These two days, an article titled
A Father's Choice:
Desperate to take risks, or wait for the child to die"
Articles swipe the network
High school educated father
The feat of risking self-medication to save his son
Attracted heated discussion among netizens
Xu Wei, 30 years old this year
I never thought that I would one day attract a large area of attention
Xu Wei's 2-and-a-half-year-old son, Yang Yangyang, was diagnosed with Menkes syndrome in the first half year of his birth. At present, the incidence of the disease in newborns is about one in 100,000, and no drug can cure it.
In the article "A Father's Choice: Desperate to Take Risks and Drugs, Or Wait for the Child to Die", it is mentioned that internationally, copper supplementation with histidine is often used as a symptomatic treatment for Menkes. Offshore, this simple compound usually exists in the form of in-hospital preparations. There are also doctors in China who have tried to promote copper histidine as a hospital preparation, but due to the overall tightening of reagents in the hospital, cumbersome administrative approval procedures, and stricter requirements for children's injections, it was eventually not resolved. Therefore, the common choice of mainland parents is to go to hospitals in Taiwan and the United States to get medicines.
However, a new crown epidemic cut off the channels for overseas medical treatment and medicine, and Xu Wei began to embark on the dangerous road of self-medicine.
After the preparation of the agent is completed
Experiment with injecting yourself first
The father, who had no medical experience and scientific research background and only had a high school education, after consulting five or six hundred medical papers, purchased his own RAW materials to prepare histidine copper preparations according to the proportion of papers. After the preparation of the agent was completed, he first injected himself for experimentation.
At present, he has transformed a 20-square-meter utility room into a closed laboratory, bought equipment and raw materials, and will further study gene therapy.
After the preparation of histidine copper was completed, Xu Wei poured the residual liquid into a Petri dish for sterility testing (pictured from the "Eight Points Jianwen" WeChat public account, photo by Chen Xin)
Xu Wei said:
"For me, the father,
Maybe I couldn't save his life,
But I can't let him come to this world in vain. ”
After reading the story of this father
Netizens are full of emotions
Hit by the father's love of the mountain
Some netizens said that they did not support imitation
If not helpless to the extreme
And reluctant to have children
Who's going to get this
I can understand this feeling
Hopefully there will be a miracle
Intravenous injection production is very strict
Experts have expressed concerns about the safety of drugs
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." ”
After hearing about his father's experience in pharmaceuticals, Zhang Jichun also sighed: "It can be said that it is a miracle that he can do this step." 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 there is no cure, so 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 yesterday
And express the intention of asking for help
Yesterday, Weibo certified as "Xu Wei, the father of self-taught gene editing for his son" user @ live up to my time, came forward to speak out, sent 2 Weibo messages in a row to respond to relevant questions, and expressed the intention of asking for help.
In the first Weibo, @Live Up to My Time thanks everyone for their concern and encouragement for the child's condition.
In the second Weibo post, @Live Up to My Time first focused on responding to some of the questions people cared about. Regarding the safety of the compound, @ live up to my time said that it has achieved the cleanliness of the environment as much as possible according to the pharmacopoeia, the space has also been sterilized with peracetic acid, the air sedimentation bacteria and planktonic bacteria have passed the test, the preparation has finally passed the filtration sterilization of 0.22um, and all raw materials are used in medicinal grades, and finally the sterility will be verified by TSA inoculation, and the bacterial endotoxin will be detected by the reagent. Regarding the relevant situation of the child after taking the drug, @ live up to my time said that the compound can only play a role in alleviating the son's condition, so I began to cultivate mesenchymal stem cells, hoping to better improve the child's condition through stem cells.
After that, @Live Up to My Time expressed his desire to ask for help. On the one hand, he said that the detection of mycoplasma, human viruses, cell surface markers, telomerase and other related tests of biological agents is expensive, and all sources of income after the child is sick are cut off, plus the construction of laboratories and the development of genetic drugs in the later stages, the pocket is shy, and I hope that relevant testing institutions can provide some help.
On the other hand, @ live up to my time also hope to have expert support, he said that he is not very thorough about the AAV9 vector immunity problems and multiple delivery problems, I hope that the relevant AAV vector experts can help answer some questions. In addition, he also wants to design the prime editor's RNA according to the child's mutation site, and verify the editing efficiency by constructing plasmids, and hopes that relevant experts can help in this regard.
Finally, @ live up to my time once again expressed my gratitude, saying that I have never heard so much encouragement and support along the way, "I believe that there is no difficulty that cannot be overcome, the figure is greater than its detail, the figure is difficult to be easy, I hope there is always there, the child will be fine!" ”
Children are babies and motivators
For you
Parents will voluntarily transform into Superman
Only hope there is a miracle
Source | Comprehensive reporting by the Morning News
Editor: Liu Mengge Shi Shangjing