Chapter 775

The photos on the platform had a great impact on Yang Rui.

Severe thalassemia is a very serious fatal disease, and because it is a genetic disease, it will cause terrible harm to very young children.

Pale complexion and dysplasia will appear in the first few months. Then, bone marrow compensatory hyperplasia will make the bones bigger. Metacarpal bones, ribs and long bones are just one of them. The change of skull will make a one-year-old child different. The raised forehead and cheekbones, collapsed bridge of nose and widened distance between eyes, Will let anyone see after the heart can not bear and uncomfortable.

The swollen liver and spleen and other iron deposits in the internal organs cause internal organ damage, but also make the skin and bones of the children's abdomen bulge, making the whole person like a horror zombie in the movie.

If it's just a picture, or something that Yang Rui doesn't understand, it won't make Yang Rui's mood fluctuate.

However, in Yang Rui's knowledge system, although thalassemia has not been cured, it is very likely to be controlled.

Modern medicine does not have the magic power of traditional medicine. In fact, few diseases can be cured by modern medicine. For most diseases, the goal of modern medicine is to control rather than cure.

If we can increase the life expectancy of patients with severe thalassemia whose average age of premature death is less than 20 years old by 10 years, or even 20 years, and improve their quality of life, then severe thalassemia will become a terrible disease from a terrible disease.

Among them, increasing life expectancy is only one of them, and the most important thing is to improve the quality of life of patients.

Nowadays, patients with severe and medium-sized thalassemia need long-term blood transfusion. In order to remove the iron enrichment produced by long-term blood transfusion, they also need long-term injection of deferoxamine. Think about it. A child should have frequent blood transfusion and drug injection from the age of two or three. What's that like. When other children were in kindergarten, the children with severe thalassemia had been deformed and had no complete skin.

Yang Rui said that deferoxane can not completely replace the role of deferoxamine, but one thing is very important. Deferoxane can be taken orally and is more effective. Although it can not solve the problem that patients need long-term blood transfusion, which requires bone marrow transplantation, it can solve the problem of long-term drug injection.

So, for this improvement, is Yang Rui worth investing in the drug development of thalassemia?

The development of any drug is not a simple matter. There are so many pharmaceutical companies all over the world. How many kinds of drugs are listed in European and American countries every year? Taking the United States as a benchmark, it is usually a double-digit number below 50, sometimes only a dozen.

In the United States, where there are more than 10000 biological companies and more than 1000 pharmaceutical companies, and where pharmaceutical companies all over the world want to land, only double-digit new drugs are on the market every year, so the difficulty of developing a new drug can be imagined.

Maybe the development itself is not difficult for Yang Rui, but phase I clinical trial, phase II clinical trial, phase III clinical trial and FDA or FDA approval are not easy.

Even a super pharmaceutical company like Pfizer needs to peel off more than one layer of skin in a round of clinical trials of new drugs, and the painful process of peeling often lasts for three or even ten years.

Is it worth it?

Intellectually speaking, Yang Rui actually believes in the importance of basic research.

For example, in the treatment of thalassemia, the development of deferoxamine depends on the discovery of iron chelating agent, which is a natural product extracted from Streptococcus fermentation broth and a joint research achievement of Microbiology and biochemistry. Yang Rui's deferrone, as he said, is also a long-term study of carboxyl pyridinone compounds before the emergence of related drugs.

As for Yang Rui himself, the ion channel research he made in advance, the PCR research, or the interaction protein in the laboratory, or the G protein research, or even the clone group research that has not yet been formally put into operation, will produce countless important drugs in different periods of time, It can influence the world for 50 or even 100 years.

It can be said that if Yang Rui focuses on basic biological research, he can create greater value, play a greater role, and lay the foundation for more people to solve their pain.

But emotionally, Yang Rui can't easily convince himself.

He did not devote all his spirit and time to scientific research. Even the part he devoted to basic research was not devoted to basic research.

Therefore, it is not appropriate to just discuss greater value and greater role.

Or do you leave the research and development of deferonone to others?

Yang Rui just thought about it and gave up.

If other people do it, they have to do research and development in a regular way. That is to say, in addition to Yang Rui's clinical trials and drug access system, they have to spend more energy on drug development.In history, when did desferridone come into the market?

1995 In March of this year.

Since the discovery of hydroxypyridinone as a new type of iron chelator in 1982, it took another 13 years for Indian pharmaceutical companies to complete drug research and clinical trials.

13 How many patients with severe thalassemia will be able to wait for desferridone after five years?

In fact, 13 years is not enough, because 95 is the date when desferridone will be listed in India, it will take two or three years to get approved and listed by European and American drug regulatory authorities, and it will take another five years to get listed in China, which is the time to speed up.

In other words, if Yang Rui doesn't do it himself, but does it to others, the emergence of desferridone will not be too early, maybe five or six years in advance, but for Chinese patients, it will still take a lifetime to wait.

Yang Rui couldn't bear it.

His thoughts were in a state of confusion.

Is it worth it? Yang Rui asked himself more than once.

If we talk about interests, it is certainly not worth it.

Of course, deferrone can make money, but it costs so much time and energy. If it has the same value, Yang Rui can use the same time and energy to make money many times.

But people can't talk about interests. The reason why Yang Rui chose scientific research instead of business is that he didn't talk about interests.

In the balance of interests and feelings, Yang Rui usually chooses the middle left route, but this time, it seems to be the middle right position.

"I want to make this medicine." At the end of the meeting, Yang Rui suddenly said a word to Xu Zhengping.

At the same time, the doctor on stage also said the conclusion: "in general, Yin! Dao effervescent tablet has obvious effect, convenient use, no systemic toxic and side effects, and definite curative effect. It can be used for a long time.... "

"It's not right for us to do this." Xu Zhengping was confused.

Wang Sisheng coughed and said, "you don't have to put it in the mouth, just put it in."

……