Key

• Menkes syndrome: A rare genetic copper deficiency disorder that leads to developmental delays and neurodegenerative changes.
• Copper ions: Essential trace elements in the human body. Patients with Menkes syndrome cannot effectively absorb them due to gene mutations.
• Histidine copper: A compound used to alleviate Menkes syndrome, which can supplement copper ions.
• Ellis-van Creveld syndrome: Another compound for treating Menkes syndrome, capable of carrying copper ions into the brain.
• Gene therapy: A potential method to cure Menkes syndrome by replacing the mutated gene.

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Abstract

Facing the desperate situation where his son was diagnosed with the rare disease Menkes syndrome and there was no available treatment in China, Xu Wei, a father with only a high - school education, resolutely embarked on the path of making drugs on his own. He started from scratch to learn medical knowledge, successfully synthesized histidine copper in a shared laboratory, and took the risk of treating his son, which temporarily relieved the condition. To further improve his son's brain development, he also made Ellis - van Creveld syndrome on his own. Xu Wei's story has attracted wide social attention and made more people aware of the difficulties and hopes of families with rare - disease patients. He hopes that through his efforts, he can promote the research progress of Menkes syndrome and bring the hope of life to more children.

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Insights

Xu Wei's story demonstrates the perseverance and never - giving - up spirit of families with rare - disease patients when facing difficulties. Although his actions are full of risks, they also reflect the current dilemma in rare - disease drug research and development and the urgent needs of patient families. This has not only drawn people's attention to rare - disease drug research and development but also prompted us to think about how to provide more hope for rare - disease patients through policy support, scientific research investment, etc. In addition, the emergence of shared laboratories has also provided new possibilities for individuals to conduct scientific research exploration.

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Views

01 "The Power of Fatherly Love"

Xu Wei learned medical knowledge from scratch and made drugs by himself to save his son, demonstrating the greatness and power of fatherly love.

02 "The Dilemma in Rare - Disease Drug Research and Development"

Rare diseases have a low incidence rate, and the cost of drug research and development is high, resulting in a lack of motivation for pharmaceutical companies to conduct research and development. As a result, patients face the dilemma of having no available drugs.

03 "The Value of Shared Laboratories"

Shared laboratories lower the threshold for scientific research and provide possibilities for individuals to conduct drug research and development. However, safety issues also need to be taken into consideration.

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In - Depth

A Rare - Disease Father's Journey as a "Drug God": The Miracles of Life and Ethical Dilemmas Behind Self - Made Drugs

Kunming, Yunnan - Recently, a news story about "a father with a high - school education making drugs for a rare disease to save his son" has attracted wide social attention. The protagonist, Xu Wei, transformed from an online shop owner into a "drug god" to save his son Haoyang, who suffers from the rare disease Menkes syndrome. He taught himself medical knowledge and synthesized histidine copper and Ellis - van Creveld syndrome in a shared laboratory to extend his son's life.

The Despair Caused by Rare Diseases

Menkes syndrome is a rare genetic copper deficiency disorder with a very low incidence rate, approximately one in 50,000 to 250,000. Due to gene mutations, children with this disease cannot effectively absorb copper ions, leading to serious problems such as developmental delays and neurodegenerative changes. What's even more despairing is that there is currently no specific drug for this disease in China.

In 2019, Haoyang was born and was diagnosed with Menkes syndrome at the age of six months. The doctor told Xu Wei that there was no cure for this disease, and children with this disease usually did not live past the age of three. This news was like a thunderbolt to Xu Wei.

Surviving in Desperation: A Self - Taught "Drug God"

Facing this desperate situation, Xu Wei did not give up. He began to consult a large number of medical literatures to understand the pathogenesis and treatment methods of Menkes syndrome. Through the Internet, he learned that histidine copper was a compound that could relieve the condition, but it was not available in China.

To save his son, Xu Wei decided to make histidine copper by himself. He found a shared laboratory, purchased experimental equipment and raw materials, and carried out the synthesis according to the methods in the papers. Due to his lack of a medical background, Xu Wei faced huge challenges. He needed to learn a large number of professional terms and experimental operations and often stayed up until the early hours of the morning.

After countless attempts, Xu Wei finally successfully synthesized histidine copper. To verify the safety of the drug, he first conducted experiments on rabbits. After confirming that there were no toxic side effects, he dared to use it on his son.

From Histidine Copper to Ellis - van Creveld Syndrome: An Unstoppable Exploration

The use of histidine copper temporarily relieved Haoyang's condition, but it could not improve his brain development. Xu Wei continued his research and found that Ellis - van Creveld syndrome could carry copper ions into the brain, which was helpful for brain development.

In order to enable his son to grow up like a normal child, Xu Wei challenged himself again and made Ellis - van Creveld syndrome on his own. He overcame greater technical difficulties and finally successfully synthesized this compound. By using it in combination with histidine copper, the abnormal brain waves of Haoyang were effectively improved.

The Gray Area of Ethics and Law

Xu Wei's story has touched countless people, but at the same time, it has also sparked discussions about rare - disease drug research and development, ethics, and the law.

The drugs made by Xu Wei have not been approved by the national drug regulatory department, so there are certain safety risks. In addition, his actions may also violate the law. However, in the context of the lag in rare - disease drug research and development and the lack of available drugs for patients, Xu Wei's actions also have a certain degree of rationality.

Shared Laboratories: New Possibilities for Scientific Research

Xu Wei's success is inseparable from the support of the shared laboratory. The emergence of shared laboratories has lowered the threshold for scientific research and provided possibilities for individuals to conduct drug research and development. However, shared laboratories also have problems such as insufficient supervision and safety risks, which need to be taken seriously.

The Dilemma and Way Out in Rare - Disease Drug Research and Development

Menkes syndrome is just one of many rare diseases. Due to the low incidence rate and high research - and - development costs, the research and development of rare - disease drugs has always faced difficulties.

To solve this problem, the government, pharmaceutical companies, scientific research institutions, and all sectors of society need to work together. The government can introduce relevant policies to encourage pharmaceutical companies to conduct research and development on rare - disease drugs; pharmaceutical companies can increase research - and - development investment and reduce research - and - development costs; scientific research institutions can strengthen basic research to provide theoretical support for drug research and development; all sectors of society can pay attention to rare - disease patients and provide them with help.

Forward - Looking Thinking

Xu Wei's story is a miracle about fatherly love, hope, and life. It shows us the perseverance and never - giving - up spirit of families with rare - disease patients in desperate situations and also makes us think about how to bring more hope to rare - disease patients through technological innovation, policy support, and social care.

In the future, with the continuous development of emerging technologies such as gene therapy and cell therapy, there is hope for new breakthroughs in the treatment of rare diseases. We look forward to the emergence of more "Xu Weis" who can create more life miracles for rare - disease patients with technology and love.