In the past two days, an article titled "A Father's Choice: Desperately Taking Risks to Take Drugs, Or Waiting for The Child to Die" swept the Internet. In the text, the story of Xu Wei, who graduated from high school, taught himself gene editing and self-medicated grade compounds to treat rare diseases in order to save his son, which triggered many discussions. On the 29th, the party Xu Wei posted through Weibo, thanking the public for their concern and encouragement, responding to the safety of the compound, and seeking help from experts and relevant testing institutions to provide support and help.
△ The preparation of histidine copper was completed, and Xu Wei poured the residual liquid into a Petri dish for sterility testing
This rare disease is little known, and the epidemic has blocked the use of drugs
On June 6, 2019, Xu Wei's second child, Xiao Haoyang, was born. At 6 months old, the growth trajectory of Xiao Haoyang was significantly behind that of his peers. He doesn't lie on his stomach and roll over, and his head can't be lifted steadily. After 3 months, Kohaoyang's genetic test report showed: atp7a gene defect, menkes syndrome. This is the first case of menkes syndrome in Yunnan, and the first doctor told Xu Wei that there is no rule of law.
Xu Wei told reporters in an interview that atp7a is responsible for the operation of trace elements such as copper throughout the body, and once a genetic mutation occurs, the child's body cannot absorb such trace elements from food, which in turn affects the development of the whole body and brain. In the patient group, he learned that there are currently only thirty or forty similar patients in the country. According to statistics from all over the world, the mortality rate of this disease within 3 years of age is close to 99%. At present, there are no treatable drugs in the country.
△ The refrigerator contains histidine copper (blue) and elismomolec copper (brown)
Internationally, histidine supplementation with copper is often used as a symptomatic treatment for menkes. For children, although copper histidine can only alleviate symptoms, it seems to be the only hope for prolonging the child's life.
Helplessly, the sudden outbreak of the new crown epidemic has cut off the channels for overseas medical treatment and medicine. At first, he was able to find patients to get expired copper histidine, but soon the expired medicine was gone.
Xu Wei, who only has a high school education, embarked on the road of self-medicine
Xu Wei, who only has a high school education, looked up the information from scratch for his children, used the translation software to translate the literature of a medical term and a medical term, and read five or six hundred papers in more than a month with amazing perseverance. Because the number of patients is too small, the market for rare disease drugs is limited, and it is not profitable to do so, and few pharmaceutical companies are willing to develop and produce. Xu Wei decided that this matter could only be done by himself.
Pharmaceuticals are not simple, no one believes that he can succeed, his wife does not believe, his friends do not believe, someone reminds him that it is too dangerous to do so, and he is not going to die; someone reminds him that he may break the law. But he didn't have time to think about it, and the child couldn't do without medicine.
△ Xu Wei wrote down the process of preparing copper histidine and the amount of notes
At the beginning, Xu Wei found that there was a shared laboratory in a building, and the cost of manual work in the venue was rented for more than 14,000 yuan a day, and it could synthesize histidine copper in two days, with a shelf life of 56 days.
Xu Wei said that when he first went to the drug, he first bought raw materials according to the methods mentioned in foreign papers, sent them to a laboratory in Shanghai, and then participated in the preparation of copper histidine on August 18, 2020. At that time, I did not know that I had to pass the sterility test, etc., but let the laboratory maintain a sterile state during the preparation process. After the end, he took the "medicine" back to Yunnan.
To ensure a safe range of doses. Xu Wei bought three small rabbits for experiments, but the dose of injection was too large, and the rabbit died. "Then it was replaced by a big rabbit, and this time it was fine." But I still did not dare to inject the child, so I first injected myself and observed that there was no abnormality in the feeling for three days before injecting the child. ”
△ Xu Wei's simple laboratory, converted from a utility room
Now at 11 o'clock every night, 2 years and 3 months old, Xiao Haoyang is held in his lap by his grandmother, and the blue liquid is gradually pushed into the body. It has been injected more than 300 times since the pharmaceutical.
In order to minimize the cost as much as possible, Xu Wei eventually purchased all the domestic equipment in his utility room and built a simple dispensing room, all of which added up to less than 20,000. Although Xu Wei's efforts can only maintain the child's status quo and cannot be cured, what comforts him most is the smile that the child occasionally appears.
Hot searches on the topic, since the pharmaceutical behavior triggered a discussion
With the wide dissemination of this article, Xu Wei was praised by netizens for completing the impossible task, and the topic of #He is not a medicine god but a father has also rushed to the hot search.
After reading the story of this father, netizens were full of emotions. Hit by the father's love of the mountain.
Some netizens said that this story is touching, but do not support people in similar situations to follow suit.
Some netizens also said that if it is not helpless to the extreme, who will take such a big risk to make their own pharmaceuticals.
For Xu Wei's attempt, Zhang Jichun, director of the former Pharmacy Department of Peking Union Medical College Hospital, expressed concerns about the safety of the drug, "As an intravenous injection production is very strict, whether the added co-solvent is a medicinal specification, the amount of addition has strict regulations, and no one who can use the drugs themselves will not say that they can use it." ”
Zhang Jichun also sighed: "Being able to do this step can be said to be a miracle." At the same time, she also repeatedly stressed that this method, understandable, is not compliant. As an individual, Zhang Jichun sympathizes with the plight of families with rare diseases, and because there is no cure, he can only let patients "use dead horses as live horse doctors". But from a professional perspective, she believes that this behavior will not be encouraged, either at the level of drug regulation or clinical use.
Xu Wei sent 2 weibo responses in a row and expressed his intention to ask for help
On the 29th, Weibo was certified as "Xu Wei, the father of self-taught gene editing to save his son", the user @ live up to my time, sent 2 Weibo messages in a row to respond to related questions and expressed the intention of asking for help.
In the first Weibo, Xu Wei thanked everyone for their concern and encouragement for the child's condition.
In the second Weibo, Xu Wei first responded to questions about drug safety and the current situation of the child, and then expressed his intention to ask for help. On the one hand, he expressed the hope that relevant testing agencies can provide some help. On the other hand, it is also desirable to have expert support.
Finally, Xu Wei once again expressed his gratitude, saying that he had never heard so much encouragement and support along the way, "I hope there is always there, the child will be fine!" ”
△ University textbooks for biochemical medicine purchased by Xu Wei
According to reports, while studying self-pharmaceuticals, Xu Wei picked up textbooks and enrolled in the adult college entrance examination, eager to go to the real laboratory and learn about all aspects of his son's disease. He hopes that systematic learning will lay the foundation for future gene therapy for his son's disease.
Source: Beijing Youth Daily, Eight Points, Red Star News, Observer Network