The photos on the podium had a great impact on Yang Rui. Pen "Fun" Pavilion www.biquge.info

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

Pale and dysplasia will appear in the first few months, and then the compensatory proliferation of the bone marrow will make the bones bigger, the metacarpal bones, ribs and long bones are just one of them, but the changes in the skull will make the one-year-old child become different, the bulging forehead and cheekbones, the collapsed nose bridge, and the widened distance between the eyes will make anyone feel unbearable and uncomfortable after looking at it.

The enlarged liver, spleen and other iron deposits in the internal organs cause internal organ damage, and externally make the skinny child's abdomen bulge, making the whole person look like a terrifying zombie in a movie.

If it was just a photo, or an incurable disease that Yang Rui didn't understand, it wouldn't make Yang Rui's mood fluctuate.

However, in Yang Rui's knowledge system, although thalassemia has not yet been cured, it has a great possibility of being controlled.

Modern medicine does not have the divine power of traditional medicine, and modern medicine can cure very few diseases, and for most diseases, the goal of modern medicine is to control rather than cure.

If we can increase the life expectancy of thalassemia major patients with an average age of less than 20 years old, or even increase their life expectancy by 20 years, and improve their quality of life, then thalassemia major will turn from a terrible disease to a terrible disease.

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

Nowadays, patients with severe and moderate thalassemia need long-term blood transfusions, in order to remove the iron enrichment produced by long-term blood transfusions, and long-term injections of deferoxamine, think about what it is like for a child to have frequent blood transfusions and frequent injections of drugs from the age of two or three. When other children were in kindergarten, children with thalassemia major were already deformed and did not have a single intact piece of skin.

Deferiprone does not completely replace the effects of deferoxamine, but it is important to note that deferiprone can be taken orally, and it is more potent, and although it does not solve the problem of patients needing long-term blood transfusions - which requires bone marrow transplantation - it can solve the problem of long-term drug injections in patients.

So, for the sake of this improvement, is it worth investing in the drug development of thalassemia?

The development of any drug is not a simple matter, there are so many pharmaceutical companies in the world, and how many drugs are marketed in Europe and the United States every year? If the United States is the benchmark, it is usually less than 50 double digits, and sometimes there are only more than 10 kinds of drugs.

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

Perhaps the development itself is not difficult for Yang Rui, but the approval of phase I clinical trials, phase II clinical trials, phase III clinical trials and FDA or the Food and Drug Administration is not an easy task.

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

Is it worth it?

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

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

As for Yang Rui himself, the research on ion channels he did in advance, as well as the research on PCR, or the research on interacting proteins in the laboratory, or the research on G proteins, and even the research on the clone group that has not yet been officially put into operation, will derive countless important drugs in varying lengths of time, and in terms of past experience, these important basic studies 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 diseases.

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

He did not devote all his energy and time to scientific research, and even the part he invested was not all used to do basic research.

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

Or, leave the R&D of deferiprone to someone else?

Yang Rui just thought about it and gave up.

If someone else does it, then it is necessary to do research and development in a proper manner, that is, in addition to the clinical trial and drug access system of Yang Rui's headache, more energy should be spent on the development of drugs.

Historically, when was deferiprone marketed?

March 1995.

Since 1982, when hydroxypyridones were discovered as a new type of iron chelator with good effect, it took another 13 years for Indian pharmaceutical companies to complete drug development and clinical trials.

After 13 years, how many people with thalassemia major can wait to get deferiprone?

In fact, 13 years is simply not enough, because 95 is the date of the launch of deferiprone in India, and it will take two or three years to be approved by the European and American drug regulatory authorities, and another five years will be released to China, and this is already the time to speed up.

In other words, if Yang Rui did not do it himself, but faked it to others, the appearance of deferiprone would not be too much earlier, maybe five or six years earlier, but for Chinese patients, it would still take a lifetime to wait.

Yang Rui couldn't bear it.

His mind was in turmoil.

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

If it's about profit, it's definitely not worth it.

Of course, it is possible to make money by deferione, but it takes so much time and energy, if it is of the same value, Yang Rui can use the same time and energy to earn an unknown number of times the money.

But people can't talk purely about profits, and the reason why Yang Rui chose scientific research instead of business is because he doesn't talk purely about interests.

Between the balance of interests and feelings, Yang Rui usually chooses the center-left route, but this time, it seems to be a center-right position.

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

At the same time, the doctor on the stage also said the conclusion part: "In general, the effect of Yindao effervescent tablets is remarkable, easy to use, local medication has no systemic toxic side effects, the curative effect is certain, and it can be used ......for a long time."

"Let's do this, right? Xu Zhengping was a little confused.

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

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