Rebirth of the God-level Xueba

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

Thalassemia major is a very serious and fatal disease, and because it is a genetic disease, it can cause terrible harm to very young children.

Pale complexion and dysplasia will appear in the first few months. Then, the compensatory hyperplasia of bone marrow will make the bones larger. Metacarpals, ribs and long bones are just one of them, but the changes in the skull will It makes a one-year-old child unique. The raised forehead and cheekbones, the collapsed bridge of the nose, and the widened distance between the eyes will make anyone feel unbearable and uncomfortable after seeing it.

The enlarged liver and spleen and other iron-deposited internal organs damage the internal organs and bulge the skinny children's abdomen, making the whole portrait look like a terrifying zombie in the movie.

If it's just a photo or an incurable disease that Yang Rui doesn't understand, it won't make Yang Rui's mood ups and downs.

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

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

If patients with thalassemia major whose average age of premature death is less than 20 years old can be increased by 10 years, or even 20 years, while improving their quality of life, then thalassemia major will be a terrible disease. Become a terrible disease.

Here, 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 thalassemia require long-term blood transfusions. In order to remove the iron enrichment caused by long-term blood transfusions, long-term injections of deferoxamine are required. Think about it, a child needs to be injected from the age of two or three. What does it feel like to have frequent blood transfusions and frequent injections of drugs? When the other children were in kindergarten, the children with thalassemia major had deformed their whole bodies and had no complete skin.

The deferiprone mentioned by Yang Rui cannot completely replace the effect of deferoxamine, but it is very important that deferiprone can be taken orally and is more effective. Although it cannot solve the problem of patients requiring long-term blood transfusion-this requires bone marrow. Transplantation-but it can solve the patient's long-term injection of drugs.

So, for this improvement, is it worthwhile for Yang Rui to invest his energy in drug development for thalassemia?

The development of any drug is not a simple matter. With so many pharmaceutical companies in the world, how many drugs are marketed in European and American countries every year? If the United States is the benchmark, it is usually a double digit below 50, and sometimes there are only more than a dozen.

In the United States, where biological companies break 10,000, pharmaceutical companies break 1,000, and pharmaceutical companies all over the world want to land, only double-digit new drugs are launched every year. The difficulty of developing a new drug can be imagined.

Perhaps the development itself is not a difficult task for Yang Rui, but it is not an easy task for phase one clinical trial, phase two clinical trial, phase three clinical trial and fda or approval from the Food and Drug Administration.

Even a super-pharmaceutical company like Pfizer has to take 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.

does it worth?

In terms of reason, Yang Rui actually believes more in the importance of basic research.

For example, the drug for the treatment of thalassemia, the development of deferoxamine relies on the discovery of iron chelator, which is a natural product extracted from the fermentation broth of streptococcus, and is also a joint research result of microbiology and biochemistry. The deferiprone proposed by Yang Rui, as he said, is the long-term research of carboxypyridone compounds that led to the production of related drugs.

As for Yang Rui himself, the research on ion channels he made in advance, and the research on the interaction protein in the laboratory, or the research on protein g, or even the research on the cloning group that has not been formally put into operation, will vary in length. In the past time, countless important drugs have been derived. As far as past experience is concerned, these important basic research can completely affect 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 illnesses.

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

He did not devote all his energy and time to scientific research, and not all of the part he invested in doing basic research.

Therefore, it is not appropriate to only discuss greater value and greater effect.

Or, leave the research and development of deferiprone to someone else?

Yang Rui just gave up after thinking about it.

If it is done by others, it must be done in a regular manner. In other words, in addition to Yang Rui's headache of clinical trials and drug access system, more energy must be spent on drug development.

In history, when was desferiprone marketed?

March 1995.

Since the discovery of hydroxypyridone compounds as a new type of iron chelator with good effects in 1982, it took another 13 years for Indian pharmaceutical companies ~www.NovelMTL.com~ to complete drug R&D and clinical trials.

13 years later, how many patients with thalassemia major can wait to get deferiprone?

In fact, 13 years is not enough, because 1995 is the date for desferiprone to be marketed in India. It will take two or three years for the European and American drug regulatory authorities to approve the listing, and it will take another five years for the listing in China. This is already an acceleration Time is up.

In other words, if Yang Rui did not do it himself, but instead used his hands with others, the appearance of deferiprone would not be too early, perhaps five or six years earlier, but for Chinese patients, it would still take a lifetime to wait.

Yang Rui couldn't bear it.

His thoughts were turbulent.

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

If you talk about benefits, it is definitely not worth it.

Of course, desferiprone can make money, but it takes so much time and energy. If it is the same value, Yang Rui can use the same time and energy to make money that is not known how many times.

But people can't purely talk about interests. Yang Rui chose scientific research instead of doing business because he didn't talk about interests purely.

As for the balance point between interests and feelings, Yang Rui usually chooses the center-left route, but this time, it seems to be the center-right position.

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

At the same time, the doctor on the stage also said the conclusion part: "In general, Yin! Dao effervescent tablets are effective, easy to use, topical medications have no systemic side effects, and the curative effect is definite, and they can be used for a long time..."

"Let's do this, it's not right." Xu Zhengping was a little confused.

Wang Sisheng coughed and said, "You don't need to match it, just put it inside."