interview with his real name and face.

His father, Kyohei, told Mr. Kaichi, "Let's get everyone to know and help us," and he accepted the interview.

I don't think anyone would oppose Kyohei's appeal to "deliver medicine to children as soon as possible."

However, in reality, while the development of cancer treatment drugs is progressing rapidly, the current situation is that the system has not sufficiently caught up.

It is said that in Europe and the United States, the delay in the development of cancer treatment drugs for children has led to a great deal of controversy, and the system has been expanded.

Now, in Japan as well, the people and doctors who have faced the drug lag are starting to raise their voices, and the country has started to take action.

I would like to convey the voices of the people on the ground so that the discussion will grow and lead to concrete measures.

Chiba Broadcasting Station Narita Branch Reporter

Shintaro Sakurai Joined the station

in 2015 Covering

the activities of a patient support group in Chiba and continuing to cover issues surrounding childhood cancer