# Case: Xu Wei, the father who made a drug for his son

> The true story of a Chinese father with a high school education who taught himself through research papers and synthesized a drug at home for his son with a rare disease. From a homemade drug to gene therapy, and the path the father took afterward: the most extreme case of a non-expert.

[Sid](/en/start/case-sid/) was a founder, and [Paul](/en/start/case-rosie/) was a data scientist. This story's protagonist had **the fewest resources**. **Xu Wei** (徐伟) is a father born in the 1990s from Kunming, China, with a **high school education** and no specialized scientific training. When his two-year-old son **Haoyang** (颢洋) was diagnosed with a rare disease and the treatment was inaccessible, he decided to **make the medicine himself**.

This case differs somewhat from the previous two. Rather than sequencing and target discovery, it is mainly a story of **reading research papers and synthesizing a chemical directly**. But the underlying attitude, *"I will learn it and do it myself,"* is the same. As we will see, it eventually led all the way to **gene therapy**.

## 1. What happened: the diagnosis

In 2020, Haoyang was diagnosed with **Menkes syndrome**[^menkes]. This genetic disease prevents the body from **transporting copper properly**. Copper is essential for brain and nervous-system development, so a deficiency can be fatal. It mostly affects boys and is very rare, but prevalence estimates vary widely by population and method. According to [NCBI GeneReviews](https://www.ncbi.nlm.nih.gov/books/NBK1413/), children with classic Menkes syndrome who are not treated early typically die between seven months and three and a half years of age.

The problem was access to treatment. **Copper histidine**[^cuhis], used for early Menkes syndrome, was **unavailable in China** at the time. The COVID-19 pandemic also blocked routes for importing it or traveling abroad for treatment. The circumstances were documented in 2021 by [AFP](https://medicalxpress.com/news/2021-11-chinese-dad-medicine-dying-son.html) and the [South China Morning Post](https://www.scmp.com/news/people-culture/social-welfare/article/3152336/chinese-father-could-not-find-drugs-treat-his).

## 2. What he did: turning a gym into a laboratory

With no route to obtain the treatment, Xu decided to make the drug himself.

- **Self-study through research papers**: although his English was limited, he used **translation software** to read overseas medical papers one line at a time and learned how to prepare copper histidine.
- **A home laboratory**: he converted the **gym his father operated** into an experimental space and obtained the necessary reagents and equipment.
- **Direct synthesis**: he combined **copper chloride (CuCl₂·2H₂O), histidine, sodium hydroxide, and water** to make a copper histidine solution. He tested it first on rabbits and then on himself before giving it to his son. [AFP's contemporary account](https://medicalxpress.com/news/2021-11-chinese-dad-medicine-dying-son.html) documents the preparation and sequence of testing.

Xu told AFP that **some of his son's blood-test values returned to normal ranges after about two weeks**. This was the father's observation reported to the press, not evidence that overall clinical improvement or treatment efficacy had been established.

## 3. Continuing onward: all the way to gene therapy

Xu did not stop with a homemade drug. Copper histidine supplements copper but does not solve the **fundamental problem of transporting copper into the brain**.

- He turned to **elesclomol**[^eles], a candidate compound that carries copper into the brain.
- He then pursued **gene therapy**[^genetherapy] targeting **ATP7A**[^atp7a], the gene that causes Menkes syndrome.
- In the summer of 2022, Haoyang received an AAV gene-therapy candidate developed with clinicians, **VectorBuilder**, and Lantu Biotech. [The participating organizations and contemporary reports](https://vectorbuilder.biomart.cn/news/3086389.htm) described it as the **first gene-therapy attempt for Menkes syndrome worldwide**. The study was registered at [ClinicalTrials.gov as NCT05507996](https://clinicaltrials.gov/study/NCT05507996); the registry lists one actual participant and says the study was terminated in November 2022 by the investigator's decision.

A father with a high school education took his son's disease from **a homemade drug → compound exploration → a single-patient gene-therapy study with research organizations**.

## 4. Update (2024)

- **His son**: in [2022 follow-up reporting](https://static.nfapp.southcn.com/content/202208/23/c6819291.html), Xu said that after gene therapy Haoyang could **turn his head from side to side and lift his limbs higher than before**. A [Chinese biopharma publication's 2024 update](https://www.pharnexcloud.com/zixun/shiye/zjgd_9103) also reported that his condition was relatively stable and his movement had improved. Both accounts describe progress reported publicly by his caregiver.
- **His father**: according to the same [2024 update](https://www.pharnexcloud.com/zixun/shiye/zjgd_9103), Xu earned a **bachelor's degree after taking China's adult college entrance examination** and said he planned to study genetics further. He also said he intended to make his laboratory available to other families affected by rare diseases.
- **Broader impact**: Xu's story received international coverage from AFP, the South China Morning Post, and other outlets in 2021. After learning of the case, [**VectorBuilder's** researchers](https://vectorbuilder.biomart.cn/news/3086389.htm) joined Lantu Biotech and clinicians in developing the gene-therapy candidate, turning an individual's effort into a formal research collaboration.

