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

Sid was a founder, and Paul 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 syndrome¹. 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, 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 histidine², 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 and the South China Morning Post.

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 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 elesclomol³, a candidate compound that carries copper into the brain.
He then pursued gene therapy⁴ targeting ATP7A⁵, 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 described it as the first gene-therapy attempt for Menkes syndrome worldwide. The study was registered at ClinicalTrials.gov as 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, 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 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, 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 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.

Sid, Paul, 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.

Sources

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.” ↩
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. ↩
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. ↩
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. ↩
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. ↩