## 5. Honest limitations

Menkes syndrome is a **very severe** disease. The neurological benefit of copper histidine depends heavily on whether treatment begins **within the first weeks of life**. It does not reverse damage that has already occurred. Xu's homemade drug carried major risks around purity, dosage, and contamination, and the risk and regulatory burden he assumed as an individual are part of the case. Changes observed in one child also cannot establish the gene therapy's general efficacy or safety.

## 6. What the three cases share

[Sid](/en/start/case-sid/), [Paul](/en/start/case-rosie/), and Xu came from different backgrounds and had different resources, but they lie on the same line.

| | Sid | Paul | Xu Wei |
| --- | --- | --- | --- |
| Background | Founder, capital, and a team | Data scientist | **High school education, minimal resources** |
| Subject | His own body | His dog | **His own son** |
| Central action | Find targets through data | Find targets with AI | **Self-study papers → make a drug → gene therapy** |

Despite their different backgrounds and resources, the same sentence runs through all three: **"Do not hand it off to someone else. Learn it, do it yourself, see it through, and make it public."** Xu started with **the least of the three** and ultimately reached formal education and clinical work.

## 7. How it connects to this study

Xu's story illuminates the subject of this handbook from **another angle**. Sid and Paul *read data to find targets*, while Xu *taught himself from primary papers and carried out the work directly*. But in the end, he too arrived at **a gene, ATP7A, and gene therapy**.

What this handbook aims to develop is ultimately the same ability: **the ability to read and understand primary sources, including data and research papers, for yourself.** These three people show in their own ways that this ability lets you take **ownership** of your problem, whatever your background.

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## Sources

- [NCBI GeneReviews: ATP7A-Related Copper Transport Disorders](https://www.ncbi.nlm.nih.gov/books/NBK1413/)
- [AFP: Chinese dad makes medicine for dying son](https://medicalxpress.com/news/2021-11-chinese-dad-medicine-dying-son.html)
- [South China Morning Post: Chinese father could not find drugs to treat his son](https://www.scmp.com/news/people-culture/social-welfare/article/3152336/chinese-father-could-not-find-drugs-treat-his)
- [The World of Chinese: I Made My Own Medicine to Keep My Son Alive](https://www.theworldofchinese.com/2021/11/i-made-my-own-medicine-to-keep-my-son-alive/)
- [ClinicalTrials.gov: NCT05507996](https://clinicaltrials.gov/study/NCT05507996)
- [VectorBuilder: development history of the Menkes gene-therapy candidate](https://vectorbuilder.biomart.cn/news/3086389.htm)
- [Nandu: one-month follow-up after gene-therapy administration](https://static.nfapp.southcn.com/content/202208/23/c6819291.html)
- [PharnexCloud: 2024 public update on Xu Wei and Haoyang](https://www.pharnexcloud.com/zixun/shiye/zjgd_9103)

[^menkes]: **Menkes syndrome** is a genetic disease in which the body **cannot transport copper into and out of cells properly**. Copper is a trace element essential to the development of the brain, nervous system, and connective tissue, so a deficiency can severely impair neurological development. Variants in the X-chromosome gene **ATP7A** cause the disease, so it mostly affects boys. Its characteristic pale, kinky hair also gives it the name "kinky hair disease."

[^cuhis]: **Copper histidine** is a compound that binds copper ions to the amino acid **histidine**. It is injected to supplement the copper lacking in patients with Menkes syndrome. It must be started **very early, in early infancy**, to slow some neurological damage, and it cannot reverse damage that has already progressed or **cure the disease**.

[^atp7a]: **ATP7A** is the gene that provides the blueprint for a **copper-transport protein** that carries copper where it is needed inside cells. Variants in this gene prevent copper from being distributed properly and cause Menkes syndrome. Gene therapy attempts to supply the body with a **normal copy of this defective gene**.

[^eles]: **Elesclomol** is a compound that binds copper ions and helps deliver copper into cells, especially **into the brain**, making it a copper ionophore. It was originally studied as an anticancer candidate and drew attention for its potential to improve delivery in diseases such as Menkes syndrome, where copper cannot reach the brain.

[^genetherapy]: **Gene therapy** treats a disease by **repairing or replacing the gene that causes it**. A common approach loads a copy of a normal gene into a carrier such as a virus and delivers it into the body so it can take over the work the defective gene cannot perform. Unlike treating symptoms alone, this approach targets the **underlying cause**.